Scientific publications

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

2026

Empl MT, Paul P, Moriarty O, Ponzano S, Beken S, Adler-Flindt S, et al. 3Rs at the European Medicines Agency: Past and future activities. NAM J. 2026;2:100086. doi: 10.1016/j.namjnl.2026.100086.

Tenhunen O, Penttinen K, Voltz C, Kuitunen H, Turpeinen M. Age and frailty in anticancer drug regulatory assessment: a comprehensive cohort study of European marketing authorisations 2012-2023. ESMO Real World Data Digit Oncol. 2026;11:100663. doi: 10.1016/j.esmorw.2025.100663.

Bright M, Kontsioti E, Pirmohamed M, Zhou Y, Maskell S. MCSG: A Method for Simultaneous Disproportionality Analysis and Background Rate Estimation in Large Pharmacovigilance Databases. Drug Saf. 2026;49(4):481-491. doi: 10.1007/s40264-025-01632-8.

Giuliani R, Kronidou-Horst N, Yap C, Sosa Mejia A, Leyens L, Framke T, et al. Innovative oncology trial designs: Time to act - A review with recommendations of the Cancer Drug Development Forum. J Cancer Policy. 2026;47:100696. doi: 10.1016/j.jcpo.2025.100696.

Guizzaro L, Bałkowiec-Iskra E, Haberkamp M, Mol PGM, Moreau A, Mueller-Berghaus J, et al. Balancing benefit and risk in early Alzheimer's disease: the European Medicines Agency (EMA) assessment of lecanemab and donanemab. Lancet Reg Health Eur. 2026;63:101644. doi: 10.1016/j.lanepe.2026.101644.

Bonkat G, Burgos JG, Ruepp R, Browne K. The European Association of Urology Guidelines on Urological Infections: Bridging Regulatory Strategy with Proactive Clinical Leadership. Eur Urol Focus. 2026;12(2):297-298. doi: 10.1016/j.euf.2025.11.005.

Vogel M, Carlsson MJ, Skowron C, Felske C, Whomsley R, Fahrer J. Nitrosamine drug substance-related impurities cause DNA methylation adducts in vitro and in primary hepatocytes upon Cytochrome P450-dependend metabolic activation. Regul Toxicol Pharmacol. 2026;167:106036. doi: 10.1016/j.yrtph.2026.106036.

Gratton J, Arlett P, Ehmann F, Prieto-Alhambra D, Verpillat P, Cormier E, et al. Advancing pharmacogenomics in medicines regulation and clinical practice: a call for collaborative action. Nat Rev Drug Discov. 2026;25(2):79-80. doi: 10.1038/d41573-025-00178-9.

Pignatti F, El-Galaly TC, Kaiser M, Porkka K, Doeswijk R, Mol P, et al. Assessing Overall Survival Benefits in Advanced Cancers: The Role of External Comparator Cohort Studies with Real-World Data. Clin Pharmacol Ther. 2026;119(4):1080-1087. doi: 10.1002/cpt.70213.

Rosenberg N, Hollak CEM, Haverhoek VA, Stoyanova-Beninska V, Pasmooij AMG, De Visser SJ. Tracing the path of non-industry-driven medicine development for rare diseases through regulatory interactions at the European Medicines Agency. Drug Discov Today. 2026;31(2):104631. doi: 10.1016/j.drudis.2026.104631.

Lacombe D, Cardoso Borges F, Amariutei AE, Booth CM, Brusselle G, Casolino R, et al. Accelerating cancer treatment optimisation: A multistakeholder roadmap from the Cancer Medicines Forum. J Cancer Policy. 2026;47:100700. doi: 10.1016/j.jcpo.2025.100700.

Barchuk A, Barboza C, Politi J, Raventós B, Prinsen P, Evers J, et al. Characteristics, treatment and survival of patients with chondrosarcoma in five European countries: a DARWIN EU® cohort study. Acta Oncol. 2026;65:193-200. doi: 10.2340/1651-226X.2026.45117.

Bouygues C, Tavridou A, Herold R, Nuevo-Ordoñez Y. Stimulating medicines repurposing in the EU: a pilot project. Nat Rev Drug Discov. 2026;25(1):12-13. doi: 10.1038/d41573-025-00198-5.

Kristyanto H, González-Tomé MI, Vlcek V, Josephson F, Vrijlandt P, Perombelon N. Global regulatory collaboration for the assessment of lenacapavir as a long-acting pre-exposure prophylaxis against HIV infection. Front Public Health. 2026;14:1760130. doi: 10.3389/fpubh.2026.1760130.

Keyter A, Sithole T, Broojerdi AK, Nwokike J, Harvey-Allchurch M, Sillo H. The World Health Organization Coalition of Interested Parties Network: a unified and coordinated approach to regulatory system strengthening. Front Med (Lausanne). 2026;13:1725720. doi: 10.3389/fmed.2026.1725720.

Liu X, Thomas NH, Asikanius E, Pothet C, Großhennig A, Koch A. A Statistical Concept for Conditional Marketing Authorisation Based on the Intermediate and Final Outcomes of a Single Confirmatory Randomised Clinical Trial. Pharm Stat. 2026;25(2):e70078. doi: 10.1002/pst.70078.

Jonker CJ, Plueschke K, Breen KC, de Lemus Belmonte M, Verpillat P, Pacurariu A. Learnings from a registry-based cohort study for spinal muscular atrophy disease. Neuromuscul Disord. 2026;59:106332. doi: 10.1016/j.nmd.2025.106332.

Baron A, Broekman E, van Gils M, Poplavska E, Mol P, Saliete I, et al. The European medicines agency review of belzutifan (Welireg) for the treatment of adult patients with von Hippel-Lindau disease-associated tumors. Oncologist. 2026;31(2):oyaf433. doi: 10.1093/oncolo/oyaf433.

Hersey S, McGuire K, Kholmanskikh O, Bivi N, Bandukwala A, Gong B, et al. 2025 White Paper on Recent Issues in Bioanalysis: Biomarkers Calibrators & Stability; Evaluation of NULISA; Neurofilament & Autoantibody Biomarker Assays; Removing IgM Interference; ELISpot & FluoroSpot Best Practices; Modular HD Cytometry; Single-cell Analysis Imaging Cytometry (PART 2A - Recommendations on Biomarkers Discovery, Development, Validation & Regulatory Approval, Ligand-Binding Assays (LBA) and Cell-Based Assays (CBA) PART 2B - Regulatory Agencies' Input on Biomarkers, IVD/CDx and Biomarker Assay Validation (BAV)). Bioanalysis. 2026;17(23):1481-1533. doi: 10.1080/17576180.2025.2599698.

Reviriego-Gordejo F, Minne D, Claassen I, Cavitte JC, Bouma A, Debaere O, et al. Preparedness and response to emerging veterinary disease outbreaks - A meeting report. Biologicals. 2026;93:101873. doi: 10.1016/j.biologicals.2026.101873.

Mondéjar-Parreño G, Moriarty O, Ponzano S, Kyriakopoulou E, Beken S, Tognana E. New approaches in assessing potential proarrhythmic risk and cardiovascular safety during medicines development: opportunities for 3Rs application. NAM J. 2026;2:100083. doi: 10.1016/j.namjnl.2026.100083.

Drai C, van der Woude HJ, Lexmond AJ, van den Hoorn T, Mol PGM, Leacy FP, et al. The European Medicines Agency's review of elexacaftor/tezacaftor/ivacaftor: extending its use to all people with cystic fibrosis aged 2 years and older who do not have two class I CFTR variants. Eur Respir J. 2026;67(1):2501506. doi: 10.1183/13993003.01506-2025.

de Bruin O, Maisonneuve E, Hurley E, Nordeng HME, Bérard A, Sheehy O, et al. Medications Used Among Nonhospitalized Pregnant Women With COVID-19: A Prospective Individual Patient Data Meta-Analysis in Europe and North America. Pharmacoepidemiol Drug Saf. 2026;35(1):e70303. doi: 10.1002/pds.70303.

Hiruy H, Bala S, Byrne JM, Roche KG, Jang SH, Kim P, et al. US Food and Drug Administration, Centers for Disease Control and Prevention, and National Institutes of Health Co-Sponsored Public Workshop Summary-Development Considerations of Antimicrobial Drugs for the Treatment of Gonorrhea. Clin Infect Dis. 2026;81(6):1201-1208. doi: 10.1093/cid/ciae386.

Kalimuddin S, Waickman AT, Avirutnan P, Alam MS, Chiu C, Young BE, et al. Proceedings of the inaugural symposium on dengue human challenge studies "Challenging the norm: Accelerating dengue countermeasures through human studies". PLoS Negl Trop Dis. 2026;20(1):e0013889. doi: 10.1371/journal.pntd.0013889.

Portela JMD, Paul P, Moriarty O, Theunissen P, Beken S, Brendler-et al. Review on organs-on-chips for medicines safety assessment: A European regulatory perspective. ALTEX. 2026;43(1):98-112. doi: 10.14573/altex.2502061.

Spaenig M, Vogel M, Hansen T, Elenschneider L, Londenberg A, Whomsley R, et al. Estimation of bioavailable concentration of endogenously formed N-nitrosamines by physiologically based kinetic modelling. ALTEX. 2026;43(1):49-64. doi: 10.14573/altex.2412061.

Hermans SJF, Versluis J, van Werkhoven ED, van Norden Y, Janssen JJWM, Huls GA, et al. Comparability of external and internal control patients for the prospective randomized HOVON-103 trial in older AML patients. Br J Haematol. 2026;208(1):179-188. doi: 10.1111/bjh.20185.

2025

Pe M, Voltz-Girolt C, Bell J, Bhatnagar V, Bogaerts J, Booth C, et al. Using patient-reported outcomes and health-related quality of life data in regulatory decisions on cancer treatment: highlights from an EMA-EORTC workshop. Lancet Oncol. 2025;26(6):687-690. doi: 10.1016/S1470-2045(25)00150-0.

Nett RJ, Brault AC, Lambert AJ, Agnihothram S, Barrett ADT, Barzon L, et al. Summary of human West Nile virus vaccine meeting, 2024: Investigating barriers to development. Vaccine. 2025;68:127938. doi: 10.1016/j.vaccine.2025.127938.

Arlett P, Umuhire D, Verpillat P, Foggi P, Wändel Liminga U, Sepodes B, et al. Clinical Evidence 2030. Clin Pharmacol Ther. 2025;117(4):884-886. doi: 10.1002/cpt.3596.

Edwards M, Blanquie O, Ehmann F. Insights into new approach methodology innovation: an EMA perspective. Nat Rev Drug Discov. 2025;24(5):325-326. doi: 10.1038/d41573-025-00052-8.

Poulard IC, Ausborn S, Allchurch MH, Palmi V, Ganan A, Joos A, et al. Reliance into Action: Understanding EMA Documents to Streamline Reliance for Marketing Authorization Applications. Ther Innov Regul Sci. 2025;59(5):1032-1041. doi: 10.1007/s43441-025-00824-9.

Edwards M, Blanquie O, Moriarty O, Beken S, Ehmann F. New approach methodologies: EU regulatory horizons. Nat Rev Drug Discov. 2025;24(8):571-572. doi: 10.1038/d41573-025-00053-7.

Pignatti F, Mol P, Quinten C, Postmus D, Schiel A, Sasseville M, et al. Use of patient-reported outcomes to inform symptom and functional outcomes in cancer drug regulatory decisions: challenges and future directions. Lancet Oncol. 2025;26(6):664-666. doi: 10.1016/S1470-2045(25)00151-2.

Cooke E, Sepodes B. Thirty years of Europe's centralized procedure for approving medicines. Nat Med. 2025;31(9):2831-2832. doi: 10.1038/s41591-025-03826-0.

Tampach S, Vidal A, Marnane I, De Coen W, Heuer O, das Neves CG, et al. Scientific cooperation among Europe's One Health agencies. One Health. 2025;21:101129. doi: 10.1016/j.onehlt.2025.101129.

Kristyanto H, Rey Torre S, Cunney A, Dunder K, Bego M, Sepodes B, et al. The European Medicines Agency evaluation of nemolizumab (Nemluvio) for the treatment of moderate-to-severe atopic dermatitis and prurigo nodularis. Br J Dermatol. 2025;193(5):996-998. doi: 10.1093/bjd/ljaf259.

