Scientific publications

The staff and experts of the European Medicines Agency (EMA) publish articles on the Agency's scientific activities in scientific publications, such as journals or textbooks.

Access to the full text of the publications may be restricted to those who have an individual or institutional subscription.


Abed I, Garcia Burgos J, Knudsen Y, Public information on shortages in the EU/EEA: improvements made between 2018 and 2020. European Journal of Hospital Pharmacy. 2023 February 8, doi: 10.1136/ejhpharm-2022-003554

Arca M., Chaves J., Himmelmann A., James S., Jonker C., Kotecha D., Lee C., Lordereau-Richard I., O'Kelly J., Plueschke K., Ryś A., Segec A., Veltrop R., Wallentin L., Weidinger F., Szymański P., Real world evidence: Perspectives from a European Society of Cardiology Cardiovascular Round Table with contribution from the European Medicines Agency - PubMed (, doi: 10.1093/ehjqcco/qcad009

Butlen-Ducuing F, McCulloch DE, Haberkamp M, Mattila T, Bałkowiec-Iskra E, Aislaitner G, Balabanov P, Lundberg J, Stenbæk DS, Elferink A, Knudsen GM, Thirstrup S, The therapeutic potential of psychedelics: the European regulatory perspective. The Lancet. 2023 February 10, doi:

Durand J, Dogné JM, Cohet C, et al. Safety Monitoring of COVID-19 Vaccines: Perspective from the European Medicines Agency Clin Pharmacol Ther. 2022;10.1002/cpt.2828. doi: 10.1002/cpt.2828

New Hofner B., Asikanius, E., Jacquet W., Framke, T., Oude Rengerink, K.,Aguirre Dávila, L., Grünewald, M., Klinglmüller F., Posch, M., Leacy, F P., Lang T., Koch, A., Zinserling, J., Roes, K., Vaccine development during a pandemic: General lessons for clinical trial design. Statistics in Biopharmaceutical Research. 2023 May 9, doi:

Salmikangas P, Carlsson B, Klumb C, Reimer T, Thirstrup S, Potency testing of cell and gene therapy products. Front Med (Lausanne). 2023 May, doi: 10.3389/fmed.2023.1190016

Tesileanu CMS, Michaleas S, Gonzalo Ruiz R, Mariz S, Fabriek BO, van Hennik PB, Dedorath J, Dekic B, Unkrig C, Brandt A, Koenig J, Enzmann H, Delgado J, Pignatti F., The EMA Assessment of Asciminib for the Treatment of Adult Patients With Philadelphia Chromosome-Positive Chronic Myeloid Leukemia in Chronic Phase Who Were Previously Treated With At Least 2 Tyrosine Kinase Inhibitors. Oncologist. 2023 May 4:oyad119. doi: 10.1093/oncolo/oyad119. Epub ahead of print. PMID: 37141403

Verbaanderd C, Trullás Jimeno A, Engelbergs J, Zander H, Reischl I, Moreno Oliver A, Vamvakas S, Vleminckx C, Bouygues C, Girard T, Day F, Frias Z, Biomarker-Driven Developments in the Context of the New Regulatory Framework for Companion Diagnostics in the European Union. National Library of Medicine. 2023 May 3, doi:


Abed I., Gonzalez-Quevedo R., Mura M., Dias M., da Rocha Dias S., García Burgos J., Commentary on the European Medicines Agency's extended mandate: Protecting public health in times of crisis and improving availability of medicines and medical devices, British Journal of Clinical Pharmacology. 2022 November 7, doi: 10.1111/bcp.15567

Bakker E., Plueschke K., Jonker C. J., Kurz X., Starokozhko V., Mol P. G. M., Contribution of Real-World Evidence in European Medicines Agency's Regulatory Decision Making. Clin Pharmacol Ther, 2022 October 17, doi: 10.1002/cpt.2766

Candore G, Monzon S, Slattery J, Piccolo L, Postigo R, Xurz X, Strauss S, Arlett P,  The Impact of Mandatory Reporting of Non-Serious Safety Reports to EudraVigilance on the Detection of Adverse ReactionsDrug Saf 45, 83–95 (2022). doi:10.1007/s40264-021-01137-0

De Wilde B, Barry E, Fox E, Karres D, Kieran M, Manlay J, Ludwinski D, Reaman G, Kearns P, The Critical Role of Academic Clinical Trials in Pediatric Cancer Drug Approvals: Design, Conduct, and Fit for Purpose Data for Positive Regulatory Decisions, Journal of Clinical Oncology, 2022 August 10. doi: 10.1200/JCO.22.00033.PMID: 35947814

Facile R, Muhlbradt EE, Gong M, Li Q, Popat V, Pétavy F, Cornet R, Ruan Y, Koide D, Saito TI, Hume S, Rockhold F, Bao W, Dubman S, Jauregui, Use of Clinical Data Interchange Standards Consortium (CDISC) Standards for Real-world Data: Expert Perspectives From a Qualitative Delphi Survey. Wurst B.JMIR Med Inform. 2022 Jan 27;10(1):e30363. doi: 10.2196/30363.PMID: 35084343

Jonker CJ, Bakker E, Kurz X, Plueschke K, Contribution of patient registries to regulatory decision making on rare diseases medicinal products in Europe. Front. Pharmacol. 13:924648. doi: 10.3389/fphar.2022.924648

Keire DA, Bream R, Wollein U, Schmaler-Ripcke J, Burchardt A, Conti M, Zmysłowski A, Keizers P, Morin J, Poh J, George M, Wierer M, International Regulatory Collaboration on the Analysis of Nitrosamines in Metformin-Containing Medicines, AAPS J 24, 56 (2022). doi: 10.1208/s12248-022-00702-4

Lasch F, Guizzaro L. Estimators for handling COVID-19-related intercurrent events with a hypothetical strategy. Pharm Stat. 2022 Jun 28. doi: 10.1002/pst.2244. Epub ahead of print. PMID: 35762230. 

Lasch F, Guizzaro L, Pétavy F, Gallo C. A simulation study on the estimation of the effect in the hypothetical scenario of no use of symptomatic treatment in trials for disease-modifying agents for Alzheimer’s disease. Statistics in Biopharmaceutical Research. 2022 Apr 28:1-4.

Lasch F, Psarelli EE, Herold R, Mattsson A, Guizzaro L, Pétavy F, Schiel A. The Impact of COVID-19 on the Initiation of Clinical Trials in Europe and the United States. Clin Pharmacol Ther. 2022 Jan 28. doi: 10.1002/cpt.2534. 

Michaleas S, Moreno Oliver A, Mueller-Berghaus J, Sarac SB, van der Elst ME, Müller-Egert S, Zander H, Enzmann H, Pignatti F, The European Medicines Agency review of sacituzumab govitecan for the treatment of triple-negative breast cancer, ESMO Open, Volume 7, Issue 3, 2022, 100497, ISSN 2059-7029, doi: 10.1016/j.esmoop.2022.100497 (

Murphy A, Bere N, Vamvakas S, Mavris M, The Added Value of Patient Engagement in Early Dialogue at EMA: Scientific Advice as a Case StudyFront. Med., 2022 January 20. doi: 10.3389/fmed.2021.811855

Palmi Reig V., Durvy B., Harvey Allchurch M., EU Mobilizes Cooperation with the African Medicines Agency, Drug Information Association., 2022 July.

Pearson A DJ, Gaspar N, Janeway K, Campbell-Hewson Q, Lawlor Elizabeth R, Copeland C, Karres D, Norga K, Benzaghou F, Weiner S, Weigel B, Weiss A R, Strauss S J, Smith M, Setty B A, Seibel N, Scobie N, Pappo A, Okpara C E, Nysom K, McDonough J, Marshall L V, Ludwinski D, Ligas F, Lesa G, Knudsen S, Kauh J, Hsieh A, Heenen D, Hawkins D S, Graham A, Garmey E, DuBois S G, Fox E, Donoghue M, de Rojas T, Chung J, Casanova M, Brennan B, Bishop M, Buenger V, Reaman G, Vassal G, Paediatric Strategy Forum for medicinal product development of multi-targeted kinase inhibitors in bone sarcomas ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration. ScienceDirect, 2022 September. doi:10.1016/j.ejca.2022.06.008

Pearson A DJ, Rossig C, Mackall C, Shah N N, Baruchel A, Reaman G, Ricafort R, Heenen D, Bassan A, Berntgen M, Bird N, Bleickardt E, Bouchkouj N, Bross P, Brownstein C, Beaussant Cohen S, de Rojas T, Ehrlich L, Fox E, Gottschalk S, Hanssens L, Hawkins D S, Horak I D, Taylor Danielle H, Johnson C, Karres D, Ligas F, Ludwinski D, Mamonkin M, Marshall L, Masouleh B K, Matloub Y, Maude S, McDonough J, Minard-Colin V, Norga K, Nysom K, Pappo A, Pearce L, Pieters R, Pule M, Quintás-Cardama A, Richardson N, Schüßler-Lenz, Scobie N, Sersch M A, Smith M A, Sterba J, Tasian S K, Weigel B, Weiner S L, Zwaan C M, Lesa G, Vassal G, Paediatric Strategy Forum for medicinal product development of chimeric antigen receptor T-cells in children and adolescents with cancer: ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration, ScienceDirect, 2021 November 21. doi:org/10.1016/j.ejca.2021.10.016

Plueschke K., Flynn R., Hedenmalm K., et al. Prescribing Patterns of Codeine and Alternative Medicines in Children in Europe. Drug Saf., 2022. doi: 10.1007/s40264-022-01214-y

Plueschke K., Jonker C., Strassmann V., et al. Collection of Data on Adverse Events Related to Medicinal Products: A Survey Among Registries in the ENCePP Resources Database. Drug Saf 45, 747-754 (2022), doi: 10.107/s40264-022-01188-x

Provansal C, Dooley D, Ziogas C, Pharmaceutical Innovation Sourcing, Nature Reviews Drug Discovery, 12 August 2022.

Schneiderova K, Bere N, Stenver DI, Straus SMJM, Patient Preferences for Rituximab Additional Risk Minimization Measures: Results From an International Online Survey, J Patient Saf, 2022 Jan 28, doi: 10.1097/PTS.0000000000000919. Online ahead of print

Tsigkos S, Mariz S, Sheean ME, Larsson K, Magrelli A, Stoyanova-Beninska V. Regulatory Standards in Orphan Medicinal Product Designation in the EU. Front Med (Lausanne). 2021 Jun 25:8:698524. doi: 10.3389/fmed.2021.698534. PMID: 34249982; PMCID: PMC8268149

van Riet-Nales D. A., van den Bemt B., van Bodegom D.,Cerreta F.,  Dooley B., Eggenschwyler D., Hirschlérova B., A. F. Jansen P., Karapinar-Çarkit F., Moran A., Span J., Stegemann S., Sundberg K., Commentary on the EMA reflection paper on the pharmaceutical development of medicines for use in the older population, Br J Clin Pharmacol. 2022;1–15. doi: 10.1111/bcp. 15207

Vaz A, Roldão Santos M, Gwaza L, Mezquita González E, Pajewska Lewandowska M, Azatyan S, Saint-Raymond A., WHO collaborative registration procedure using stringent regulatory authorities' medicine evaluation: reliance in action?, Expert Rev Clin Pharmacol. 2022 Feb 12:1-7. doi: 10.1080/17512433.2022.2037419. Epub ahead of print. PMID: 35130803


Arlett P., Kjær J., Broich K., Cooke E., Real-World Evidence in EU Medicines Regulation: Enabling Use and Establishing Value, Clinical Pharmacology & Therapeutics, 2021 November 19, doi:

Bahri, P., Pariente, A., Systematising Pharmacovigilance Engagement of Patients, Healthcare Professionals and Regulators: A Practical Decision Guide Derived from the International Risk Governance Framework for Engagement Events and Discourse, Drug Saf. 2021 September; 44, 1193-1208, doi:

Butler D., Vucic K., Straus S., Cupelli A., Micaleff B., Serracino-Inglott A., Borg B., PDF icon Regulatory experience of handling Risk Management Plans (RMPs) for medicinal products in the EU , Expert Opinion on Drug Safety, DOI: 10.1080/14740338.2021.1909569 

Candore G, Monzon S, Slattery J, Piccolo L, Postigo R, Xurz X, Strauss S, Arlett P,  The Impact of Mandatory Reporting of Non-Serious Safety Reports to EudraVigilance on the Detection of Adverse ReactionsDrug Saf 45, 83–95 (2022). doi:10.1007/s40264-021-01137-0

Cavalieri M, Enzmann H, Strauss S, Cooke E,  The European Medicines Agency's EU conditional marketing authorisations for COVID-19 vaccines, The Lancet, 2021 January 13, doi: 10.1016/S0140-6736(21)00085-4.

Cavaleri M., Sweeney F., Gonzalez-Quevedo R., Carr M., Shaping EU medicines regulation in the post COVID-19 era, The Lancet Regional Health Europe, 2021 October 1, doi: 10.1016/j.lanepe.2021.100192.

Flynn R., Plueschke K., Quinten C., Strassmann V, Duijnhoven R. G., Gordillo-Marañon M., Rueckbeil M.,CCohet C., Kurz X., Marketing Authorization Applications Made to the European Medicines Agency in 2018–2019: What was the Contribution of Real-World Evidence?, 2021 October 24, doi:

Liu Q, Schwartz JB, Slattum PW, Lau SWJ, Guinn D, Madabushi R, Burckart G, Califf R, Cerreta F, Cho C, Cook J, Gamerman J, Goldsmith P, van der Graaf PH, Gurwitz JH, Haertter S, Hilmer S, Huang S-M, Inouye SK, Kanapuru B, Pirmohamed M, Posner P, Radziszewska B, Keipp Talbot H, Temple R, Roadmap to 2030 for Drug Evaluation in Older Adults, Clinical Pharmacology & Therapeutics, October 2021, doi: 10.1002/cpt.2452 

de la Casa-Resino I, Empl MT, Villa S, Kolar B, Fabrega J, Lillicrap AD, Karamanlis XN, Carapeto-García R. Environmental risk assessment of veterinary medicinal products intended for use in aquaculture in Europe: the need for developing a harmonised approach. Environ Sci Eur. 2020;33:84.

Eichler HG, Adams R, Andreassen E, Arlett P, van de Casteele M, Chapman SJ, Goettsch WG, Martinsson JL, Llinares-Garcia J, Nachtnebel A, Pean E, Rasi G, Reksten TR, Timmers L, Vreman RA, van de Vijver I, Wenzl M. Exploring the opportunities for alignment of regulatory postauthorization requirements and data required for performance-based managed entry agreements. Int J Technol Assess Health Care. 2021 Aug 23;37(1):e83. doi: 10.1017/S026646232100057X. PMID: 34424152

Hines P.A , Agricola E., Llinares Garcia J., O’Dwyer L., Herold R., Therapeutic genome editing: regulatory horizons, ev Drug Discov. 2021 Jul 29. doi: 10.1038/d41573-021-00130-7. Online ahead of print.

del Seppia I, Schalansky J, Claassen I, PDF icon From collection to connection – the EMA veterinary data strategy , TOPRA Regulatory Rapporteur, Vol. 18, No 6, June 2021.

Pelfrene E, Botgros R, Cavaleri M., Antimicrobial multidrug resistance in the era of COVID-19: a forgotten plight?, Antimicrob Resist Infect Control. 2021 Jan 29;10(1):21. doi: 10.1186/s13756-021-00893-z. PMID: 33514424.

Ruepp R, Frötschl R, Bream R, Filancia M, Girard T, Spinei A, Weise M and Whomsley R (2021) The EU Response to the Presence of Nitrosamine Impurities in MedicinesFront. Med. 8:782536. doi: 10.3389/fmed.2021.782536

Selker HP, Cohen T, D'Agostino RB, Dere WH, Ghaemi SN, Honig PK, Kaitin KI, Kaplan HC, Kravitz RL, Larholt K, McElwee NE, Oye KA, Palm ME, Perfetto E, Ramanathan C, Schmid CH, Seyfert-Margolis V, Trusheim M, Eichler HG, A Useful and Sustainable Role for N-of-1 Trials in the Healthcare Ecosystem, Clin Pharmacol Ther. 2021 Sep 22. doi: 10.1002/cpt.2425, Epub ahead of print. PMID: 34551122.

Siapkara A., Fracasso C., Egger G.F., Rizzari C., Serén Trasorras C., Athanasiou D., Turner M.A., Recommendations by the European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA) Working Group on preparedness of clinical trials about paediatric medicines process, BMJ. Epub ahead of print. doi:10.1136/ archdischild-2020-321433

Stephenson D., Badawy R., Mathur S., Tome M., Rochester L., Digital Progression Biomarkers as Novel Endpoints in Clinical Trials: A Multistakeholder Perspective, Journal of Parkinson's Disease, vol. 11, no. s1, pp. S103-S109, 2021, doi: 10.3233/JPD-202428

Trullas A, Delgado J, Koenig J, Fuerstenau U, Dedorath J, Hausmann S, Stock T, Enzmann H, Pignatti F. The EMA assessment of encorafenib in combination with cetuximab for the treatment of adult patients with metastatic colorectal carcinoma harbouring the BRAFV600E mutation who have received prior therapy. ESMO Open. 2021 Feb;6(1):100031. doi: 10.1016/j.esmoop.2020.100031. Epub 2021 Jan 8.PMID: 33422765 Free PMC article. Review.

Trullas A, Delgado J, Genazzani A, Mueller-Berghaus J, Migali C, Müller-Egert S, Zander H, Enzmann H, Pignatti F. The EMA assessment of pembrolizumab as monotherapy for the first-line treatment of adult patients with metastatic microsatellite instability-high or mismatch repair deficient colorectal cancer, ESMO Open. 2021 Jun;6(3):100145. doi: 10.1016/j.esmoop.2021.100145. Epub 2021 Apr 30.PMID: 33940347

Trullas-Jimeno A, Delgado J, Garcia-Ochoa B, Wang I, Sancho-Lopez A, Payares-Herrera C, Dalhus M L, Strøm B O, Egeland E J, Enzmann H, Pignatti F. The EMA assessment of avapritinib in the treatment of gastrointestinal stromal tumours harbouring the PDGFRA D842V mutation, doi: 10.1016/j.esmoop.2021.100159.

Verhagen H, Alonso-Andicoberry C, Assunção R, Cavaliere F, Eneroth H, Hoekstra J, Koulouris S, Kouroumalis A, Lorenzetti S, Mantovani A, Menozzi D, Nauta M, Poulsen M, Rubert J, Siani A, Sirot V, Spaggiari G, Thomsen ST, Trevisan M, Cozzini P., Risk-benefit in food safety and nutrition - Outcome of the 2019 Parma Summer School, Food Research International, Volume 141, 2021, 110073, ISSN 0963-9969

Wang SV, Pinheiro S, Hua W, Arlett P, Uyama Y, Berlin JA, Bartels DB, Kahler KH, Bessette LG, Schneeweiss S., STaRT-RWE: structured template for planning and reporting on the implementation of real world evidence studies., BMJ. 2021 Jan 12;372:m4856. doi: 10.1136/bmj.m4856. PMID: 33436424.


Arlett P, Kurz X, Soltys K, Blum MD., International Collaboration in Real-World Evidence Generation for Direct Acting Oral Anti-Coagulants, Clin Pharmacol Ther. 2020 Aug 28. doi: 10.1002/cpt.1999. Online ahead of print.

Bahri P, Morales DR, Inoubli A, Dogné JM, Straus SMJM., Proposals for Engaging Patients and Healthcare Professionals in Risk Minimisation from an Analysis of Stakeholder Input to the EU Valproate Assessment Using the Novel Analysing Stakeholder Safety Engagement Tool (ASSET). Drug Saf. 2020 Oct 30. doi: 10.1007/s40264-020-01005-3. Epub ahead of print. PMID: 33125664.

Bell SC, Mall MA, Gutierrez H, Macek M, Madge S, Davies JC, Burgel PR, Tullis E, Castaños C, Castellani C, Byrnes CA, Cathcart F, Chotirmall SH, Cosgriff R, Eichler I, Fajac I, Goss CH, Drevinek P, Farrell PM, Gravelle AM, Havermans T, Mayer-Hamblett N, Kashirskaya N, Kerem E, Mathew JL, McKone EF, Naehrlich L, Nasr SZ, Oates GR, O'Neill C, Pypops U, Raraigh KS, Rowe SM, Southern KW, Sivam S, Stephenson AL, Zampoli M, Ratjen F.v, The future of cystic fibrosis care: a global perspective, Lancet Respir Med. 2020 Jan;8(1):65-124. doi: 10.1016/S2213-2600(19)30337-6. Epub 2019 Sep 27.

Bhasale AL, Sarpatwari A, De Bruin ML, Lexchin J, Lopert R, Bahri P, Mintzes BJ., Postmarket Safety Communication for Protection of Public Health: A Comparison of Regulatory Policy in Australia, Canada, the European Union, and the United States., Clin Pharmacol Ther. 2020 Aug 7. doi: 10.1002/cpt.2010. Epub ahead of print. PMID: 32767557.

Cavaller Bellaubi M, Harvey Allchurch M, Lagalice C, Saint-Raymond A., The European Medicines Agency facilitates access to medicines in low- and middle-income countries, Expert Rev Clin Pharmacol. 2020 Feb 13:1-5. doi: 10.1080/17512433.2020.1724782.

Cerreta F, Ritzhaupt A, Metcalfe T, Askin S, Duarte J, Berntgen M, Vamvakas S., Digital Technologies for Medicines: Shaping a Framework for Success, Nat Rev Drug Discov. 2020 May 12. doi: 10.1038/d41573-020-00080-6. Online ahead of print.

Correia Pinheiro L, Durand J, Dogné JM, An application of machine learning in pharmacovigilance: estimating likely patient genotype from phenotypical manifestations of fluoropyrimidine toxicity, Clin Pharmacol Ther. 2020 Jan 19. doi: 10.1002/cpt.1789. [Epub ahead of print]

Ehmann F., Aerssens J., Blanchard R., Commentary on ICH Guideline on Genomic Sampling and Data Management-Enabling Opportunities in Drug Development and Patient Treatment,
Br J Clin Pharmacol. 2020 Apr 19. doi: 10.1111/bcp.14305. Online ahead of print,

Eichler HG, Cavaleri M, Enzmann H, Scotti F, Sepodes B, Sweeney F, Vamvakas S, Rasi G, Clinical trials for Covid‐19: can we better use the short window of opportunity?, Clinical Pharmacology & Therapeutics, doi:10.1002/cpt.1891

Eichler HG, Rasi G.,Clinical trial publications: A sufficient basis for healthcare decisions?, Eur J Intern Med. 2020 Jan 13. pii: S0953-6205(19)30425-X. doi: 10.1016/j.ejim.2019.11.022. [Epub ahead of print]

Eichler HG, Pignatti F, Schwarzer‐Daum B, Hidalgo‐Simon A, Eichler I, Arlett P, Brun N, Humphreys A, Vamvakas S, Rasi G. (2020), Randomised controlled trials versus real world evidence: neither magic nor myth. Clin Pharmacol Ther. Accepted Author Manuscript. doi:10.1002/cpt.2083.

Fabrega, J.,Carapeto, R., Regulatory review of the environmental risk assessment of veterinary medicinal products in the European Union, with particular focus on the centralised authorisation procedure. Environ Sci Eur 32, 99 (2020). doi: 10.1186/s12302-020-00374-x

Gonzalez-Quevedo R., Ziogas C., Silva I., Vegter R., Humphreys A., Advancing development of medicines by academia and non-profit research organizations in the European Union, Nat Rev Drug Discov. 2020 Nov 23. doi: 10.1038/d41573-020-00205-x. Epub ahead of print. PMID: 33230307.

Gonzalez-Quevedo, R., et al, PDF icon EMA stakeholder workshop: advancing animal health through multi-stakeholder dialogue , TOPRA Regulatory Rapporteur April 2020

Greco M, Bere N., Patients’ emotions matter in the regulation of medicines. Blog post on BMJopinion, 11 August

Herold R., 360° Onkologie, Infopaket 2020, Ausgabe 10 : Aus Daten Lernen (4)

Hines P.A., Guy R.H., Brand A., Humphreys A.J., Papaluca-Amati M., Regulatory Science and Innovation Programme for Europe (ReScIPE): A proposed model, Br J Clin Pharmacol. 2019 Aug 19. doi: 10.1111/bcp.14099. Online ahead of print, PMID: 31426120

Hines P.A., Janssens R., Gonzalez-Quevedo R., Lambert A.I.O.M., Humphreys A.J., A future for regulatory science in the European Union: the European Medicines Agency's strategy, Nat Rev Drug Discov. 2020 May;19(5):293-294. doi: 10.1038/d41573-020-00032-0

Holtkamp F, Gudmundsdottir H, Maciulaitis R, Benda N, Thomson A, Vetter T., Change in Albuminuria and Estimated GFR as End Points for Clinical Trials in Early Stages of CKD: A Perspective From European Regulators., Am J Kidney Dis. 2020 Jan;75(1):6-8. doi: 10.1053/j.ajkd.2019.07.019. Epub 2019 Oct 28.

Januskiene J, Segec A, Slattery J, Genov G, Plueschke K, Kurz X, Arlett P., What are the patients' and health care professionals' understanding and behaviors towards adverse drug reaction reporting and additional monitoring?, Pharmacoepidemiol Drug Saf. 2020 Oct 25. doi: 10.1002/pds.5162. Online ahead of print. PMID: 33099846

Kurz X, Arlett P, Eichler HG, Nolte A, Straus S, Rasi G, Increasing the impact of Post Authorisation Safety Studies: transparency is key., Eur J Intern Med. 2020 Dec 1:S0953-6205(20)30429-5. doi: 10.1016/j.ejim.2020.11.019. Epub ahead of print. PMID: 33277138.

Kush RD, Warzel D, Kush MA, Sherman A, Navarro EA, Fitzmartin R, Pétavy F, Galvez J, Becnel LB, Zhou FL, Harmon N, Jauregui B, Jackson T, Hudson L., FAIR Data Sharing: The Roles of Common Data Elements and Harmonization., J Biomed Inform. 2020 May 11:103421. doi: 10.1016/j.jbi.2020.103421. [Online ahead of print]

Mantua V, Arango C, Balabanov P, Butlen-Ducuing F., Digital health technologies in clinical trials for central nervous system drugs: an EU regulatory perspective., Nat Rev Drug Discov. 2020 Sep 29, doi: 10.1038/d41573-020-00168-z, Epub ahead of print. PMID: 32994577

Mariz S, Westermark K, Sepodes B., PDF icon Designation of orphan conditions in Europe: regulatory observations and considerations after implementation of regulation 141/2000 , Expert Opinion on Orphan Drugs, DOI: 10.1080/21678707.2020.1784720

Michaleas S, Penninga E, Hovgaard D, Dalseg AM, Rosso A, Sarac SB, Jimenez JC, Fernández LL, Fernández CP, Mangas-SanJuan V, Garcia I, Payares-Herrera C, Sancho-López A, Enzmann H, de Castro Lopes Silva MSS, Duarte S, Pignatti F, EMA Review of Daratumumab (Darzalex) for the Treatment of Adult Patients Newly Diagnosed with Multiple Myeloma, Oncologist. 2020 Dec;25(12):1067-1074. doi: 10.1002/onco.13554. Epub 2020 Oct 16. PMID: 33026700; PMCID: PMC7938407

Monnier A.A., Tacconelli E., Årdal C., Cavaleri M., Gyssens I.C., A case study on Staphylococcus aureus bacteraemia: available treatment options, antibiotic R&D and responsible antibiotic-use strategies, JAC-Antimicrobial Resistance, Volume 2, Issue 2, June 2020, dlaa034

Moseley J, Vamvakas S, Berntgen M, Cave A, Kurz X, Arlett P, Acha V,, Bennett S, Cohet C, Corriol-Rohou S, Du Four E, Lamoril C, Langeneckert A, Koban M, Pasté M, Sandler S, Van Baelen K, Cangini A, García S, Obach M, Gimenez Garcia E, Varela Lema L, Jauhonen HM, Rannanheimo P, Morrison D, Van De Casteele M, Strömgren A, Viberg A, Makady A, Guilhaume C., Regulatory and health technology assessment advice on postlicensing and postlaunch evidence generation is a foundation for lifecycle data collection for medicines., Br J Clin Pharmacol. 2020 Mar 11. doi: 10.1111/bcp.14279. [Epub ahead of print]

Nambiar S., Cavaleri M., Sato J., Achieving Regulatory Alignment for Anti-Infective Clinical Trials, ACS Infect Dis. 2020 Jun 12;6(6):1308-1310. doi: 10.1021/acsinfecdis.0c00270

Pottegård A, Kurz X, Christiansen Ch F, Klungel O, Considerations for pharmacoepidemiological analyses in the SARS-CoV-2 pandemic, Wiley, May 2020, DOI: 10.1002/pds.5029

Radawski CA, Hammad TA, Colilla S, Coplan P, Hornbuckle K, Freeman E, Smith MY, Sobel RE, Bahri P, Arias AE, Bennett D., The utility of real-world evidence for benefit-risk assessment, communication, and evaluation of pharmaceuticals: Case studies., Pharmacoepidemiol Drug Saf. 2020 Nov 4. doi: 10.1002/pds.5167. Epub ahead of print. PMID: 33146901.