Gonzalez-Quevedo R, Garcia Burgos J, Greco M, Straus S, Liminga UW, Sepodes B. European Medicines Agency seeks stakeholders' views on its reflection paper on patient experience data. Br J Clin Pharmacol. 2025;91(12):3301-3303. doi: 10.1002/bcp.70330.

Garcia Burgos J, Jirsova E, Kranz J, Bonkat G. A Call To Prevent Inappropriate Prescribing of Fluoroquinolones from the European Medicines Agency and the European Association of Urology Guidelines Panel for Urological Infections. Eur Urol. 2025;88(5):427-429. doi: 10.1016/j.eururo.2025.07.015.

Essink SCM, Zomerdijk IM, Goedecke T, Straus SMJM, Gardarsdottir H, De Bruin ML. Duration of Time Intervals for Risk Minimization Measure Effectiveness Studies. Clin Pharmacol Ther. 2025;117(4):1106-1114. doi: 10.1002/cpt.3569.

Pandya L, Norstedt I, Seychell M, Cooke E, de la Mata Barranco I, Blomberg N, et al. Europe-Africa collaboration: working together to strengthen health research, implementation, and equitable systems. Lancet Glob Health. 2025;13(8):e1338-e1340. doi: 10.1016/S2214-109X(25)00201-3.

Moorthy V, Devereaux PJ, Sui-Lin Teh C, Rooslamiati I, Adeyemi OD, Hamid S, et al. Tracking maturity of the enabling environment for clinical trials. Lancet. 2025;406(10499):113-116. doi: 10.1016/S0140-6736(25)01279-6.

Broekman KE, van Gils MPMQ, Baron A, Poplavska E, Mol PGM. Letter to the editor re: fatal intracranial haemorrhage after belzutifan initiation. ESMO Open. 2025;10(8):105537. doi: 10.1016/j.esmoop.2025.105537.

Fink M, Lutters B, O'Connor DB, Ganan Jimenez A, Holborow R. Navigating global regulatory pathways for orphan medical devices. RF Quarterly 2025;5(4):4-12.

Cooke E, Rendi-Wagner P. Rising antimicrobial resistance in Europe: EMA and ECDC outline urgent actions to achieve EU targets. Lancet Reg Health Eur. 2025;59:101539. doi: 10.1016/j.lanepe.2025.101539.

Ruepp R, Botgros R, Le Ber V, Brandt F, Pantke E, Fuerst-Wilmes M, et al. Changes to the use of medicines containing the antibiotic azithromycin. Fam Med Community Health. 2025;13(4):e003699. doi: 10.1136/fmch-2025-003699.

Moon K, Coxon C, Årdal C, Botgros R, Djebara S, Durno L, et al. Considerations and perspectives on phage therapy from the transatlantic taskforce on antimicrobial resistance. Nat Commun. 2025;16(1):10883. doi: 10.1038/s41467-025-64608-3.

Dijkstra JN, Henzi BC, Mathews KD, Erasmus CE, Knox R, Willis T, et al. 279th ENMC international workshop: Classification, clinical care, outcome measures and biomarkers in childhood onset facioscapulohumeral dystrophy: towards standardizing clinical care and ensuring clinical trial readiness. Hoofddorp, The Netherlands, 1-3 November 2024. Neuromuscul Disord. 2025;55:106236. doi: 10.1016/j.nmd.2025.106236.

Pariser AR, Stoyanova-Beninska V, Iliach O, Jundi R, Lee KJ, Morikawa H, et al. Non-oncologic orphan drug approvals across the world: Types of evidence required and time to approval. Drug Discov Today. 2025;30(12):104529. doi: 10.1016/j.drudis.2025.104529.

Destoop M, Mohr P, Butlen F, Kéri P, Samochowiec J, De Picker L, et al. Use of psychedelic treatments in psychiatric clinical practice: an EPA policy paper. Eur Psychiatry. 2025;68(1):e3. doi: 10.1192/j.eurpsy.2024.1806.

Mitroiu M, Teerenstra S, Oude Rengerink K, Pétavy F, Roes KCB. Possible Data-Generating Models of Longitudinal Continuous Outcomes and Intercurrent Events to Investigate Estimands. Stat Biopharm Res. 2025;17(2),247–259. doi: 10.1080/19466315.2024.2369266.

Amdal CD, Falk RS, Alanya A, Schlichting M, Roychoudhury S, Bhatnagar V, et al. SISAQOL-IMI consensus-based guidelines to design, analyse, interpret, and present patient-reported outcomes in cancer clinical trials. Lancet Oncol. 2025;26(12):e683-e693. doi: 10.1016/S1470-2045(25)00520-0.

Yusufujiang M, Damen JAA, Idema DL, Schuit E, Moons KGM, de Jong VMT. Network meta-analysis of prediction models using aggregate or individual participant data - a scoping review and recommendations for reporting and conduct. J Clin Epidemiol. 2025;188:112006. doi: 10.1016/j.jclinepi.2025.112006.

DuBois SG, Moreno L, Bagatell R, Cheung NK, Gray JC, Locatelli F, et al. Paediatric Strategy Forum for medicinal product development of agents targeting GD2 ganglioside in children and adolescents with cancer. Eur J Cancer. 2025;231:116093. doi: 10.1016/j.ejca.2025.116093.

Sepodes B, Garcia Burgos J, Thirstrup S. EMA approvals of new medicinal products and new biosimilar products in September and October 2025. Lancet Reg Health Eur. 2025; 59:101542. doi:10.1016/j.lanepe.2025.101542.

Buoninfante A, Cavaleri M. Reply to "Investigating a role for NSAIDs in the pathophysiology of vaccine-associated myocarditis". NPJ Vaccines. 2025;10(1):249. doi: 10.1038/s41541-025-01285-9.

Berdel WE, Davy JM, Al M, Haan RR, Cavaleri M. Centralised ethics review of clinical trials in a public health emergency in the EU. Lancet. 2025;406(10518):2418. doi: 10.1016/S0140-6736(25)02054-9.

Zanello G, Stoyanova-Beninska V, Iliach O, Scherman D, Parker S, Pearce DA. Regulatory sandboxes: A new frontier for rare disease therapies. Rare 2025;3:100110. doi: 10.1016/j.rare.2025.100110.

Kurz X, Cohet C, Perez-Gutthann S, Rao S, Gardarsdottir H. Strengthening Pharmacoepidemiology in a Changing Research Environment: The European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP). Pharmacoepidemiol Drug Saf. 2025;34(11):e70263. doi: 10.1002/pds.70263.

Browne K, Garcia Burgos J, Jirsová E, Ruepp R, Bonkat G, Vinker S, et al. The case for more prudent prescribing of fluoroquinolones in primary care. Eur J Gen Pract. 2025;31(1):2584496. doi: 10.1080/13814788.2025.2584496.

García-Arieta A, Tam A, Tiseyra F, Fernandes EAF, Soares KCC, Pereira RS, et al. A survey of regulatory recommendations for waivers of in vivo bioequivalence studies of generic products for certain dosage forms by participating regulators and organisations of the International Pharmaceutical Regulators Programme. Part 2. J Pharm Pharm Sci. 2025;28:14721. doi: 10.3389/jpps.2025.14721.

Zafiropoulos N, Pignatti F, Kouroumalis A, Guizzaro L, Karres D, Demolis P, et al. A model structure for describing uncertainties in benefit-risk assessment of oncology drug applications. Front Med (Lausanne). 2025;12:1589578. doi: 10.3389/fmed.2025.1589578.

Arlett P, Ehmann F, Herold R, Korakianiti E, Moscariello P, Barbier L, et al. A Fast Path from Innovation to Safe and Effective Medicines. Clin Pharmacol Ther. 2025;118(6):1392-1404. doi: 10.1002/cpt.70107.

Abed I, Bennett V, Cuddy B, Dang S, García Burgos J, Di Giorgio D, et al. The impact of the European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) to address shortages of human and veterinary medicines across Europe. Front Public Health. 2025;13:1673681. doi: 10.3389/fpubh.2025.1673681.

Morales DR, Gardarsdottir H. From Evidence to Impact: Closing the Loop in Pharmacovigilance With Real-World Data. Pharmacoepidemiol Drug Saf. 2025;34(11):e70244. doi: 10.1002/pds.70244.

Costa E, Ajith V, Al Khaldi AF, Isgrò A, Lee KJ, Luigetti R, et al. Addressing global regulatory challenges in rare disease drug development. Drug Discov Today. 2025;30(10):104462. doi: 10.1016/j.drudis.2025.104462.

Loedy N, Gonzalez Dorta H, Abrams S, Crèvecoeur J, Morales DR, Cohet C, et al. An extension of the Benefit Risk Assessment of VaccinEs toolkit to evaluate Comirnaty and Spikevax vaccination in the European Union. Eur J Public Health. 2025;35(5):1007-1013. doi: 10.1093/eurpub/ckaf135.

Flory E, Walter M, Closson-Carella V, Celis P, Schuessler-Lenz M, Reischl I. Medicinal Products Based on Adeno-Associated Viral Vectors: A Regulatory Perspective on the Potential Risk of Insertion-Mediated Tumorigenesis. Hum Gene Ther. 2025;36(21-22):1405-1414. doi: 10.1177/10430342251366314.

Stacchiotti S, Pantziarka P, Leonard H, Voltz C, Abatedaga L, Bouche G, et al. How to foster new treatment development in ultra-rare tumours? Joint EMA-EORTC multi-stakeholder workshops on ultra-rare sarcomas as a model for rare cancers. Cancer Treat Rev. 2025;140:103003. doi: 10.1016/j.ctrv.2025.103003.

Ahnfelt E, Lagerlund O, Klint J, Fladvad M, Jarvis C, Chen TJ, et al. Advancing Global Harmonization: Implementing Global Dose Form Attributes for Medicinal Products Identification. Ther Innov Regul Sci. 2025;59(6):1306-1315. doi: 10.1007/s43441-025-00838-3.

De Lisa R, de Andres-Trelles F, Bax R, Galluzzo S, Alward B, Thomson A, et al. New medicines for childhood-onset systemic lupus erythematosus: an EU perspective on paediatric drug development. Front Med (Lausanne). 2025;12:1583140. doi: 10.3389/fmed.2025.1583140.

Carmona Araújo A, Guerreiro JP, Bulhosa C, Martins AP, Alves da Costa F, Goulão J. Morbimortality consequences of use and misuse of psychoactive medicines in Portugal. Br J Clin Pharmacol. 2025;91(11):3225-3235. doi: 10.1002/bcp.70171.

Pignatti F, Herold R, Demolis P, Rohr UP. Novel PET RANO BM 1.0 criteria: Regulatory perspectives on new endpoint definitions in brain metastases. Neuro Oncol. 2025;27(6):1407-1408. doi: 10.1093/neuonc/noaf067.

Norén GN, Donegan K, Muñoz MA, Alshammari TM, Pratt N, Candore G, et al. Recommendations to Enable Broader Use of Real-World Evidence to Inform Decision-Making Throughout Pharmacovigilance Signal Management. Pharmacoepidemiol Drug Saf. 2025;34(10):e70231. doi: 10.1002/pds.70231.

Bergman J, Guizzaro L, Abellan JJ, Lasch F. Estimands in equivalence trials and non-inferiority trials: a cross-sectional study of EMA scientific advice to drug developers. Trials. 2025;26(1):348. doi: 10.1186/s13063-025-09068-2.

Sepodes B, Garcia Burgos J, Thirstrup S. EMA approvals of new medicinal products and new biosimilar products in July and August 2025. Lancet Reg Health Eur. 2025;57:101469. doi:10.1016/j.lanepe.2025.101469.

Del Giacco S, Canonica GW, Agache I, Price D, Roche N, Schunemann H, et al. Expert Perspectives on Next Generation Health Guidelines: How to Integrate RWE in EBM. Pragmat Obs Res. 2025;16:169-177. doi: 10.2147/POR.S487342.

Schneider KL, Alves de Sousa L, Sava I, Heß S, Umuhire D, Morales DR. The European Health Data Space in communicable diseases surveillance and monitoring of medicines and vaccines: achievements of a pilot project examining the user journey. Eur J Public Health. 2025;35(S3):iii5-iii10. doi: 10.1093/eurpub/ckaf115.

Barbier L, Herold R, Thirstrup S. Delivering on the Promises of Medicines Innovation - Promoting and Achieving Excellence in Regulatory Science in Europe. DIA Global Forum 2025; 17 (9).