Reginster JY, Beaudart C, Al-Daghri N, Avouac B, Bauer J, Bere N, Bruyère O, Cerreta F, Cesari M, Rosa MM, Cooper C, Cruz Jentoft AJ, Dennison E, Geerinck A, Gielen E, Landi F, Laslop A, Maggi S, Prieto Yerro MC, Rizzoli R, Sundseth H, Sieber C, Trombetti A, Vellas B, Veronese N, Visser M, Vlaskovska M, Fielding RA., Update on the ESCEO recommendation for the conduct of clinical trials for drugs aiming at the treatment of sarcopenia in older adults. Aging Clin Exp Res. 2020 Jul 31. doi: 10.1007/s40520-020-01663-4.

Ritzhaupt A, Hayes I, Ehmann F., Implementing the EU in vitro diagnostic regulation - a European regulatory perspective on companion diagnostics., Expert Rev Mol Diagn. 2020 Jan 28:1-3. doi: 10.1080/14737159.2020.1720653. [Epub ahead of print]

Santoro A., Caplanusi I., Sweeney F., Cappelli B., Nolan L., Straus S., Arlett P., Navigating stormy waters: 10 years of operation of the European Union Regulatory Network Incident Management Plan for Medicines for Human Use, Pharmacoepidemiology and Drug Safety, 2020 Sept. 21,

Segec A, Slattery J, Morales DR, Januskiene J, Kurz X, Arlett P., Does additional monitoring status increase the reporting of adverse drug reactions? An interrupted time series analysis of EudraVigilance data., Pharmacoepidemiol Drug Saf. 2020 Nov 16. doi: 10.1002/pds.5174. Epub ahead of print. PMID: 33197106.

Strampelli A, Cerreta F, Vučić K., Medication use among older people in Europe: Implications for regulatory assessment and co-prescription of new medicines., Br J Clin Pharmacol. 2020 Oct;86(10):1912-1920. doi: 10.1111/bcp.14462. Epub 2020 Jul 26. PMID: 32644249; PMCID: PMC7495283.

Tambuyzer E, Vandendriessche B, Austin CP, Brooks PJ, Larsson K, Needleman KIM, Valentine J, Davies K, Groft SC, Preti R, Oprea TI, Prunotto M. Correction to: Therapies for rare diseases: therapeutic modalities, progress and challenges ahead., Nat Rev Drug Discov. 2020 Jan 8. doi: 10.1038/s41573-019-0059-7. [Epub ahead of print]

Tavridou A, Rogers D, Bonelli M, Schiel A, Hidalgo-Simon A.,Towards a better use of scientific advice for developers of advanced therapies., Br J Clin Pharmacol. 2020 Nov 25. doi: 10.1111/bcp.14672. Epub ahead of print. PMID: 33237580.

Vreman RA, de Ruijter AS, Zawada A, Tafuri G, Stoyanova-Beninska V, O’Connor D, Naumann-Winter F, Wolter F, Mantel-Teeuwisse AK, Leufkens HGM, Sidiropoulos I, Larsson K, Goettsch WG., Assessment of significant benefit for orphan medicinal products by European regulators may support subsequent relative effectiveness assessments by health technology assessment organizations

Wieringa F.P., Sheldon M.I., Hidalgo Simon A., Regulatory Approaches to Stimulate Innovative Renal Replacement Therapies, Nat Rev Nephrol. 2020 Apr 2. doi: 10.1038/s41581-020-0275-8. Online ahead of print


Arlett P., Building an Evidence Base on the Place of Industry-Sponsored Programs in Drug Safety Surveillance, Drug Saf. 2019 May;42(5):581-582. doi: 10.1007/s40264-018-00791-1.

Arlett P, Straus S, Rasi G, Pharmacovigilance 2030, Clin Pharmacol Ther, November 2019, doi: 10.1002/cpt.1689

Brown P, Bahri P, Engagement’ of patients and healthcare professionals in regulatory pharmacovigilance: establishing a conceptual and methodological framework, Eur J Clin Pharmacol (2019).

Cave A., Kurz X., Arlett P, Real world data for regulatory decision making: challenges and possible solutions for Europe, Clin Pharmacol Ther. 2019 Apr 10. doi: 10.1002/cpt.1426. [Epub ahead of print]

Cerreta F, EMA geriatric expert drafting group., New harmonized considerations on the evaluation instruments for baseline characterisation of frailty in the European Union., Br J Clin Pharmacol. 2019 Jul 5. doi: 10.1111/bcp.14044. [Epub ahead of print]

Coppola P, Andersson A, Cole S., The Importance of the Human Mass Balance Study in Regulatory Submissions., CPT Pharmacometrics Syst Pharmacol. 2019 Sep 12. doi: 10.1002/psp4.12466. [Epub ahead of print]

De Wit M, Cooper C, Tugwell P, Bere N, Kirwan J, Conaghan PG, Roberts C, Aujoulat I, Al-Daghri N, Araujo de Carvalho I, Barker M, Bedlington N, Brandi ML, Bruyere O, Burlet N, Halbout P, Hiligsmann M, Jiwa F, Kanis JA, Laslop A, Lawrence W, Pinto D, Prieto Yerro C, Rabenda V, Rizzoli R, Scholte-Voshaar M, Vlaskoyska M. Reginster JY, Practical guidance for engaging patients in health research, treatment guidelines and regulatory processes: Results of an expert group meeting organized by the World Health Organization (WHO) and the European Society for Clinical and Economic Aspects of Osteoporosis, Osteoarthritis and Musculoskeletal Diseases (ESCEO), Aging Clin Exp Res (2019)

Eichler H-G, Enzmann H, Rasi G, Added therapeutic benefit and drug licensing, NRDD April 2019, doi: 10.1038/d41573-019-00068-x

Eichler H G, Koenig F,  Arlett P,  Enzmann H,  Humphreys A, Pétavy F, Schwarzer‐Daum B, Sepodes B, Vamvakas S, Rasi G Are novel, non‐randomised analytic methods fit for decision‐making? The need for prospective, controlled and transparent validation Clin Pharm Ther, 2019, doi:10.1002/cpt.1638

Gini R, Fournie X, Dolk H, Kurz X, Verpillat P, Simondon F, Strassmann V, Apostolidis K, Goedecke T., The ENCePP Code of Conduct: A best practise for scientific independence and transparency in noninterventional postauthorisation studies, Pharmacoepidemiol Drug Saf. 2019 Apr;28(4):422-433. doi: 10.1002/pds.4763.

Haberkamp M, Moseley J, Athanasiou D, de Andres-Trelles F, Elferink A, Rosa MM, Magrelli A., European regulators’ views on a wearable-derived performance measurement of Ambulation for DMD regulatory trials, Neuromuscul Disord. 2019 Jul;29(7):514-516. doi: 10.1016/j.nmd.2019.06.003.

Hedenmalm K, Blake K, Donegan K, Macia MA, Gil M, Williams J, Montero D, Candore G, Morales D, Kurz X, Arlett P., A European multi-centre drug utilisation study of the impact of regulatory measures on prescribing of codeine for pain in children, Pharmacoepidemiol Drug Saf. 2019;1-11

Hedenmalm K, Slattery J, Skibicka-Stepien I, Kurz X, Morales D, Prescribing patterns of tramadol in adults in IMS® primary care databases in France and Germany between 1 January  2006 and 30 June 2016, Eur J Clin Pharmacol. 2019 Jan 23. doi: 10.1007/s00228-018-02622-9. 

Hedenmalm K, Kurz X, Morales D, Effect of withdrawal of fusafungine from the market on prescribing of antibiotics and other alternative treatments in Germany: a pharmacovigilance impact study, Eur J Clin Pharmacol. 2019 Mar 5. doi: 10.1007/s00228-019-02650-z. [Epub ahead of print]

Herold R, Camarero J, Melchiorri D, Sebris Z, Enzmann H, Pignatti F., Revocation of the conditional marketing authorisation of a cancer medicine: The olaratumab experience, Eur J Cancer. 2019 Oct 23;123:25-27. doi: 10.1016/j.ejca.2019.09.020. [Epub ahead of print]

Hidalgo-Simon A., From laboratory to patient - the evolving path to become a medicine, MOLECULAR FRONTIERS JOURNAL, Volume 3, Number 2, December 2019

Hines PA, Guy RH, Humphreys AJ, Papaluca-Amati M., The European Medicines Agency’s goals for regulatory science to 2025, Nat Rev Drug Discov. 2019 Jun;18(6):403-404. doi: 10.1038/d41573-019-00071-2.

Hines P, Hiu Yu L, Guy RH, Brand A, Papaluca-Amati M., Scanning the horizon: a systematic literature review of methodologies, BMJ Open. 2019 May 27;9(5):e026764. doi: 10.1136/bmjopen-2018-026764.

Jauregui B, Hudson LD, Becnel LB, Navarro Almario E, Fitzmartin R, Pétavy F, Seigneuret N, Malone JK, Zhou FL, Galvez J, Jackson T, Harmon N, Kush RD, The Turning Point for Clinical Research: Global Data Standardization, Applied Clinical Trials Online, 22 January 2019.

Kashoki M, Hanaizi Z, Yordanova S, Veselý R, Bouygues C, Llinares J, Kweder SL., A Comparison of EMA and FDA Decisions for New Drug Marketing Applications 2014-2016: Concordance, Discordance, and Why, Clin Pharmacol Ther. 2019 Jul 15. doi: 10.1002/cpt.1565. [Epub ahead of print]

Kehoe T, Blind E, Janssen H. Regulatory Aspects of the Development of Drugs for Metabolic Bone Diseases, Br J Clin Pharmacol. 2019 Jun;85(6):1208-1212. doi: 10.1111/bcp.13791. Epub 2018 Dec 3.

Lenić I, Blake K, Garcia-Arieta A, Potthast H, Welink J., Overview of the European Medicines Agency's Experience With Biowaivers in Centralized Applications,Clin Transl Sci. 2019 Sep;12(5):490-496. doi: 10.1111/cts.12642.

Lienhardt C, Vernon AA, Cavaleri M, Nambiar S, Nahid P., Development of new TB regimens: Harmonizing trial design, product registration requirements, and public health guidance., PLoS Med. 2019 Sep 6;16(9):e1002915. doi: 0.1371/journal.pmed.1002915. eCollection 2019 Sep.

Mavris M, Furia Helms A, Bere N, Engaging patients in medicines regulation: a tale of two agencies., Nature Reviews Drug Discovery, ISSN 1474-1784 (online)

McGettigan P, Alonso Olmo C, Plueschke K, Castillon M, Nogueras Zondag D, Bahri, Kurz X, Mol PGM, Patient Registries: An Underused Resource for Medicines Evaluation, Drug Saf. 2019 Jul 13. doi: 10.1007/s40264-019-00848-9. [Epub ahead of print]

Morales D, Pacurariu A, Slattery J, Pinheiro L, McGettigan P, Kurz X., Association Between Peripheral Neuropathy and Exposure to Oral Fluoroquinolone or Amoxicillin-Clavulanate Therapy, JAMA Neurol. 2019 Apr 29. doi: 10.1001/jamaneurol.2019.0887. [Epub ahead of print]

Morales DR, Slattery J, Pacurariu A, Pinheiro L, McGettigan P, Kurz X, Correction to: Relative and Absolute Risk of Tendon Rupture with Fluoroquinolone and Concomitant Fluoroquinolone/Corticosteroid Therapy: Population-Based Nested Case-Control Study, Clin Drug Investig. 2019 Feb;39(2):215. doi: 10.1007/s40261-019-00755-y.

Morgan EM, Munro JE, Horonjeff J, Horgan B, Shea B, Feldman BM, Clairman H, Bingham CO 3rd, Thornhill S, Strand V, Alongi A, Magni-Manzoni S, van Rossum MAJ, Vesely R, Vojinovic J, Brunner HI, Harris JG, Horton DB, Lovell DJ, Mannion M, Rahimi H, Ravelli A, Ringold S, Ruperto N, Schrandt MS, Shenoi S, Shiff NJ, Toupin-April K, Tzaribachev N, Weiss P, Consolaro A., Establishing an Updated Core Domain Set for Studies in Juvenile Idiopathic Arthritis: A Report from the OMERACT 2018 JIA Workshop., J Rheumatol. 2019 Aug;46(8):1006-1013. doi: 10.3899/jrheum.181088. Epub 2019 Feb 15.

Nielsen SM, Tugwell P, de Wit MPT, Boers M, Beaton DE, Woodworth TG, Escorpizo R, Shea B, Toupin-April K, Guillemin F, Strand V, Singh JA, Kloppenburg M, Furst DE, Wells GA, Smolen JS, Veselý R, Boonen A, Storgaard H, Voshaar M, March L, Christensen R; Contextual Factors Working Group, Identifying Provisional Generic Contextual Factor Domains for Clinical Trials in Rheumatology: Results from an OMERACT Initiative, J Rheumatol. 2019 Sep;46(9):1159-1163. doi: 10.3899/jrheum.181081. Epub 2019 Jan 15.

Nordenmalm S, Kimland E, Ligas F, Lehmann B, Claverol J, Nafria B, Tötterman AM, Pelle B.,Children’s views on taking medicines and participating in clinical trials, Arch Dis Child. 2019 Sep;104(9):900-905. doi: 10.1136/archdischild-2018-31651

Ollivier C, Sun H, Amchin W, Beghetti M, Berger RMF, Breitenstein S, Garnett C, Gullberg N, Hassel P, Ivy D, Kawut SM, Klein A, Lesage C, Migdal M, Nije B, Odermarsky M, Strait J, de Graeff PA, Stockbridge N. New strategies for the conduct of clinical trials in pediatric Pulmonary Arterial Hypertension (PAH): Outcome of a multi-stakeholder meeting with patients, academia, industry and regulators held at the European Medicines Agency on Monday 12th June 2017, J Am Heart Assoc. 2019 May 21;8(10):e011306.

Ollivier C, Thomson A, Manolis E, Blake K, Karlsson KE, Knibbe CAJ, Pons G, Hemmings R, Commentary on the EMA Reflection Paper on the use of extrapolation in the development of medicines for paediatrics, Br J Clin Pharmacol. 2019 Feb 1. doi: 10.1111/bcp.13883. [Epub ahead of print]

Olmo CA, McGettigan P, Kurz X, Barriers and opportunities for use of patient registries in medicines regulation, Clin Pharmacol Ther. 2019 Apr 10. doi: 10.1002/cpt.1414. [Epub ahead of print]

Pearson ADJ, Scobie N, Norga K, Ligas F, Chiodin D, Burke A, Minard-Colin V, Adamson P, Marshall LV, Balakumaran A0, Benettaib B, Bhargava P, Bollard CM, Bolotin E, Bomken S, Buechner J, Burkhardt B, Caron H, Copland C, Demolis P, Egorov A, Farhan M, Zugmaier G, Gross T, Horton-Taylor D, Klapper W, Lesa G, Marcus R, Miles RR, Nottage K, Pacaud L, Ricafort R, Schrappe M, Sterba J, Vezan R, Weiner S, Kim SY, Reaman G, Vassal G, ACCELERATE and European Medicine Agency Paediatric Strategy Forum for medicinal product development for mature B-cell malignancies in children, Eur J Cancer. 2019 Feb 14;110:74-85. doi: 10.1016/j.ejca.2019.01.013. [Epub ahead of print]

Pelfrene E, Harvey Allchurch M, Ntamabyaliro N, Nambasa V, Ventura FV, Nagercoil N, et al. The European Medicines Agency’s scientific opinion on oral fexinidazole for human African trypanosomiasis. PLoS Negl Trop Dis 13(6): e0007381.

Pelfrene E, Sebris Z, Cavaleri M, Comment on Fauconnier, A. Phage Therapy Regulation: From Night to Dawn, Viruses 2019, 11, 771; doi:10.3390/v11090771

Potts J, Genov G, Segec A, Raine J, Straus S, Arlett P, Improving the Safety of Medicines in the European Union: From Signals to Action , Clin Pharmacology Ther., Dec 2019, doi: 10.1002/cpt.1678

Schneeweiss S, Rassen JA, Brown JS, Rothman KJ, Happe L, Arlett P, Dal Pan G, Goettsch W, Murk W, Wang SV., Graphical Depiction of Longitudinal Study Designs in Health Care Databases, Ann Intern Med. 2019 Mar 19;170(6):398-406. doi: 10.7326/M18-3079.

Schuessler‐Lenz M, Enzmann H, Vamvakas S, Regulators' Advice Can Make a Difference: European Medicines Agency Approval of Zynteglo for Beta Thalassemia, Clin Pharmacol Ther., Nov 2019, doi: 10.1002/cpt.1639

Selker HP, Eichler HG, Stockbridge NL, McElwee NE, Dere WH, Cohen T, Erban JK, Seyfert-Margolis VL, Honig PK, Kaitin KI, Oye KA, D'Agostino RB Sr, Efficacy and Effectiveness Too (EE2) Trials:Clinical Trial Designs to Generate Evidence on Efficacy and on Effectiveness in Wide Practice, Clin Pharmacol Ther. 2019 Jan 4. doi: 10.1002/cpt.1347. [Epub ahead of print]

Sheean ME, Malikova E, Duarte D, Capovilla G, Fregonese L, Hofer MP, Magrelli A, Mariz S, Mendez-Hermida F, Nistico R, Leest T, Sipsas NV, Tsigkos S, Vitezic D, Larsson K, Sepodes B, Stoyanova-Beninska V., Nonclinical data supporting orphan medicinal product designations in the area of rare infectious diseases., Drug Discov Today. 2019 Nov 5. pii: S1359-6446(19)30419-2. doi: 10.1016/j.drudis.2019.10.015. [Epub ahead of print]

Stephenson D, Hill D, Cedarbaum JM, Tome M, Vamvakas S, Romero K, Conrado DJ, Dexter DT, Seibyl J, Jennings D, Nicholas T, Matthews D, Xie Z, Imam S, Maguire P, Russell D, Gordon MF, Stebbins GT, Somer E, Gallagher J, Roach A, Basseches P, Grosset D, Marek K; Critical Path for Parkinson’s Consortium, The Qualification of an Enrichment Biomarker for Clinical Trials Targeting Early Stages of Parkinson's Disease, J Parkinsons Dis. 2019;9(3):553-563. doi: 10.3233/JPD-191648.

Sturkenboom M, Bahri P, Chiucchiuini A, Grove Krause T, Hahné S, Khromava A, Kokki M, Kramarz P, Kurz X, Larson HJ, de Lusignan S, Mahy P, Torcel-Pagnon L, Titievsky L, Bauchau V; ADVANCE consortium, Why we need more collaboration in Europe to enhance post-marketing surveillance of vaccines, Vaccine. 2019 Oct 31. pii: S0264-410X(19)31002-3. doi: 10.1016/j.vaccine.2019.07.081. [Epub ahead of print]

Sultana J, Zaccaria C, de Lisa R, Rossi F, Capuano A, Ferrajolo C., Good Pharmacovigilance Practice in Paediatrics: An Overview of the Updated European Medicines Agency Guidelines, Paediatr Drugs. 2019 Oct;21(5):317-321. doi: 10.1007/s40272-019-00350-w.

Tanniou J, Smid SC, van der Tweel I, Teerenstra S, Roes KCB, Level of evidence for promising subgroup findings: the case of trends and multiple subgroups, Stat Med. 2019 Mar 13. doi: 10.1002/sim.8133. [Epub ahead of print]

Tervonen T, Angelis A, Hockley K, Pignatti F, Phillips LD., Quantifying Preferences in Drug Benefit-Risk Decisions, Clin Pharmacol Ther. 2019 Nov;106(5):955-959. doi: 10.1002/cpt.1447. Epub 2019 Apr 24. PMID: 30929257

Torcel-Pagnon L, Bauchau V, Mahy P, Tin Tin Htar M, van der Sande M, Mahé C, Krause TG, Charrat A, Simondon F, Kurz X; ADVANCE Consortium. Guidance for the governance of public-private collaborations in vaccine post-marketing settings in Europe. Vaccine. 2019 May 6. pii: S0264-410X(19)30561-4. doi: 10.1016/j.vaccine.2019.04.073. [Epub ahead of print]

Tzogani K, Florez B, Markey G, Caleno M, Olimpieri OM, Melchiorri D, Hovgaard DJ, Sarac SB, Penttilä K, Lapveteläinen T, Salmonson T, Bergh J, Gisselbrecht C, Pignatti F., European Medicines Agency review of ixazomib (Ninlaro) for the treatment of adult patients with multiple myeloma who have received at least one prior therapy, ESMO Open. 2019 Sep 8;4(5):e000570. doi: 10.1136/esmoopen-2019-000570. eCollection 2019.

Vannice KS, Cassetti MC, Eisinger RW, Hombach J, Knezevic I, Marston HD, Wilder-Smith A, Cavaleri M, Krause PR, Demonstrating vaccine effectiveness during a waning epidemic: a WHO/NIH meeting report on approaches to development and licensure of Zika vaccine candidates, Vaccine. 2019 Jan 10. pii: S0264-410X(18)31713-4. doi: 10.1016/j.vaccine.2018.12.040. [Epub ahead of print]

van der Laan JW, van Malderen K, de Jager N, Duarte D, Egger GF, Lavergne F, Roque CG, Vieira I, Wiesner L, Carleer J., Evaluation of Juvenile Animal Studies for Pediatric CNS-Targeted Compounds: A Regulatory Perspective, Int J Toxicol. 2019 Nov/Dec;38(6):456-475. doi: 10.1177/1091581819883569. [Epub 2019 Oct 29.]

van Stekelenborg J, Ellenius J, Maskell S, Bergvall T, Caster O, Dasgupta N, Dietrich J, Gama S, Lewis D, Newbould V, Brosch S, Pierce CE, Powell G, Ptaszyńska-Neophytou A, Wiśniewski AFZ, Tregunno P, Norén GN, Pirmohamed M., Recommendations for the Use of Social Media in Pharmacovigilance: Lessons from IMI WEB-RADR, Drug Saf. 2019 Dec;42(12):1393-1407. doi: 10.1007/s40264-019-00858-7.

Whomsley R, Brendler-Schwaab S, Griffin E, Jensen J, Moermond C, Scholz B, Sortvik Nilssen L, Stemplewski H, Roennefahrt I., Commentary on the draft revised guideline on the environmental risk assessment of medicinal products for human use, Environmental Sciences Europe volume 31, Article number: 17 (2019)

Wolff-Holz E, Tiitso K, Vleminckx C, Weise M., Evolution of the EU Biosimilar Framework: Past and Future, BioDrugs. 2019 Dec;33(6):621-634. doi: 10.1007/s40259-019-00377-y.


Aartsma-Rus A, Mercuri E, Vroom E, Balabanov P, Meeting report of the "Regulatory Exchange Matters" session at the 5th International TREAT-NMD Conference: Lessons in communication: How an early dialogue between patients, regulators and academics can further therapy development for neuromuscular disorders Freiburg, Germany, 27-29 November 2017, Neuromuscul Disord. 2018 Jul;28(7):619-623. doi: 10.1016/j.nmd.2018.04.009. Epub 2018 Apr 20

Alteri E, Guizzaro L., Be open about drug failures to speed up research, Nature. 2018 Nov;563(7731):317-319

Amaouche N., Casaert Salome H., Collignon O., Roldao Santos M., Ziogas C., Marketing authorisation applications submitted to the European Medicines Agency by small and medium-sized enterprises: an analysis of major objections and their impact on outcomes, Drug Discovery Today, Vol 23 (10), pp. 1801-1805, October 2018

Arlett P, Rapid response to: Pandemrix vaccine: why was the public not told of early warning signs?, BMJ 2018;362:k3948

Bahri P, Rägo L; CIOMS Working Group on Vaccine Safety, CIOMS Guide To Vaccine Safety Communication - Executive summary, Vaccine. 2019 Jan 14;37(3):401-408. doi: 10.1016/j.vaccine.2018.11.082. Epub 2018 Dec 13.

Bahri P., Castillon Melero M., Listen to the public and fulfil their information interests - translating vaccine communication research findings into guidance for regulators, Br J Clin Pharmacol. 2018 Mar 24. doi: 10.1111/bcp.13587. [Epub ahead of print]

Barkholt L., Voltz-Girolt C., Raine J., Salmonson T., Schüssler-Lenz M., Regulatory watch: European regulatory experience with advanced therapy medicinal products, Nat Rev Drug Discov. 2018 Nov 30. doi: 10.1038/nrd.2018.200. [Epub ahead of print]

Bec G., Strecenski I., Castelnovo T., MAH compliance with post-authorisation obligations in the European Union: a five year review, Regulatory Rapporteur - Vol 15, No 11: 27-32

Bernabe RDLC, van Thiel GJMW, Breekveldt NS, Gispen-de Wied CC, van Delden JJM, Ethics in clinical trial regulation: ethically relevant issues from EMA inspection reports, 2008 to 2012, Curr Med Res Opin. 2018 Oct 29:1-9. doi: 10.1080/03007995.2018.1528214. [Epub ahead of print]

Bhattacharyya A, Gallo P, Crisp A, LaVange L, Molenberghs G, Pétavy F, Seltzer J, The changing landscape of data monitoring committees - Perspectives from regulators, members and sponsors, Biom J. 2018 Dec 27. doi: 10.1002/bimj.201700307. [Epub ahead of print]

Blind E., Janssen H., Dunder K., de Graeff P.A., The European Medicines Agency's approval of new medicines for type 2 diabetes, Diabetes Obes Metab, 2018 May 8. doi: 10.1111/dom.13349. [Epub ahead of print]

Borysowski J, Saxena A, Bateman-House A, Papaluca M, Różyńska J, Wnukiewicz-Kozłowska A, Górski A, Expanded access: growing importance to public health, J Epidemiol Community Health. 2018 Jul;72(7):557-558. doi: 10.1136/jech-2017-210409. Epub 2018 Apr 7.

Cerreta, F., Padrão, A., Skibicka-Stepien, I. et al., Medicines for older people. Assessment and transparency at the European Medicines Agency regarding cardiovascular and antithrombotic medicinal products, Eur Geriatr Med (2018) 9: 415.