Devane D, Briel M, Bhagani S, Boesten N, Buchholz S, De Luca EC, et al. Protocol for a core outcome set for pharmacological treatments in hospitalised patients with acute viral respiratory infections (COSAVRI). PLoS One. 2025;20(9):e0330288. doi: 10.1371/journal.pone.0330288.

Postmus D, Cornelissen JJ, Bogaerts J, Roes KC, Hillege HL, Pignatti F. Beyond traditional endpoints: A multi-state modelling approach to integrating morbidity and mortality in cancer treatments evaluation. Eur J Cancer. 2025;228:115704. doi: 10.1016/j.ejca.2025.115704.

Hernán Pérez de la Ossa D, Haas F, Bream RN, Kotzagiorgis E, Tiitso K, Jekerle V. Learning from the EMA experience: common CMC deficiencies in marketing authorisation applications over the past decade. Drug Discov Today. 2025;30(9):104444. doi: 10.1016/j.drudis.2025.104444.

Sepodes B, Garcia Burgos J, Thirstrup S. EMA approvals of new medicinal products and new biosimilar products in May and June 2025. Lancet Reg Health Eur. 2025;55:101389. doi:10.1016/j.lanepe.2025.101389.

Excler JL, Saluja T, Wilder-Smith A, Kaminski RW, MacLennan CA, Cavaleri M, et al. Non-typhoidal Salmonella combination vaccines: clinical development plan and regulatory considerations. Vaccine. 2025 Aug 30;62:127515. doi: 10.1016/j.vaccine.2025.127515.

Zebachi S, Tanniou J, Bakker E, de Vries ST, Di Bidino R, Xoxi E, et al. Navigating the Real World: A Scoping Review of Structured Frameworks to Effectively Identify, Evaluate, and Select Real-World Data Sources for Fit-for-Purpose Studies. Clin Pharmacol Ther. 2025 Oct;118(4):894-905. doi: 10.1002/cpt.3746.

Sepodes B, Garcia Burgos J, Thirstrup S. EMA approvals of new medicinal products and new biosimilar products in March and April 2025. Lancet Reg Health Eur. 2025;54:101330. doi:10.1016/j.lanepe.2025.101330.

Vogel M, Escher SE, Weiler E, Londenberg A, Deppenmeier U, Whomsley R. Quantitative Investigation of Nitrosamine Drug Substance-Related Impurities (NDSRIs) Under Artificial Gastric Conditions by Liquid Chromatography-Tandem Mass Spectrometry and Structure-Activity Relationship Analysis. Drug Test Anal. 2025;17(9):1772-1784. doi: 10.1002/dta.3874.

Gonzalez Tome M, Gonzalez-Quevedo R, Escudeiro Dos Santos M, Dornbusch HJ, Straus S, Cooke E. Meningococcal B Vaccines as a Paradigm of Safe and Effective Vaccines for Children. Vaccines (Basel). 2025;13(7):770. doi: 10.3390/vaccines13070770.

Aarum S, Popp C, Hunter A, Skvarc M, Reischl I, Zander H, et al. Multistakeholder scientific advice for medicinal products used in combination with a medical device or a companion diagnostic in the EU-summary of a Focus group discussion. Front Med (Lausanne). 2025;12:1593644. doi: 10.3389/fmed.2025.1593644.

Magrelli A, O'Connor DJ, Stoyanova-Beninska V. Editorial: The changing focus of regulatory frameworks around the globe and the opportunities for harmonization. Front Med (Lausanne). 2025;12:1645278. doi: 10.3389/fmed.2025.1645278.

Browne K, Bałkowiec-Iskra E, Elferink A, Haberkamp M, Straus S, Sepodes B, et al. Applying the EU Regulatory Framework to Determine the Benefit-Risk Profile of Psychedelics. ACS Pharmacol Transl Sci. 2025;8(8):2830-2838. doi: 10.1021/acsptsci.5c00456.

Pearson AD, Chi S, Laetscht TW, Marshall L, Raetz E, George RE, et al. Paediatric strategy forum for medicinal product development of cyclin-dependent kinase inhibitors in children and adolescents ACCELERATE in collaboration with the European Medicines Agency With participation of the Food and Drug Administration. Eur J Cancer. 2025;226:115629. doi: 10.1016/j.ejca.2025.115629.

Mbaelachi N. Celebrating its past, European Medicines Agency looks to the future of public communication. Medical Writing. 2025; 34(2):18-20. doi: 10.56012/ofdv3305.

Kas MJH, Penninx BWJH, Knudsen GM, Cuthbert B, Falkai P, Sachs GS, et al. Precision psychiatry roadmap: towards a biology-informed framework for mental disorders. Mol Psychiatry. 2025;30(8):3846-3855. doi: 10.1038/s41380-025-03070-5.

Plueschke K, Jonker C, Kankanen H, Vetter T, Sepodes B, Naehrlich L, et al. Optimizing Patient Registries for Regulatory Decision Making - Key Learnings From an HMA/EMA Multistakeholder Workshop. Clin Pharmacol Ther. 2025;118(3):551-560. doi: 10.1002/cpt.3733.

Pignatti F, Josephson F, Demolis P, Tenhunen O, Péan E, Postmus D. Expedited Approval in Oncology: A Study of European Regulators' Perspectives and Trade-Offs. Clin Pharmacol Ther. 2025;118(3):642-648. doi: 10.1002/cpt.3708.

Walder L, Pallocca G, Bastos LF, Beekhuijzen M, Busquet F, Constantino H, et al. EU roadmap for phasing out animal testing for chemical safety assessments: Recommendations from a multi-stakeholder roundtable. ALTEX. 2025;42(3):435-450. doi: 10.14573/altex.2503241.

Crisafulli S, Bate A, Brown JS, Candore G, Chandler RE, Hammad TA, et al. Real-World Evidence and Big Data Special Interest Group of the International Society of Pharmacovigilance. Interplay of Spontaneous Reporting and Longitudinal Healthcare Databases for Signal Management: Position Statement from the Real-World Evidence and Big Data Special Interest Group of the International Society of Pharmacovigilance. Drug Saf. 2025;48(9):959-976. doi: 10.1007/s40264-025-01548-3.

Barbier L, Moscariello P, Leufkens HG, Herold R, Pasmooij AMG. A new European platform for advancing regulatory science research. Nat Rev Drug Discov. 2025;24(7):485-486. doi: 10.1038/d41573-025-00024-y.

Smith MY, Davis R, Bahri P, Saragoussi D, Nguyen V, Toyserkani GA, et al. Using Mixed Methods to Evaluate Risk Minimisation Programs in Europe and the USA: An Innovative Blueprint. Drug Saf. 2025;48(7):821-838. doi: 10.1007/s40264-025-01533-w.

Buoninfante A, Cavaleri M. T cells responses after vaccination: a regulatory perspective. Front Immunol. 2025;16:1584738. doi: 10.3389/fimmu.2025.1584738.

Palomo GM, Pose-Boirazian T, Naumann-Winter F, Costa E, Duarte DM, Kalland ME, et al. Navigating the orphan medicinal product designation: Evidence requirements for gene therapies in Europe. Mol Ther. 2025;33(6):2834-2841. doi: 10.1016/j.ymthe.2024.10.015.

Thanarajasingam G, Major A, Bhatnagar V, Dueck AC, Smedby KE, El-Galaly TC, et al. Beyond maximum grade: introduction to The Lancet Haematology Adverse Events Reporting Series. Lancet Haematol. 2025;12(6):e403-e406. doi: 10.1016/S2352-3026(25)00027-4.

Zosso-Pavic M, Pignatti F, Wolfer A, Li Q, Demolis P, Rohr UP, et al. Navigating the complexity: reflections on the development of perioperative cancer treatments. Lancet Oncol. 2025;26(6):675-678. doi: 10.1016/S1470-2045(25)00217-7.

Le Doare K, Benassi V, Cavaleri M, Enwere G, Giersing B, Goldblatt D, et al. Clinical and regulatory development strategies for GBS vaccines intended for maternal immunisation in low- and middle-income countries. Vaccine. 2025;58:127131. doi: 10.1016/j.vaccine.2025.127131.

Garcia Burgos J, Thirstrup S, Cooke E. The European Medicines Agency and The Lancet Regional Health-Europe join forces to protect public health. Lancet Reg Health Eur. 2025;53:101331. doi: 10.1016/j.lanepe.2025.101331.

Cardoso Borges F, van der Graaf WTA, Saesen R, Aebi S, Amariutei AE, Bekelman J, et al. Defining the role of pragmatic clinical trials in cancer clinical research: outcomes of a collaborative workshop hosted by the European Organisation for Research and Treatment of Cancer. Lancet Oncol. 2025;26(5):e253-e263. doi: 10.1016/S1470-2045(24)00756-3.

Meln I, Cnossen V, Corti N, Andeweg A, Baay M, Chiu C, et al. Regulatory workshop on standardisation of clinical procedures, endpoints and data robustness of human challenge studies - A stakeholder meeting report. Biologicals. 2025;90:101818. doi: 10.1016/j.biologicals.2025.101818.

Ibarz-Pavon AB, Bielsky MC, Bose R, Cavaleri M, Crump JA, Hombach J, et al. Consultation report - considerations for a regulatory pathway for bivalent Salmonella Typhi/Paratyphi A vaccines for use in endemic countries. Vaccine. 2025;56:127189. doi: 10.1016/j.vaccine.2025.127189.

Bonertz A, Drai C, Hartenstein D, Kaul S, Mitrevski M, Zubeldia JM. European Medicines Agency (EMA) commentary on EMA/CHMP Guideline on allergen products development for immunotherapy and allergy diagnosis in moderate to low-sized study populations. Br J Clin Pharmacol. 2025;91(5):1293-1296. doi: 10.1002/bcp.70048.

Guillen E, Barry S, Jost N, Ekman N, Knippel V, Kuhlmann-Gottke J, et al. The Tailored Biosimilar Approach: Expectations and Requirements. Drugs. 2025;85(5):601-608. doi: 10.1007/s40265-025-02168-y.

Kaminski RW, Pavlinac PB, Platts-Mills JA, Rogawski McQuade ET, Hausdorff WP, Isbrucker RA, et al. WHO Workshop Report: Regulatory Science to Inform Clinical Pathways for Shigella Vaccines Intended for Use in Children in Low- and Middle-Income Countries. Vaccines (Basel). 2025;13(5):439. doi: 10.3390/vaccines13050439.

Gonzalez Dorta H, Verbeeck J, Crevecoeur J, Morales DR, Loedy N, Cohet C, et al. Utilising the Benefit Risk Assessment of Vaccines (BRAVE) toolkit to evaluate the benefits and risks of Vaxzevria in the EU: a population-based study. Lancet Digit Health. 2025;7(5):100861. doi: 10.1016/j.landig.2025.02.001.

Hidalgo-Simon A, Pothet C, Cooke E, Thirstrup S. Academic development of advanced therapies-How to foster their future in the clinic. Br J Clin Pharmacol. 2025;91(4):1080-1083. doi: 10.1002/bcp.70024

Szmigiel A, da Rocha MM, Browne K, Morales D, Olsen DB, Warren-Gash C, et al. Association Between β-Adrenoreceptor Agonists and Antagonists and Parkinson's Disease: Systematic Review and Meta-Analysis. Pharmacoepidemiol Drug Saf. 2025;34(4):e70140. doi: 10.1002/pds.70140

Huber C, Zinserling J, Benda N, Vetter T, Rueckbeil M. Methodological Insights on Biomarker-Based Patient Selection: A Review of Scientific Advice Procedures at the European Medicines Agency. Clin Pharmacol Ther. 2025;117(5):1226-1235. doi: 10.1002/cpt.3558

Bivi N, Graham D, Joglekar L, McGuire K, Stoop J, Zoghbi J, et al. 2024 White paper on recent issues in bioanalysis: Impact of LDT in US and IVDR in EU; AI/ML for High Parameter Flow Cytometry; The rise of Olink Technology; CDx for AAV Gene Therapies; Integrative Bioanalysis by Multiple Platforms; Super Sensitive ADA/NAb LBA (PART 2A - Recommendations on Advanced Strategies for Biomarkers, IVD/CDx Assays (BAV), Cell Based Assays (CBA), and Ligand-Binding Assays (LBA) PART 2B - Regulatory Agencies' Input on Biomarkers, IVD/CDx, and Biomarker Assay Validation). Bioanalysis. 2025;17(4):211-248. doi: 10.1080/17576180.2024.2442218

Garcia Burgos J, Thirstrup S. EMA approvals of new medicinal products and new biosimilar products in January and February 2025. Lancet Reg Health Eur. 2025; 51:101279. doi: 10.1016/j.lanepe.2025.101279.

Cavaleri M, de Sousa CMA, Hacker A, Higgs ES, Lumpkin MM, Maia CS, et al. A roadmap for fostering timely regulatory and ethics approvals of international clinical trials in support of global health research systems. Lancet Glob Health. 2025;13(4):e769-e777. doi: 10.1016/S2214-109X(24)00515-1.

Hermans AMM, Bakker E, Starokozhko V, den Otter L, Elferink AJA, Bradshaw A, et al. Biomarkers for neurodegenerative diseases in regulatory decision-making by the European Medicines Agency. Alzheimers Dement (N Y). 2025;11(1):e70072. doi: 10.1002/trc2.70072.

Gobat N, Slack C, Hannah S, Salzwedel J, Bladon G, Garcia Burgos J, et al. Better engagement, better evidence: working in partnership with patients, the public, and communities in clinical trials with involvement and good participatory practice. Lancet Glob Health. 2025;13(4):e716-e731. doi: 10.1016/S2214-109X(24)00521-7.

Silva F, Butlen-Ducuing F, Guizarro L, Balabanov P. A review of psychedelics trials completed in depression, informed by European regulatory perspectives. Neurosci Appl. 2025;4:105516. doi: 10.1016/j.nsa.2025.105516.

Bell J, Drury T, Mütze T, Pipper CB, Guizzaro L, Mitroiu M, et al. Estimation Methods for Estimands Using the Treatment Policy Strategy; a Simulation Study Based on the PIONEER 1 Trial. Pharm Stat. 2025;24(2):e2472. doi: 10.1002/pst.2472.

Fuggle N, Laslop A, Rizzoli R, Al-Daghri N, Alokail M, Balkowiec-Iskra E, et al. Treatment of Osteoporosis and Osteoarthritis in the Oldest Old. Drugs. 2025;85(3):343-360. doi: 10.1007/s40265-024-02138-w.

Lasch F, Carvalho JRB, Pothet C. Demonstration of Major Therapeutic Advantage From a Review of EU Conditional Marketing Authorizations in Oncology and Hematology. Clin Pharmacol Ther. 2025;117(4):1098-1105. doi: 10.1002/cpt.3554.

Barniol J, Rugo K, Hendrix M, Harvey Allchurch M, Tubic B, Ahmeti A, et al. Accession to the EU: How Can the EMA Pre-Accession Assistance Program Help? DIA Global Forum 2025;17 (2)

Kuaban A, Croker AK, Keefer J, Valentino LA, Bierer BE, Boateng S, et al. The roadmap to integrate diversity, equity, and inclusion in hematology clinical trials: an American Society of Hematology initiative. Blood Adv. 2025;9(4):687-695. doi: 10.1182/bloodadvances.2024013945.

Butlen-Ducuing F, Silva F, Silva I, Balabanov P, Thirstrup S. Applying the EU regulatory framework for the clinical use of psychedelics. Lancet Psychiatry. 2025;12(1):7-9. doi: 10.1016/S2215-0366(24)00203-7.

Lynggaard H, McKendrick S, Baird M, Kerwash E, Lanius V, Lasch F, et al. Applying the estimand framework to clinical pharmacology trials with a case study in bioequivalence. Br J Clin Pharmacol. 2025;91(2):310-324. doi: 10.1111/bcp.16347.

Pearson AD, Mueller S, Filbin MG, Grill J, Hawkins C, Jones C, et al. Paediatric strategy forum for medicinal product development in diffuse midline gliomas in children and adolescents ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration. Eur J Cancer. 2025;217:115230. doi: 10.1016/j.ejca.2025.115230.

Schaefer R, Donaldson L, Chigome A, Escudeiro Dos Santos M, Lamprianou S, Ndembi N, et al. Antiretroviral Use for HIV Prevention During Pregnancy: The Need to Strengthen Regulatory and Surveillance Systems in Africa. Drug Saf. 2025;48(3):209-216. doi: 10.1007/s40264-024-01494-6.

Stewart B, Lepola P, Egger GF, Ali F, Allen AJ, Croker AK, et al. Requirements and special considerations for drug trials with children across six jurisdictions: 1. Clinical trial application review in the regulatory approval process. Front Med (Lausanne). 2025;12:1542408. doi: 10.3389/fmed.2025.1542408.

Stewart B, Lepola P, Egger GF, Ali F, Allen AJ, Croker AK, et al. Requirements and special considerations for drug trials with children across six jurisdictions: 2. Ethics review in the regulatory approval process. Front Med (Lausanne). 2025;12:1539787. doi: 10.3389/fmed.2025.1539787.

Tavares D, Chien H-T, Sheean ME, Theunissen P, van Meer P, Van Malderen K. From guideline to practice: three years of ICH S11 insights and recommendations. Front Med (Lausanne). 2025;12:1518936. doi: 10.3389/fmed.2025.1537001.

Paul P, Colin PJ, Musuamba Tshinanu F, Versantvoort C, Manolis E, et al. Current Use of Physiologically Based Pharmacokinetic modeling in New Medicinal Product Approvals at EMA. Clin Pharmacol Ther. 2025;117(3):808-817. doi: 10.1002/cpt.3525.

Gravanis I, Berntgen M, Vamvakas S, Demolis P, Foggi P. Challenges and ongoing initiatives towards better integrated EU scientific advice. Front Med (Lausanne). 2025;12:1473346. doi: 10.3389/fmed.2025.1473346.

Karres D, Pino-Barrio MJ, Benchetrit S, Benda N, Cochat P, Galluzzo S, et al. Evidence generation throughout paediatric medicines life cycle: findings from collaborative work between European Medicines Agency (EMA) and EUnetHTA on use of extrapolation. Br J Pharmacol. 2025;182(3):484-494. doi: 10.1111/bph.17396

Beck AE, Kampman M, Huynh C, Simon C, Plueschke K, Cohet C, et al. Collaborative Real-World Evidence Among Regulators: Lessons and Perspectives. Clin Pharmacol Ther. 2025;117(2):368-373. doi: 10.1002/cpt.3457

Correia Pinheiro L, Arlett P, Roes K, Musuamba Tshinanu F, Westman G, Frias Z, et al. Artificial Intelligence in European Medicines Regulation: From Vision to Action. Harnessing the Capabilities of Artificial Intelligence for the Benefit of Public and Animal Health. Clin Pharmacol Ther. 2025;117(2):335-336. doi: 10.1002/cpt.3494

Blanquie O, Guizzaro L, Herold R, Haberkamp M, Elferink A, Balabanov P, et al. EU regulatory horizons for Alzheimer's disease. Lancet Neurol. 2025; 24(2):96-97. doi:10.1016/S1474-4422(25)00004-3

Tannergren C, Arora S, Babiskin A, Borges L, Chatterjee P, Cheng YH, et al. Current State and New Horizons in Applications of Physiologically Based Biopharmaceutics Modeling (PBBM): A Workshop Report. Mol Pharm. 2025;22(1):5-27. doi: 10.1021/acs.molpharmaceut.4c01148

Zaragoza Domingo S, Pinkham A, Tinoco D, Kottner J, Balabanov P, Butlen-Ducuing F. Use of Standard Methodology for Novel Outcomes in Clinical Trials: Advantages for New Therapeutics in Neurosciences. Clin Schizophr Relat Psychoses. 2025;19(1).

2024

Brivio E, Karres D, Lesa G, Ligas F. Approved medicines for paediatric solid tumours in Europe: Lessons from the life cycle of a paediatric investigation plan. EJC Paed Oncol 2024;4,100190. Doi: 10.1016/j.ejcped.2024.100190.

Prunas O, Willemsen J, Warren JL, Bont L, Schwartz JL, Atwell J, et al. Workshop on the design and use of clinical trials with multiple endpoints, with a focus on prevention of RSV. Vaccine X. 2024;19:100509. doi: 10.1016/j.jvacx.2024.100509.

Agricola E, Auriche-Benichou C, Baiao H, Blanquie O, Bodea T, Boráň T, et al. The European Innovation Network as a hub for medicines innovation in Europe. Nat Rev Drug Discov. 2024;23(6):405-406. doi: 10.1038/d41573-024-00039-x.

Dessy F, Sonderegger I, Wagner L, Buoninfante A, Wadhwa M, Agnes J, et al. Harmonization of Vaccine Ligand Binding Assays Validation. Bioanalysis. 2024;16(19-20):1067-1091. doi: 10.1080/17576180.2024.2411925.

Brinkhuis F, Ruof J, van den Ham H, Gianfrate F, Strammiello V, Berntgen M, et al. Evaluating progress towards implementation of the European HTA Regulation: Insights generated from the European Access Academy's multi-stakeholder survey. Health Policy and Technology. 2024;13(5):100930. doi: 10.1016/j.hlpt.2024.100930

Manolis E, Musuamba FT, de Vries CS, Colin PJ, Oleksiewicz MB. EMA perspective on the value of model-informed drug development for labeling recommendations regarding medicine use during pregnancy and breastfeeding. CPT Pharmacometrics Syst Pharmacol. 2024;13(11):1820-1823. doi: 10.1002/psp4.13214.

Larsson K. What Is Rare and What Is Orphan? A Guide to the Regulatory Terminology. Clin Pharmacol Ther. 2024 Sep 17. doi: 10.1002/cpt.3446.

Curtis EM, Miguel M, McEvoy C, Ticinesi A, Torre C, Al-Daghri N, et al. Impact of dementia and mild cognitive impairment on bone health in older people. Aging Clin Exp Res. 2024;37(1):5. doi: 10.1007/s40520-024-02871-y.

Martirosyan L, Satta MG, Garcia Burgos J, Wändel-Liminga U, Straus S. Commentary/Response to Damkier et al. Pharmacoepidemiol Drug Saf. 2024;33(12):e70058. doi: 10.1002/pds.70058.

Taams AC, Herberts CA, Egberts ACG, Zafiropoulos N, Pignatti F, Bloem LT. Uncertainties about the benefit-risk balance of oncology medicines assessed by the European Medicines Agency. ESMO Open. 2024;9(12):103991. doi: 10.1016/j.esmoop.2024.103991.

Windfuhr F, Larsson K, Framke T, Lasch F. Which clinical trial designs and statistical approaches have been used in assessments of orphan maintenance by the European Medicines Agency between 2012 and 2022? A cross-sectional study. BMJ Open. 2024;14(12):e086171. doi: 10.1136/bmjopen-2024-086171.

Bertagnolio S, Dobreva Z, Centner CM, Olaru ID, Donà D, Burzo S, et al. WHO global research priorities for antimicrobial resistance in human health. Lancet Microbe. 2024;5(11):100902. doi: 10.1016/S2666-5247(24)00134-4.