Cilia M, Ruiz S, Richardson P, Salmonson T, Serracino-Inglott A, Wirth F, Borg JJ, Quality Issues Identified During the Evaluation of Biosimilars by the European Medicines Agency's Committee for Medicinal Products for Human Use, AAPS PharmSciTech. 2018 Feb;19(2):489-511

Collignon O, Koenig F, Koch A, Hemmings RJ, Pétavy F, Saint-Raymond A, Papaluca-Amati M, Posch M, Adaptive designs in clinical trials: from scientific advice to marketing authorisation to the European Medicine Agency, Trials. 2018 Nov 20;19(1):642. doi: 10.1186/s13063-018-3012-x

Collignon O, Pétavy F., Statistical considerations about the design and endpoints of randomized clinical trials for children with irritable bowel syndrome. Neurogastroenterol Motil. 2018 May;30(5):e13266

Day S, Jonker AH, Lau LPL, Hilgers RD, Irony I, Larsson K, Roes KC, Stallard N, Recommendations for the design of small population clinical trials, Orphanet J Rare Dis. 2018 Nov 6;13(1):195. doi: 10.1186/s13023-018-0931-2

Ecker A, Mariz S, Naumann-Winter F, Norga K, Barisic I, Girard T, Tomasi P, Mentzer D, Sepodes B, Comparative analysis of the scope of European Union paediatric investigation plans with corresponding orphan designations, Arch Dis Child. 2018 May;103(5):427-430. doi: 10.1136/archdischild-2017-313352. Epub 2017 Oct 31

Eichler HG, Barker R, Bedlington N, Bouvy JC, Broekmans AW, Bucsics A, Cerreta F, Corriol-Rohou S, Granados A, Le Cam Y, Schuurman A., The evolution of adaptiveness: balancing speed and evidence. Nat Rev Drug Discov. 2018 Jul 6. doi: 10.1038/nrd.2018.90. [Epub ahead of print]

Eichler HG, Bedlington N, Boudes M, Bouvy JC, Broekmans AW, Cerreta F, Faulkner SD, Forda S, Joos A, Le Cam Y, Mayer M, Pirard V, Corriol-Rohou S; ADAPT SMART Consortium., Medicines Adaptive Pathways to Patients: Why, when and how to engage?.Clin Pharmacol Ther. 2018 Jun 14. doi: 10.1002/cpt.1121. [Epub ahead of print]

Eichler HG, Bloechl-Daum B, Broich K, Kyrle PA, Oderkirk J, Rasi G, Santos Ivo R, Schuurman A, Senderovitz T, Slawomirski L, Wenzl M, Paris V. Data Rich, Information Poor: Can We Use Electronic Health Records to Create a Learning Healthcare System for Pharmaceuticals?, Clin Pharmacol Ther. 2019 Apr;105(4):912-922. doi: 10.1002/cpt.1226. Epub 2018 Oct 14

Eichler H.G., Sweeney F., The evolution of clinical trials: Can we address the challenges of the future? Clin Trials. 2018 Feb;15(1_suppl): 27-32.

Fregonese L, Greene L, Hofer M, Magrelli A, Naumann-Winter F, Larsson K, Sheean M, Stoyanova-Beninska V, Tsigkos S, Westermark K, Sepodes B., Demonstrating significant benefit of orphan medicines: analysis of 15 years of experience in Europe, Drug Discov Today. 2018 Jan;23(1):90-100. doi: 10.1016/j.drudis.2017.09.010.

Gaspar N, Marshall LV, Binner D, Herold R, Rousseau R, Blanc P, Capdeville R, Carleer J, Copland C, Kerloeguen Y, Norga K, Pacaud L, Sevaux MA, Spadoni C, Sterba J, Ligas F, Taube T, Uttenreuther-Fischer M, Chioato S, O'Connell MA, Geoerger B, Blay JY, Soria JC, Kaye S, Wulff B, Brugières L, Vassal G, Pearson ADJ; Members of Working Group 1 of the Paediatric Platform of ACCELERATE, 
Joint adolescent-adult early phase clinical trials to improve access to new drugs for adolescents with cancer: proposals from the multi-stakeholder platform-ACCELERATE, Ann Oncol. 2018 Mar 1;29(3):766-771. doi: 10.1093/annonc/mdy002.

Goedecke T, Morales D, Pacurariu A, Kurz X, Response to Comment on `Measuring the impact of medicines regulatory interventions – Systematic review and methodological considerations`, Br J Clin Pharmacol. 2018 Sep;84(9):2169-2170. doi: 10.1111/bcp.13664. Epub 2018 Jul 3.

Goedecke T., Morales D., Pacurariu A., Kurz X., Measuring the impact of medicines regulatory interventions – systematic review and methodological considerations, Br J Clin Pharmacol. 2018 Mar;84(3):419-433

Hofer MP, Hedman H, Mavris M, Koenig F, Vetter T, Posch M, Vamvakas S, Regnstrom J, Aarum S, Marketing authorisation of orphan medicines in Europe from 2000 to 2013, Drug Discov Today. 2018 Feb;23(2):424-433. doi: 10.1016/j.drudis.2017.10.012. Epub 2017 Oct 23.

Hwang T.J., Tomasi P.A., Bourgeois F.T., Delays in completion and results reporting of trials under the Paediatric Regulation in the European Union: cohort study, PLoS Med. 2018 Mar 1;15(3)

Hwang TJ, Tomasi PA, Saint-Raymond A, Bourgeois FT, Availability of paediatric information in European Medicines Agency approvals, Lancet Child Adolesc Health. 2018 May;2(5):e9. doi: 10.1016/S2352-4642(18)30101-9. Epub 2018 Apr 12.

Ioannou F, Burnsteel C, Mackay DKJ, Gay CG, Regulatory pathways to enable the licencing of alternatives to antibiotics, Biologicals. 2018 May;53:72-75. doi: 10.1016/j.biologicals.2018.03.003. Epub 2018 Mar 31

Kondo H, Saint-Raymond A, Yasuda N, What to Know About Medicines With New Active Ingredients Approved in FY 2016 / 2016 in Japan and EU: A Brief Comparison of New Medicines Approved in Japan and the EU in 2016, Ther Innov Regul Sci. 2018 Mar;52(2):214-219. doi: 10.1177/2168479017720248. Epub 2017 Jul 21.

Kurz X., Perez‐Gutthann S., ENCePP Steering Group, Strengthening standards, transparency, and collaboration to support medicine evaluation: Ten years of the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP), Pharmacoepidemiol Drug Saf. 2018;1-11.

Kälviäinen R., Straus S., Dogne J.M., Bakchine S., Haas M., Reducing valproate use in women with epilepsy, The Lancet Neurology , Volume 17 , Issue 7 , 580-581

Lim R, Lee DK, Sabourin P, Ferguson J, Metcalf M, Smith M, Corriol-Rohou S, Eichler HG, Lumpkin M, Hirsch G, Chen IM, O'Rourke B, Schiel A, Crabb N, Aronson N, Pezalla E, Boutin M, Binder L, Wilhelm L, Recognizing that Evidence is Made, Not Born, Clin Pharmacol Ther. 2018 Nov 25. doi: 10.1002/cpt.1317. [Epub ahead of print]

Marshall S, Madabushi R, Manolis E, Krudys K, Staab A, Dykstra K, Visser SAG, Model-informed Drug Discovery and Development (MID3): Current Industry Good Practice & Regulatory Expectations and Future Perspectives, CPT Pharmacometrics Syst Pharmacol. 2018 Nov 8. doi: 10.1002/psp4.12372. [Epub ahead of print]

Mazzaglia G., Straus S.M.J., Arlett P., da Silva D., Janssen H., Raine J., Alteri E., Study Design and Evaluation of Risk Minimization Measures: A Review of Studies Submitted to the European Medicines Agency for Cardiovascular, Endocrinology, and Metabolic Drugs, Drug Saf. 2018 Feb;41(2): 191-202

McCarthy D., Bahri P., Barnes J., Delumeau J.C., Edwards B., Harrison-Woolrych M., An Update on ISoP Special Interest Groups (SIGs), Drug Saf. 2018 Jan;41(1): 1-6

Moellenhoff K, Dette H, Kotzagiorgis E, Volgushev S, Collignon O., Regulatory assessment of drug dissolution profiles comparability via maximum deviation. Statistics in Medicine. 2018;1–14.

Morales D.R., Slattery J., Evans S., Kurz X., Antidepressant use during pregnancy and risk of autism spectrum disorder and attention deficit hyperactivity disorder: systematic review of observational studies and methodological considerations, BMC Med. 2018 Jan 15;16(1):6

Morales D.R., Slattery J., Pinheiro L., Kurz X., Hedenmalm K., Indications for Systemic Fluoroquinolone Therapy in Europe and Prevalence of Primary Care Prescribing in France, Germany and the UK: Descriptive Population Based Study, Clin Drug Investig.2018 Aug 24. doi: 10.1007/s40261-018-0684-7 [Epub ahead of print]

Morales DR, Slattery J, Pacurariu A, Pinheiro L, McGettigan P, Kurz X. Relative and Absolute Risk of Tendon Rupture with Fluoroquinolone and Concomitant Fluoroquinolone/Corticosteroid Therapy: Population-Based Nested Case-Control Study, Clin Drug Investig. 2019 Feb;39(2):205-213. doi: 10.1007/s40261-018-0729-y.

Newbould V, Le Meur S, Goedecke T, Kurz X, Correction to: Medication Errors – a characterisation of spontaneously reported cases in EudraVigilance, Drug Saf. 2018 Dec;41(12):1439-1440. doi: 10.1007/s40264-018-0700-0

Nordenmalm S., Tomasi P., Pallidis C., More medicines for children - impact of the EU Paediatric Regulation, Arch Dis Child. 2018 Feb 28. pii: archdischild-2017-313309. doi: 10.1136/archdischild-2017-313309. [Epub ahead of print]

O'Connor DJ, Sheean ME, Hofer MP, Tsigkos S, Mariz S, Fregonese L, Larsson K, Hivert V, Westermark K, Naumann-Winter F, Stoyanova-Beninska V, Barišić I, Capovilla G, Magrelli A, Sepodes B, Defining orphan conditions in the context of the European orphan regulation: challenges and evolution, Nat Rev Drug Discov. 2019 Mar 22. doi: 10.1038/nrd.2018.128. [Epub ahead of print]

O'Sullivan J, Ponzano S, Bonelli M, Safety Pharmacology Study Results and their Impact on the Design of First-in-human Trials for Authorised Oncology Therapies, Pharmaceutical Medicine, 32(5), 335-341, 2018

Ollivier C, Mulugeta YL, Ruggieri L, Saint-Raymond A, Yao L, Paediatric extrapolation: A necessary paradigm shift, Br J Clin Pharmacol. 2019 Apr;85(4):675-679. doi: 10.1111/bcp.13809. Epub 2018 Dec 18

Pacurariu A., Plueschke K., McGettigan P., et al., Electronic healthcare databases in Europe: descriptive analysis of characteristics and potential for use in medicines regulation, BMJ Open 2018;8:e023090. doi: 10.1136/bmjopen-2018-023090

Pacurariu A., Plueschke K., Olmo C.A., Kurz X., Imposed registries within the European postmarketing surveillance system: Extended analysis and lessons learned for regulators, Pharmacoepidemiol Drug Saf. 2018 May 11. doi: 10.1002/pds.4449 [Epub ahead of print]

Pacurariu AC, Hoeve CE, Arlett P, Genov G, Slattery J, Sturkenboom MCJM, Straus SMJM, Is patient exposure pre and post-approval a determinant of the timing and frequency of occurrence of safety issues?, Pharmacoepidemiol Drug Saf. 2018 Feb;27(2):168-173

Pelfrene E, Mura M, Cavaleiro Sanches A, Cavaleri M, Monoclonal antibodies targeting infectious agents: a promising future?, Clin Microbiol Infect. 2019 Jan;25(1):60-64. doi: 10.1016/j.cmi.2018.04.024. Epub 2018 Apr 30.

Peschel W, Monedero Alvarez B, Harmonised European standards as basis for the safe use of herbal medicinal products and their marketing authorisation in EU member states, Pharmaceutical Medicine; August 2018, Volume 32, Issue 4,  pp 275–293

Pinheiro LC, Candore G, Zaccaria C, Slattery J, Arlett P, An algorithm to detect unexpected increases in frequency of reports of adverse events in EudraVigilance, Pharmacoepidemiol Drug Saf. 2018 Jan;27(1):38-45

Plueschke K., McGettigan P., Pacurariu A., Kurz X., Cave A., EU-funded initiatives for real world evidence: descriptive analysis of their characteristics and relevance for regulatory decision-making, BMJ Open. 2018 Jun 14;8(6)

Pomba C, Catry B, Edo JT, Jukes H, Licensing and Approval of Antimicrobial Agents for Use in Animals, Microbiol Spectr. 2018 Aug;6(4). doi: 10.1128/microbiolspec.ARBA-0016-2017.

Postigo R., Brosch S., Slattery J., van Haren A., Dogné J.M., Kurz X., Candore G., Domergue F., Arlett P., EudraVigilance Medicines Safety Database: Publicly Accessible Data for Research and Public Health Protection, Drug Saf. 2018 Mar 9. doi: 10.1007/s40264-018-0647-1. [Epub ahead of print]

Ponzano S, Blake K, Bonelli M, Enzmann H; European Medicines Agency Committee for Human Medicinal Products “First-in-Human Guideline Drafting Group”, Promoting Safe Early Clinical Research of Novel Drug Candidates: A European Union Regulatory Perspective, Clin Pharmacol Ther. 2018 Apr;103(4):564-566;

Ponzano S, Nigrelli G, Fregonese L, Eichler I, Bertozzi F, Bandiera T, Galietta LJV, Papaluca M, A European regulatory perspective on cystic fibrosis: current treatments, trends in drug development and translational challenges for CFTR modulators, Eur Respir Rev. 2018 Apr 13;27(148). pii: 170124. doi: 10.1183/16000617.0124-2017. Print 2018 Jun 30.

Postmus D., Richard S., Bere N., van Valkenhoef G., Galinsky J., Low E., Moulon I., Mavris M., Salmonsson T., Flores B., Hillege H., Pignatti F., Individual Trade-Offs Between Possible Benefits and Risks of Cancer Treatments: Results from a Stated Preference Study with Patients with Multiple Myeloma., Oncologist. 2018 Jan;23(1):44-51

Sharma RA, Fumi L, Audisio RA, Denys A, Wood BJ, Pignatti F, Commentary: How will interventional oncology navigate the "valleys of death" for new medical devices?, Br J Radiol. 2018 Feb;91(1083):20170643

Sheean ME, Stoyanova-Beninska V, Capovilla G, Duarte D, Hofer MP, Hoffmann M, Magrelli A, Mariz S, Tsigkos S, Shaili E, Polsinelli B, Ricciardi M, Bonelli M, Balabanov P, Larsson K, Sepodes B, Nonclinical data supporting orphan medicinal product designations: lessons from rare neurological conditions, Drug Discov Today. 2018 Jan;23(1):26-48

Slattery J., Morales D., Pinheiro L., Kurz X.,Cohort Study of Psychiatric Adverse Events Following Exposure to Levonorgestrel-Containing Intrauterine Devices in UK General Practice, Drug Saf. 2018 May 21. doi: 10.1007/s40264-018-0683-x. [Epub ahead of print]

Slikker W Jr, de Souza Lima TA, Archella D, de Silva JB Junior, Barton-Maclaren T, Bo L, Buvinich D, Chaudhry Q, Chuan P, Deluyker H, Domselaar G, Freitas M, Hardy B, Eichler HG, Hugas M, Lee K, Liao CD, Loo LH, Okuda H, Orisakwe OE, Patri A, Sactitono C, Shi L, Silva P, Sistare F, Thakkar S, Tong W, Valdez ML, Whelan M, Zhao-Wong A, Emerging technologies for food and drug safety, Regul Toxicol Pharmacol. 2018 Oct;98:115-128. doi: 10.1016/j.yrtph.2018.07.013. Epub 2018 Jul 23.

Smith LEH, Hellström A, Stahl A, Fielder A, Chambers W, Moseley J, Toth C, Wallace D, Darlow BA, Aranda JV, Hallberg B, Davis JM; Retinopathy of Prematurity Workgroup of the International Neonatal Consortium, Development of a Retinopathy of Prematurity Activity Scale and Clinical Outcome Measures for Use in Clinical Trials, JAMA Ophthalmol. 2018 Dec 13. doi: 10.1001/jamaophthalmol.2018.5984. [Epub ahead of print]

Smith M.Y., Russell A., Bahri P., Mol P.G.M., Frise S., Freeman E., Morrato E.H., The RIMES Statement: A Checklist to Assess the Quality of Studies Evaluating Risk Minimization Programs for Medicinal Products, Drug Saf. 2018 Apr;41(4):389-4

Suarez-Sharp S, Cohen M, Kesisoglou F, Abend A, Marroum P, Delvadia P, Kotzagiorgis E, Li M, Nordmark A, Bandi N, Sjögren E, Babiskin A, Heimbach T, Kijima S, Mandula H, Raines K, Seo P, Zhang X, Applications of clinically relevant dissolution testing:  workshop summary report, AAPS J. 2018 Aug 27;20(6):93. doi: 10.1208/s12248-018-0252-3

Sullivan JO, Blake K, Berntgen M, Salmonson T, Welink J; Pharmacokinetics Working Party, Overview of the European Medicines Agency's Development of Product-Specific Bioequivalence Guidelines, Clin Pharmacol Ther. 2018 Sep;104(3):539-545. doi: 10.1002/cpt.957. Epub 2018 Jan 9.

Tacconelli E, Carrara E, Savoldi A, Harbarth S, Mendelson M, Monnet DL, Pulcini C, Kahlmeter G, Kluytmans J, Carmeli Y, Ouellette M, Outterson K, Patel J, Cavaleri M, Cox EM, Houchens CR, Grayson ML, Hansen P, Singh N, Theuretzbacher U, Magrini N; WHO Pathogens Priority List Working Group, Discovery, research, and development of new antibiotics: the WHO priority list of antibiotic-resistant bacteria and tuberculosis, Lancet Infect Dis. 2018 Mar;18(3):318-327

Tafuri G., Lucas I., Estevão S., Moseley J., d'Andon A., Bruehl H., Gajraj E., Garcia S., Hedberg N., Massari M., Molina A., Obach M., Osipenko L., Petavy F., Petschulies M., Pontes C., Russo P., Schiel A., Van de Casteele M., Zebedin-Brandl E.M., Rasi G., Vamvakas S., The impact of parallel regulatory-HTA scientific advice on clinical development. Assessing the uptake of regulatory and HTA recommendations, Br J Clin Pharmacol. 2018 Jan 25. doi: 10.1111/bcp.13524.

Thanarajasingam G, Minasian LM, Baron F, Cavalli F, De Claro RA, Dueck AC, El-Galaly TC, Everest N, Geissler J, Gisselbrecht C, Gribben J, Horowitz M, Ivy SP, Jacobson CA, Keating A, Kluetz PG, Krauss A, Kwong YL, Little RF, Mahon FX, Matasar MJ, Mateos MV, McCullough K, Miller RS, Mohty M, Moreau P, Morton LM, Nagai S, Rule S, Sloan J, Sonneveld P, Thompson CA, Tzogani K, van Leeuwen FE, Velikova G, Villa D, Wingard JR, Wintrich S, Seymour JF, Habermann TM, Beyond maximum grade: modernising the assessment and reporting of adverse events in haematological malignancies, Lancet Haematol. 2018 Nov;5(11):e563-e598. doi: 10.1016/S2352-3026(18)30051-6. Epub 2018 Jun 18.

Tsigkos S, Hofer MP, Sheean ME, Mariz S, Larsson K, Naumann-Winter F, Fregonese L, Sepodes B, Establishing rarity in the context of orphan medicinal product designation in the European Union, Drug Discov Today. 2018 Mar;23(3):681-686

Tzogani K, Penninga E, Schougaard Christiansen ML, Hovgaard D, Sarac SB, Camarero Jimenez J, Garcia I, Lafuente M, Sancho-López A, Salmonson T, Gisselbrecht C, Pignatti F, EMA Review of Daratumumab for the Treatment of Adult Patients with Multiple Myeloma, Oncologist. 2018 May;23(5):594-602. doi: 10.1634/theoncologist.2017-0328. Epub 2018 Jan 25

Tzogani K, van Hennik P, Walsh I, De Graeff P, Folin A, Sjöberg J, Salmonson T, Bergh J, Laane E, Ludwig H, Gisselbrecht C, Pignatti F, The European Medicines Agency Review of Panobinostat (Farydak) for the Treatment of Adult Patients with Relapsed and/or Refractory Multiple Myeloma, Oncologist. 2018 May;23(5):631-636. doi: 10.1634/theoncologist.2017-0301. Epub 2017 Nov 30. Erratum in: Oncologist. 2018 Jul;23(7):870.

van Gerven J, Bonelli M, Commentary on the EMA Guideline on strategies to identify and mitigate risks for first-in-human and early clinical trials with investigational medicinal products, Br J Clin Pharmacol. 2018 Jul;84(7):1401-1409. doi: 10.1111/bcp.13550. Epub 2018 May 30

Vannice KS, Wilder-Smith A, Barrett ADT, Carrijo K, Cavaleri M, de Silva A, Durbin AP, Endy T, Harris E, Innis BL, Katzelnick LC, Smith PG, Sun W, Thomas SJ, Hombach J, Clinical development and regulatory points for consideration for second-generation live attenuated dengue vaccines, Vaccine. 2018 Jun 7;36(24):3411-3417. doi: 10.1016/j.vaccine.2018.02.062. Epub 2018 Mar 7

Vatzaki E., Straus S., Dogne H.M., Garcia Burgos J., Girard T., Martelletti P., Latest clinical recommendations on valproate use for migraine prophylaxis in women of childbearing age: overview from European Medicines Agency and European Headache Federation, The Journal of Headache and Pain (2018) 19:68

Vermeer N, Giezen T, Zastavnik S, Wolff-Holz E, Hidalgo-Simon A, Identifiability of biologicals in ADR reports received from European clinical practice, Clin Pharmacol Ther. 2018 Nov 21. doi: 10.1002/cpt.1310. [Epub ahead of print]


Aartsma-Rus A., Balabanov P., Binetti L., Haas M., Haberkamp M., Mitchell J., Mário M.R., Muntoni F., Finkel R., Mercuri E., Stakeholder collaboration for spinal muscular atrophy therapy development, The Lancet Neurology , Vol 16(4), p. 264

Aartsma-Rus A., Straub V., Hemmings R., Haas M., Schlosser-Weber G., Stoyanova-Beninska V., Mercuri E., Muntoni F., Sepodes B., Vroom E., Balabanov P., Development of exon skipping therapies for Duchenne muscular dystrophy: a critical review and a perspective on the outstanding issues, Nucleic Acid Ther. 2017 Aug 10. doi: 10.1089/nat.2017.0682. [Epub ahead of print]

Aisen P, Touchon J, Amariglio R, Andrieu S, Bateman R, Breitner J, Donohue M, Dunn B, Doody R, Fox N, Gauthier S, Grundman M, Hendrix S,Ho C, Isaac M, Raman R, Rosenberg P, Schindler R, Schneider L, Sperling R, Tariot P, Welsh-Bohmer K, Weiner M, Vellas B, EU/US/CTAD Task Force: Lessons Learned from Recent and Current Alzheimer's PreventionTrials, J Prev Alzheimers Dis. 2017;4(2):116-124

Anton R., Haas M., Arlett P., Weise M., Balabanov P., Mazzaglia G., Prieto L., Keller-Stanislawski B., Raine J., Drug-induced progressive multifocal leukoencephalopathy in multiple sclerosis: European regulators' perspective, Clin Pharmacol Ther. 2016 Dec 21. doi: 10.1002/cpt.604

Bahri P., Fogd J., Morales D., Kurz X., ADVANCE consortium, Application of real-time global media monitoring and 'derived questions' for enhancing communication by regulatory bodies: the case of human papillomavirus vaccines, BMC Med. Vol. 15(1), p. 91, doi: 10.1186/s12916-017-0850-4.

Banovac M, Candore G, Slattery J, Houÿez F, Haerry D, Genov G, Arlett P, Patient reporting in the EU: Analysis of EudraVigilance data, Drug Saf. 2017 Jul;40(7):629-645

Berger M.L., Sox H., Willke R.J., Brixner D.L., Eichler H.G., Goettsch W., Madigan D., Makady A., Schneeweiss S., Tarricone R., Wang S.V., Watkins J., Daniel Mullins C., Good practices for real-world data studies of treatment and/or comparative effectiveness: Recommendations from the joint ISPOR-ISPE Special Task Force on real-world evidence in health care decision making, Pharmacoepidemiol Drug Saf. 2017 Sep;26(9):1033-1039. doi: 10.1002/pds.4297.

Bonini S., Rasi G., Inhibitor of Fatty Acid Amide Hydrolase - Learning from Tragic Failures, N Engl J Med., Vol. 376(4):394.

Bonini S, Bonini M, Biosimilars and drug development in allergic and immunologic diseases, J Allergy Clin Immunol. 2017 May;139(5):1461-1464. doi: 10.1016/j.jaci.2017.03.008. Epub 2017 Mar 27

Boráň T, Menezes-Ferreira M, Reischl I, Celis P, Ferry N, Gänsbacher B, Krafft H, Lipucci di Paola M, Sladowski D, Salmikangas P, Clinical Development and Commercialization of Advanced Therapy Medicinal Products (ATMPs) in EU: how are the product pipeline and regulatory framework evolving?, Hum Gene Ther Clin Dev. 2017 Sep;28(3):126-135

Bouvy J.C., Blake K., Slattery J., De Bruin M.L., Arlett P., Kurz X., Registries in European post-marketing surveillance: a retrospective analysis of centrally approved products, 2005-2013, Pharmacoepidemiol Drug Saf. 2017 Mar 26.

Bueno H., de Graeff P., Richard-Lordereau I., Emmerich J., Fox K. A., Friedman C. P., Gaudin C., El-Gazayerly A., Goldman S., Hemmrich M., Henderson R. A., Himmelmann A., Irs A., Jackson N., James S. K., Katus H. A., Laslop A., Laws I., Mehran R., Ong S., Prasad K., Roffi M., Rosano G. M., Rose M., Sinnaeve P. R., Stough W. G., Thygesen K., Van de Werf F., Varin C., Verheugt F. W., de Los Angeles Alonso García M., Report of the European Society of Cardiology Cardiovascular Round Table regulatory workshop update of the evaluation of new agents for the treatment of acute coronary syndrome: Executive summary, Eur Heart J Acute Cardiovasc Care, pii: 2048872616649859

Cole S, Hay JL, Luzon E, Nordmark A, Skottheim Rusten I., European regulatory perspective on pediatric physiologically based pharmacokinetic models, Int J of Pharmacokinetics. May 2017, doi 10.4155/ipk-2016-0025

Collignon O., Veselý R., Should baseline-dependent cut-offs really be used to define disease improvement in juvenile idiopathic arthritis? And few other considerations, Rheumatology (Oxford), Vol. 56(1), pp.165-167.

Collignon O., Veselý R., Statistical considerations in the development of clinical predictive scores: comment on the article by Domsic et al, Arthritis Rheumatol., Vol. 69(1), pp. 241-242. doi: 10.1002/art.39854

Cowie MR, Filippatos GS, Alonso Garcia MLA, Anker SD, Baczynska A, Bloomfield DM, Borentain M, Bruins Slot K, Cronin M, Doevendans PA, El-Gazayerly A, Gimpelewicz C, Honarpour N, Janmohamed S, Janssen H, Kim AM, Lautsch D, Laws I, Lefkowitz M, Lopez-Sendon J, Lyon AR, Malik FI, McMurray JJV, Metra M, Figueroa Perez S, Pfeffer MA, Pocock SJ, Ponikowski P, Prasad K, Richard-Lordereau I, Roessig L, Rosano GMC, Sherman W, Stough WG, Swedberg K, Tyl B, Zannad F, Boulton C, De Graeff P, New medicinal products for chronic heart failure: advances in clinical trial design and efficacy assessment, Eur J Heart Fail. 2017 Jun;19(6):718-727

Davis JM, Baer GR, Portman R, Nelson R, Storari L, Aranda JV, Bax R, Zajicek A, Klein A, Turner M, Baygani S, Thomson M, Allegaert K; International Neonatal Consortium, Enrollment of Neonates in More Than One Clinical Trial, Clin Ther. 2017 Oct;39(10):1959-1969

De Briyne N., Gopal R., Diesel G., Iatridou D., O'Rourke D., Veterinary pharmacovigilance in Europe: a survey of veterinary practitioners, Veterinary Record Open, Vol. 4, Issue 1: e000224. doi: 10.1136/vetreco-2017-000224.

Dunder K, de Graeff PA, Blind E, Comment on the editorial by Turner et al. on Assessment of cardiovascular risk of new drugs for the treatment of diabetes mellitus: risk assessment versus risk aversion by Zannad et al, Eur Heart J Cardiovasc Pharmacother. 2017 Jul 1;3(3):129

Eichler I, Manolis E, Chest Imaging in CF Studies  -  Commentary, J Cyst Fibros. 2017 Mar;16(2):173-174

Farkas AM, Mariz S, Stoyanova-Beninska V, Celis P, Vamvakas S, Larsson K, Sepodes B, Advanced Therapy Medicinal Products for Rare Diseases: State of Play of Incentives Supporting Development in Europe, Front Med (Lausanne). 2017 May 16;4:53

Grimaldi-Bensouda L, Nordon C, Rossignol M, Jardon V, Boss V, Warembourg F, Reynolds R, Kurz X, Rouillon F, Abenhaim L; PROTECT-WP6 study group, Antiepileptic drugs and risk of suicide attempts: a case-control study exploring the impact of underlying medical conditions, Pharmacoepidemiol Drug Saf. 2017 Mar;26(3):239-247

Goedecke T., Ord K., Newbould V., Brosch S., Arlett P., Medication Errors: New EU Good Practice Guide on Risk Minimisation and Error Prevention, Drug Saf. 2017 Dec;40(12):1241-1248

Heininger U, Holm K, Caplanusi I, Bailey SR; CIOMS Working Group on Vaccine Safety, Guide to active vaccine safety surveillance: Report of CIOMS working group on vaccine safety - executive summary, Vaccine. 2017 Jul 13;35(32):3917-3921

Isaac MB, Vamvakas S, Swings and Roundabouts in CNS Drug Biomarkers, J Prev Alzheimers Dis. 2017;4(3):134-135

Isaac MT, Vamvakas S, Isaac MB, Diagnostic biomarkers for Alzheimer's disease: a regulatory view, Lancet Neurol. 2017 Aug;16(8):580-581

Jonsson B, Martinalbo J, Pignatti F, European Medicines Agency Perspective on Oncology Study Design for Marketing Authorization and Beyond, Clin Pharmacol Ther. 2017 May;101(5):577-579

Kollb-Sielecka M, Demolis P., Emmerich J., Markey G., Salmonson T., Haas M., The European Medicines Agency review of pitolisant for treatment of narcolepsy: summary of the scientific assessment by the Committee for Medicinal Products for Human Use, sleepmedicine, Vol 33, pp. 125-129

Kurz X., Advancing regulatory science, advancing regulatory practice, Pharmacoepidemiol Drug Saf., doi: 10.1002/pds.4181.

Kurz X, Bauchau V, Mahy P, Glismann S, van der Aa LM, Simondon F; ADVANCE consortium, The ADVANCE Code of Conduct for Collaborative Vaccine Studies, Vaccine. 2017 Apr 4;35(15):1844-1855

Landray MJ, Bax JJ, Alliot L, Buyse M, Cohen A, Collins R, Hindricks G, James SK, Lane S, Maggioni AP, Meeker-O'Connell A, Olsson G, Pocock SJ, Rawlins M, Sellors J, Shinagawa K, Sipido KR, Smeeth L, Stephens R, Stewart MW, Stough WG, Sweeney F, Van de Werf F, Woods K, Casadei B, Improving public health by improving clinical trial guidelines and their application, Eur Heart J. 2017 Jun 1;38(21):1632-1637. doi: 10.1093/eurheartj/ehx086

Lombardi D, Squires L, Sjostedt P, Thompson C, Turner M, Eichler I, Industry and Patient Perspectives on Child Participation in Clinical Trials. The Pediatric Assent Initiative Survey Report, Therapeutic Innovation& Regulatory Science 2017, Vol. 52 (1) 29-37

Luigetti R, Bachmann P, Cooke E, Salmonson T, Collaboration not Competition: Developing New Reliance Models, WHO Drug Information Vol. 30, No. 4, 2016

Manolis E, Brogren J, Cole S, Hay JL, Nordmark A, Karlsson KE, Lentz F, Benda N, Wangorsch G, Pons G, Zhao W, Gigante V, Serone F, Standing JF, Dokoumetzidis A, Vakkilainen J, van den Heuvel M, Mangas Sanjuan V, Taminiau J, Kerwash E, Khan D, Musuamba FT, Skottheim Rusten I, EMA Modelling and Simulation Working Group, Commentary on the MID3 Good Practices Paper, CPT Pharmacometrics Syst Pharmacol. 2017 Jul;6(7):416-417. doi: 10.1002/psp4.12223. Epub 2017 Jul 22

Medem A.V., Seidling H.M., Eichler H.G., Kaltschmidt J., Metzner M., Hubert C.M., Czock D., Haefeli W.E., Definition of variables required for comprehensive description of drug dosage and clinicalpharmacokinetics, Eur J Clin Pharmacol. Eur J Clin Pharmacol.Vol 73(5), pp. 633-641

Moreno L, Caron H, Geoerger B, Eggert A, Schleiermacher G, Brock P, Valteau-Couanet D, Chesler L, Schulte JH, De Preter K, Molenaar J, Schramm A, Eilers M, Van Maerken T, Johnsen JI, Garrett M, George SL, Tweddle DA, Kogner P, Berthold F, Koster J, Barone G, Tucker ER, Marshall L, Herold R, Sterba J, Norga K, Vassal G, Pearson AD, Accelerating drug development for neuroblastoma - New Drug Development Strategy: an Innovative Therapies for Children with Cancer, European Network for Cancer Research in Children and Adolescents and International Society of Paediatric Oncology Europe Neuroblastoma project, Expert Opin Drug Discov. 2017 Aug;12(8):801-811

Musuamba, Manolis E, Holford N, Cheung S, Friberg LE, Ogungbenro K, Posch M, Yates J, Berry S, Thomas N, Corriol-Rohou S, Bornkamp B, Bretz F, Hooker AC, Van der Graaf PH, Standing JF, Hay J, Cole S, Gigante V, Karlsson K, Dumortier T, Benda N, Serone F, Das S, Brochot A, Ehmann F, Hemmings R, Rusten IS, Advanced Methods for Dose and Regimen Finding During Drug Development: Summary of the EMA/EFPIA Workshop on Dose Finding (London 4-5 December 2014), CPT Pharmacometrics Syst Pharmacol. 2017 Jul;6(7):418-429. doi: 10.1002/psp4.12196. Epub 2017 Jul 19

Newbould V, Le Meur S, Goedecke T, Kurz X., Medication Errors: A Characterisation of Spontaneously Reported Cases in EudraVigilance, Drug Saf. 2017 Jul 11. doi: 10.1007/s40264-017-0569-3

Newbould V, Le Meur S, Goedecke T, Kurz X, Correction to: Medication Errors – a characterisation of spontaneously reported cases in EudraVigilance, Drug Saf. 2017 Dec;40(12):1293. doi: 10.1007/s40264-017-0609-z

Ohmann C, Banzi R, Canham S, Battaglia S, Matei M, Ariyo C, Becnel L, Bierer B, Bowers S, Clivio L, Dias M, Druml C, Faure H, Fenner M, Galvez J, Ghersi D, Gluud C, Groves T, Houston P, Karam G, Kalra D, Knowles RL, Krleža-Jerić K, Kubiak C, Kuchinke W, Kush R, Lukkarinen A, Marques PS, Newbigging A, O'Callaghan J, Ravaud P, Schlünder I, Shanahan D, Sitter H, Spalding D, Tudur-Smith C, van Reusel P, van Veen EB, Visser GR, Wilson J, Demotes-Mainard J, Sharing and reuse of individual participant data from clinical trials: principles and recommendations, BMJ Open. 2017 Dec 14;7(12)

Pauwels K., Huys I., Casteels M., Larsson K., Voltz C., Penttila K., More T., Simoens S., Are products with an orphan designation for oncology indications different from products for other rare indications? A retrospective analysis of European orphan designations granted between 2002-2012, Orphanet Journal of Rare Diseases, doi: 10.1186/s13023-017-0578-4

Penkov D, Tomasi P, Eichler I, Murphy D, Yao LP, Temeck J, Pediatric Medicine Development: An Overview and Comparison of Regulatory Processes in the European Union and United States, Therapeutic Innovation & Regulatory Science 2017, Vol. 51(3) 360-371

Polsinelli B, Tsigkos S, Naumann-Winter F, Mariz S, Sepodes B, Evolving prevalence of haematological malignancies in orphan designation procedures in the European Union, Orphanet J Rare Dis. 2017 Jan 21;12(1):17

Pomba C., Rantala M., Greko C., Baptiste K. E., Catry B., van Duijkeren E., Mateus A., Moreno M. A., Pyörälä S., Ružauskas M., Sanders P., Teale C., Threlfall E. J., Kunsagi Z., Torren-Edo J., Jukes H., Törneke K., Public health risk of antimicrobial resistance transfer from companion animals, J Antimicrob Chemother, Vol. 72(4), pp. 957-968, doi: 10.1093/jac/dkw481

Raimi-Abraham BT, de Orbe Izquierdo MS, Collignon O, Cerreta F, Regulatory considerations on the enrollment of older adults in oncology clinical trials, J Geriatr Oncol. 2017 May; 8(3):151-153. doi: 10.1016/j.jgo.2017.02.006, Epub 2017 Mar 9

Rohner E, Grabik M, Tonia T, Jüni P, Pétavy F, Pignatti F, Bohlius J, Does access to clinical study reports from the European Medicines Agency reduce reporting biases? A systematic review and meta-analysis of randomized controlled trials on the effect of erythropoiesis-stimulating agents in cancer patients, PLoS One. 2017 Dec 11;12(12)

Salmonson T., Dogné J. M., Janssen H., Garcia Burgos J., Blake P., Non-vitamin-K oral anticoagulants and laboratory testing: now and in the future: Views from a workshop at the European Medicines Agency (EMA), Eur Heart J Cardiovasc Pharmacother., Vol. 3(1), pp. 42–47.

Santoro A, Genov G, Spooner A, Raine J, Arlett P., Promoting and Protecting Public Health: How the European Union Pharmacovigilance System Works, Drug Saf. 2017 Jul 22. doi: 10.1007/s40264-017-0572-8

Stanel SC, Sjöberg J, Salmonson T, Foggi P, Caleno M, Melchiorri D, Gravanis I, Tzogani K, Pignatti F, European Medicines Agency Approval Summary: Zaltrap For The Treatment Of Patients With Oxaliplatin-Resistant Metastatic Colorectal Cancer, ESMO Open. 2017 May 2;2(2):e000190

Stefanska A.M., Distlerová D., Musaus J., Olski T.M., Dunder K., Salmonson T., Mentzer D., Müller-Berghaus J., Hemmings R., Veselý R., Extrapolation in the development of paediatric medicines: examples from approvals for biological treatments for paediatric chronic immune-mediated inflammatory diseases, Arch Dis Child. 2017 May 29. pii: archdischild-2016-312259. doi: 10.1136/archdischild-2016-312259

Tomasi P.A., Egger G.F., Saint-Raymond A, Enabling development of paediatric medicines in Europe: 10 years of the EU Paediatric Regulation, Paediatr Drugs. 2017 Sep 12. doi: 10.1007/s40272-017-0261-1.

Tzogani K, Camarero Jiménez J, Garcia I, Sancho-López A, Martin M, Moreau A, Demolis P, Salmonson T, Bergh J, Laane E, Ludwig H, Gisselbrecht C, Pignatti F, The European Medicines Agency review of carfilzomib (Kyprolis) for the treatment of adult patients with multiple myeloma who have received at least one prior therapy, Oncologist. 2017 Nov;22(11):1339-1346

Veselý R., Richardson P., The switch to infliximab biosimilars, The Lancet, doi:

Wickström K., Moseley J., Biomarkers and Surrogate Endpoints in Drug Development: A European Regulatory View, Invest Ophthalmol Vis Sci, Vol.58 (6), BIO27-BIO33.


Andrews EB, Arellano FM, Avorn J, Bortnichak EA, Chen R, Dai WS, de Abajo FJ, Dieck GS, de Vries C, Edlavitch S, Freiman J, Hallas J, Jones J, Koo L, Kaufman DW, Kurz X, Lanes S, Mitchell AA, Moride Y, Nelson RC, Neutel I, Park BJ, Perez-Gutthann S, Reynolds R, Sacks S, Santanello N, Stang P, Stergachis A, Strom BL, Stürmer T, Toh D, Trontell A, Walker AM, Waller P, Watson DJ, West S, Wilcock K, Wise RP, Guidelines for good pharmacoepidemiology practice (GPP), Pharmacoepidemiol Drug Saf. 2016 Jan;25(1):2-10. doi: 10.1002/pds.3891. Epub 2015 Nov 5.

Andrews EB, Arellano FM, Avorn J, Bortnichak EA, Chen R, Dai WS, de Abajo FJ, Dieck GS, de Vries C, Edlavitch S, Freiman J, Hallas J, Jones J, Koo L, Kaufman DW, Kurz X, Lanes S, Mitchell AA, Moride Y, Nelson RC, Neutel I, Park BJ, Perez-Gutthann S, Reynolds R, Sacks S, Santanello N, Stang P, Stergachis A, Strom BL, Stürmer T, Toh D, Trontell A, Walker AM, Waller P, Watson DJ, West S, Wilcock K, Wise RP, Guidelines for good pharmacoepidemiology practice (GPP), Pharmacoepidemiol Drug Saf. 2016 Jan;25(1):2-10. doi: 10.1002/pds.3891. Epub 2015 Nov 5.

Blake KV, Saint-Raymond A, Zaccaria C, Domergue F, Pelle B, Slattery J, Enhanced Paediatric Pharmacovigilance at the European Medicines Agency: A Novel Query Applied to Adverse Drug Reaction Reports, Paediatr Drugs. 2016 Feb;18(1):55-63

Bonini S., Rasi G., First-in-Human Clinical Trials - What We Can Learn from Tragic Failures, N Engl J Med. 2016 Nov 3;375(18):1788-1789.

Brauer R, Ruigómez A, Downey G, Bate A, Garcia Rodriguez LA, Huerta C, Gil M,de Abajo F, Requena G, Alvarez Y, Slattery J, de Groot M, Souverein P, Hesse U,Rottenkolber M, Schmiedl S, de Vries F, Tepie MF, Schlienger R, Smeeth L, Douglas I, Reynolds R, Klungel O, Prevalence of antibiotic use: a comparison across various European health care data sources, Pharmacoepidemiol Drug Saf. 2016 Mar;25 Suppl 1:11-20

Breckenridge A, Eichler HG, Jarow JP, Precision medicine and the changing role of regulatory agencies, Nat Rev Drug Discov. 2016 Dec;15(12):805-806

Butlen-Ducuing F., Pétavy F., Guizzaro L., Zienowicz m., Haas M, Alteri E., Salmonson T., Corruble E., Regulatory watch: Challenges in drug development for central nervous system disorders: a European Medicines Agency perspective, Nat Rev Drug Discov., doi:10.1038/nrd.2016.237.

Cavaleri M, Thomson A, Salmonson T, Hemmings RJ, A viewpoint on European Medicines Agency experience with investigational medicinal products for Ebola, Clin Trials. 2016 Feb;13(1):101-4

Cerreta F., Ankri J., Bowen D., Cherubini A., Cruz Jentoft A.J., Guðmundsson A., Haberkamp M., Jansen P., Marchionni N., Morgan S., Rönnemaa E., Petrovic M., Pilotto A., Rosa M., Wildiers H., Baseline frailty evaluation in drug development, J Frailty Aging., vol. 5(3), pp.139-140.
Ciani O., Buyse M., Drummond M., Rasi G., Saad E. D., Taylor R. S., Use of surrogate end points in healthcare policy: a proposal for adoption of a validation framework, Nat Rev Drug Discov. Vol 15(7), p. 516

Cook G, France G, Holte Ø, Lorenti G, Tainsh D, Summary of the EMA Joint Regulators/Industry QbD workshop (London, UK; 28-29 January 2014), PDA J Pharm Sci Technol. 2016 Mar-Apr;70(2):163-76

Cox E., Cavaleri M., Eichler H.G., Woodcock J., Borio L., Facilitating Antibacterial Drug Development in a Time of Great Need, Clin Infect Dis. 63(suppl. 2), pp. S27-S28., doi: 10.1093/cid/ciw257

de Groot MC, Candore G, Uddin MJ, Souverein PC, Ali MS, Belitser SV, Huerta C, Groenwold RH, Alvarez Y, Slattery J, Korevaar J, Hoes AW, Roes KC, de Boer A, Douglas IJ, Schlienger RG, Reynolds R, Klungel OH, Gardarsdottir H, Case-only designs for studying the association of antidepressants and hip or femur fracture, Pharmacoepidemiol Drug Saf. 2016 Mar;25 Suppl 1:103-13

Donnelly H., Alemayehu D., Botgros R., Comic-Savic S., Eisenstein B., Lorenz B., Merchant K., Pelfrene E., Reith C., Santiago J., Tiernan R., Wunderink R., Tenaerts P., Knirsch C., Streamlining Safety Data Collection in Hospital-Acquired Bacterial Pneumonia and Ventilator-Associated Bacterial Pneumonia Trials: Recommendations of the Clinical Trials Transformation Initiative Antibacterial Drug Development Project Team, Clin Infect Dis. Vol. 63(suppl. 2), pp. S39-45., doi: 10.1093/cid/ciw316.

Donnelly H, Alemayehu D, Botgros R, Comic-Savic S, Eisenstein B, Lorenz B, Merchant K, Pelfrene E, Reith C, Santiago J, Tiernan R, Wunderink R, Tenaerts P, Knirsch C, Streamlining Safety Data Collection in Hospital-Acquired Bacterial Pneumonia and Ventilator-Associated Bacterial Pneumonia Trials: Recommendations of the Clinical Trials Transformation Initiative Antibacterial Drug Development Project Team, Clin Infect Dis. 2016 Aug 15;63 Suppl 2:S39-45.

Ehmann F, Pita R, The EU is ready for non-biological complex medicinal products, Generics and Biosimilars Initiative Journal (GaBI Journal). 2016;5(1):30-5

Eichler H.G., Hurts H., Broich K., Rasi G., Drug Regulation and Pricing - Can Regulators Influence Affordability?, N Engl J Med, Vol. 374, pp. 1807-1809. doi: 10.1056/NEJMp1601294.

Eichler HG, Bloechl-Daum B, Bauer P, Bretz F, Brown J, Hampson LV, Honig P, Krams M, Leufkens H, Lim R, Lumpkin MM, Murphy MJ, Pignatti F, Posch M, Schneeweiss S, Trusheim M, Koenig F, "Threshold-crossing": A Useful Way to Establish the Counterfactual in Clinical Trials?, Clin Pharmacol Ther. 2016 Dec;100(6):699-712.

Egger G.F., Wharton G.T., Malli S., Temeck J., Murphy M.D., Tomasi P., A Comparative Review of Waivers Granted in Pediatric Drug Development by FDA and EMA from 2007-2013, Therapeutic Innovation & Regulatory Science, doi: 10.1177/2168479016646809

Gadroen K, Straus SM, Pacurariu A, Weibel D, Kurz X, Sturkenboom MC, Patterns of spontaneous reports on narcolepsy following administration of pandemic influenza vaccine; a case series of individual case safety reports in Eudravigilance, Vaccine. 2016 Sep 22;34(41):4892-7

Garcia-Larsen V., Del Giacco S.R., Moreira A., Bonini M., Haahtela T., Bonini S., Carlsen K.H., Agache I., Fonseca J., Papadopoulos N.G., Delgado L., Dietary intake and risk of asthma in children and adults: protocol for a systematic review and meta-analysis, Clin Transl Allergy. 2016 Apr 28;6:17. doi: 10.1186/ s13601-016-0106-y.

Grimaldi-Bensouda L, Klungel O, Kurz X, de Groot MC, Maciel Afonso AS, de Bruin ML, Reynolds R, Rossignol M, Calcium channel blockers and cancer risk: an analysis using the UK Clinical Practice Research Datalink (CPRD), BMJ Open. 2016 Jan 8;6(1):e009147.

Gupta A., Mathad J. S., Abdel-Rahman S. M., Albano J. D., Botgros R., Brown V., Browning R. S., Dawson L., Dooley K. E., Gnanashanmugam D., Grinsztejn B., Hernandez-Diaz S., Jean-Philippe P., Kim P., Lyerly A. D., Mirochnick M., Mofenson L. M., Montepiedra G., Piper J., Sahin L., Savic R., Smith B., Spiegel H., Swaminathan S., Watts D. H., White A., Toward Earlier Inclusion of Pregnant and Postpartum Women in Tuberculosis Drug Trials: Consensus Statements From an International Expert Panel, Clin Infect Dis., doi: 10.1093/cid/civ991.
Gravanis I., Tzogani K., van Hennik P., de Graeff P., Schmitt P., Mueller-Berghaus J., Salmonson T., Gisselbrecht C., Laane E., Bergmann L., Pignatti F., The European Medicines Agency review of brentuximab vedotin (Adcetris) for the treatment of adult patients with relapsed or refractory CD30+ Hodgkin lymphoma or systemic anaplastic large cell lymphoma, Oncologist, Vol. 21(1), pp. 102-9.

Hallgreen CE, Mt-Isa S, Lieftucht A, Phillips LD, Hughes D, Talbot S, Asiimwe A, Downey G, Genov G, Hermann R, Noel R, Peters R, Micaleff A, Tzoulaki I, Ashby D; PROTECT Benefit-Risk group, Literature review of visual representation of the results of benefit-risk assessments of medicinal products, Pharmacoepidemiol Drug Saf. 2016 Mar;25(3):238-50

Healy MJ, Tong W, Ostroff S, Eichler HG, Patak A, Neuspiel M, Deluyker H, Slikker W Jr, Regulatory bioinformatics for food and drug safety, Regul Toxicol Pharmacol. 2016 Oct;80:342-7

Huerta C, Abbing-Karahagopian V, Requena G, Oliva B, Alvarez Y, Gardarsdottir H, Miret M, Schneider C, Gil M, Souverein PC, De Bruin ML, Slattery J, De Groot MC, Hesse U, Rottenkolber M, Schmiedl S, Montero D, Bate A, Ruigomez A, García-Rodríguez LA, Johansson S, de Vries F, Schlienger RG, Reynolds RF, Klungel OH, de Abajo FJ, Exposure to benzodiazepines (anxiolytics, hypnotics and related drugs) in seven European electronic healthcare databases: a cross-national descriptive study from the PROTECT-EU Project, Pharmacoepidemiol Drug Saf. 2016 Mar;25 Suppl 1:56-65.

Jutel M., Agache I., Bonini S., Burks A.W., Calderon M., Canonica W., Cox L., Demoly P., Frew A.J., O'Hehir R., Kleine-Tebbe J., Muraro A., Lack G., Larenas D., Levin M., Martin B.L., Nelson H., Pawankar R., Pfaar O., van Ree R., Sampson H., Sublett J.L., Sugita K., Du Toit G., Werfel T., Gerth van Wijk R., Zhang L., Akdis M., Akdis C.A., International Consensus on Allergen Immunotherapy II: Mechanisms, standardization, and pharmacoeconomics, J Allergy Clin Immunol. 2016 Feb;137(2):358-68. doi: 10.1016/j.jaci.2015.12.1300.

Kelly LE, Davies EH, Saint-Raymond A, Tomasi P, Offringa M, Important issues in the justification of a control treatment in paediatric drug trials, Arch Dis Child. 2016 Oct;101(10):962-7

Klungel OH, Kurz X, de Groot MC, Schlienger RG, Tcherny-Lessenot S, Grimaldi L, Ibáñez L, Groenwold RH, Reynolds RF, Multi-centre, multi-database studies with common protocols: lessons learnt from the IMI PROTECT project, Pharmacoepidemiol Drug Saf. 2016 Mar;25 Suppl 1:156-65.

Knirsch C., Alemayehu D., Botgros R., Comic-Savic S., Friedland D., Holland T.L., Merchant K., Noel G.J., Pelfrene E., Reith C., Santiago J., Tiernan R., Tenearts P., Goldsack J.C., Fowler V.G. Jr., Improving Conduct and Feasibility of Clinical Trials to Evaluate Antibacterial Drugs to Treat Hospital-Acquired Bacterial Pneumonia and Ventilator-Associated Bacterial Pneumonia: Recommendations of the Clinical Trials Transformation Initiative Antibacterial Drug Development Project Team, Clin Infect Dis., Vol. 63 Suppl 2:S29-36. doi: 10.1093/cid/ciw258.

Krischke M, Hempel G, Völler S, André N, D'Incalci M, Bisogno G, Köpcke W, Borowski M, Herold R, Boddy AV, Boos J, Pharmacokinetic and pharmacodynamic study of doxorubicin in children with cancer: results of a "European Pediatric Oncology Off-patents Medicines Consortium" trial, Cancer Chemother Pharmacol. 2016 Dec;78(6):1175-1184.

Lengsavath M, Dal Pra A, de Ferran AM, Brosch S, Härmark L, Newbould V, Goncalves S, Social media monitoring and adverse drug reaction reporting in pharmacovigilance: an overview of the regulatory landscape, Ther Innov Regul Sci. 2017 Jan;51(1):125-131. doi: 10.1177/2168479016663264. Epub 2016 Sep 27.

Luzon E., Blake K., Cole S., Nordmark A., Versantvoort C., Benlund E. G., Physiologically-Based Pharmacokinetic modelling in regulatory decision making at the European Medicines Agency, Clin Pharmacol Ther., doi: 10.1002/cpt.539

Maciá-Martínez M.A., de Abajo F. J., Roberts G., Slattery J., Thakrar B., Wisniewski A. F., An empirical approach to explore the relationship between measures of disproportionate reporting and relative risks from analytical studies, Drug Saf., Vol. 39(1), pp. 29-43.

Martinalbo J., Bowen D., Camarero J., Chapelin M., Démolis P., Foggi P., Jonsson B., Llinares J., Moreau A., O'Connor D., Oliveira J., Vamvakas S., Pignatti F., Early market access of cancer drugs in the EU, Ann Oncol., Vol. 27(1).

Mariz S, Reese JH, Westermark K, Greene L, Goto T, Hoshino T, Llinares-Garcia J, Sepodes B, Worldwide collaboration for orphan drug designation, Nat Rev Drug Discov. 2016 Jun 1;15(6):440-1.

Mazzaglia G., Mol P., Drug Utilization research and risk management, in Elseviers M. et al., Drug Utilization Research - Methods and Applications, Wiley-Blackwell, May 2016, pp. 392-398.

Meeker-O'Connell A., Glessner C., Behm M., Mulinde J., Roach N., Sweeney F., Tenaerts P., Landray M. J., Enhancing clinical evidence by proactively building quality into clinical trials, Clin Trials., doi: 1740774516643491

Molinuevo JL, Cami J, Carné X, Carrillo MC, Georges J, Isaac MB, Khachaturian Z, Kim SY, Morris JC, Pasquier F, Ritchie C, Sperling R, Karlawish J, Ethical challenges in preclinical Alzheimer's disease observational studies and trials: Results of the Barcelona summit, Alzheimers Dement. 2016 May;12(5):614-22.

Morel T, Lhoir A, Picavet E, Mariz S, Sepodes B, Llinares J, Cassiman D, Regulatory watch: The orphan drug pipeline in Europe, Nat Rev Drug Discov. 2016 Jun 1;15(6):376

Nair H., Ramilo O., Eichler I., Pelfrene E., Mejias A., Polack F. P., Pouwels K. B., Langley J. M., Nunes M., Van der Maas N., Kragten–Tabatabaie L., Baraldi E., Heikkinen T., Fauroux B., Sharland M., Park C., Manzoni P., Papadopoulos N. G., Martinón–Torres F., Stein R., Bont L., in collaboration with Respiratory Syncytial Virus Network (ReSViNET), Harmonization of RSV therapeutics – from design to performance, J. of Global Health, Vol. 6(1)., doi: 10.7189/jogh.06.010205

Nieto M, Demolis P, Béhanzin E, Moreau A, Hudson I, Flores B, Stemplewski H, Salmonson T, Gisselbrecht C, Bowen D, Pignatti F, The European Medicines Agency review of decitabine (Dacogen) for the treatment of adult patients with acute myeloid leukaemia (AML): Summary of the scientific assessment of the Committee for Medicinal Products for Human Use (CHMP), Oncologist. 2016 Jun;21(6):692-700.

Oye KA, Eichler HG, Hoos A, Mori Y, Mullin TM, Pearson M, Pharmaceuticals licensing and reimbursement in the European Union, United States and Japan, Clin Pharmacol Ther. 2016 Dec;100(6):626-632

Papathanasiou P., Brassart L., Blake P., Hart A., Whitbread L., Pembrey R., Kieffer J., Transparency in drug regulation: public assessment reports in Europe and Australia, Drug Discovery Today, doi: 10.1016/j.drudis.2016.06.025.