Drmić A, Saccà R, Vetter T, Ehmann F. Identifying and overcoming challenges in the EMA's qualification of novel methodologies: a two-year review. Front Pharmacol. 2024;15:1470908. doi: 10.3389/fphar.2024.1470908

Du M, Dernie F, Català M, Delmestri A, Man WY, Brash JT, et al. Treatment of systemic lupus erythematosus: Analysis of treatment patterns in adult and paediatric patients across four European countries. Eur J Intern Med. 2024;130:106-117. doi: 10.1016/j.ejim.2024.08.008

Zaccaria C, Piccolo L, Gordillo-Marañón M, Touraille G, de Vries C. Identification of Pregnancy Adverse Drug Reactions in Pharmacovigilance Reporting Systems: A Novel Algorithm Developed in EudraVigilance. Drug Saf. 2024;47(11):1127-1136. doi: 10.1007/s40264-024-01448-y

Dernie F, Corby G, Robinson A, Bezer J, Mercade-Besora N, Griffier R, et al. Standardised and Reproducible Phenotyping Using Distributed Analytics and Tools in the Data Analysis and Real World Interrogation Network (DARWIN EU). Pharmacoepidemiol Drug Saf. 2024;33(11):e70042. doi: 10.1002/pds.70042

Alcalde-Herraiz M, Xie J, Newby D, Prats C, Gill D, Gordillo-Marañón M, et al. Effect of genetically predicted sclerostin on cardiovascular biomarkers, risk factors, and disease outcomes. Nat Commun. 2024;15(1):9832. doi: 10.1038/s41467-024-53623-5

Lee SH, Hotaki LT, Oh K, Samuel J, Villiers K de, Eshetie K, et al. Index of application status transparency and availability of public information for Project Orbis agencies. Int. J. Drug Reg. Affairs [Internet]. 2024; 12(3):55-65. doi: 10.22270/ijdra.v12i3.699

Cerreta F, Iskra EB, Cupelli A, Sepodes B, Rönnemaa E, Rosa MM, et al. Medicines for an aging population: The EMA perspective and policies. J Am Geriatr Soc. 2024;72(9):2921-2927. doi: 10.1111/jgs.18953

Bell K, White S, Diaz A, Bahri P, Sima F, Al-Delaimy WK, et al. Can evidence drive health equity in the COVID-19 pandemic and beyond? J Public Health Policy. 2024;45(1):137-151. doi: 10.1057/s41271-023-00452-3

Shivji R, Grabski E, Jekerle V. Scientific and Regulatory Lessons Learnt on Building a Chemistry, Manufacturing, and Controls (CMC) Package for COVID-19 Variant Vaccine Updates in the EU—A Regulator’s Perspective. Vaccines. 2024;12(11):1234. doi: 10.3390/vaccines12111234

Bronzwaer S, de Coen W, Heuer O, Marnane I, Vidal A. The framework for action of the Cross-agency One Health Task Force. One Health. 2024;19:100925. doi:10.1016/j.onehlt.2024.100925

Grupstra RJ, Goedecke T, Gardarsdottir H. Limitations Reported in Evaluating Effectiveness of Risk Minimization Measures in the EU during 2018-2021: A Qualitative Analysis of Industry-Sponsored Post-Authorization Safety Studies. Clin Pharmacol Ther. 2024;116(5):1252-1258. doi: 10.1002/cpt.3369

Uster DW, Cordo' V, Cormier E, Ehmann F. Insights into Early Interactions on Innovative Developments with European Regulators. Ther Innov Regul Sci. 2024;58(6):1108-1119. doi: 10.1007/s43441-024-00686-7

Schoenmakers DH, van den Berg S, Timmers L, Adang LA, Bäumer T, Bosch A, et al. Framework for Multistakeholder Patient Registries in the Field of Rare Diseases: Focus on Neurogenetic Diseases. Neurology. 2024 Sep 24;103(6):e209743. doi: 10.1212/WNL.0000000000209743

Prilla S, Groeneveld S, Pacurariu A, Restrepo-Méndez MC, Verpillat P, Torre C, et al. Real-World Evidence to Support EU Regulatory Decision Making-Results From a Pilot of Regulatory Use Cases. Clin Pharmacol Ther. 2024;116(5):1188-1197. doi: 10.1002/cpt.3355.

Moseley J, Leest T, Larsson K, Magrelli A, Stoyanova-Beninska V. Inherited retinal dystrophies and orphan designations in the European Union. Eur J Ophthalmol. 2024;34(6):1631-1641. doi: 10.1177/11206721241236214.

Rippin G, Sanz H, Hoogendoorn WE, Ballarini NM, Largent JA, Demas E, et al. Examining the Effect of Missing Data and Unmeasured Confounding on External Comparator Studies: Case Studies and Simulations. Drug Saf. 2024 Dec;47(12):1245-1263. doi: 10.1007/s40264-024-01467-9

Hermans SJF, van der Maas NG, van Norden Y, Dinmohamed AG, Berkx E, Huijgens PC, et al. Externally Controlled Studies Using Real-World Data in Patients With Hematological Cancers: A Systematic Review. JAMA Oncol. 2024;10(10):1426-1436. doi: 10.1001/jamaoncol.2024.3466.

Bahri P, Genov G, Arlett P, Šarinić VM, Korakianiti E, Nolte A, et al. The STAR Compass to Guide Future Pharmacovigilance Based on a 10-Year Review of the Strengthened EU System.Drug Saf. 2024;47(10):941-956. doi: 10.1007/s40264-024-01451-3.

Tazare J, Wang SV, Gini R, Prieto-Alhambra D, Arlett P, Morales Leaver DR, et al. Sharing Is Caring? International Society for Pharmacoepidemiology Review and Recommendations for Sharing Programming Code. Pharmacoepidemiol Drug Saf. 2024;33(9):e5856. doi: 10.1002/pds.5856.

Lawler M, Keeling P, Kholmanskikh O, Minnaard W, Moehlig-Zuttermeister H, Normanno N, et al. Empowering effective biomarker-driven precision oncology: A call to action. Eur J Cancer. 2024;209:114225. doi: 10.1016/j.ejca.2024.114225.

O'Connor TF, Chatterjee S, Lam J, de la Ossa DHP, Martinez-Peyrat L, Hoefnagel MHN, et al. An examination of process models and model risk frameworks for pharmaceutical manufacturing. Int J Pharm X. 2024;8:100274. doi: 10.1016/j.ijpx.2024.100274.

Lienhardt C, Dooley KE, Nahid P, Wells C, Ryckman TS, Kendall EA, et al. Target regimen profiles for tuberculosis treatment. Bull World Health Organ. 2024;102(8):600-607. doi: 10.2471/BLT.24.291881.

Haberkamp M, Aislaitner G, Martínez-Lapiscina EH, Weise M. Tofersen for SOD-1-associated amyotrophic lateral sclerosis. Lancet Neurol. 2024;23(8):772-773. doi: 10.1016/S1474-4422(24)00259-X.

Gini R, Pajouheshnia R, Gutierrez L, Swertz MA, Hyde E, Sturkenboom M, et al. Metadata for Data dIscoverability aNd Study rEplicability in obseRVAtional Studies (MINERVA): Lessons Learnt From the MINERVA Project in Europe. Pharmacoepidemiol Drug Saf. 2024;33(8):e5884. doi: 10.1002/pds.5884.

Pepin X, Arora S, Borges L, Cano-Vega M, Carducci T, Chatterjee P, et al. Parameterization of Physiologically Based Biopharmaceutics Models: Workshop Summary Report. Mol Pharm. 2024;21(8):3697-3731. doi: 10.1021/acs.molpharmaceut.4c00526.

Pajouheshnia R, Gini R, Gutierrez L, Swertz MA, Hyde E, Sturkenboom M, et al. Metadata for Data dIscoverability aNd Study rEplicability in obseRVAtional Studies (MINERVA): Development and Pilot of a Metadata List and Catalogue in Europe. Pharmacoepidemiol Drug Saf. 2024;33(8):e5871. doi: 10.1002/pds.5871.

Gordillo-Marañón M, Szmigiel A, Yalmanová V, Caplanusi I, Genov G, Olsen DB, et al. COVID-19 Vaccines and Heavy Menstrual Bleeding: The Impact of Media Attention on Reporting to EudraVigilance. Drug Saf. 2024;47(8):783-798. doi: 10.1007/s40264-024-01426-4.

Buchholz S, Di Meco E, Bałkowiec-Iskra EZ, Sepodes B, Cavaleri M. Generating clinical evidence for treatment and prevention options for long COVID. Nat Med. 2024;30(8):2109-2110. doi: 10.1038/s41591-024-03031-5.

Pearson AD, DuBois SG, Macy ME, de Rojas T, Donoghue M, Weiner S, et al. Paediatric strategy forum for medicinal product development of PI3-K, mTOR, AKT and GSK3β inhibitors in children and adolescents with cancer. Eur J Cancer. 2024;207:114145. doi: 10.1016/j.ejca.2024.114145.

Van der Schueren B, Vrijlandt P, Thomson A, Janssen H, Dunder K. New guideline of the European Medicines Agency (EMA) on the clinical investigation of medicinal products in the treatment and prevention of diabetes mellitus. Diabetologia. 2024;67(7):1159-1162. doi: 10.1007/s00125-024-06162-z.

Kalland ME, Pose-Boirazian T, Palomo GM, Naumann-Winter F, Costa E, Matusevicius D, et al. Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation. Gene Ther. 2024;31(7-8):366-377. doi: 10.1038/s41434-024-00446-0.

Brinkhuis F, Julian E, van den Ham H, Gianfrate F, Strammiello V, Berntgen M, et al. Navigating the path towards successful implementation of the EU HTA Regulation: key takeaways from the 2023 Spring Convention of the European Access Academy. Health Res Policy Syst. 2024;22(1):74. doi: 10.1186/s12961-024-01154-2.

Gordillo-Marañón M, Candore G, Hedenmalm K, Browne K, Flynn R, Piccolo L, et al. Lessons Learned on Observed-to-Expected Analysis Using Spontaneous Reports During Mass Vaccination. Drug Saf. 2024;47(7):607-615. doi: 10.1007/s40264-024-01422-8.

Buoninfante A, Andeweg A, Genov G, Cavaleri M. Myocarditis associated with COVID-19 vaccination. NPJ Vaccines. 2024;9(1):122. doi: 10.1038/s41541-024-00893-1.

Silva I, Gabrielli G, Garcia Burgos J, Moulon I, Calvo Rojas G, Jaeger U, et al. A decade of collaboration in medicines regulation: healthcare professionals engaging with the European Medicines Agency. Front Med (Lausanne). 2024;11:1399947. doi: 10.3389/fmed.2024.1399947.

Fangusaro J, Avery RA, Fisher MJ, Packer RJ, Walsh KS, Schouten-van Meeteren A, et al. Considering Functional Outcomes as Efficacy Endpoints in Pediatric Low-Grade Glioma Clinical Trials: An FDA Educational Symposium. Clin Cancer Res. 2024;30(11):2303-2308. doi: 10.1158/1078-0432.CCR-23-3386.

Kruhlak NL, Schmidt M, Froetschl R, Graber S, Haas B, Horne I, et al. Determining recommended acceptable intake limits for N-nitrosamine impurities in pharmaceuticals: Development and application of the Carcinogenic Potency Categorization Approach (CPCA). Regul Toxicol Pharmacol. 2024;150:105640. doi: 10.1016/j.yrtph.2024.105640.

Zaragoza Domingo S, Alonso J, Ferrer M, Acosta MT, Alphs L, Annas P, et al. Methods for Neuroscience Drug Development: Guidance on Standardization of the Process for Defining Clinical Outcome Strategies in Clinical Trials. Eur Neuropsychopharmacol. 2024;83:32-42. doi: 10.1016/j.euroneuro.2024.02.009.

Almeida D, Umuhire D, Gonzalez-Quevedo R, António A, Burgos JG, Verpillat P, et al. Leveraging patient experience data to guide medicines development, regulation, access decisions and clinical care in the EU. Front Med (Lausanne). 2024;11:1408636. doi: 10.3389/fmed.2024.1408636.

Taskén K, Haj Mohammad SF, Fagereng GL, Sørum Falk R, Helland Å, Van Waalwijk van Doorn-Khosrovani SB, et al. PCM4EU and PRIME-ROSE: Collaboration for implementation of precision cancer medicine in Europe. Acta Oncol. 2024;63:385-391. doi: 10.2340/1651-226X.2024.34791.

Paixao P, Garcia Arieta A, Silva N, Petric Z, Bonelli M, Morais JAG, et al. A Two-Way Proposal for the Determination of Bioequivalence for Narrow Therapeutic Index Drugs in the European Union. Pharmaceutics. 2024;16(5):598. doi: 10.3390/pharmaceutics16050598.

Goedecke T, Martirosyan L, Gault N, Seifert K, Morales DR, Bahri P, et al. Studying the impact of European Union regulatory interventions for minimising risks from medicines: lessons learnt and recommendations. Pharmacoepidemiol Drug Saf. 2024;33(8):e5874. doi: 10.1002/pds.5874.