Pearson A. D., Herold R., Rousseau R., Copland C., Bradley-Garelik B., Binner D., Capdeville R., Caron H., Carleer J., Chesler L., Geoerger B., Kearns P., Marshall L. V., Pfister S. M., Schleiermacher G., Skolnik J., Spadoni C., Sterba J., van den Berg H., Uttenreuther-Fischer M., Witt O., Norga K., Vassal G., Members of Working Group 1 of the Paediatric Platform of ACCELERATE; Implementation of mechanism of action biology-driven early drug development for children with cancer, Eur J Cancer, Vol 62, pp. 124-131

Pelfrene E., Willebrand E., Cavaleiro Sanches A., Sebris Z., Cavaleri M., Bacteriophage therapy: a regulatory perspective, J. Antimicrob. Chemother.,
doi: 10.1093/jac/dkw083.

Perrone F, Jommi C, Di Maio M, Gimigliano A, Gridelli C, Pignata S, Ciardiello F, Nuzzo F, de Matteis A, Del Mastro L, Bryce J, Daniele G, Morabito A, Piccirillo MC, Rocco G, Guizzaro, Gallo C, The association of financial difficulties with clinical outcomes in cancer patients: secondary analysis of 16 academic prospective clinical trials conducted in Italy, Ann Oncol. 2016 Dec;27(12):2224-2229. 

Pignatti F, Martinalbo J, Jonsson B, Foggi P, Reply to the letter to the editor 'Number-Needed-To-Treat for pricing costly anti-cancer drugs. The example of Regorafenib in metastatic colorectal cancer' by Graziano et al, Ann Oncol. 2016 May;27(5):958.

Pinheiro L., Blake K., Januskiene J., Yue Q. Y., Arlett P., Geographical variation in reporting Interstitial Lung Disease as an adverse drug reaction: findings from an European Medicines Agency analysis of reports in EudraVigilance, doi: 10.1002/pds.3998.

Pita R., Ehmann F., Papaluca M., Nanomedicines in the EU - Regulatory Overview, AAPS J., doi: 10.1208/s12248-016-9967-1

Postmus D., Mavris M., Hillege H.L., Salmonson T., Ryll B., Plate A., Moulon I., Eichler H.G., Bere N., Pignatti F., Incorporating Patient Preferences Into Drug Development and Regulatory Decision Making: Results From a Quantitative Pilot Study With Cancer Patients, Carers, and Regulators, Clin Pharmacol Ther., Vol. 99(5), pp. 548-54.

Reynolds RF, Kurz X, de Groot MC, Schlienger RG, Grimaldi-Bensouda L, Tcherny-Lessenot S, Klungel OH, The IMI PROTECT project: purpose, organizational structure, and procedures, Pharmacoepidemiol Drug Saf. 2016 Mar;25 Suppl 1:5-10.

Richardson JL, Stephens S, Thomas SHL, Jamry-Dziurla A, de Jong-van den Berg L, Zetstra-van der Woude P, Laursen M, Hliva V, Mt-Isa S, Bourke A, Dreyer NA, Blackburn SC, An International Study of the Ability and Cost-Effectiveness of Advertising Methods to Facilitate Study Participant Self-Enrolment Into a Pilot Pharmacovigilance Study During Early Pregnancy, JMIR Public Health Surveill. 2016 Mar 18;2(1):e13. doi: 10.2196/publichealth.5366. eCollection 2016 Jan-Jun.

Saint-Raymond A., Pelle B., Zaccaria C., Sennwitz M., Branch S., Usage of unpublished paediatric data, Arch Dis Child., Vol. 101(1), pp. 81-4.

Saint-Raymond A, de Vries CS, Medicine safety in pregnancy and ambitions for the EU medicine regulatory framework, Clin Pharmacol Ther. 2016 Jul;100(1):21-3

Salmonson T, Janssen H, Sudhop T, Stahl E, Regulatory Reply to the ATMOSPHERE Data Monitoring Committee, N Engl J Med. 2016 Apr 21;374(16):1585-6.

Schneeweiss S, Eichler HG, Garcia-Altes A, Chinn C, Eggimann AV, Garner S, Goettsch W, Lim R, Löbker W, Martin D, Müller T, Park BJ, Platt R, Priddy S, Ruhl M, Spooner A, Vannieuwenhuyse B, Willke RJ, Real World Data in Adaptive Biomedical Innovation: A Framework for Generating Evidence Fit for Decision-Making, Clin Pharmacol Ther. 2016 Dec;100(6):633-646.

Schuerch M, Gasse C, Robinson NJ, Alvarez Y, Walls R, Mors O, Christensen J, Hesse U, de Groot M, Schlienger R, Reynolds R, Klungel O, de Vries F, Impact of varying outcomes and definitions of suicidality on the associations of antiepileptic drugs and suicidality: comparisons from UK Clinical Practice Research Datalink (CPRD) and Danish national registries (DNR), Pharmacoepidemiol Drug Saf. 2016 Mar;25 Suppl 1:142-55

Schüssler-Lenz M, Beuneu C, Menezes-Ferreira M, Jekerle V, Bartunek J, Chamuleau S, Celis P, Doevendans P, O'Donovan M, Hill J, Hystad M, Jovinge S, Kyselovič J, Lipnik-Stangelj M, Maciulaitis R, Prasad K, Samuel A, Tenhunen O, Tonn T, Rosano G, Zeiher A, Salmikangas P, Cell-based therapies for cardiac repair: a meeting report on scientific observations and European regulatory viewpoints, Eur J Heart Fail. 2016 Feb;18(2):133-41.

Seabroke S., Candore G., Juhlin K., Quarcoo N., Wisniewski A., Arani R., Painter J., Tregunno P., Noren N., Slattery J., Performance of Stratified and Subgrouped Disproportionality. Analyses in Spontaneous Databases, Drug Saf., Vol. 39(4), pp. 355–364.

Sepodes B, Regulation Of Orphan Medicinal Products In Europe: History Trends And Challenges, Clin Ther. 2016 Oct 6;38(10S):e7-e8

Slattery J., Measuring Signal Detection Performance: Can We Trust Negative Controls and Do We Need Them?, Vol. 39(5), pp. 371-3.

Souverein PC, Abbing-Karahagopian V, Martin E, Huerta C, de Abajo F, Leufkens HG, Candore G, Alvarez Y, Slattery J, Miret M, Requena G, Gil MJ, Groenwold RH, Reynolds R, Schlienger RG, Logie JW, de Groot MC, Klungel OH, van Staa TP, Egberts TC, De Bruin ML, Gardarsdottir H, Understanding inconsistency in the results from observational pharmacoepidemiological studies: the case of antidepressant use and risk of hip/femur fractures, Pharmacoepidemiol Drug Saf. 2016 Mar;25 Suppl 1:88-102. doi: 10.1002/pds.3862.

Spindler P., Bach K. F., Schmiegelow M., Bedlington N., Eichler H. G., Innovation of medical products: the evolution of regulatory science, research and education, Therapeutic Innovation & Regulatory Science, Vol. 50(1), pp. 44-48.

Straub V., Balabanov P., Bushby K., Ensini M., Goemans N., De Luca A., Pereda A., Hemmings R., Campion G., Kaye E., Arechavala-Gomeza V., Goyenvalle A., Niks E., Velldhuizen O., Furlong P., Stoyanova-Beninska V., Wood M. J., Johnson A., Mercuri E., Muntoni F., Sepodes B., Haas M., Vroom E., Aartsma-Rus A.. Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy,The Lancet Neurology, Vol. 15(8), pp. 882–890.

Sun H, Vesely R, Lee KJ, Klein A, Ikima M, Mulberg AE; International Inflammatory Bowel Disease (i-IBD) Working Group, Pediatric Crohn's Disease Clinical Outcome Assessments and Biomarkers: Current State and Path Forward for Global Collaboration, J Pediatr Gastroenterol Nutr. 2016 Jun 2.

Tafuri G., Pagnini M., Moseley J., Massari M., Petavy F., Behring A., Catalan A., Gajraj E., Hedberg N., Obach M., Osipenko L., Russo P., Van De Casteele M., Zebedin EM., Rasi G., Vamvakas S., How aligned are the perspectives of EU regulators and HTA bodies? A comparative analysis of regulatory-HTA parallel scientific advice, Br J Clin Pharmacol, doi: 10.1111/bcp.13023.

Tamborlane W.V., Haymond M.W., Dunger D., Shankar R., Gubitosi-Klug R., Bethin K., Karres J., Tomasi P., Libman I., Hale P.H., Portman R., Klingensmith G., Reed M., Blumer J., Giacoia G., Expanding Treatment Options for Youth with Type 2 Diabetes: Current Problems and Proposed Solutions, Diabetes Care, Vol. 39(3), pp. 323–329.

Turner MA, Davis JM, McCune S, Bax R, Portman RJ, Hudson LD, The International Neonatal Consortium:  Collaborating to Advance Regulatory Science for Neonates, Pediatr Res. 2016 Oct;80(4):462-4

Uddin MJ, Groenwold RH, de Boer A, Gardarsdottir H, Martin E, Candore G, Belitser SV, Hoes AW, Roes KC, Klungel OH, Instrumental variables analysis using multiple databases: an example of antidepressant use and risk of hip fracture, Pharmacoepidemiol Drug Saf. 2016 Mar;25 Suppl 1:122-31.

Udo R, Tcherny-Lessenot S, Brauer R, Dolin P, Irvine D, Wang Y, Auclert L, Juhaeri J, Kurz X, Abenhaim L, Grimaldi L, De Bruin ML, The risk of acute liver injury associated with the use of antibiotics--evaluating robustness of results in the pharmacoepidemiological research on outcomes of therapeutics by a European consortium (PROTECT) project, Pharmacoepidemiol Drug Saf. 2016 Mar;25 Suppl 1:47-55.

Van Riet-Nales D.A., Hussain N., Sundberg K.A., Eggenschwyler D., Ferris C., Robert J.L., Cerreta F., Regulatory incentives to ensure better medicines for older people: from ICH E7 to the EMA reflection paper on quality aspects, Int J Pharm. 2016 May 2. pii: S0378-5173(16)30367-2.

Vicini P., Fields O., Lai E., Litwack E. D., Martin A. M., Morgan T. M., Pacanowski M. A., Papaluca M., Perez O. D., Ringel M. S., Robson M., Sakul H., Vockley J., Zaks T., Dolsten M., Søgaard M., Precision Medicine in the Age of Big Data: The Present and Future Role of Large Scale Unbiased Sequencing in Drug Discovery and Development, Clin Pharmacol Ther., Vol. 99(2), pp. 198-207.

Willkommen H, Blümel J, Brorson K, Chen D, Chen Q, Gröner A, Kreil TR, Ruffing M, Ruiz S, Scott D, Silvester G, Meeting Report: 2015 PDA Virus & TSE Safety Forum, PDA J Pharm Sci Technol. 2016 Mar-Apr;70(2):177-88.

Wisniewski A.F.Z., Bate A., Bousquet C., Brueckner A., Candore G., Juhlin K., Marcia-Martinez M.A., Manlik K., Quarcoo N., Seabroke S., Slattery J., Southworth H., Thakrar B., Tregunno P., Van Holle L., Kayser M., Noren G.N., Good Signal Detection Practices: Evidence from IMI PROTECT, Drug Safety, doi: 10.1007/s40264-016-0405-1.

Zannad F, Stough WG, Lipicky RJ, Tamargo J, Bakris GL, Borer JS, Alonso García Mde L, Hadjadj S, Koenig W, Kupfer S, McCullough PA, Mosenzon O, Pocock S, Scheen AJ, Sourij H, Van der Schueren B, Stahre C, White WB, Calvo G, Assessment of cardiovascular risk of new drugs for the treatment of diabetes mellitus: risk assessment vs. risk aversion, Eur Heart J Cardiovasc Pharmacother. 2016 Jul;2(3):200-5

Ziogas C, A regulator’s insights for SMEs in the biologics and advanced therapies sectors, Regulatory Rapporteur – Vol 13, No 2, February 2016


Bahri P., Dodoo A. N., Edwards B. D., Edwards I. R., Fermont I., Hagemann U., Hartigan-Go K., Hugman B., Mol P. G., 'The ISoP CommSIG for Improving Medicinal Product Risk Communication: A New Special Interest Group of the International Society of Pharmacovigilance', Drug Saf., Vol. 38(7), pp. 621-627

Barnes P.J., Bonini S., Seeger W., Belvisi M.G., Ward B., Holmes A., Barriers to new drug development in respiratory disease, Eur Respir J. 2015 May;45(5):1197-207. doi: 10.1183/09031936.00007915

Bartlett J. A., Brewster M., Brown P., Cabral-Lilly D., Cruz C. N., David R., Eickhoff W. M., Haubenreisser S., Jacobs A., Malinoski F., Morefield E., Nalubola R., Prud'homme R. K., Sadrieh N., Sayes C. M., Shahbazian H., Subbarao N., Tamarkin L., Tyner K., Uppoor R., Whittaker-Caulk M., Zamboni W., 'Summary report of PQRI workshop on nanomaterial in drug products: current experience and management of potential risks', The AAPS Journal, Vol. 17(1), pp. 44-64.

Blind E., de Graeff P. A., Meurs I., Holtkamp F., Baczynska A., Janssen H., The European Medicines Agency's approval of proprotein convertase subtilisin/kexin type 9 inhibitors, Eur Heart J. pii: ehv673.

Bonelli M., Di Giuseppe F., Beken S., 'Impact analysis of ICH S9 on non-clinical development of anticancer drugs', Regul Toxicol Pharmacol., Vol. 73(1), pp. 361-366

Bonini M., Gramiccioni C., Fioretti D., Ruckert B., Rinaldi M., Akdis C., Todaro A., Palange P., Carlsen K.H., Pelliccia A., Rasi G., Bonini S., Asthma, allergy and the Olympics: a 12-year survey in elite athletes, AIDA and the Italian Unit of the GA2LEN Olympic Study, Curr Opin Allergy Clin Immunol. 2015 Apr;15(2):184-92. doi: 10.1097/ACI.0000000000000149

Both L., Botgros R., Cavaleri M., Analysis of licensed over-the-counter (OTC) antibiotics in the European Union and Norway, 2012, Euro Surveill., Vol. 20(34).

Bousquet J., Kuh D., Bewick M., Standberg T., Farrell J., Pengelly R., Joel M.E., Rodriguez Mañas L., Mercier J., Bringer J., Camuzat T., Bourret R., Bedbrook A., Kowalski M.L., Samolinski B., Bonini S. et al., Operational Definition of Active and Healthy Ageing (AHA): A Conceptual Framework, J Nutr Health Aging. 2015 Nov;19(9):955-60. doi: 10.1007/s12603-015-0589-6

Butlen-Ducuing F., Zienowicz M., Pétavy F., Haas M., Salmonson T., Eichler H.G., Rasi G., European regulatory experience with drugs for central nervous system disorders, Nat Rev Drug Discov., Vol14(2), pp. 89-90.

Candore G, Juhlin K, Manlik K, Thakrar B, Quarcoo N, Seabroke S, Wisniewski A, Slattery J, Comparison of statistical signal detection methods within and across spontaneous reporting databases, Drug Saf. 2015 Jun;38(6):577-87

Catry B., Cavaleri M., Baptiste K., Grave K., Grein K., Holm A., Jukes H., Liebana E., Lopez Navas A., Mackay D., Magiorakos A. P., Moreno Romo M. A., Moulin G., Muñoz Madero C., Matias Ferreira Pomba M. C., Powell M., Pyörälä S., Rantala M., Ružauskas M., Sanders P., Teale C., Threlfall E. J., Törneke K., van Duijkeren E., Torren Edo J., Use of colistin-containing products in animals within the European Union and European Economic Area (EU/EEA): development of resistance and possible impact on human and animal health, Int J Antimicrob Agents, Vol 46(3), pp. 297-306.

Cavaleri M, Manolis E, Hollow Fibre System Model for Tuberculosis: The European Medicines Agency Experience, Clin Infect Dis. 2015 Aug 15;61 Suppl 1:S1-4.

Celis P, Ferry N, Hystad M, Schüßler-Lenz M, Doevendans PA, Flory E, Beuneu C, Reischl I, Salmikangas P, Advanced Therapy Medicinal Products – How to bring cell-based medicinal products successfully to the market. Report from the CAT-DGTI-GSCN workshop at the DGTI Annual Meeting 2014, Transfus Med Hemother. 2015 May;42(3):194-9

Cohen J., Vincent J. L., Adhikari N. K. J., Machado F. R. , Angus D. C., Calandra T., Jaton K., Giulieri S., Delaloye J., Opal S., Tracey K., van der Poll T., Pelfrene E., 'Sepsis: a roadmap for future research', Lancet Infect Dis., doi:

Dal Pan G. J., Arlett P. R., 'The US Food and Drug Administration-European Medicines Agency collaboration in pharmacovigilance: common objectives and common challenges', Drug Saf., Vol. 38, pp. 13-15.

Dodt J., Hubbard A. R., Wicks S. J., Gray E., Neugebauer B., Charton E., Silvester G., 'Potency determination of factor VIII and factor IX for new product labelling and postinfusion testing: challenges for caregivers and regulators', Haemophilia, doi: 10.1111/hae.12634.

Dreyer NA, Blackburn S, Hliva V, Mt-Isa S, Richardson J, Jamry-Dziurla A, Bourke A, Johnson R, Balancing the interests of patient data protection and medication safety monitoring in a public-private partnership, JMIR Med Inform. 2015 Apr 15;3(2):e18. doi: 10.2196/medinform.3937.

Dreyer NA, Blackburn SC, Mt-Isa S, Richardson JL, Thomas S, Laursen M, Zetstra-van der Woude P, Jamry-Dziurla A, Hliva V, Bourke A, de Jong-van den Berg L, Direct-to-Patient Research: Piloting a New Approach to Understanding Drug Safety During Pregnancy, JMIR Public Health Surveill. 2015 Dec 22;1(2):e22. doi: 10.2196/publichealth.4939. eCollection 2015 Jul-Dec.

Ehmann F., Caneva L., Prasad K., Paulmichl M., Maliepaard M., Llerena A., Ingelman-Sundberg M., Papaluca-Amati M., 'Pharmacogenomic information in drug labels: European Medicines Agency perspective', Pharmacogenomics J. 2015 Jun;15(3):201-10

Ehmann F., Papaluca M., Di Giuseppe F., Pani L., Eskova A., Manolis E., Herold R., 'Changes and determination of dosing recommendations for medicinal products recently authorised in the European Union', Expert Opin Pharmacother, Vol.16(6), pp. 903-11.

Eichler H. G., Baird L., Barker R., Bloechl-Daum B., Børlum-Kristensen F., Brown J., Chua R., Del Signore S., Dugan U., Ferguson J., Garner S., Goettsch W., Haigh J., Honig P., Hoos A., Huckle P., Kondo T., Le Cam Y., Leufkens H., Lim R., Longson C., Lumpkin M., Maraganore J., O'Rourke B., Oye K., Pezalla E., Pignatti F., Raine J., Rasi G., Salmonson T., Samaha D., Schneeweiss S., Siviero P., Skinner M., Teagarden J., Tominaga T., Trusheim M., Tunis S., Unger T., Vamvakas S., Hirsch G., 'From adaptive licensing to adaptive pathways: delivering a flexible life span approach to bring new drugs to patients', Clin Pharmacol Ther., Vol. 97(3), pp. 234-46.

Eichler H.G, Thomson A., Eichler I., Schneeweiss S., 'Assessing the relative efficacy of new drugs: an emerging opportunity', Nature Reviews Drug Discovery, Vol. 14, pp. 443–444.

Flory E, Gasparini P, Jekerle V, Palomäki T, Celis P, Boráň T, McBlane JW, Borg JJ, Kyselovic J, Lipnik–Stangelj M, Maimets T, Menezes-Ferreira M, Pante G, Prilla S, Riekstina U, Schneider CK, Tsiftsoglou A, Salmikangas P, Regulatory viewpoints on the development of advanced stem cell–based medicinal products in light of the first EU-approved stem cell product, Cell Gene Therapy Insights 2015; 1(1), 109-127.

Fregonese L, Eichler I, The future of the development of medicines in idiopathic pulmonary fibrosis, BMC Med. 2015 Sep 24;13:239

Goldman M., Seigneuret N., Eichler H. G., 'The Innovative Medicines Initiative: An engine for regulatory science', Nat Rev Drug Discov., Vol. 14(1), pp.1-2

Haas M., Mantua V., Haberkamp M., Pani L., Isaac M., Butlen-Ducuing F., Vamvakas S., Broich K., The European Medicines Agency's strategies to meet the challenges of Alzheimer disease, Nat Rev Drug Discov. 2015 Apr;14(4):221-2. doi: 10.1038/nrd4585

Haas M., Vlcek V., Balabanov P., Salmonson T., Bakchine S., Markey G., Weise M., Schlosser-Weber G., Brohmann H., Yerro C. P., Mendizabal M. R., Stoyanova-Beninska V., Hillege H. L., 'The European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene: Summary of the scientific assessment of the Committee for Medicinal Products for Human Use', Neuromuscul Disord. Vol. 25(1), pp.5-13

Hanaizi Z., Flores B., Hemmings R., Camarero J., Sancho-Lopez A., Salmonson T., Gisselbrecht C., Laane E., Pignatti F.,' The European Medicines Agency review of pomalidomide for the treatment of adult patients with multiple myeloma: Summary of the scientific assessment of the Committee for Medicinal Products for Human Use', Oncologist, Vol. 20(3), pp. 329-334.

Haubenreisser s., Harvey-Allchurch M., Chapter 3 - The European Medicines Agency and the Regulation of Medicines in the European Union, Food and Drug Regulation in an Era of Globalised Marekts, pp. 25-36.

Hofer M.P., Jakobsson C., Zafiropoulos N., Vamvakas S., Vetter T., Regnstrom J., Hemmings R.J., Regulatory watch: Impact of scientific advice from the European Medicines Agency, Nature Reviews Drug Discovery, Vol 14(5), pp. 302-303.

Huerta C, Abbing-Karahagopian V, Requena G, Oliva B, Alvarez Y, Gardarsdottir H, Miret M, Schneider C, Gil M, Souverein PC, De Bruin ML, Slattery J, De Groot MC, Hesse U, Rottenkolber M, Schmiedl S, Montero D, Bate A, Ruigomez A, García-Rodríguez LA, Johansson S, de Vries F, Schlienger RG, Reynolds RF, Klungel OH, de Abajo FJ, (included in 2014 report under incorrect title), Pharmacoepidemiol Drug Saf. 2015 Jul 7        

Klug B., Celis P., Ruepp R., Robertson J S., EU regulatory guidelines for the clinical evaluation of adjuvants, Clin Res Pr Drug Regul Aff, doi:10.3109/10601333.2015.1001899

Koenig F., Slattery S., Groves T., Lang T., Benjamini Y., Day S., Bauer P., and Posch M., Sharing clinical trial data on patient level: Opportunities and challenges, Biometrical Journal 57, 8-26, (2015) [bib]

Krause P. R., Cavelari M., Coleman G., Gruber M. F., Approaches to Demonstration of Ebola Virus Vaccine Efficacy, Lancet, Vo. 15(6), pp. 615-746.

Loth E., Spooren W., Ham L. M., Isaac M. B., Auriche-Benichou C., Banaschewski T., Baron-Cohen S., Broich K., Bölte S., Bourgeron T., Charman T., Collier D., de Andres-Trelles F., Durston S., Ecker C., Elferink A., Haberkamp M., Hemmings R., Johnson M. H., Jones E. J., Khwaja O. S., Lenton S., Mason L., Mantua V., Meyer-Lindenberg A., Lombardo M. V., O'Dwyer L., Okamoto K., Pandina G. J., Pani L., Persico A. M., Simonoff E., Tauscher-Wisniewski S., Llinares-Garcia J., Vamvakas S., Williams S., Buitelaar J. K., Murphy D. G., Identification and Validation of Biomarkers for Autism Spectrum Disorders, Nat Rev Drug Discov. Vol 15(1), pp. 70-3.

Luciani F., Galluzzo S., Gaggioli A., Kruse N. A., Venneugues P., Schneider C. K., Pini C., Melchiorri D., Implementing quality by design for biotech products: are regulators on track?, Vol 7(3), pp. 451-455

Luigetti R., Cooke E., Cuddy B., Goux S., Rees I., GMP Oversight of Medicines Manufacturers in the European Union, PDA Letter

Murray S., McKenna L., Pelfrene E., Botgros R., Accelerating clinical drug development for children with tuberculosis, International Journal of Tuberculosis and Lung Disease., 2015 Dec;19 Suppl 1, pp. 69-74.

Nachman S., Ahmed A., Amanullah F., Becerra M. C., Botgros R., Brigden G., Browning R., Gardiner E., Hafner R., Hesseling A., How C., Jean-Philippe P., Lessem E., Makhene M., Mbelle N., Marais B., McIlleron H., McNeeley D. F., Mendel C., Murray S., Navarro E., Anyalechi E. G., Porcalla A. R., Powell C., Powell M., Rigaud M, Rouzier V., Samson P., Schaaf H. S., Shah S., Starke J., Swaminathan S., Wobudeya E., Worrell C., Towards early inclusion of children in tuberculosis drugs trials: a consensus statement, Lancet Infect Dis., Vol. 15(6), pp. 711–720.

Norga KK, Kankergeneesmiddelen voor kinderen: impact van de Europese “Paediatric Regulation”

Offringa M., Davis J.M., Turner M.A., Ward R., Bax R., Maldonado S., Sinha V., McCune S.K., Zajicek A., Benjamin D.K., Bucci-Rechtsweg C., Nelson R.M., Applying Regulatory Science to Develop Safe and Effective Medicines for Neonates: Report of the US Food and Drug Administration First Annual Neonatal Scientific Workshop, October 28-29, 2014, Therapeutic Innovation & Regulatory Science, 7 August 2015

Oye K.A., Pearson M., Eichler H.G., Mullin T., Hoos A., Managing uncertainty in drug development and use informed regulation: Enhancing adaptability and flexibility in pharmaceuticals regulation. In: Improving Risk Regulation, International Risk Governance Council (IRGC). Lausanne: IRGC, 2015 (63-80).

Pacurariu A.C., Straus S.M., Trifirò G., Schuemie M.J., Gini R., Herings R., Mazzaglia G., Picelli G., Scotti L., Pedersen L., Arlett P., Van der Lei J., Sturkenboom M.C., Coloma P.M., Useful Interplay Between Spontaneous ADR Reports and Electronic Healthcare Records in Signal Detection, Drug Saf., doi:10.​1007/​s40264-015-0341-5, doi:10.1586/17512433.2015.1028918

Pani L, Pecorelli S, Rosano G, Anker SD, Peracino A, Fregonese L, Prasad K, Rasi G, Steps Forward In Regulatory Pathways For Acute and Chronic Heart Failure, Eur J Heart Fail. 2015 Jan;17(1):3-8. doi: 10.1002/ejhf.209.

Papaluca M., Grecoa M., Tognana E., Ehmann F., Saint-Raymond A., White spots in pharmaceutical pipelines–EMA identifies potential areas of unmet medical needs, Expert Review of Clinical Pharmacology, Vol.8, Issue 3, 2015

Pelfrene E., Pinheiro M. H., Cavaleri M., 'Artemisinin-based combination therapy in the treatment of uncomplicated malaria: review of recent regulatory experience at the European Medicines Agency', Int Health, doi: 10.1093/inthealth/ihv017.

Pignatti F., Ashby D., Brass E. P., Eichler H. G., Frey P., Hillege H. L., Hori A., Levitan B., Liberti L., Löfstedt R. E., McAuslane N., Micaleff A., Noel R. A., Postmus D., Renn O., Sabourin B. J., Salmonson T., Walker S., Structured Frameworks to Increase the Transparency of the Assessment of Benefits and Risks of Medicines: Current Status and Possible Future Directions, Vol. 98(5), pp. 522-33.