Zaratin P, Samadzadeh S, Seferoğlu M, Ricigliano V, Dos Santos Silva J, Tunc A, et al. The global patient-reported outcomes for multiple sclerosis initiative: bridging the gap between clinical research and care - updates at the 2023 plenary event. Front Neurol. 2024;15:1407257. doi: 10.3389/fneur.2024.1407257

Billingham L, Brown L, Framke T, Greystoke A, Hovig E, Mathur S, et al. Histology independent drug development - Is this the future for cancer drugs? Cancer Treat Rev. 2024;123:102674. doi: 10.1016/j.ctrv.2023.102674

Capone G, Eriksson B, Al M, van Belkum S, Broich K, Lamas M, et al. Accelerating clinical trials in the EU (ACT EU): transforming the EU clinical trials landscape. Nat Rev Drug Discov. 2024. doi: 10.1038/d41573-024-00085-5

Gordillo-Marañón M, Candore G, López-Fauqued M, Deli KC, Piccolo L, Alcini P, et al. Predicting the submission frequency of periodic safety update reports: development and application of the EURD tool. Front Med (Lausanne). 2024;11:1299190. doi: 10.3389/fmed.2024.1299190

Tavridou A, Rogers D, Farinelli G, Gravanis I, Jekerle V. Genome-editing medicinal products: the EMA perspective. Nat Rev Drug Discov. 2024;23(4):242-243. doi: 10.1038/d41573-024-00050-2

Parciak T, Geys L, Helme A, van der Mei I, Hillert J, Schmidt H, et al. Introducing a core dataset for real-world data in multiple sclerosis registries and cohorts: Recommendations from a global task force. Mult Scler. 2024;30(3):396-418. doi: p10.1177/13524585231216004p

Pesiou S, Barcelo R, Papazisis G, Torres F, Pontes C. Prevalence of use of on-label and off-label psychotropics in the Greek pediatric population. Front. Pharmacol. 2024;15:1348887. doi:10.3389/fphar.2024.1348887

McCulloch DE, Liechti ME, Kuypers KPC, Nutt D, Lundberg J, Stenbæk DS, et al. Knowledge gaps in psychedelic medicalisation: Clinical studies and regulatory aspects. Neuroscience Applied. 2024;3:103938. doi: https://doi.org/10.1016/j.nsa.2024.103938

Van Spall HGC, Bastien A, Gersh B, Greenberg B, Mohebi R, Min J, et al. The role of early-phase trials and real-world evidence in drug development. Nature Cardiovasc Res. 2024;3(2):110-7. doi: 10.1038/s44161-024-00420-4

Caplanusi I, Szmigiel A, van der Elst M, Schougaard Christiansen ML, Thirstrup S, Zaccaria C, et al. The Role of the European Medicines Agency in the Safety Monitoring of COVID-19 Vaccines and Future Directions in Enhancing Vaccine Safety Globally. Drug Saf. 2024 May;47(5):405-418. doi: 10.1007/s40264-024-01405-9

Aiyegbusi OL, Cruz Rivera S, Roydhouse J, Kamudoni P, Alder Y, Anderson N, et al. Recommendations to address respondent burden associated with patient-reported outcome assessment. Nat Med. 2024 Mar;30(3):650-659. doi: 10.1038/s41591-024-02827-9

Horan WP, Sachs G, Velligan DI, Davis M, Keefe RSE, Khin NA, et al. Current and Emerging Technologies to Address the Placebo Response Challenge in CNS Clinical Trials: Promise, Pitfalls, and Pathways Forward. Innov Clin Neurosci. 2024 Mar 1;21(1-3):19-30

Stacchiotti S, Bouche G, Herold R, Pantziarka P, Schuster K, Wilson R, et al. How to develop new systemic treatments in ultra-rare cancers with high unmet needs? The case of alveolar soft-part sarcoma. Eur J Cancer. 2024 May;202:114003. doi: 10.1016/j.ejca.2024.114003

Shea B, Pardo JP, Grosskleg S, Beaton DE, Conaghan P, Goettsch W, et al. Increasing uptake through collaboration in the development of core outcome sets: Lessons learned at OMERACT 2023. Semin Arthritis Rheum. 2024 Jun;66:152438. doi: 10.1016/j.semarthrit.2024.152438

Hermans SJF, van Norden Y, Versluis J, Rijneveld AW, van der Holt B, de Weerdt O, et al. Benefits and risks of clofarabine in adult acute lymphoblastic leukemia investigated in depth by multi-state modeling. Cancer Med. 2024 May;13(9):e6756. doi: 10.1002/cam4.6756

Xie J, Mothe B, Alcalde Herraiz M, Li C, Xu Y, Jödicke AM, et al. Relationship between HLA genetic variations, COVID-19 vaccine antibody response, and risk of breakthrough outcomes. Nat Commun. 2024 May 13;15(1):4031. doi: 10.1038/s41467-024-48339-5

Mackie C, Arora S, Seo P, Moody R, Rege B, Pepin X, et al. Physiologically Based Biopharmaceutics Modeling (PBBM): Best Practices for Drug Product Quality, Regulatory and Industry Perspectives: 2023 Workshop Summary Report. Mol Pharm. 2024 May 6;21(5):2065-2080. doi: 10.1021/acs.molpharmaceut.4c00202

Baratta M, Jian W, Hengel S, Kaur S, Cunliffe J, Boer J, et al. 2023 White Paper on Recent Issues in Bioanalysis: Deuterated Drugs; LNP; Tumor/FFPE Biopsy; Targeted Proteomics; Small Molecule Covalent Inhibitors; Chiral Bioanalysis; Remote Regulatory Assessments; Sample Reconciliation/Chain of Custody (PART 1A - Recommendations on Mass Spectrometry, Chromatography, Sample Preparation Latest Developments, Challenges, and Solutions and BMV/Regulated Bioanalysis PART 1B - Regulatory Agencies' Inputs on Regulated Bioanalysis/BMV, Biomarkers/IVD/CDx/BAV, Immunogenicity, Gene & Cell Therapy and Vaccine). Bioanalysis. 2024;16(9):307-364. doi: 10.1080/17576180.2024.2347153.

Celis P, Farinelli G, Hidalgo-Simon A, Meij P, Tihaya M, Schüssler-Lenz M, et al. EMA commentary on the guideline on quality, nonclinical and clinical aspects of medicinal products containing genetically modified cells. Br J Clin Pharmacol. 2024;90(5):1203-1212. doi: 10.1111/bcp.16047.

Osborne V, Goodin A, Brown J, Winterstein AG, Bate A, Cohet C, Pont L, Moeny D, Klungel O, Pinheiro S, Seeger J, Chan KA, Edlavitch S, Tilson H, Layton D. Updated core competencies in pharmacoepidemiology to inform contemporary curricula and training for academia, government, and industry. Pharmacoepidemiol Drug Saf. 2024;33(4):e5789. doi: 10.1002/pds.5789.

van der Maas NG, Versluis J, Nasserinejad K, van Rosmalen J, Pabst T, Maertens J, et al. Bayesian interim analysis for prospective randomized studies: reanalysis of the acute myeloid leukemia HOVON 132 clinical trial. Blood Cancer J. 2024;14(1):56. doi: 10.1038/s41408-024-01037-3.

Desmet T, Julian E, Van Dyck W, Huys I, Simoens S, Giuliani R, et al. An Inclusive Civil Society Dialogue for Successful Implementation of the EU HTA Regulation: Call to Action to Ensure Appropriate Involvement of Stakeholders and Collaborators. J Mark Access Health Policy. 2024;12(1):21-34. doi: 10.3390/jmahp12010004.

Cavaleri M, Kaslow D, Boateng E, Chen WH, Chiu C, Choy RKM, et al. Fourth Controlled Human Infection Model (CHIM) meeting, CHIM regulatory issues, May 24, 2023. Biologicals. 2024;85:101745. doi: 10.1016/j.biologicals.2024.101745.

Mészáros L, Lasch F, Delafont B, Guizzaro L. Estimands in CNS trials - A review of strategies for addressing intercurrent events. Contemp Clin Trials Commun. 2024;38:101266. doi: 10.1016/j.conctc.2024.101266.

Ehmann F, Kuhn A, Pasmooij AMG, Humphreys A, Van Hengel A, Dooley B, et al. Report of the European Medicines Agency Conference on RNA-Based Medicines. Nucleic Acid Ther. 2024;34(1):4-11. doi: 10.1089/nat.2023.0021.

Pearson ADJ, de Rojas T, Karres D, Reaman G, Scobie N, Fox E, et al. Impact of ACCELERATE Paediatric Strategy Forums: a review of the value of multi-stakeholder meetings in oncology drug development. J Natl Cancer Inst. 2024;116(2):200-207. doi: 10.1093/jnci/djad239.

Lo Re III V, Cocoros NM, Hubbard RA, Dutcher SK, Newcomb CW, Connolly JG, et al. Risk of Arterial and Venous Thrombotic Events Among Patients with COVID-19: A Multi-National Collaboration of Regulatory Agencies from Canada, Europe, and United States. Clin Epidemiol. 2024;16:71-89. doi: 10.2147/CLEP.S448980.

Silva M, Moseley J, Vetter T, Regnstrom J, Tome M, Aarum S, et al. Patient-reported, observer-reported and performance outcomes in qualification procedures at the European Medicines Agency 2013-2018. Br J Clin Pharmacol. 2024;90(1):299-312. doi: 10.1111/bcp.15907.

Postmus D, Litiere S, Bogaerts J, Versluis J, Cornelissen JJ, Pignatti F. Attitudes of healthcare professionals and drug regulators about progression-free survival as endpoint in the advanced cancer setting. Eur J Cancer. 2024;197:113496. doi: 10.1016/j.ejca.2023.113496.

Mészáros L, Guizzaro L. Developing medicines for the pre-symptomatic stage of degenerative neurological conditions: Challenges and opportunities. Rev Neurol (Paris). 2024;180(3):141-146. doi: 10.1016/j.neurol.2023.06.002.

Cortese S, Purper-Ouakil D, Apter A, Arango C, Baeza I, Banaschewski T, et al. Psychopharmacology in children and adolescents: unmet needs and opportunities. Lancet Psychiatry. 2024;11(2):143-154. doi: 10.1016/S2215-0366(23)00345-0.

Cannata C, Backhaus T, Bramke I, Caraman M, Lombardo A, Whomsley R, et al. Prioritisation of data-poor pharmaceuticals for empirical testing and environmental risk assessment. Environ Int. 2024;183:108379. doi: 10.1016/j.envint.2023.108379.

Cernuschi T, Malvolti S, Hall S, Debruyne L, Bak Pedersen H, Rees H, et al. The quest for more effective vaccine markets - Opportunities, challenges, and what has changed with the SARS-CoV-2 pandemic. Vaccine. 2024;42(S1):S64-S72. doi: 10.1016/j.vaccine.2022.07.032.

Chlebus M, Thirstrup S, Ganan Jimenez A, Nothelfer K, Franco P, Bakker A, et al. How “Team Europe” Can Co-Create the Healthcare R&D Framework of Tomorrow. DIA Global Forum 2024; 16(8)

Jjingo CJ, Bala S, Waack U, Needles M, Bensman TJ, McMaster O, et al. FDA Public Workshop Summary-Addressing Challenges in Inhaled Antifungal Drug Development. Clin Infect Dis. 2024;78(6):1564-1570. doi: 10.1093/cid/ciad607.

Muñoz J, Efthimiou O, Audigier V, de Jong VMT, Debray TPA. Multiple imputation of incomplete multilevel data using Heckman selection models. Stat Med. 2024;43(3):514-533. doi: 10.1002/sim.9965.

Bhatnagar M, Burckart G, Snyder D, Bax R, Yao L, Green D. Regulatory considerations in the design and conduct of pediatric clinical trials. In: Gasthuys E, Allegaert K, Dossche L, Turner M, editors. Essentials of Translational Pediatric Drug Development: From Past Needs to Future Opportunities. Elsevier Health Sciences; 2024. pp. 385-419.

Abed I, Garcia Burgos J, Knudsen Y. Public information on shortages in the EU/EEA: improvements made between 2018 and 2020. Eur J Hosp Pharm. 2024 Jun 21;31(4):344-347. doi: 10.1136/ejhpharm-2022-003554.

2023

Colloud S, Metcalfe T, Askin S, Belachew S, Ammann J, Bos E, et al. Evolving regulatory perspectives on digital health technologies for medicinal product development. NPJ Digit Med. 2023;6(1):56. doi: 10.1038/s41746-023-00790-2.

Bahri P, Bowring G, Edwards BD, Anton C, Aronson JK, Caro-Rojas A, et al. Communicating for the Safe Use of Medicines: Progress and Directions for the 2020s Promoted by the Special Interest Group of the International Society of Pharmacovigilance. Drug Saf. 2023;46(6):517-532. doi: 10.1007/s40264-023-01285-5.

Croft NM, de Ridder L, Griffiths AM, Hyams JS, Ruemmele FM, et al. Paediatric Inflammatory Bowel Disease: A Multi-Stakeholder Perspective to Improve Development of Drugs for Children and Adolescents. J Crohns Colitis. 2023;17(2):249-258. doi: 10.1093/ecco-jcc/jjac135.