Pignatti F., Jonsson B., Blumenthal G., Justice R., 'Assessment of benefits and risks in development of targeted therapies for cancer - The view of regulatory authorities', Mol Oncol., pii: S1574-7891(14)00242-7

Radawski C, Morrato E, Hornbuckle K, Bahri P, Smith M, Juhaeri J, Mol P, Levitan B, Huang H-Y, Coplan P, Li H (on behalf of the ISPE BRACE SIG), Benefit-Risk Assessment, Communication and Evaluation (BRACE) throughout the Life Cycle of Therapeutic Products: Overall Perspective and Role of the Pharmacoepidemiologist, Pharmacoepidemiol Drug Saf. 2015; 24: 1233-1240

Rasi G., Bonini S., Innovative medicines: new regulatory procedures for the third millennium, Expert Opin Biol Ther. 2015;15 Suppl 1:S5-8. doi: 10.1517/14712598.2015.1026322

Roca I, Akova M, Baquero F, Carlet J, Cavaleri M, Coenen S, Cohen J, Findlay D, Gyssens I, Heure OE, Kahlmeter G, Kruse H, Laxminarayan R, Liébana E, López-Cerero L, MacGowan A, Martins M, Rodríguez-Baño J, Rolain JM, Segovia C, Sigauque B, Taconelli E, Wellington E, Vila J, The global threat of antimicrobial resistance: science for intervention, New Microbes New Infect. 2015 Apr 16;6:22-9.

Romero K, Ito K, Rogers J, Polhamus D, Qiu R, Stephenson D, Mohs R, Lalonde R, Sinha V, Wang Y, Brown D, Isaac M, Vamvakas S, Hemmings R, Pani L, Bain Lj, Corrigan B; Alzheimer's Disease Neuroimaging Initiative for the Coalition Against Major Diseases, The Future Is Now:  Model-Based Clinical Trial Design for Alzheimer’s Disease, Clin Pharmacol Ther. 2015 Mar;97(3):210-4.

Rottenkolber M., Voogd E., van Dijk L., Primatesta P., Becker C., Schlienger R., de Groot M. C., Alvarez Y., Durand J., Slattery J., Afonso A., Requena G., Gil M., Alvarez A., Hesse U., Gerlach R., Hasford J., Fischer R., Klungel O. H., Schmiedl S., 'Time trends of period prevalence rates of patients with inhaled long-acting Beta-2-agonists-containing prescriptions: a European comparative database study', PLoS One, Vol.10(2):e0117628.

Rottenkolber M, Voogd E, van Dijk L, Primatesta P, Becker C, de Groot MC, Plana E, Alvarez Y, Durand J, Slattery J, Afonso A, Requena G, Huerta C, Alvarez A, de Abajo F, Tauscher M, Hasford J, Fischer R, Reynolds R, Schmiedl S, Seasonal changes in prescribing of long-acting beta-2-agonists-containing drugs, Respir Med. 2015 Jul;109(7):828-37

Sabaté M, Ferrer P, Ballarín E, Rottenkolber M, Amelio J, Schmiedl S, Reynolds R, Klungel O, Ibáñez L; PROTECT Work Package 2, Inpatient drug utilization in Europe: nationwide data sources and a review of publications on a selected group of medicines (PROTECT project), Basic Clin Pharmacol Toxicol. 2015 Mar;116(3):201-11. doi: 10.1111/bcpt.12358. Epub 2014 Dec 29.

Salmikangas P., Menezes-Ferreira M., Reischl I., Tsiftsoglou A., Kyselovic J., Borg J. J., Ruiz S., Flory E., Trouvin J. H., Celis P., Ancans J., Timon M., Pante G., Sladowski D., Lipnik-Stangelj M., Schneider C. K., 'Manufacturing, characterisation and control of cell-based medicinal products: challenging paradigms towards commercial use', Regen. Med., Vol. 10(1), pp. 65-78.

Salmikangas P, Schuessler-Lenz M, Ruiz S, Celis P, Reischl I, Menezes-Ferreira M, Flory E, Renner M, Ferry N, Marketing Regulatory Oversight  of Advanced Therapy Medicinal Products (ATMPs) in Europe: The EMA/CAT Perspective, Adv Exp Med Biol. 2015;871:103-30

Samardzic J., Turner M.A., Bax R., Allegaert K., Neonatal Medicines Research: Challenges and Opportunities, Expert Opin Drug Metab Toxicol. 2015 Jul;11(7):1041-52

Segec A., Keller-Stanislawski B., Vermeer N.S., Macchiarulo C., Straus S.M., Hidalgo-Simon A., De Bruin M.L., Strategy in Regulatory Decision-Making for Management of Progressive Multifocal Leukoencephalopathy, Clinical Pharmacology & Therapeutics, Vol. 98(5), pp. 502-505.

Torneke K., Torren-Edo J., Grave K., Mackay D. K., The management of risk arising from the use of antimicrobials in veterinary medicine in EU/EEA countries – a review, J Vet Pharmacol Ther. doi: 10.1111/jvp.12226.

Torren E. J., Grave K., Mackay D., '“One Health”: the regulation and consumption of antimicrobials for animal use in the EU', IAHJ, Vol. 2, pp.14-16.

Tzogani K, Skibeli V, Westgaard I, Dalhus M, Thoresen H, Slot KB, Damkier P, Hofland K, Borregaard J, Ersbøll J, Salmonson T, Pieters R, Sylvester R, Mickisch G, Bergh J, Pignatti F, The European Medicines Agency approval of axitinib (Inlyta) for the treatment of advanced renal cell carcinoma after failure of prior treatment with sunitinib or a cytokine: summary of the scientific assessment of the committee for medicinal products for human use, Oncologist. 2015 Feb;20(2):196-201

Vermeer N.S., Straus S.M.J.M., Mantel-Teeuwisse A.K., Hidalgo-Simon A., Egberts A.C.G., Leufkens H.G.M., De Bruin M.L., Drug-induced progressive multifocal leukoencephalopathy: Lessons learned from contrasting natalizumab and rituximab, Clinical Pharmacology and Therapeutics, Vol. 98(5), pp. 542-550.

Walton M. K., Powers J. H. 3rd, Hobart J., Patrick D., Marquis P., Vamvakas S., Isaac M., Molsen E., Cano S., Burke L. B., Clinical Outcome Assessments: Conceptual Foundation-Report of the ISPOR Clinical Outcomes Assessment - Emerging Good Practices for Outcomes Research Task Force, Value Health Vol18, pp. 741-52.


Aartsma-Rus A., Ferlini A., Goemans N., Pasmooij A. M., Wells D. J., Bushby K., Vroom E., Balabanov P., 'Translational and regulatory challenges for exon skipping therapies', Human Gene Therapy, Vol. 25(10), pp. 885-92.

Abbing-Karahagopian V., Huerta C., Souverein P. C., de Abajo F., Leufkens H. G., Slattery J., Alvarez Y., Miret M., Gil M., Oliva B., Hesse U., Requena G., de Vries F., Rottenkolber M., Schmiedl S., Reynolds R., Schlienger R. G., de Groot M. C., Klungel O. H., van Staa T. P., van Dijk L., Egberts A. C., Gardarsdottir H., De Bruin M. L., 'Antidepressant prescribing in five European countries: application of common definitions to assess the prevalence, clinical observations, and methodological implications', Eur J Clin Pharmacol, Vol. 70(7), pp. 849-857.

Abbing-Karahagopian V., Kurz X., de Vries F., van Staa T. P., Alvarez Y., Hesse U., Hasford J., van Dijk L., de Abajo F. J., Weil J. G., Grimaldi-Bensouda L., Egberts A. C., Reynolds R. F., Klungel O. H., 'Bridging differences in outcomes of pharmacoepidemiological studies: Design and first results of the Protect project', Curr Clin Pharmacol, Vol 9, pp. 130-138.

Abed I, The approval process of medicines in Europe, Medical writing (2014)Vol. 23, No 2; p117 – 121

Abou-El-Enein M, Bauer G, Reinke P, Renner M, Schneider CK, A roadmap toward clinical translation of genetically-modified stem cells for treatment of HIV, Trends Mol Med. 2014 Nov;20(11):632-42.

Arlett P., Portier G., de Lisa R., Blake K., Wathion N., Dogne J. M., Spooner A., Raine J., Rasi G., 'Proactively managing the risk of marketed drugs: experience with the EMA Pharmacovigilance Risk Assessment Committee', Nat Rev Drug Discov, Vol. 13(5), pp. 395-397.

Arlett .P, Postigo R., Janssen H., Spooner A., 'Periodic Benefit-Risk Evaluation Report: A European Union Regulatory Perspective'. Pharmaceutical Medicine', Vol. 28(6), pp. 309-315.

Arlett P., Sarac S. B., Thomson A., Davies C., Teixeira T., Blake K. V., Stenver D., 'The European Medicines Agency's use of prioritised independent research for best evidence in regulatory action on diclofenac', Pharmacoepidemiol Drug Saf, Vol. 23(4), pp. 431-434.

Baird L. G., Banken R., Eichler H. G., Kristensen F. B., Lee D. K., Lim J. C., Lim R., Longson C., Pezalla E., Salmonson T., Samaha D., Tunis S., Woodcock J., Hirsch G., 'Accelerated access to innovative medicines for patients in need', Clin Pharmacol Ther, Vol. 96(5), pp. 559–571.

Balabanov P., Haas M., Elferink A., Bakchine S., Broich K., 'Addressing the regulatory and scientific challenges in multiple sclerosis - a statement from the EU regulators', Mult Scler, Vol. 20(10), pp. 1282-1287.

Beckmann J., Hagemann U., Bahri P., Bate A., Boyd I. W., Dal Pan G. J., Edwards B. D., Edwards I. R., Hartigan-Go K., Lindquist M., McEwen J., Moride Y., Olsson S., Pal S. N., Soulaymani-Bencheikh R., Tuccori M., Vaca C. P., Wong I. C. K., 'Teaching pharmacovigilance: the WHO-ISoP core elements of a comprehensive modular curriculum', Drug Saf, Vol. 37(10), pp. 743-759.

Berntgen M., Gourvil A., Pavlovic M., Goettsch W., Eichler H.-G., Kristensen F. B., 'Improving the contribution of regulatory assessment reports to health technology assessments - a collaboration between the European Medicines Agency and the European Network for Health Technology Assessment', Value in Health, Vol. 17(5), pp. 634-641.

Blake K. V., Zaccaria C., Domergue F., La Mache E., Saint-Raymond A., Hidalgo-Simon A., 'Comparison between paediatric and adult suspected adverse drug reactions reported to the European Medicines Agency: implications for pharmacovigilance', Pediatric Drugs, Vol. 16(4), pp. 309-319.

Bonini S., Eichler H. G., Wathion N., Rasi G., 'Transparency and the European Medicines Agency – sharing of clinical trial data', N Engl J Med, Vol. 371(26), pp. 2452-2455.

Borg J. J., Tomasi P., Pani L., Aislaitner G., Pirozynski M., Leufkens H., Melchiorri D., 'Licensing of generic medicines: are there any challenges left? A pharmaceutical regulatory perspective', Sci Pharm, Vol. 82, pp. 847-856.

Borg JJ, Laslop A, Pani L, Maciulaitis R, Melchiorri D, Reflections on Decisions Made on the Well-Established Use of Medicinal Products by EU Regulators and the ECJ, Sci Pharm. 2014; 82: 655–663

Caneva L., Bonelli M., Papaluca-Amati M., Vidal J. M., 'Critical review on the environmental risk assessment of medicinal products for human use in the centralised procedure', Regul Toxicol Pharmacol, Vol. 68(3), pp. 312-316.

Cerreta F., Temple R., Asahina Y., Connaire C., 'Regulatory activities to address the needs of older patients', J Nutr Health Aging, doi: 10.1007/s12603-014-0494-4.

Clini EM, Fregonese L, A conceptual framework for reporting experience of physical activity in COPD, Eur Respir J. 2014 Nov;44(5):1110-2

Cortes-Blanco .A, Prieto-Yerro C., Martinez-Lazaro R., Zamora J., Jiménez-Huete A., Haberkamp M., Pohly J., Enzmann H., Zinserling J., Strassmann V., Broich K.. 'Florbetapir (18F) for brain amyloid positron emission tomography: Highlights on the European marketing approval', Alzheimers Dement., Vol. 10(5 Suppl), pp. S395-9.

de Groot M. C., Schuerch M., de Vries F., Hesse U., Oliva B., Gil M., Huerta C., Requena G., de Abajo F., Afonso A. S., Souverein P. C., Alvarez Y., Slattery J., Rottenkolber M., Schmiedl S., Van Dijk L., Schlienger R. G., Reynolds R., Klungel O. H., 'Antiepileptic drug use in seven electronic health record databases in Europe: A methodologic comparison', Epilepsia, Vol. 55(5), pp. 666-673.

Egan A. G., Blind E., Dunder K., de Graeff P. A., Hummer B. T., Bourcier T., Rosebraugh C., 'Pancreatic safety of incretin-based drugs — FDA and EMA assessment', N Engl J Med, Vol. 370(9), pp. 794-797.

Egger G. F., Manent N., 'Re: Non-publication and delayed publication of randomized trials on vaccines: survey', BMJ, 2014, Vol. 348, g3058.

Franco P., Porta N., Holliday J. D., Willett P., 'The use of 2D fingerprint methods to support the assessment of structural similarity in orphan drug legislation', J Cheminform, Vol. 6(1): 5.

Giezen TJ, Schneider CK, Safety assessment of biosimilars in Europe: a regulatory perspective, GaBI Journal 2014;4:180-3.    

Gravanis I., Vleminckx C., Jonsson B., Pignatti F., 'The changing world of oncology dug development: the regulatory bodies' perspective', Chin Clin Oncol, Vol. 3(2), p. 22.

Grave K., Mackay D., Muller A., Torren-Edo J., 'European Surveillance of Veterinary Antimicrobial Consumption (ESVAC): Establishment of the project and key outputs', Regulatory Rapporteur, Vol. 11(9), pp. 4-9.

Grave K., Torren-Edo J., Muller A., Greko C., Moulin G., Mackay D., ESVAC Group, 'Variations in the sales and sales patterns of veterinary antimicrobial agents in 25 European countries', J Antimicrob Chemother, Vol. 69(8), pp. 2284-2291.

Hampson L. V., Herold R., Posch M., Saperia J., Whitehead A., 'Bridging the gap: A review of dose-investigations in paediatric investigation plans', Br J Clin Pharmacol, Vol. 78(4), pp. 898–907.

Hanaizi Z., Unkrig C., Enzmann H., Camarero J., Sancho-Lopez A., Salmonson T., Gisselbrecht C., Laane E., Pignatti F., 'The European Medicines Agency review of bosutinib for the treatment of adult patients with chronic myelogenous leukaemia: Summary of the scientific assessment of the Committee for Medicinal Products for Human Use', Oncologist., Vol.19(4), pp. 421-5.

Herold R., Saint-Raymond A., Norga K., 'Too few medicines for children with cancer', JAMA Pediatr., Vol.168(6), p. 583.

Hill D. L., Schwarz A. J., Isaac M., Pani L., Vamvakas S., Hemmings R., Carrillo M. C., Yu P., Sun J., Beckett L., Boccardi M., Brewer J., Brumfield M., Cantillon M., Cole P. E., Fox N., Frisoni G. B., Jack C., Kelleher T., Luo F., Novak G., Maguire P., Meibach R., Patterson P., Bain L., Sampaio C., Raunig D., Soares H., Suhy J., Wang H., Wolz R., Stephenson D., 'Coalition Against Major Diseases / European Medicines Agency biomarker qualification of hippocampal volume for enrichment of clinical trials in predementia stages of Alzheimer's disease', Alzheimers Dement, Vol. 10(4), pp. 421-429.

Karres J, Pratt V, Guettier JM, Temeck J, Tamborlane WV, Dunger D, Bejnariu C, De Beaufort C, Tomasi P, Joining forces: a call for greater collaboration to study new medicines in children and adolescents with type 2 diabetes, Diabetes Care. 2014 Oct;37(10):2665-7

Kurki P., Bielsky M.C.; Working Party on Similar Biological (Biosimilar) Medicinal Products (BMWP) of Committee for Medicinal Products for Human Use (CHMP), 'Letter to the editor: ECCO position challenged by European drug regulators', J Crohns Colitis. 2014 Mar;8(3):258

Lincker H., Ziogas C., Carr M., Porta N., Eichler, H. G., 'Regulatory watch: Where do new medicines originate from in the EU?', Nat Rev Drug Discov, Vol. 13(2), pp. 92-93.

Mackay D., Grave K., Muller A., Torren-Edo J., 'Key success factors for surveys of antimicrobial sales and use in the European Union', in proceeding: 'Responsible and prudent use of antimicrobial agents for animals. International solidarity to fight against antimicrobial resistance', OIE Global Conference, Paris, France, 13-15 March 2013, pp. 28-31.

Maignen F, Hauben M, Hung E, Holle LV, Dogne JM, A conceptual approach to the masking effect of measures of disproportionality, Pharmacoepidemiol Drug Saf. 2014 Feb;23(2):208-17

Mariz S., Tsigkos S., Fregonese L., Aarum S., Dehlink R., Llinares J., Sepodes B., 'The orphan framework as a new opportunity: an expert opinion', Informa healthcare, Vol. 2(11 ), pp. 1181-1186.

Mentzer D., Desfontaine E., Tomasi P. A., 'The importance and challenge of pediatric trials of hemophilia drugs', Nat Med., doi:10.1038/nm.3546

Narayan G, Cossu G, Galli MC, Flory E, Ovelgonne H, Salmikangas P, Schneider CK, Trouvin JH, Clinical Development of Gene Therapy Needs a Tailored Approach: A Regulatory Perspective from the European Union, Hum Gene Ther Clin Dev. 2014 Mar;25(1):1-6

Nofziger C., Papaluca M., Terzic A., Waldman S., Paulmichl M., 'Policies to aid the adoption of personalized medicine', Nat Rev Drug Discov, Vol. 13(3), pp. 159-160.

Pacurariu A.C., Coloma P. M., van Haren A., Genov G., Sturkenboom M. C., Straus S. M., 'A description of signals during the first 18 months of the EMA Pharmacovigilance Risk Assessment Committee', Drug Saf., Vol. 37(12), pp.1059-66.

Peschel W., 'The use of community herbal monographs to facilitate registrations and authorisations of herbal medicinal products in the European Union 2004-2012', J Ethnopharmacol, doi: 10.1016/j.jep

Petricciani J, Sheets R, Griffiths E, Knezevic I. Written with contributions from EMA staff Trullas and expert Fauconnier. No co-authors, so not included in AR, Adventitious agents in viral vaccines: Lessons learned from 4 case studies, Biologicals. 2014 Sep;42(5):223-36    

Pfleiderer M., Trouvin J. H., Brasseur D., Gränstrom M., Shivji R., Mura M., Cavaleri M., 'Brief Report for Vaccine - Summary of knowledge gaps related to quality and efficacy of current influenza vaccines', Vaccine., Vol 32(35), pp. 4586-91.

Pignatti F., Ehmann F., Hemmings R., Jonsson B., Nuebling M., Papaluca-Amati M., Posch M., Rasi G., 'Cancer drug development and the evolving regulatory framework for companion diagnostics in the European Union', Clin Cancer Res. Vol. 20(6), pp.1458-68.

Quijano Ruiz B., Desfontaine E., Arenas-López S., Wang S., 'Pediatric formulation issues identified in paediatric investigation plans', Expert Rev Clin Pharmacol, Vol. 7(1), pp. 25-30.

Requena G., Abbing-Karahagopian V., Huerta C., De Bruin M. L., Alvarez Y., Miret M., Hesse U., Gardarsdottir H., Souverein P. C., Slattery J., Schneider C., Rottenkolber M., Schmiedl S., Gil M., De Groot M. C., Bate A., Ruigómez A., García Rodríguez L. A., Johansson S., de Vries F., Montero D., Schlienger R., Reynolds R., Klungel O. H., de Abajo F. J., 'Incidence rates and trends of hip/femur fractures in five European countries: comparison using e-healthcare records databases', Calcif Tissue Int, Vol. 94(6), pp. 580-589.

Sabaté M, Pacheco JF, Ballarín E, Ferrer P, Petri H, Hasford J, Schoonen MW, Rottenkolber M, Fortuny J, Laporte JR, Ibáñez L1; PROTECT Work Package 2, A compilation of research working groups on drug utilisation across Europe, BMC Res Notes. 2014 Mar 13;7:143. doi: 10.1186/1756-0500-7-143.

Selker H. P., Oye K. A., Eichler H. G., Stockbridge N. L., Mehta C. R., Kaitin K. I., McElwee N. E., Honig P. K., Erban J. K., D'Agostino R. B. 'A proposal for integrated efficacy-to-effectiveness (E2E) clinical trials', Clin Pharmacol Ther, Vol. 95(2), pp. 147-153.

Shankar G, Arkin S, Cocea L, Devanarayan V, Kirshner S, Kromminga A, Quarmby V, Richards S, Schneider CK, Subramanyam M, Swanson S, Verthelyi D, Yim S; American Association of Pharmaceutical Scientists, Assessment and Reporting of the Clinical Immunogenicity of Therapeutic Proteins and Peptides - Harmonized Terminology and Tactical Recommendations, AAPS J. 2014 Jul;16(4):658-73

Snyder PJ, Kahle-Wrobleski K, Brannan S, Miller DS, Schindler RJ, DeSanti S, Ryan JM, Morrison G, Grundman M, Chandler J, Caselli RJ, Isaac M, Bain L, Carrillo MC, Assessing Cognition and Function in Alzheimer's Disease Clinical Trials: Do We Have the Right Tools?, Alzheimers Dement. 2014 Nov;10(6):853-60 

Sun H., Vesely R., Nelson R. M., Taminiau J., Szitanyi P., Isaac M., Klein A., Uzu S., Griebel D., Mulberg A. E., International Inflammatory Bowel Disease Working Group, 'Steps toward harmonization for clinical development of medicines in pediatric ulcerative colitis-a global scientific discussion, part 2: data extrapolation, trial design, and pharmacokinetics', J Pediatr Gastroenterol Nutr, Vol. 58(6), pp. 684-688.

Sun H., Vesely R., Taminiau J., Szitanyi P., Papadopoulos E. J., Isaac M., Klein A., Uzu S., Griebel D., Mulberg A. E., International Inflammatory Bowel Disease Working Group, 'Steps toward harmonization for clinical development of medicines in pediatric ulcerative colitis-a global scientific discussion, part 1: efficacy endpoints and disease outcome assessments', J Pediatr Gastroenterol Nutr, Vol. 58(6), pp. 679-683.

Tomasi P, Success of European Commission's paediatric regulation is established, BMJ. 2014 Oct 14;349

Toussi M, Lis Y, Qizilbash N, Blake KV, Ehrenstein V, Kurz X, Moore N, Sinclair M, Encepp-HTA Working Group Survey on Capacity To Conduct Research in Support Of Health Technology Assessment, Value Health. 2014 Nov;17(7):A440. doi: 10.1016/j.jval.2014.08.1151. Epub 2014 Oct 26.   

Tsiftsoglou AS, Trouvin JH, Calvo G, Ruiz S, Demonstration of Biosimilarity, Extrapolation of Indications and Other Challenges Related to Biosimilars in Europe, BioDrugs. 2014 Dec;28(6):479-86

Tsigkos S., Llinares J., Mariz S., Aarum S., Fregonese L., Dembowska-Baginska B., Elbers R., Evers P., Foltanova T., Lhoir A., Corrêa-Nunes A., O'Connor D., Voordouw A., Westermark K., Sepodes B., 'Use of biomarkers in the context of orphan medicines designation in the European Union', Orphanet J Rare Dis, Vol. 9(1), 13.

Tsigkos S., Mariz S., Llinares J., Fregonese L., Aarum S., Naumann-Winter F., Westermark K., Sepodes B., 'Establishing medical plausibility in the context of orphan medicines designation in the European Union', Orphanet Journal of Rare Diseases, Vol. 9: 175.

Tzogani K, Nagercoil N, Hemmings RJ, Samir B, Gardette J, Demolis P, Salmonson T, Pignatti F, The European Medicines Agency approval of ingenol mebutate (Picato) for the cutaneous treatment of non hyperkeratotic, non hypertrophic actinic keratosis in adults: Summary of the scientific assessment of the Committee for Medicinal Products for Human Use (CHMP), Eur J Dermatol. 2014 Jul-Aug;24(4)

The Expert Panel on Therapeutic Products for Infants, Children, and Youth, 'Improving Medicines for Children in Canada', the Council of Canadian Academies.

van Aerts L. A., De Smet K., Reichmann G., Willem van der Laan J., Schneider C. K., 'Biosimilars entering the clinic without animal studies: A paradigm shift in the European Union', MAbs 6(5), pp. 1155-1162.

van Riet-Nales D. A., Römkens E. G., Saint-Raymond A., Kozarewicz P., Schobben A. F., Egberts T. C., Rademaker C. M., 'Oral medicines for children in the European paediatric investigation plans', PLoS One., Vol. 9(6), p. e98348

Vetter T., GFR Decline as an End Point for Clinical Trials in CKD: A View From Europe, Am J Kidney Dis, Vol. 64(6), pp. 838-840.

Vermeer NS, Duijnhoven RG, Straus SM, Mantel-Teeuwisse AK, Arlett PR, Egberts AC, Leufkens HG, De Bruin ML, Risk management plans as tool for proactive pharmacovigilance: a cohort study of newly approved drugs in Europe, Clin Pharmacol Ther. 2014 Dec;96(6):723-31. doi: 10.1038/clpt.2014.184. Epub 2014 Sep 15.


Baird L. G., Trusheim M. R., Eichler H. G., Berndt E. R., Hirsch G., 'Comparison of stakeholder metrics for traditional and adaptive development and licensing approaches to drug development', Therapeutic Innovation & Regulatory Science, Vol. 47(4), pp. 474-483.

Barkholt L., Flory E., Jekerle V., Lucas-Samuel S., Ahnert P., Bisset L., Büscher D., Fibbe W., Foussat A., Kwa M., Lantz O., Mačiulaitis R., Palomäki T., Schneider C. K., Sensebé L., Tachdjian G., Tarte K., Tosca L., Salmikangas P., 'Risk of tumorigenicity in mesenchymal stromal cell-based therapies - bridging scientific observations and regulatory viewpoints', Cytotherapy, Vol. 15(7), pp. 753-759.

Berntgen M., Enzmann H., Schabel E., Prieto Yerro C., Gómez-Outes A., Salmonson T., Musaus J., 'Linaclotide for treatment of irritable bowel syndrome - the view of European regulators', Dig Liver Dis, Vol. 45(9), pp. 724–726.

Berrut G., Andrieu S., Araujo de Carvalho I., Baeyens J. P., Bergman H., Cassim B., Cerreta F., Cesari M., Cha H. B., Chen L. K., Cherubini A., Chou M. Y., Cruz-Jentoft A. J., De Decker L., Du P., Forette B., Forette F., Franco A., Guimaraes R., Guttierrez-Robledo L. M., Jauregui J., Khavinson V., Lee W. J., Peng L. N., Perret-Guillaume C., Petrovic M., Retornaz F., Rockwood K., Rodriguez-Manas L., Sieber C., Spatharakis G., Theou O., Topinkova E., Vellas B., Benetos A., 'Promoting access to innovation for frail old persons. IAGG (International Association of Gerontology and Geriatrics), WHO (World Health Organization) and SFGG (Société Française de Gériatrie et de Gérontologie) Workshop - Athens January 20-21, 2012 Tool (GFST)', J Nutr Health Aging, Vol. 17(8), pp. 688-693.

Beyer A. R., Fasolo B., Phillips L. D., de Graeff P. A., Hillege H. L., 'Risk perception of prescription drugs: results of a survey among experts in the European regulatory network', Med Decis Making, Vol. 33(4), pp. 579-592.

Blackburn SCF, Arlett PR, Benefit Risk Management Plans?, Pharmaceutical Medicine. 2013;27(1):5-8

Brassart, L., Skarlatos A., Papaluca M., Camanas Saez L., Cerreta F., 'Product information and older people: how to assist informed prescription and safe therapeutic use?', Regulatory Rapporteur, Vol. 10(7/8).

Breckenridge A., Eichler H. G., 'Towards a prevention model of health care', Nat Rev Drug Discov, Vol. 2(8), pp. 563-564.

Butlen-Ducuing F., Guelfi J. D., 'Méthodes d'étude chez l'homme', in: Les antidépresseurs, Lavoisier, January 2013.

Callréus T., Schneider C. K., 'The emergence of regulatory science in pharmaceutical medicine', Pharm Med, Vol. 27(6), pp. 345-351.

da Rocha Dias S., Salmonson T., van Zwieten-Boot B., Jonsson B., Marchetti S., Schellens J. H., Giuliani R., Pignatti F., 'The European Medicines Agency review of vemurafenib (Zelboraf®) for the treatment of adult patients with BRAF V600 mutation-positive unresectable or metastatic melanoma: summary of the scientific assessment of the Committee for Medicinal Products for Human Use', Eur J Cancer, Vol. 49(7), pp. 1654-1661.

Egger G. F., Herold R., Rodriguez A., Manent N., Sweeney F., Saint-Raymond A., 'European Union Clinical Trials Register: on the way to more transparency of clinical trial data', Expert Rev Clin Pharmacol, Vol. 6(5), pp. 457-459.

Ehmann F., Papaluca Amati M., Salmonson T., Posch M., Vamvakas S., Hemmings R., Eichler H. G., Schneider C. K., 'Gatekeepers and enablers: how drug regulators respond to a challenging and changing environment by moving toward a proactive attitude', Clin Pharmacol Ther, Vol. 93(5), pp. 425–432.

Ehmann F., Sakai-Kato K., Duncan R., Hernán Pérez de la Ossa D., Pita R., Vidal J. M., Kohli A., Tothfalusi L., Sanh A., Tinton S., Robert J. L., Silva Lima B., Papaluca Amati M., 'Next-generation nanomedicines and nanosimilars: EU regulators' initiatives relating to the development and evaluation of nanomedicines', Nanomedicine (Lond), Vol. 8(5), pp. 849-856.

Eichler H. G., Bloechl-Daum B., Brasseur D., Breckenridge A., Leufkens H., Raine J., Salmonson T., Schneider C. K., Rasi G., 'The risks of risk aversion in drug regulation', Nat Rev Drug Discov, Vol. 12(12), pp. 907-916.

Eichler H. G., Pétavy F., Pignatti F., Rasi G. 'Access to patient-level trial data - a boon to drug developers', N Engl J Med, Vol. 369(17), pp. 1577-1579.

Elmgren L., Li X., Wilson C., Ball R., Wang J., Cichutek K., Pfleiderer M., Kato A., Cavaleri M., Southern J., Jivapaisarnpong T., Minor P., Griffiths E., Sohn Y., Wood D., 'A global regulatory science agenda for vaccines', Vaccine, Vol. 31 Suppl 2, pp. B163-B175.

Franco P., 'Orphan drugs: the regulatory environment', Drug Discov Today, Vol. 18(3-4), pp. 163-172.

Gravanis I., 'Geriatric oncology: European Union regulatory perspectives and initiatives', J Geriatr Oncol, Vol. 4(2), pp. 202-204.

Gravanis I., Lopez A. S., Hemmings R. J., Jiménez J. C., Garcia-Carbonero R., Gallego I. G., Giménez E. V., O'Connor D., Giuliani R., Salmonson T., Pignatti F., 'The European Medicines Agency review of abiraterone for the treatment of metastatic castration-resistant prostate cancer in adult men after docetaxel chemotherapy and in chemotherapy-naive disease: summary of the scientific assessment of the Committee for Medicinal Products for Human Use', Oncologist, Vol. 18(9), pp. 1032-1042.

Henshall C., Sansom L., Eichler H.-G., Lemgruber A., Longson C., O'Rourke B., Tunis S., 'Understanding the role and evidence expectations of health technology assessment and coverage/payer bodies. What are they looking for, and how and why does this differ from what regulators require?', Therapeutic Innovation & Regulatory Science, 28 November 2013.

Hilger A., Arras-Reiter C., Keller-Stanislawski B., Ljungberg B., Male C., Mentzer D., Seitz R., Silvester G., 'Comment on: Mannucci, P. M. Evolution of the European guidelines for the clinical development of factor VIII products', Haemophilia, Vol. 19(3), pp. 349-350.

Janssens E., 'Pharmaceuticals: EU initiative to tackle medication errors – Proposals and challenges', Eur J Risk Regulation, Vol. 3/2013, pp. 388-391.

Karres J., Tomasi P., 'New medicines for type 2 diabetes in adolescents: many products, few patients', Expert Rev Clin Pharmacol, Vol. 6(3), pp. 227-229.

Köhne C. H., Bedenne L., Carrato A., Bouché O., Popov I., Gaspà L., Valladares M., Rougier P., Gog C., Reichardt P., Wils J., Pignatti F., Biertz F., 'A randomised phase III intergroup trial comparing high-dose infusional 5-fluorouracil with or without folinic acid with standard bolus 5-fluorouracil/folinic acid in the adjuvant treatment of stage III colon cancer: the Pan-European Trial in Adjuvant Colon Cancer 2 study', Eur J Cancer, Vol. 49(8), pp. 1868-1875.

Liberti L., McAuslane N., Patel P., Breckenridge A., Eichler H. G., Peterson R., 'Regulatory review: How do agencies ensure the quality of decision making?', Clin Pharmacol Ther, Vol. 94(3), pp. 305-308.

Lionetti G., Kimura Y., Schanberg L. E., Beukelman T., Wallace C. A., Ilowite N. T., Winsor J., Fox K., Natter M., Sundy J. S., Brodsky E., Curtis J. R., Del Gaizo V., Iyasu S., Jahreis A., Meeker-O'Connell A., Mittleman B. B., Murphy B. M., Peterson E. D., Raymond S. C., Setoguchi S., Siegel J. N., Sobel R. E., Solomon D., Southwood T. R., Vesely R., White P. H., Wulffraat N. M., Sandborg C. I., 'Using registries to identify adverse events in rheumatic diseases', Pediatrics, Vol. 132(5), e1384-e1394.

Maignen F., Hauben M., Hung E., Holle L. V., Dogne J. M., 'A conceptual approach to the masking effect of measures of disproportionality', Pharmacoepidemiol Drug Saf, Vol. 23(2), pp. 208-217.

Maignen F., Hauben M., Hung E., Van Holle L., Dogne J. M., 'Assessing the extent and impact of the masking effect of disproportionality analyses on two spontaneous reporting systems databases', Pharmacoepidemiol Drug Saf, Vol. 23(2), pp. 195-207.

Maliepaard M., Nofziger C., Papaluca M., Zineh I., Uyama Y., Prasad K., Grimstein C., Pacanowski M., Ehmann F., Dossena S., Paulmichl M., 'Pharmacogenetics in the evaluation of new drugs: a multiregional regulatory perspective', Nat Rev Drug Discov, Vol. 12(2), pp. 103-115.

Manolis E., Rohou S., Hemmings R., Salmonson T., Karlsson M., Milligan P. A.. 'The role of modeling and simulation in development and registration of medicinal products: Output from the EFPIA / EMA Modeling and Simulation Workshop', CPT Pharmacometrics Syst Pharmacol, Vol. 2, e31.

Marshall S. F., Hemmings R., Josephson F., Karlsson M. O., Posch M., Steimer J. L., 'Modeling and simulation to optimize the design and analysis of confirmatory trials, characterize risk-benefit, and support label claims', CPT Pharmacometrics Syst Pharmacol, Vol. 2, e27.

Melchiorri D., Pani L., Gasparini P., Cossu G., Ancans J., Borg J. J., Drai C., Fiedor P., Flory E., Hudson I., Leufkens H. G., Müller-Berghaus J., Narayanan G., Neugebauer B., Pokrotnieks J., Robert J. L., Salmonson T., Schneider C. K., 'Regulatory evaluation of Glybera in Europe - two committees, one mission', Nat Rev Drug Discov, Vol. 12(9), p. 719.

Péan E., Flores B., Hudson I., Sjöberg J., Dunder K., Salmonson T., Gisselbrecht C., Laane E., Pignatti F., 'The European Medicines Agency review of pixantrone for the treatment of adult patients with multiply relapsed or refractory aggressive non-Hodgkin's B-cell lymphomas: summary of the scientific assessment of the Committee for Medicinal Products for Human Use', Oncologist, Vol. 18(5), pp. 625-633.

Phillips L. D., Fasolo B., Zafiropoulous N., Eichler H. G., Ehmann F., Jekerle V., Kramarz P., Nicoll A., Lönngren T., 'Modelling the risk-benefit impact of H1N1 influenza vaccines', Eur J Public Health, Vol. 23(4), pp. 674-678.

Posch M, Comment to: Group sequential tests for delayed responses, Hampson, Lisa V., and Christopher Jennison. "Group sequential tests for delayed responses (with discussion)." Journal of the Royal Statistical Society: Series B (Statistical Methodology) 75.1 (2013): 3-54.

Putzeist M., Mantel-Teeuwisse A. K., Llinares J., Gispen-De Wied C. C., Hoes A. W., Leufkens H. G., 'EU marketing authorization review of orphan and non-orphan drugs does not differ', Drug Discov Today, Vol. 18(19–20), pp. 1001–1006.

Ruperto N., Vesely R., Saint-Raymond A., Martini A., 'Impact of the European paediatric legislation in paediatric rheumatology: past, present and future', Ann Rheum Dis, Vol. 72(12), pp.1893-1896.

Saint Raymond A, Llinares J, Orphan medicines: a success with a challenging future, Expert Opinion on Orphan Drugs, 2013 1:3, 185-187

Schneider CK, 'The ethics of biosimilars', GaBI J, Vol. 2(1), pp. 6-7.

Schneider CK, Biosimilars in rheumatology: the wind of change, Ann Rheum Dis. 2013 Mar;72(3):315-8

Slattery J., Alvarez Y., Hidalgo A., 'Choosing thresholds for statistical signal detection with the proportional reporting ratio', Drug Saf, Vol. 36(8), pp. 687-692.

Staab A., Rook E., Maliepaard M., Aarons L., Benson C., 'Modeling and simulation in clinical pharmacology and dose finding', CPT Pharmacometrics Syst Pharmacol, Vol. 2, e29.

Stephenson D., Aviles E., Bain L. J., Brumfield M., Carrillo M., Comery T. A., Compton C., Corrigan B., Forrest Gordon M., Jack C. R., Katz R., Logovinsky V., Satlin A., Marek K., Nicholas T., Polhamus D., Angersbach B. S., Raghavan N., Romano G., Romero K., Shaw L., Woodcock J., Vradenburg G., Isaac M., 'Coalition Against Major Diseases:
precompetitive collaborations and regulatory paths to accelerating drug development for neurodegenerative diseases
', Therapeutic Innovation & Regulatory Science, Vol. 47(6), pp. 632-638.

Tomasi P., 'Paediatric clinical trials: need for capacity building', J Clin Stud, Vol. 5(3), pp. 10-16.

Tomasi P., Saint-Raymond A., 'Scope of the PIP in the EU: easier accessibility to the paediatric rewards and simplification of administrative requirements', Scrip Regulatory Affairs, January 2013, p. 5.

Tsiftsoglou AS, Ruiz S, Schneider CK, Development and regulation of biosimilars: current status and future challenges, BioDrugs. 2013 Jun;27(3):203-11

Tzogani K., Straube M., Hoppe U., Kiely P., O'Dea G., Enzmann H., Salmon P., Salmonson T., Pignatti F., 'The European Medicines Agency approval of 5-aminolaevulinic acid (Ameluz) for the treatment of actinic keratosis of mild to moderate intensity on the face and scalp: Summary of the scientific assessment of the Committee for Medicinal Products for Human Use', J Dermatolog Treat, Vol. 25(5), pp. 371-374.

van Riet Nales D. A., Kozarewicz P., Wang S., Saint-Raymond A., Robert J. L., 'Comments on the EMA draft guideline: final steps towards a harmonized view between regulators and industry', Int J Pharm, Vol. 457(1), pp. 337-339.

Vaquer G., Rivière F., Mavris M., Bignami F., Llinares-Garcia J., Westermark K., Sepodes B., 'Animal models for metabolic, neuromuscular and ophthalmological rare diseases', Nat Rev Drug Discov, Vol. 12(4), pp. 287-305.

Vermeer N. S., Straus S. M., Mantel-Teeuwisse A. K., Domergue F., Egberts T. C., Leufkens H. G., De Bruin M. L., 'Traceability of biopharmaceuticals in spontaneous reporting systems: a cross-sectional study in the FDA Adverse Event Reporting System (FAERS) and EudraVigilance databases', Drug Saf, Vol. 36(8), pp. 617-625.

Vestergaard H. T., D'Apote L., Schneider C. K., Herberts C., 'The evolution of nonclinical regulatory science: advanced therapy medicinal products as a paradigm', Mol Ther, Vol. 21(9), pp. 1644-1648.

Vidal J. M., 'Non-clinical science and regulatory approach. An introduction', Toxicologische Communicatie, Data en Documentatie, Vol. 3, pp. 14-15.

Visser S. A., Manolis E., Danhof M., Kerbusch T., 'Modeling and simulation at the interface of nonclinical and early clinical drug development', CPT Pharmacometrics Syst Pharmacol, Vol. 2, e30.


Bahri P., Harrison-Woolrych M., 'Focusing on risk communication about medicines: why now?', Drug Saf, Vol. 35(11), pp. 971-975.

Bahri P., Harrison-Woolrych M., 'How to improve communication for the safe use of medicines? Discussions on social marketing and patient-tailored approaches at the annual meetings of the WHO Programme for International Drug Monitoring', Drug Saf, Vol. 35(12), pp. 1073-1079.

Blake K. V., Devries C. S., Arlett P., Kurz X., Fitt H., 'Increasing scientific standards, independence and transparency in post-authorisation studies: the role of the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance', Pharmacoepidemiol Drug Saf, Vol. 21(7), pp. 690-696.

Blake KV, Moore N, Kurz X, ENCePP, Making available observational study protocols and results: the role of ENCePP, Circulation: Cardiovascular Quality and Outcomes. Reply to: 2012;5:4 418-419

Butlen-Ducuing F., Haas M., Pani L., van Zwieten-Boot B., Broich K., 'DSM-5 and clinical trials in psychiatry: challenges to come?', Nat Rev Drug Discov, Vol. 11(8), pp. 583-584.

Calderon M. A., Gerth van Wijk R., Eichler I., Matricardi P. M., Varga E. M., Kopp M. V., Eng P., Niggemann B., Nieto A., Valovirta E., Eigenmann P. A., Pajno G., Bufe A., Halken S., Beyer K., Wahn U., European Academy of Allergy and Clinical Immunology, 'Perspectives on allergen-specific immunotherapy in childhood: an EAACI position statement', Pediatr Allergy Immunol, Vol. 23(4), pp. 300-306.

Cerreta F., Eichler H. G., Rasi G., 'Drug policy for an aging population — the European Medicines Agency's geriatric medicines strategy', N Engl J Med, Vol. 367(21), pp. 1972-1974.

Cesari M., Fielding R. A., Pahor M., Goodpaster B., Hellerstein M., van Kan G. A., Anker S. D., Rutkove S., Vrijbloed J. W., Isaac M., Rolland Y., M'rini C., Aubertin-Leheudre M., Cedarbaum J. M., Zamboni M., Sieber C. C., Laurent D., Evans W. J., Roubenoff R., Morley J. E., Vellas B., International Working Group on Sarcopenia, 'Biomarkers of sarcopenia in clinical trials-recommendations from the International Working Group on Sarcopenia', J Cachexia Sarcopenia Muscle, Vol. 3(3), pp. 181-190.

Eichler H. G., Abadie E., Baker M., Rasi G., 'Fifty years after thalidomide; what role for drug regulators?', Br J Clin Pharmacol, Vol. 74(5), pp. 731-733.

Eichler H. G., Abadie E., Breckenridge A., Leufkens H., Rasi G., 'Open clinical trial data for all? A view from regulators', PLoS Med, Vol. 9(4), e1001202.

Eichler H. G., Oye K., Baird L. G., Abadie E., Brown J., Drum C. L., Ferguson J., Garner S., Honig P., Hukkelhoven M., Lim J. C., Lim R., Lumpkin M. M., Neil G., O'Rourke B., Pezalla E., Shoda D., Seyfert-Margolis V., Sigal E. V., Sobotka J., Tan D., Unger T. F., Hirsch G., 'Adaptive licensing: taking the next step in the evolution of drug approval', Clin Pharmacol Ther, Vol. 91(3), pp. 426-437.

Galli MC, Long-term follow-up of cancer patients treated with gene therapy medicinal products, J Gene Med. 2012 Jun;14(6):440-2

Gaydos B., Koch A., Miller F., Posch M., Vandemeulebroecke M., Wang S.-J., 'Perspective on adaptive designs: 4 years European Medicines Agency reflection paper, 1 year draft US FDA guidance – where are we now?', Clin Invest, Vol. 2(3), pp. 235-240.

Gispen-de Wied C., Stoyanova V., Yu Y., Isaac M., Pani L., de Andres-Trelles F., 'The placebo arm in clinical studies for treatment of psychiatric disorders: a regulatory dilemma', Eur Neuropsychopharmacol, Vol. 22(11), pp. 804-811.

Grave K., Greko C., Kvaale M. K., Torren-Edo J., Mackay D., Muller A., Moulin G., ESVAC Group, 'Sales of veterinary antibacterial agents in nine European countries during 2005-09: trends and patterns', J Antimicrob Chemother, Vol. 67(12), pp. 3001-3008.

Grein K., 'Responsibilities of regulatory agencies in the marketing of antimicrobials', Rev Sci Tech, Vol. 31(1), pp. 289-298.

Hidalgo-Simon A., Arlett P., 'Pharmacovigilance in Europe: direction of travel in a changing environment', Expert Rev Clin Pharmacol, Vol. 5(5), pp. 485-488.

Isaac M., Pani L., Gispen-de Wied C., Koch A., 'European licensing of maintenance treatment in schizophrenia', Lancet, Vol. 380(9841), pp. 562-563.

Isai A., Durand J., Le Meur S., Hidalgo-Simon A., Kurz X., 'Autoimmune disorders after immunisation with Influenza A/H1N1 vaccines with and without adjuvant: EudraVigilance data and literature review', Vaccine, Vol. 30(49), pp. 7123-7129.

Klug B., Celis P., Carr M., Reinhardt J., 'Regulatory structures for gene therapy medicinal products in the European Union', In: Freeman T. (ed.), Gene transfer vectors for clinical application, United Kingdom, Academic Press, pp. 337-354.

Knöss W, Chinou I, Regulation of medicinal plants for public health--European community monographs on herbal substances, Planta Med. 2012 Aug;78(12):1311-6 

Lumpkin M. M., Eichler H. G., Breckenridge A., Hamburg M. A., Lönngren T., Woods K., 'Advancing the science of medicines regulation: the role of the 21st-century medicines regulator', Clin Pharmacol Ther, Vol. 92(4), pp. 486-493.

Maciulaitis R., D'Apote L., Buchanan A., Pioppo L., Schneider C. K., 'Clinical development of advanced therapy medicinal products in Europe: evidence that regulators must be proactive', Mol Ther, 20(3), pp. 479-482.

Maxwell S., Eichler H. G., Bucsics A., Haefeli W. E., Gustafsson L. L., e-SPC Consortium, 'e-SPC - delivering drug information in the 21st century: developing new approaches to deliver drug information to prescribers', Br J Clin Pharmacol, Vol. 73(1), pp. 12-15.

Mischak H., Ioannidis J. P., Argiles A., Attwood T. K., Bongcam-Rudloff E., Broenstrup M., Charonis A., Chrousos G. P., Delles C., Dominiczak A., Dylag T., Ehrich J., Egido J., Findeisen P., Jankowski J., Johnson R. W., Julien B. A., Lankisch T., Leung H. Y., Maahs D., Magni F., Manns M. P., Manolis E., Mayer G., Navis G., Novak J., Ortiz A., Persson F., Peter K., Riese H. H., Rossing P., Sattar N., Spasovski G., Thongboonkerd V., Vanholder R., Schanstra J. P., Vlahou A., 'Implementation of proteomic biomarkers: making it work', Eur J Clin Invest, Vol. 42(9), pp. 1027-1036.

Mullier F., Rahier J. F., Maignen F., Cornet Y., Graux C., Chatelain C., Chatelain B., Dogne J. M., 'A case of therapy-related myeloid neoplasm in a patient with Crohn's disease treated with azathioprine', Acta Haematol, Vol. 128(1), pp. 1-6.

Pean E., Demolis P., Moreau A., Hemmings R. J., O'Connor D., Brown D., Shepard T., Abadie E., Pignatti F., 'The European Medicines Agency review of cabazitaxel (Jevtana®) for the treatment of hormone-refractory metastatic prostate cancer: summary of the scientific assessment of the Committee for Medicinal Products for Human Use', Oncologist, Vol. 17(4), pp. 543-549.

Pean E., Klaar S., Berglund E. G., Salmonson T., Borregaard J., Hofland K. F., Ersbøll J., Abadie E., Giuliani R., Pignatti F., 'The European Medicines Agency review of eribulin for the treatment of patients with locally advanced or metastatic breast cancer: summary of the scientific assessment of the Committee for Medicinal Products for Human Use', Clin Cancer Res, Vol. 18(17), pp. 4491-4497.

Pelkonen O., Abass K., Wiesner J., 'Thujone and thujone-containing herbal medicinal and botanical products: toxicological assessment', Regul Toxicol Pharmacol, Vol. 65(1), pp. 100-107.

Pignatti F., Flores B., Jonsson B., 'Platelet response as the basis for approval in chronic immune (idiopathic) thrombocytopenic purpura (ITP): a regulatory perspective', Am J Hematol, Vol. 87, pp. 943-944.

Posch M., Proschan M. A., 'Unplanned adaptations before breaking the blind', Stat Med, Vol. 31(30), pp. 4146-4153.

Prieto L., Spooner A., Hidalgo-Simon A., Rubino A., Kurz X., Arlett P., 'Evaluation of the effectiveness of risk minimization measures', Pharmacoepidemiol Drug Saf, Vol. 21(8), pp. 896-899.

Putzeist M., Mantel-Teeuwisse A. K., Aronsson B., Rowland M., Gispen-de Wied C. C., Vamvakas S., Hoes A. W., Leufkens H. G., Eichler H. G., 'Factors influencing non-approval of new drugs in Europe', Nat Rev Drug Discov, Vol. 11(12), pp. 903-904.

Quigley K., 'Veterinary medicines: What is the MUMS / limited markets policy?', Regulatory Rapporteur, Vol. 10(2).

Saint-Raymond A., Herold R., 'Medicines for pediatric oncology: can we overcome the failure to deliver?', Expert Rev Clin Pharmacol, Vol. 5(5), pp. 493-495.

Sala Soriano E., Eichler I., Helms P. J., 'Joined-up thinking', European Pharmaceutical Contractor, March 2012, pp. 35-36.

Schneider C. K., Vleminckx C., Gravanis I., Ehmann F., Trouvin J. H., Weise M., Thirstrup S., 'Setting the stage for biosimilar monoclonal antibodies', Nat Biotechnol, Vol. 30(12), pp. 1179-1185.

Stegemann S., Baeyens J.-P., Cerreta F., Chanied E., Löfgren A., Maio M., Schreier G., Thesing-Bleck E., 'Adherence measurement systems and technology for medications in older patient populations', Eur Geriatr Med, Vol. 3(4), pp. 254-260.

Tomasi P., 'Writing applications for paediatric investigation plans and waivers', Medical Writing, Vol. 21(2), pp. 104-107.

van Riet-Nales D. A., Wang S., Saint-Raymond A., Robert J. L., 'The EMA quality guideline on the pharmaceutical development of medicines for paediatric use', Int J Pharm, Vol. 435(2), pp. 132-134.

Veselý R., Haf Davies E., 'Helping the medicine go down', Public Service Review: European Science & Technology, Vol. 14, pp. 1-2.

Videnova K., Mackay D. K., 'Availability of vaccines against major animal diseases in the European Union', Rev Sci Tech, Vol. 31(3), pp. 971-978.

Vinhas de Souza M., Keller-Stanislawski B., Blake K., Hidalgo-Simon A., Arlett P., Dal Pan G., 'Drug-induced PML: a global agenda for a global challenge', Clin Pharmacol Ther, Vol. 91(4), pp. 747-750.

Westermark K., Llinares J., 'Promoting the development of drugs against rare diseases: what more should be done?', Expert Rev Pharmacoecon Outcomes Res, Vol. 12(5), pp. 541-543.

Zafiropoulos N., Phillips L., Pignatti F., Luria X., 'Evaluating benefit–risk: an Agency perspective', Regulatory Rapporteur, Vol. 9(6), pp. 5-8.

Zehetmayer S., Posch M., 'False discovery rate control in two-stage designs', BMC Bioinformatics, Vol. 13, 81.

Zomerdijk I. M., Sayed-Tabatabaei F. A., Trifirò G., Blackburn S. C., Sturkenboom M. C., Straus S. M., 'Risk minimization activities of centrally authorized products in the EU: a descriptive study', Drug Saf, Vol. 35(4), pp. 299-314.


Arlett P.R., Kurz X., 'New approaches to strengthen pharmacovigilance', Drug Discov Today Technol, Vol. 8(1), e15–e19.

Arnardottir A. H., Haaijer-Ruskamp F. M., Straus S. M., Eichler H. G., de Graeff P. A., Mol P. G., 'Additional safety risk to exceptionally approved drugs in Europe?', Br J Clin Pharmacol, Vol. 72(3), pp. 490-499.

Bahri P., Mol P. G., Théophile H., Edwards I. R., Hugman B. P., 'Communication in drug safety: a report from an interactive debate held at the 10th annual meeting of the International Society of Pharmacovigilance (ISoP), 2010', Drug Saf, Vol. 34(10), pp. 881-882.

Blake K. V., Prilla S., Accadebled S., Guimier M., Biscaro M., Persson I., Arlett P., Blackburn S., Fitt H., 'European Medicines Agency review of post-authorisation studies with implications for the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance', Pharmacoepidemiol Drug Saf, Vol. 20(10), pp. 1021-1029.

Blake K. V., Smeraldi C., Kurz X., Arlett P., Blackburn S., Fitt H., 'The European Network of Centres for Pharmacoepidemiology and Pharmacovigilance: application to diabetes and vascular disease', British Journal of Diabetes & Vascular Disease, Vol. 11(6), pp. 304-307.

Büttel I. C., Chamberlain P., Chowers Y., Ehmann F., Greinacher A., Jefferis R., Kramer D., Kropshofer H., Lloyd P., Lubiniecki A., Krause R., Mire-Sluis A., Platts-Mills T., Ragheb J. A., Reipert B. M., Schellekens H., Seitz R., Stas P., Subramanyam M., Thorpe R., Trouvin J. H., Weise M., Windisch J., Schneider C. K., 'Taking immunogenicity assessment of therapeutic proteins to the next level', Biologicals, Vol. 39(2), pp. 100-109.

Carleer J., Karres J., 'Juvenile animal studies and pediatric drug development: a European regulatory perspective', Birth Defects Res B Dev Reprod Toxicol, Vol. 92(4), pp. 254-260.

De Mattia F., Chapsal J. M., Descamps J., Halder M., Jarrett N., Kross I., Mortiaux F., Ponsar C., Redhead K., McKelvie J., Hendriksen C., 'The consistency approach for quality control of vaccines - a strategy to improve quality control and implement 3Rs', Biologicals, Vol. 39(1), pp. 59-65.

Ehmann F., Schneider C. K., 'Biosimilar monoclonal antibodies: The implications', Hospital Pharmacy Europe, Vol. 56, May/June 2011.

Eichler H. G., Abadie E., Breckenridge A., Flamion B., Gustafsson L. L., Leufkens H., Rowland M., Schneider C. K., Bloechl-Daum B., 'Bridging the efficacy-effectiveness gap: a regulator's perspective on addressing variability of drug response', Nat Rev Drug Discov, Vol. 10(7), pp. 495-506.

Eichler I., Soriano E. S., 'Close collaboration between academia, industry and drug regulators is required in the development of allergen products for specific immunotherapy in children', Allergy, Vol. 66(8), pp. 999-1004.

Freire-Moran L., Aronsson B., Manz C., Gyssens I. C., So A. D., Monnet D. L., Cars O., ECDC-EMA Working Group, 'Critical shortage of new antibiotics in development against multidrug-resistant bacteria - time to react is now', Drug Resist Updat, Vol. 14(2), pp. 118-124.

Hanaizi Z., van Zwieten-Boot B., Calvo G., Lopez A. S., van Dartel M., Camarero J., Abadie E., Pignatti F., 'The European Medicines Agency review of ipilimumab (Yervoy) for the treatment of advanced (unresectable or metastatic) melanoma in adults who have received prior therapy: summary of the scientific assessment of the Committee for Medicinal Products for Human Use', Eur J Cancer, Vol. 48, pp. 237-242.