Brady LS, Larrauri CA; AMP SCZ Steering Committee. Accelerating Medicines Partnership® Schizophrenia (AMP® SCZ): developing tools to enable early intervention in the psychosis high risk state. World Psychiatry. 2023;22(1):42-43. doi: 10.1002/wps.21038.

Hofner B, Asikanius E, Jacquet W, Framke T, Oude Rengerink K, Aguirre Dávila L, et al. Vaccine Development during a Pandemic: General Lessons for Clinical Trial Design. Stat Biopharm Res 2023;16(2):158–170. doi: 10.1080/19466315.2023.2211538.

Starokozhko V, Tarrahi F, Vrijlandt PJWS, Mol PGM. Safety-Related Drug Label Changes Following Large Post-Marketing Cardiometabolic Trials: A Review of European Public Assessment Reports. Clin Pharmacol Ther. 2023;113(4):859-866. doi: 10.1002/cpt.2840.

Hedenmalm K, Quinten C, Kurz X, Bradley M, Lee H, Eworuke E. A collaborative study of the impact of N-nitrosamines presence and ARB recall on ARB utilization - results from IQVIA™ Disease Analyzer Germany. Eur J Clin Pharmacol. 2023;79(6):849-858. doi: 10.1007/s00228-022-03439-3.

Piccinin C, Basch E, Bhatnagar V, Calvert M, Campbell A, Cella D, et al. Recommendations on the use of item libraries for patient-reported outcome measurement in oncology trials: findings from an international, multidisciplinary working group. Lancet Oncol. 2023;24(2):e86-e95. doi: 10.1016/S1470-2045(22)00654-4.

Aiyegbusi OL, Cruz Rivera S, Oliver K, Manna E, Collis P, King-Kallimanis BL, et al. The opportunity for greater patient and public involvement and engagement in drug development and regulation. Nat Rev Drug Discov. 2023;22(5):337-338. doi: 10.1038/d41573-023-00031-x.

Dimitriou NG, Meuth SG, Martinez-Lapiscina EH, Albrecht P, Menge T. Treatment of Patients with Multiple Sclerosis Transitioning Between Relapsing and Progressive Disease. CNS Drugs. 2023;37(1):69-92. doi: 10.1007/s40263-022-00977-3.

Roldan Munoz S, Postmus D, de Vries ST, Gross-Martirosyan L, Bahri P, Hillege H, et al. What Factors Make EU Regulators Want to Communicate Drug Safety Issues Related to SGLT2 Inhibitors? An Online Survey Study. Drug Saf. 2023;46(3):243-255. doi: 10.1007/s40264-022-01270-4.

Zarkavelis G, Amylidi AL, Verbaanderd C, Cherny NI, Metaxas Y, de Vries EGE, et al. Off-label despite high-level evidence: a clinical practice review of commonly used off-patent cancer medicines. ESMO Open. 2023;8(1):100604. doi: 10.1016/j.esmoop.2022.100604.

Hines PA, Herold R, Pinheiro L, Frias Z, Arlett P. Artificial intelligence in European medicines regulation. Nat Rev Drug Discov. 2023;22(2):81-82. doi: 10.1038/d41573-022-00190-3.

Cherchi A, Vaz A, Coelho A, Fregonese L, Thirstrup S. Tuberculosis medicines for children in Europe: an unmet medical need. ERJ Open Res. 2023;9(4):00730-2022. doi: 10.1183/23120541.00730-2022.

van Duijkeren E, Rantala M, Bouchard D, Busani L, Catry B, Kaspar H, et al. The use of aminopenicillins in animals within the EU, emergence of resistance in bacteria of animal and human origin and its possible impact on animal and human health. J Antimicrob Chemother. 2023;78(8):1827-1842. doi: 10.1093/jac/dkad157.

Guillen E, Ekman N, Barry S, Weise M, Wolff-Holz E. A Data Driven Approach to Support Tailored Clinical Programs for Biosimilar Monoclonal Antibodies. Clin Pharmacol Ther. 2023;113(1):108-123. doi: 10.1002/cpt.2785.

Durand J, Dogné JM, Cohet C, Browne K, Gordillo-Marañón M, Piccolo L, et al. Safety Monitoring of COVID-19 Vaccines: Perspective from the European Medicines Agency. Clin Pharmacol Ther. 2023;113(6):1223-1234. doi: 10.1002/cpt.2828.

Travis J, Rothmann M, Thomson A. Perspectives on informative Bayesian methods in pediatrics. J Biopharm Stat. 2023;33(6):830-843. doi: 10.1080/10543406.2023.2170405.

Russek M, Quinten C, de Jong VMT, Cohet C, Kurz X. Assessing heterogeneity of electronic health-care databases: A case study of background incidence rates of venous thromboembolism. Pharmacoepidemiol Drug Saf. 2023;32(9):1032-1048. doi: 10.1002/pds.5631.

Horne S, Vera MD, Nagavelli LR, Sayeed VA, Heckman L, Johnson D, et al. Regulatory Experiences with Root Causes and Risk Factors for Nitrosamine Impurities in Pharmaceuticals. J Pharm Sci. 2023;112(5):1166-1182. doi: 10.1016/j.xphs.2022.12.022.

Thor S, Vetter T, Marcal A, Kweder S. EMA-FDA Parallel Scientific Advice: Optimizing Development of Medicines in the Global Age. Ther Innov Regul Sci. 2023;57(4):656-661. doi: 10.1007/s43441-023-00501-9.

Andersson NW, Thiesson EM, Baum U, Pihlström N, Starrfelt J, Faksová K, et al. Comparative effectiveness of heterologous third dose vaccine schedules against severe covid-19 during omicron predominance in Nordic countries: population based cohort analyses. BMJ. 202324;382:e074325. doi: 10.1136/bmj-2022-074325.

Vass P, Akdag DS, Broholm GE, Kjaer J, Humphreys AJ, Ehmann F. Enabling technologies driving drug research and development. Front Med (Lausanne). 2023;10:1122405. doi: 10.3389/fmed.2023.1122405.

Roldan Munoz S, de Vries ST, Lankester G, Pignatti F, van Munster BC, Radford I, et al. Preferences about Future Alzheimer's Disease Treatments Elicited through an Online Survey Using the Threshold Technique. J Prev Alzheimers Dis. 2023;10(4):756-764. doi: 10.14283/jpad.2023.84.

Khurana M, Egger GF, Yao L, Thompson A, Pallidis C, Goldstein SL, et al. Overcoming Barriers to Drug Development in Children with CKD. Clin J Am Soc Nephrol. 2023;18(8):1101-1107. doi: 10.2215/CJN.0000000000000167.

Saesen R, Machado M, Crifo B, Liu L, de Vries C, Herold R, et al. Involvement of the European Medicines Agency in multi-stakeholder regulatory science research projects: experiences of staff members and project coordinators. Front Med (Lausanne). 2023;10:1181702. doi: 10.3389/fmed.2023.1181702.

Polverejan E, O'Kelly M, Hefting N, Norton JD, Lim P, Walton MK. Defining Clinical Trial Estimands: A Practical Guide for Study Teams with Examples Based on a Psychiatric Disorder. Ther Innov Regul Sci. 2023;57(5):911-939. doi: 10.1007/s43441-023-00524-2.

Manolis E, García-Arieta A, Lindahl A, Kotzagiorgis E, Limberg J, Holte Ø, et al. Using mechanistic models to support development of complex generic drug products: European Medicines Agency perspective. CPT Pharmacometrics Syst Pharmacol. 2023;12(5):556-559. doi: 10.1002/psp4.12906.

Miele G, Cepparulo S, Abbadessa G, Lavorgna L, Sparaco M, Simeon V, et al. Clinically Manifest Infections Do Not Increase the Relapse Risk in People with Multiple Sclerosis Treated with Disease-Modifying Therapies: A Prospective Study. J Clin Med. 2023;12(3):1023. doi: 10.3390/jcm12031023.

Szymański P, Weidinger F, Lordereau-Richard I, Himmelmann A, Arca M, Chaves J, et al. Real world evidence: Perspectives from a European Society of Cardiology Cardiovascular Round Table with contribution from the European Medicines Agency. Eur Heart J Qual Care Clin Outcomes. 2023;9(2):109-118. doi: 10.1093/ehjqcco/qcad009.

Younus MM, Alkhakany M, Bahri P, Caro A, Rostom H, Ndagije HB, et al. The ISoP PatEG-SIG for Promoting Patient Engagement in Pharmacovigilance: A Change of Paradigm is Needed. Drug Saf. 2023;46(7):619-623. doi: 10.1007/s40264-023-01313-4.

Verbaanderd C, Trullás Jimeno A, Engelbergs J, Zander H, Reischl I, Moreno Oliver A, et al. Biomarker-Driven Developments in the Context of the New Regulatory Framework for Companion Diagnostics in the European Union. Clin Pharmacol Ther. 2023;114(2):316-324. doi: 10.1002/cpt.2928.

Butlen-Ducuing F, McCulloch DE, Haberkamp M, Mattila T, Bałkowiec-Iskra E, Aislaitner G, et al. The therapeutic potential of psychedelics: the European regulatory perspective. Lancet. 2023;401(10378):714-716. doi: 10.1016/S0140-6736(23)00264-7.

Pearson ADJ, Federico S, Gatz SA, Ortiz M, Lesa G, Scobie N, et al. Paediatric Strategy Forum for medicinal product development of DNA damage response pathway inhibitors in children and adolescents with cancer: ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration. Eur J Cancer. 2023;190:112950. doi: 10.1016/j.ejca.2023.112950.

de Wildt SN, Foeldvari I, Siapkara A, Lepola P, Kriström B, Ruggieri L, et al. Off-label is not always off-evidence: authorising paediatric indications for old medicines. Lancet Child Adolesc Health. 2023;7(6):371-372. doi: 10.1016/S2352-4642(23)00083-4.

Pesiou S, Barcelo R, Fradera M, Torres F, Pontes C. Utilisation of drugs for the treatment of psychiatric diseases in the pediatric population: focus on off-label use. Front Pharmacol. 2023;14:1157135. doi: 10.3389/fphar.2023.1157135.

Van Haesendonck L, Ruof J, Desmet T, Van Dyck W, Simoens S, Huys I, et al. The role of stakeholder involvement in the evolving EU HTA process: Insights generated through the European Access Academy's multi-stakeholder pre-convention questionnaire. J Mark Access Health Policy. 2023;11(1):2217543. doi: 10.1080/20016689.2023.2217543.

Asturias EJ, Excler JL, Ackland J, Cavaleri M, Fulurija A, Long R, et al. Safety of Streptococcus pyogenes Vaccines: Anticipating and Overcoming Challenges for Clinical Trials and Post-Marketing Monitoring. Clin Infect Dis. 2023;77(6):917-924. doi: 10.1093/cid/ciad311.

Salmikangas P, Carlsson B, Klumb C, Reimer T, Thirstrup S. Potency testing of cell and gene therapy products. Front Med (Lausanne). 2023;10:1190016. doi: 10.3389/fmed.2023.1190016.

Marrie RA, Chataway J, Bierer BE, Finlayson M, Martinez-Lapiscina EH, Panagoulias J, et al. Enhancing diversity of clinical trial populations in multiple sclerosis. Mult Scler. 2023;29(9):1174-1185. doi: 10.1177/13524585231189677.

Woods SW, Parker S, Kerr MJ, Walsh BC, Wijtenburg SA, Prunier N, et al; Accelerating Medicines Partnership Schizophrenia. Development of the PSYCHS: Positive SYmptoms and Diagnostic Criteria for the CAARMS Harmonized with the SIPS. Early Interv Psychiatry. 2023:1-18. doi: 10.1111/eip.13457.

van der Laan JW, Andersson M, Beken S, Bonelli M, Brendler-Schwaab S, Kane R, et al. EMA commentary on the ICH guideline for testing for carcinogenicity of pharmaceuticals. Br J Clin Pharmacol. 2023;89(8):2341-2348. doi: 10.1111/bcp.15790.

Palomo GM, Pose-Boirazian T, Naumann-Winter F, Costa E, Duarte DM, Kalland ME, et al. The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products. Mol Ther. 2023;31(12):3414-3423. doi: 10.1016/j.ymthe.2023.09.020.