Hatzakis A., Wait S., Bruix J., Buti M., Carballo M., Cavaleri M., Colombo M., Delarocque-Astagneau E., Dusheiko G., Esmat G., Esteban R., Goldberg D., Gore C., Lok A. S., Manns M., Marcellin P., Papatheodoridis G., Peterle A., Prati D., Piorkowsky N., Rizzetto M., Roudot-Thoraval F., Soriano V., Thomas H. C., Thursz M., Valla D., van Damme P., Veldhuijzen I. K., Wedemeyer H., Wiessing L., Zanetti A. R., Janssen H. L., 'The state of hepatitis B and C in Europe: report from the hepatitis B and C summit conference', J Viral Hepat, Vol. 18 Suppl 1, pp. 1-16.

Heininger U., Bachtiar N. S., Bahri P., Dana A., Dodoo A., Gidudu J., Santos E. M., 'The concept of vaccination failure', Vaccine, Vol. 30(7), pp. 1265-1268.

Isaac M., Vamvakas S., Abadie E., Jonsson B., Gispen C., Pani L., 'Qualification opinion of novel methodologies in the predementia stage of Alzheimer's disease: cerebro-spinal-fluid related biomarkers for drugs affecting amyloid burden - regulatory considerations by European Medicines Agency focusing in improving benefit', Eur Neuropsychopharmacol, Vol. 21(11), pp. 781-788.

Karres J., Tomasi P., Saint-Raymond A., 'The development of pharmacological treatment of obesity in children. A European regulatory perspective', Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz, Vol. 54(5), pp. 570-576.

Korakianiti E., Rekkas D., 'Statistical thinking and knowledge management for quality-driven design and manufacturing in pharmaceuticals', Pharm Res, Vol. 28(7), pp. 1465-1479.

Kurz X., Domergue F., Slattery J., Segec A., Szmigiel A., Hidalgo-Simon A., 'Safety monitoring of influenza A/H1N1 pandemic vaccines in EudraVigilance', Vaccine, Vol. 29(26), pp. 4378-4387.

Leufkens H. G., Eichler H. G., 'Innovative methods in drug regulatory sciences', Drug Discov Today Technol, Vol. 8(1), e1-e2.

Leufkens H. G., Schellekens H., Aronsson B., 'Post-innovation innovation of medicinal products', Drug Discov Today Technol, Vol. 8(1), e37-e41.

Manolis E., Herold R., 'Pharmacometrics for regulatory decision making: status and perspective', Clin Pharmacokinet, Vol. 50(10), pp. 625-626.

Manolis E., Osman T. E., Herold R., Koenig F., Tomasi P., Vamvakas S., Saint-Raymond A., 'Role of modeling and simulation in pediatric investigation plans', Paediatr Anaesth, Vol. 21(3), pp. 214-221.

Manolis E., Vamvakas S., Isaac M., 'New pathway for qualification of novel methodologies in the European Medicines Agency', Proteomics Clin Appl, Vol. 5(5-6), pp. 248-255.

Mariz S., Llinares J., Westermark K., 'Orphan drugs. EU regulations', BMJ, Vol. 342, d136.

Matt P., van Zwieten-Boot B., Calvo Rojas G., Ter Hofstede H., Garcia-Carbonero R., Camarero J., Abadie E., Pignatti F, 'The European Medicines Agency review of tegafur / gimeracil / oteracil (Teysuno™) for the treatment of advanced gastric cancer when given in combination with cisplatin: summary of the scientific assessment of the Committee for Medicinal Products for Human Use', Oncologist, Vol. 16(10), pp. 1451-1457.

Müllner M., Eichler H.-G., 'Current issues in drug regulation', In: M. Müllner (ed.), Clinical Pharmacology: Current Topics and Case Studies, Springer Vienna, pp. 19-31.

Nieto M., Borregaard J., Ersbøll J., ten Bosch G. J., van Zwieten-Boot B., Abadie E., Schellens J. H., Pignatti F., 'The European Medicines Agency review of pazopanib for the treatment of advanced renal cell carcinoma: summary of the scientific assessment of the Committee for Medicinal Products for Human Use', Clin Cancer Res, Vol. 17(21), pp. 6608-6614.

Nieto M., Calvo G., Hudson I., Feldschreiber P., Brown D., Lee C. C., Lay G., Valeri A., Abadie E., Thomas A., Pignatti F., 'The European Medicines Agency review of eltrombopag (Revolade) for the treatment of adult chronic immune (idiopathic) thrombocytopenic purpura: summary of the scientific assessment of the Committee for Medicinal Products for Human Use', Haematologica, Vol. 96(9), e33-e40.

Olski T. M., Lampus S. F., Gherarducci G., Saint Raymond A., 'Three years of paediatric regulation in the European Union', Eur J Clin Pharmacol, Vol. 67(3), pp. 245-252.

Phillips L. D., Fasolo B., Zafiropoulos N., Beyer A., 'Is quantitative benefit-risk modelling of drugs desirable or possible?' Drug Discov Today Technol, Vol. 8(1), e3–e10.

Pignatti F., Gravanis I., Herold R., Vamvakas S., Jonsson B., Marty M., 'The European Medicines Agency: an overview of its mission, responsibilities, and recent initiatives in cancer drug regulation', Clin Cancer Res, Vol. 17(16), pp. 5220-5225.

Pignatti F., Hemmings R., Jonsson B., 'Is it time to abandon complete blinded independent central radiological evaluation of progression in registration trials?', Eur J Cancer, Vol. 47(12), pp. 1759-1762.

Pignatti F., Luria X., Abadie E., Eichler H. G., 'Regulators, payers, and prescribers: can we fill the gaps?', Lancet Oncol, Vol. 12(10), pp. 930-931.

Pirmohamed M., Friedmann P. S., Molokhia M., Loke Y. K., Smith C., Phillips E., La Grenade L., Carleton B., Papaluca-Amati M., Demoly P., Shear N. H., 'Phenotype standardization for immune-mediated drug-induced skin injury', Clin Pharmacol Ther, Vol. 89(6), pp. 896-901.

Putzeist M., Heemstra H. E., Llinares Garcia J., Mantel-Teeuwisse A. K., Gispen-De Wied C. C., Hoes A. W., Leufkens H. G., 'Determinants for successful marketing authorisation of orphan medicinal products in the EU', Drug Discov Today, Vol. 17(7-8), pp. 352-358.

Rahmner P. B., Eiermann B., Korkmaz S., Gustafsson L. L., Gruvén M., Maxwell S., Eichler H. G., Vég A., 'Physicians' reported needs of drug information at point of care in Sweden', Br J Clin Pharmacol, Vol. 73(1), pp. 115-125.

Raine J., Wise L., Blackburn S., Eichler H. G., Breckenridge A., 'European perspective on risk management and drug safety', Clin Pharmacol Ther, Vol. 89(5), pp. 650-654.

Rocchi F., Tomasi P., 'The development of medicines for children', Pharmacol Res, Vol. 64(3), pp. 169-175.

Ruperto N., Eichler I., Herold R., Vassal G., Giaquinto C., Hjorth L., Valls-i-Soler A., Peters C., Helms P. J., Saint Raymond A., 'A European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA)', Arch Dis Child, Vol. 97(3), pp. 185-188.

Salmikangas P., Celis P., 'Current challenges in the development of novel cell-based medicinal products', Regulatory Rapporteur, Vol. 8(7-8), pp. 4-7.

Silva Lima B., 'EMA responds to criticism of reflection paper on non-clinical studies for generic nano-particle iron medicinal product applications', Scrip Regulatory Affairs, 12 August 2011.

Vamvakas S., Martinalbo J., Pita R., Isaac M., 'On the edge of new technologies (advanced therapies, nanomedicines)', Drug Discov Today Technol, Vol. 8(1), e21-e28.

Vleminckx C., Ehmann F., 'Guidelines EMA pour le développement de médicaments biosimilaires', Oncologie, Vol. 13(5), pp. 191-195.

Voltz-Girolt C., Celis P., Boucaumont M., D'Apote L., Pinheiro M. H., Papaluca-Amati M., 'The advanced therapy classification procedure. Overview of experience gained so far', Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz, Vol. 54(7), pp. 811-815.

Weise M., Bielsky M. C., De Smet K., Ehmann F., Ekman N., Narayanan G., Heim H. K., Heinonen E., Ho K., Thorpe R., Vleminckx C., Wadhwa M., Schneider C. K., 'Biosimilars - Why terminology matters', Nat Biotechnol, Vol. 29(8), pp. 690-693.

Westermark K., Holm B. B., Söderholm M., Llinares-Garcia J., Rivière F., Aarum S., Butlen-Ducuing F., Tsigkos S., Wilk-Kachlicka A., N'Diamoi C., Borvendég J., Lyons D., Sepodes B., Bloechl-Daum B., Lhoir A., Todorova M., Kkolos I., Kubáčková K., Bosch-Traberg H., Tillmann V., Saano V., Héron E., Elbers R., Siouti M., Eggenhofer J., Salmon P., Clementi M., Krieviņš D., Matulevičiene A., Metz H., Vincenti A. C., Voordouw A., Dembowska-Bagińska B., Nunes A. C., Saleh F. M., Foltánová T., Možina M., Torrent i Farnell J., Beerman B., Mariz S., Evers M. P., Greene L., Thorsteinsson S., Gramstad L., Mavris M., Bignami F., Lorence A., Belorgey C., 'European regulation on orphan medicinal products: 10 years of experience and future perspectives', Nat Rev Drug Discov, Vol. 10(5), pp. 341-349.

Wirth S., Kelly D., Sokal E., Socha P., Mieli-Vergani G., Dhawan A., Lacaille F., Saint-Raymond A., Olivier S., Taminiau J., 'Guidance for clinical trials for children and adolescents with chronic hepatitis C', J Pediatr Gastroenterol Nutr, Vol. 52(2), pp. 233-237.


Alvarez Y., Hidalgo A., Maignen F., Slattery J., 'Validation of statistical signal detection procedures in EudraVigilance post-authorization data: a retrospective evaluation of the potential for earlier signalling', Drug Saf, Vol. 33(6), pp. 475-487.

Bahri P., 'Public pharmacovigilance communication: a process calling for evidence-based, objective-driven strategies', Drug Saf, Vol. 33(12), pp. 1065-1079.

Bauer P., Koenig F., Brannath W., Posch M., 'Selection and bias - two hostile brothers', Stat Med, Vol. 29(1), pp. 1-13.

Blind E., Dunder K., de Graeff P. A., Abadie E., 'Rosiglitazone: a European regulatory perspective', Diabetologia, Vol. 54(2), pp. 213-218.

Bloechl-Daum B., Butlen-Ducuing F., Llinares-Garcia J., 'Special situations, market fragmentation: orphan drugs for rare diseases' in: Clinical pharmacology: current topics and case studies, Vienna, New York, Springer.

Brosch S., Spina A., 'Pharmaceuticals: International Conference on Harmonisation and Standardisation initiatives in the pharmaceutical domain', Eur J Risk Regulation, Vol. 3, pp. 274-276.

Butlen-Ducuing F., Rivière F., Aarum S., Llinares-Garcia J., 'European Medicines Agency support mechanisms fostering orphan drug development', Drug News Perspect, Vol. 23(1), pp. 71-81.

Butlen-Ducuing F., Tsigkos S. 'Ten years of orphan drug designation in Europe', EHJP Practice, Vol. 16(4), pp. 19-20.

Davies E. H., Ollivier C. M., Saint-Raymond A., 'Paediatric investigation plans for pain: painfully slow!', Eur J Clin Pharmacol, Vol. 66(11), pp. 1091-1097.

Del Seppia I., 'Lost in translation': scientific names of medicine in a globalized world', Eur J Risk Regulation, Vol. 4, pp. 431-435.

Dieterle F., Sistare F., Goodsaid F., Papaluca M., Ozer J. S., Webb C. P., Baer W., Senagore A., Schipper M. J., Vonderscher J., Sultana S., Gerhold D. L., Phillips J. A., Maurer G., Carl K., Laurie D., Harpur E., Sonee M., Ennulat D., Holder D., Andrews-Cleavenger D., Gu Y. Z., Thompson K. L., Goering P. L., Vidal J. M., Abadie E., Maciulaitis R., Jacobson-Kram D., Defelice A. F., Hausner E. A., Blank M., Thompson A., Harlow P., Throckmorton D., Xiao S., Xu N., Taylor W., Vamvakas S., Flamion B., Lima B. S., Kasper P., Pasanen M., Prasad K., Troth S., Bounous D., Robinson-Gravatt D., Betton G., Davis M. A., Akunda J., McDuffie J. E., Suter L., Obert L., Guffroy M., Pinches M., Jayadev S., Blomme E. A., Beushausen S. A., Barlow V. G., Collins N., Waring J., Honor D., Snook S., Lee J., Rossi P., Walker E., Mattes W., 'Renal biomarker qualification submission: a dialog between the FDA-EMEA and Predictive Safety Testing Consortium', Nat Biotechnol, Vol. 28(5), pp. 455–462.

Eichler H. G., Aronsson B., Abadie E., Salmonson T., 'New drug approval success rate in Europe in 2009', Nat Rev Drug Discov, Vol. 9(5), pp. 355-356.

Eichler H. G., Bloechl-Daum B., Abadie E., Barnett D., König F., Pearson S., 'Relative efficacy of drugs: an emerging issue between regulatory agencies and third-party payers', Nat Rev Drug Discov, Vol. 9(4), pp. 277-291.

Eichler H. G., Hemmings R. J., Vamvakas S., 'Where statisticians can contribute to address future challenges for drug regulatory agencies: a view from the European Medicines Agency (EMEA)', Stat Biopharm Res, Vol. 2(3), pp. 355-358.

Goodsaid F. M., Amur S., Aubrecht J., Burczynski M. E., Carl K., Catalano J., Charlab R., Close S., Cornu-Artis C., Essioux L., Fornace A. J. Jr, Hinman L., Hong H., Hunt I., Jacobson-Kram D., Jawaid A., Laurie D., Lesko L., Li H. H., Lindpaintner K., Mayne J., Morrow P., Papaluca-Amati M., Robison T. W., Roth J., Schuppe-Koistinen I., Shi L., Spleiss O., Tong W., Truter S. L., Vonderscher J., Westelinck A., Zhang L., Zineh I., 'Voluntary exploratory data submissions to the US FDA and the EMA: experience and impact', Nat Rev Drug Discov, Vol. 9(6), pp. 435-445.

Gravanis I., Ersbøll J., Skovlund E., Abadie E., Marty M., Pignatti F., 'The European Medicines Agency review of ofatumumab (Arzerra®) for the treatment of chronic lymphocytic leukemia in patients refractory to fludarabine and alemtuzumab: summary of the scientific assessment of the European Medicines Agency Committee for Medicinal Products for Human Use', Oncologist, Vol. 15(12), pp. 1335-1343.

Grave K., Torren Edo J., Mackay D., 'Comparison of the sales of veterinary antibacterial agents between 10 European countries', J Antimicrob Chemother, Vol. 65(9), pp. 2037-2040.

Herold R., Saint-Raymond A., 'Preamble may not improve consent and assent process', Ped Blood Cancer, Vol. 56(2), p. 327.

Isaac M., Koch A., 'The risk of death among adult participants in trials of antipsychotic drugs in schizophrenia', Eur Neuropsychopharmacol, Vol. 20(3), pp. 139-145.

Jablonski T., 'Pharmaceuticals: End of the transitional period for traditional herbal medicinal products coming soon', Eur J Risk Regulation, Vol. 2, pp. 156-159.

Liberti L., Breckenridge A., Eichler H. G., Peterson R., McAuslane N., Walker S., 'Expediting patients' access to medicines by improving the predictability of drug development and the regulatory approval process', Clin Pharmacol Ther, Vol. 87(1), 27-31.

Mackay D., Kriz N., 'Current challenges in viral safety and extraneous agent testing', Biologicals, Vol. 38(3), pp. 335-337.

Maignen F., Hauben M., Tsintis P., 'Modelling the time to onset of adverse reactions with parametric survival distributions: a potential approach to signal detection and evaluation', Drug Saf, Vol. 33(5), pp. 417-434.

Matheis K., Laurie D., Andriamandroso C., Arber N., Badimon L., Benain X., Bendjama K., Clavier I., Colman P., Firat H., Goepfert J., Hall S., Joos T., Kraus S., Kretschmer A., Merz M., Padro T., Planatscher H., Rossi A., Schneiderhan-Marra N., Schuppe-Koistinen I., Thomann P., Vidal J. M., Molac B., 'A generic operational strategy to qualify translational safety biomarkers', Drug Discov Today, Vol. 16(13-14), pp. 600-608.

Regnstrom J., Koenig F., Aronsson B., Reimer T., Svendsen K., Tsigkos S., Flamion B., Eichler H. G., Vamvakas S., 'Factors associated with success of market authorisation applications for pharmaceutical drugs submitted to the European Medicines Agency', Eur J Clin Pharmacol, Vol. 66(1), pp. 39-48.

Saint-Raymond A., 'EMEA sees benefits for children', Applied Clinical Trials Online, February 2010.

Saint-Raymond A., 'Regulatory aspects of drug development in children: change and resistance to change', Expert Rev Clin Pharmacol, Vol. 3(5), pp. 593-595.

Saint-Raymond A., Hill S., Martines J., Bahl R., Fontaine O., Bero L., 'CONSORT 2010', Lancet, Vol. 376(9737), pp. 229-230.

Seigneuret N., 'The paediatric regulation: three years on', Regulatory Rapporteur, Vol. 7(6), pp. 5-9.

Silva-Lima B., Due Theilade-Thomsen M., Carleer J., Vidal J. M., Tomasi P., Saint-Raymond A., 'Juvenile animal studies for the development of paediatric medicines: a description and conclusions from a European Medicines Agency workshop on juvenile animal testing for nonclinical assessors', Birth Defects Res B Dev Reprod Toxicol, Vol. 89(6), pp. 467-473.

Sistare F. D., Dieterle F., Troth S., Holder D.J., Gerhold D., Andrews-Cleavenger D., Baer W., Betton G., Bounous D., Carl K., Collins N., Goering P., Goodsaid F., Gu Y. Z., Guilpin V., Harpur E., Hassan A., Jacobson-Kram D., Kasper P., Laurie D., Lima B. S., Maciulaitis R., Mattes W., Maurer G., Obert L. A., Ozer J., Papaluca-Amati M., Phillips J. A., Pinches M., Schipper M. J., Thompson K. L., Vamvakas S., Vidal J. M., Vonderscher J., Walker E., Webb C., Yu Y., 'Towards consensus practices to qualify safety biomarkers for use in early drug development', Nat Biotechnol, Vol. 28(5), pp. 446–454.

Spina A. 'European networks in the regulation of biotechnologies', Eur Law Rev, Vol. 35(2), pp. 197-213.

Stoyanova-Beninska V. V., Wohlfarth T,. Isaac M., Kalverdijk L. J., van den Berg H., Gispen-de Wied C., 'The EU paediatric regulation: effects on paediatric psychopharmacology in Europe', Eur Neuropsychopharmacol, Vol. 21(8), pp. 565-570.

Surh L. C., Pacanowski M. A., Haga S. B., Hobbs S., Lesko L. J., Gottlieb S., Papaluca-Amati M., Patterson S. D., Hughes A. R., Kim M. J., Close S. L., Mosteller M., Zineh I., Dechairo B., Cohen N. A., 'Learning from product labels and label changes: how to build pharmacogenomics into drug-development programs', Pharmacogenomics, Vol. 11(12), pp. 1637-1647.

Tomasi P., 'In search of safe and effective medicines', European Pharmaceutical Contractor, winter 2010, pp. 46-49.

Vidal J. M., Kawabata T. T., Thorpe R., Silva-Lima B., Cederbrant K., Poole S., Mueller-Berghaus J., Pallardy M., van der Laan J. W., 'In vitro cytokine release assays for predicting cytokine release syndrome: the current state-of-the-science. Report of a European Medicines Agency workshop', Cytokine, Vol. 51(2), pp. 213-215.

Westermark K, Tsigkos S, Llinares J, Is it time to clarify orphan drug policies? Yes, for equity's sake, Rapid Response to BMJ 2010;341:c4777


Borg J. J., Robert J. L., Wade G., Aislaitner D., Pirożynski M., Abadie E., Salmonson T., Vella Bonanno P., 'Where is industry getting it wrong? A review of quality concerns raised at day 120 by the Committee for Medicinal Products for Human Use during European centralised marketing authorisation submissions for chemical entity medicinal products', J Pharm Pharm Sci, Vol. 12(2), pp. 181-198.

Brasseur D., Pons G., Sain-Raymond A., 'Le règlement européen de &quotmeilleurs médicaments pour les enfants en Europe&quot. Une chance pour la recherche et les soins en pédiatrie?' In: Delfosse M. L., Parizeau M. H., Amman J. P. La recherche clinique avec les enfants: à la croisée de l'éthique et du droit. Belgium, France, Quebec: Editions Anthemis, pp. 51-66.

Broich K., 'Committee for Medicinal Products for Human Use (CHMP) assessment on efficacy of antidepressants', Eur Neuropsychopharmacol, Vol. 19(5), pp. 305-308.

Celis P., Miglicacio G., Pedone E., Petracek J., Pinheiro M. H., Salmikangas P., Schneider C. K., 'Advanced therapies: Regulatory principles and practice', Regulatory Rapporteur, Vol. 6, pp. 12-16.

Celis P., Pedone E., 'The Committee for Advanced Therapies at the European Medicines Agency and the advanced therapies certification procedure', Drug Development, Vol. 4, pp. 64-67.

Eichler H. G., Abadie E., Raine J. M., Salmonson T., 'Safe drugs and the cost of good intentions', N Engl J Med, Vol. 360(14), pp. 1378-1380.

Eichler I., Saint-Raymond A., 'The EU paediatric regulation: the way forward or a barrier to progress?', RAJ Pharma, Vol. 20, pp. 347-348.

Fanciulli G., Tomasi P. A., Delitala A. P., Delitala G., 'Activation of alpha1-adrenoceptors inhibits growth hormone secretion in humans', Exp Clin Endocrinol Diabetes, Vol. 117(9), pp. 460-462.

Giezen T. J., Mantel-Teeuwisse A. K., Strauss S. M. J. M., Egberts T. C. G., Blackburn S., Persson I., Leufkens H. G. M., 'Evaluation of post-authorization safety studies in the first cohort of EU risk management plans at time of regulatory approval', Drug Saf, Vol. 32(12), pp. 1175-1187.

Goedecke T., Brosch S., Arlett P., 'EudraVigilance - the common EU database to support pharmacovigilance activities', Regulatory Rapporteur, Vol. 6(2), pp. 6-11.

Grein K., 'Pharmacovigilance and the European Medicines Agency: conduct of pharmacovigilance activities', In: Woodward, K. N. (ed.) Veterinary pharmacovigilance: adverse reactions to veterinary medicinal products. Wiley-Blackwell.

Jekerle V., Schröder C., Pedone E., 'Legal basis of the advanced therapies regulation', Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz, Vol. 53(1), pp. 4-8.

Ludolph A. C., Kassubek J., Landwehrmeyer B. G., Mandelkow E., Mandelkow E. M., Burn D. J., Caparros-Lefebvre D., Frey K. A., de Yebenes J. G., Gasser T., Heutink P., Höglinger G., Jamrozik Z., Jellinger K. A., Kazantsev A., Kretzschmar H., Lang A. E., Litvan I., Lucas J. J., McGeer P. L., Melquist S., Oertel W., Otto M., Paviour D., Reum T., Saint-Raymond A., Steele J. C., Tolnay M., Tumani H., van Swieten J. C., Vanier M. T., Vonsattel J. P., Wagner S., Wszolek Z. K., Reisensburg Working Group for Tauopathies With Parkinsonism, 'Tauopathies with parkinsonism: clinical spectrum, neuropathologic basis, biological markers, and treatment options', Eur J Neurol, Vol. 16(3), pp. 297-309.

Manolis E., Pons G., 'Proposals for model-based paediatric medicinal development within the current European Union regulatory framework', Br J Clin Pharmacol, Vol. 68(4), pp. 493–501.

Novelli G., Borgiani P., Ciccacci C., Di Daniele N., Sirugo G., Papaluca Amati M., 'Pharmacogenomics: role in medicines approval and clinical use', Public Health Genomics, Vol. 13(5), pp. 284-291.

Saint-Raymond A., 'Médicaments pédiatriques et recherche : un équilibre fragile à trouver entre risques et bénéfices', In: Delfosse M. L., Parizeau M. H., Amman J.P. La recherche clinique avec les enfants: à la croisée de l'éthique et du droit. Belgium, France, Quebec: Editions Anthemis.

Saint-Raymond A., Seigneuret N., 'The European paediatric initiative: 1 year of experience', Paediatr Drugs, Vol. 11(1), pp. 9-10.

Saint-Raymond A., Sweeney F., 'Clinical trials in developing countries: risk or opportunity?' Future Med Chem, Vol. 1(7), pp. 1193-1195.

Schneider C. K., Papaluca M., Kurki P., 'A European perspective on immunogenicity evaluation', Nat Biotechnol, Vol. 27(6), pp. 507-508.

Scientific Advisory Group on Antimicrobials of the Committee for Medicinal Products for Veterinary Use, 'Reflection paper on the use of third and fourth generation cephalosporins in food producing animals in the European Union: development of resistance and impact on human and animal health', J Vet Pharmacol Ther, Vol. 32(6), pp. 515-533.

Shah R. R., Saint Raymond A., 'Regulation of human medicinal products in the European Union', In: Griffin J. P. The textbook of pharmaceutical medicine. 6th ed. London: BMJ Books, pp. 444-499.

Shivji R., Purves J., Celis P., 'European Medicines Agency: influenza pandemic preparedness'. Risk Wise: Epidemics, ISBN: 0-9536140-6-9, pp. 36-39.

Tomasi PA, Fanciulli G, Casti T, Delitala G, Persistent hyperprolactinemia and bilateral galactocele in a male infant, Int J Pediatr Endocrinol. 2009; 2009: 578610


Blackburn S., 'The European Network of Centres in Pharmacoepidemiology and Pharmacovigilance', PRM Newsletter, Vol. 0, pp. 5-6.

Blind E., 'Twenty years of progress with parathyroid hormone (PTH): from specialized and &quotdifficult&quot measurement to common laboratory parameter and treatment option in osteoporosis', Clin Lab, Vol. 54, pp. 439-449.

De Clercq K., Goris N., Barnett P. V., Mackay D. K., 'FMD vaccines: reflections on quality aspects for applicability in European disease control policy', Transbound Emerg Dis, Vol. 55(1), pp. 46-56.

De Clercq K., Goris N., Barnett P. V., Mackay D. K., 'The importance of quality assurance/quality control of diagnostics to increase the confidence in global foot-and-mouth disease control', Transbound Emerg Dis, Vol. 55(1), pp. 35-45.

Eichler H. G., Pignatti F., Flamion B., Leufkens H., Breckenridge A., 'Balancing early market access to new drugs with the need for benefit/risk data: a mounting dilemma', Nat Rev Drug Discov, Vol. 7(10), pp. 818-826.

Hauben M., Vogel U., Maignen F., 'Number needed to detect: nuances in the use of a simple and intuitive signal detection metric', Pharm Med, Vol. 22(1), pp. 13-22.

Llinares J., 'Orphan designation in the EU: an overview and key achievements', Regulatory Rapporteur, November 2008, pp. 5-9.

Neugebauer B., Drai C., Haase M., Hilger A., Keller-Stanislawski B., Laitinen-Parkkonen P., Mentzer D., Rasmussen C., Ratignier C., Seitz R., 'Factor VIII products and inhibitor development: concepts for revision of European regulatory guidelines', Haemophilia, Vol. 14(1), pp. 142-144.

Ormarsdottir S., Reginster J. Y., Abadie E., 'European regulatory perspectives for innovative therapies', Osteoporos Int, Vol. 19(6), pp. 725-731.


Grein K., Papadopoulos O., Tollis M., 'Safe use of vaccines and vaccine compliance with food safety requirements', Rev Sci Tech, Vol. 26(2), pp. 339-350.

Mackay D., 'Authorisation within the European Union of vaccines against antigenically variable viruses responsible for major epizootic diseases', Rev Sci Tech, Vol. 26(2), pp. 421-428.

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