Sridhara R, Marchenko O, Jiang Q, Barksdale E, Alonzo TA, Amatya A, et al. Statistical Considerations in Pediatric Cancer Trials: Report of American Statistical Association Biopharmaceutical Section Open Forum Discussions, Stat Biopharm Res 2023;15:845-851. doi: 10.1080/19466315.2023.2238650.

Schönborn L, Seck SE, Thiele T, Kaderali L, Hoffmann T, Hlinka A, et al. Long-term outcome in vaccine-induced immune thrombocytopenia and thrombosis. J Thromb Haemost. 2023;21(9):2519-2527. doi: 10.1016/j.jtha.2023.06.027.

Cioc RC, Joyce C, Mayr M, Bream RN. Formation of N-Nitrosamine Drug Substance Related Impurities in Medicines: A Regulatory Perspective on Risk Factors and Mitigation Strategies. Org Process Res Dev. 2023;10:1736–1750. doi: 10.1021/acs.oprd.3c00153.

Tesileanu CMS, Michaleas S, Gonzalo Ruiz R, Mariz S, Fabriek BO, van Hennik PB, et al. 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 Two Tyrosine Kinase Inhibitors. Oncologist. 2023;28(7):628-632. doi: 10.1093/oncolo/oyad119.

Tesileanu CMS, Pignatti F, Tognana E, Humphreys A. Queries Raised During Oncology Business Pipeline Meetings at the European Medicines Agency: A 5-Year Retrospective Analysis. Clin Pharmacol Ther. 2023;114(5):1043-1049. doi: 10.1002/cpt.3015.

DeVries JH, Irs A, Hillege HL. The European Medicines Agency assessment of mavacamten as treatment of symptomatic obstructive hypertrophic cardiomyopathy in adult patients. Eur Heart J. 2023;44(37):3492-3494. doi: 10.1093/eurheartj/ehad429.

Gonzalez-Quevedo R, Pioppo L, Garcia Burgos J, Arlett P. European Medicines Agency seeks stakeholders' views on future transparency measures for clinical trials. Br J Clin Pharmacol. 2023;89(8):2338-2340. doi: 10.1111/bcp.15825.

Engeroff K, Warm D, Bittner S, Blanquie O. Different activity patterns control various stages of Reelin synthesis in the developing neocortex. Cereb Cortex. 2023;33(15):9376-9386. doi: 10.1093/cercor/bhad210.

Castro I, Van Tricht M, Bonaccorso N, Sciortino M, Garcia Burgos J, Costantino C, et al. Stakeholders' Understanding of European Medicine Agency's COVID-19 Vaccine Information Materials in EU and Regional Contexts. Vaccines (Basel). 2023;11(10):1616. doi: 10.3390/vaccines11101616.

Nicorescu V, Cyrus B. EMA activities related to antiparasitic veterinary medicinal products. Regulatory Rapporteur 2023;20(9).

Denkinger M, Knol W, Cherubini A, Simonds A, Lionis C, Lacombe D, et al. Inclusion of functional measures and frailty in the development and evaluation of medicines for older adults. Lancet Healthy Longev. 2023;4(12):e724-e729. doi: 10.1016/S2666-7568(23)00208-8.

Butlen-Ducuing F, Haberkamp M, Aislaitner G, Bałkowiec-Iskra E, Mattila T, Doucet M, et al. The new European Medicines Agency guideline on antidepressants: a guide for researchers and drug developers. Eur Psychiatry. 2023;67(1):e2. doi: 10.1192/j.eurpsy.2023.2479.

Yasinskaya Y, Bala S, Waack U, Dixon C, Higgins K, Moore JN, et al. Food and Drug Administration Public Workshop Summary-Development Considerations of Antifungal Drugs to Address Unmet Medical Need. Clin Infect Dis. 2023;77(3):380-387. doi: 10.1093/cid/ciad195.

Wilson BE, Sullivan R, Peto R, Abubakar B, Booth C, Werutsky G, et al. Global Cancer Drug Development-A Report From the 2022 Accelerating Anticancer Agent Development and Validation Meeting. JCO Glob Oncol. 2023;9:e2300294. doi: 10.1200/GO.23.00294.

Tervonen T, Veldwijk J, Payne K, Ng X, Levitan B, Lackey LG, et al. Quantitative Benefit-Risk Assessment in Medical Product Decision Making: A Good Practices Report of an ISPOR Task Force. Value Health. 2023;26(4):449-460. doi: 10.1016/j.jval.2022.12.006.

Stolz D, Matera MG, Rogliani P, van den Berge M, Papakonstantinou E, Gosens R, et al. Current and future developments in the pharmacology of asthma and COPD: ERS seminar, Naples 2022. Breathe (Sheff). 2023;19(2):220267. doi: 10.1183/20734735.0267-2022.

Smith RP, Lawes J, Davies RH, Hutchison ML, Vidal A, Gilson D, et al. UK-wide risk factor study of broiler carcases highly contaminated with Campylobacter. Zoonoses Public Health. 2023;70(6):523-541. doi: 10.1111/zph.13063.

Pe M, Alanya A, Falk RS, Amdal CD, Bjordal K, Chang J, et al. Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints in Cancer Clinical Trials-Innovative Medicines Initiative (SISAQOL-IMI): stakeholder views, objectives, and procedures. Lancet Oncol. 2023;24(6):e270-e283. doi: 10.1016/S1470-2045(23)00157-2.

Muñoz J, Hufstedler H, Gustafson P, Bärnighausen T, De Jong VMT, Debray TPA. Dealing with missing data using the Heckman selection model: methods primer for epidemiologists. Int J Epidemiol. 2023;52(1):5-13. doi: 10.1093/ije/dyac237.

Muñoz J, Efthimiou O, Audigier V, de Jong VMT, Debray TPA. Multiple imputation of incomplete multilevel data using Heckman selection models. Stat Med. 2024;43(3):514-533. doi: 10.1002/sim.9965.

Morales DR, Arlett P. RCTs and real world evidence are complementary, not alternatives. BMJ. 2023;381:736. doi: 10.1136/bmj.p736.

Marshall S, Ahamadi M, Chien J, Iwata D, Farkas P, Filipe A, et al. Model-Informed Drug Development: Steps Toward Harmonized Guidance. Clin Pharmacol Ther. 2023;114(5):954-959. doi: 10.1002/cpt.3006.

Häckl S, Koch A, Lasch F. Type-I-error rate inflation in mixed models for repeated measures caused by ambiguous or incomplete model specifications. Pharm Stat. 2023;22(6):1046-1061. doi: 10.1002/pst.2328.

Grupstra RJ, Goedecke T, Scheffers J, Strassmann V, Gardarsdottir H. Review of Studies Evaluating Effectiveness of Risk Minimization Measures Assessed by the European Medicines Agency Between 2016 and 2021. Clin Pharmacol Ther. 2023;114(6):1285-1292. doi: 10.1002/cpt.3034.

Giersing BK, Isbrucker R, Kaslow DC, Cavaleri M, Baylor N, Maiga D, et al. Clinical and regulatory development strategies for Shigella vaccines intended for children younger than 5 years in low-income and middle-income countries. Lancet Glob Health. 2023;11(11):e1819-e1826. doi: 10.1016/S2214-109X(23)00421-7.

De Meester D, Goossens M, Marco E, Claessens M, Gautier J, Annweiler C, et al. Evaluation of the Geriatric Nutritional Risk Index in predicting mortality in older patients with COVID-19 in the AgeBru cohort. Clin Nutr ESPEN. 2023;57:65-72. doi: 10.1016/j.clnesp.2023.06.025.

de Jong VMT, Hoogland J, Moons KGM, Riley RD, Nguyen TL, Debray TPA. Propensity-based standardization to enhance the validation and interpretation of prediction model discrimination for a target population. Stat Med. 2023;42(19):3508-3528. doi: 10.1002/sim.9817.

de Jong VMT, Campbell H, Maxwell L, Jaenisch T, Gustafson P, Debray TPA. Adjusting for misclassification of an exposure in an individual participant data meta-analysis. Res Synth Methods. 2023;14(2):193-210. doi: 10.1002/jrsm.1606.

Clay I, Peerenboom N, Connors DE, Bourke S, Keogh A, Wac K, et al. Reverse Engineering of Digital Measures: Inviting Patients to the Conversation. Digit Biomark. 2023;7(1):28-44. doi: 10.1159/000530413.

Busner J, Pandina G, Day S, Mahableshwarkar A, Kempf L, Sheean M, et al. Patient Centricity: Design and Conduct of Clinical Trials in Orphan Diseases: Third of Three Sets of Expanded Proceedings from the 2020 ISCTM Autumn Conference on Pediatric Drug Development. Innov Clin Neurosci. 2023;20(1-3):25-31.

Ayalew K, Ning YM, Foringer MJ, Leibenhaut S, Sellers JW, Yu B, et al. Comparison of Good Clinical Practice Inspection Processes for Marketing Applications Between the United States Food and Drug Administration and the European Medicines Agency. Ther Innov Regul Sci. 2023;57(1):79-85. doi: 10.1007/s43441-022-00441-w.

Hanaizi Z, Kweder S, Thor S, Ribeiro S, Marcal A. Considering Global Development? Insights from Applications for FDA Breakthrough Therapy and EMA PRIME Designations. Ther Innov Regul Sci. 2023;57(2):321-328. doi: 10.1007/s43441-022-00475-0.

Sridhara R, Barksdale E, Marchenko O, Jiang Q, Ando Y, Bloomquist E, et al. Cancer Clinical Trials beyond Pandemic: Report of an American Statistical Association Biopharmaceutical Section Open Forum Discussion. Stat Biopharm Res. 2023;15(2):444-449. doi: 10.1080/19466315.2022.2103181.

Bakker E, Plueschke K, Jonker CJ, Kurz X, Starokozhko V, Mol PGM. Contribution of Real-World Evidence in European Medicines Agency's Regulatory Decision Making. Clin Pharmacol Ther. 2023;113(1):135-151. doi: 10.1002/cpt.2766.

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. Br J Clin Pharmacol. 2023;89(1):5-10. doi: 10.1111/bcp.15567.

Cerreta F, Vučić K, Laslop A. Assessing Medicines for Use in the Geriatric Population. Clin Pharmacol Ther. 2023;113(3):536-540. doi: 10.1002/cpt.2793.

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;18(4):331-336. doi: 10.1097/PTS.0000000000000919.

Mitroiu M, Teerenstra S, Oude Rengerink K, Pétavy F, Roes KCB. Estimation of treatment effects in short-term depression studies. An evaluation based on the ICH E9(R1) estimands framework. Pharm Stat. 2022;21(5):1037-1057. doi: 10.1002/pst.2214.

Jansen E, Hines PA, Berntgen M, Brand A. Strengthening the Interface of Evidence-Based Decision Making Across European Regulators and Health Technology Assessment Bodies. Value Health. 2022;25(10):1726-1735. doi: 10.1016/j.jval.2022.01.026.

Sforzini L, Worrell C, Kose M, Anderson IM, Aouizerate B, Arolt V, et al. A Delphi-method-based consensus guideline for definition of treatment-resistant depression for clinical trials. Mol Psychiatry. 2022;27(3):1286-1299. doi: 10.1038/s41380-021-01381-x.

Ramanan AV, Modi N, de Wildt SN; c4c Learning from COVID-19 Group. Improving clinical paediatric research and learning from COVID-19: recommendations by the Conect4Children expert advice group. Pediatr Res. 2022;91(5):1069-1077. doi: 10.1038/s41390-021-01587-3.

Facile R, Muhlbradt EE, Gong M, Li Q, Popat V, Pétavy F, et al. Use of Clinical Data Interchange Standards Consortium (CDISC) Standards for Real-world Data: Expert Perspectives From a Qualitative Delphi Survey. JMIR Med Inform. 2022;10(1):e30363. doi: 10.2196/30363.

Asker-Hagelberg C, Boran T, Bouygues C, Eskola SM, Helmle L, Hernández C, et al. Repurposing of Medicines in the EU: Launch of a Pilot Framework. Front Med (Lausanne). 2022;8:817663. doi: 10.3389/fmed.2021.817663.

Abend AM, Zhang L, Fredro-Kumbaradzi E, Hoffelder T, Cohen MJ, Anand O, et al. Current Approaches for Dissolution Similarity Assessment, Requirements, and Global Expectations. AAPS J. 2022;24(3):50. doi: 10.1208/s12248-022-00691-4.

Lasch F, Psarelli EE, Herold R, Mattsson A, Guizzaro L, Pétavy F, et al. The Impact of COVID-19 on the Initiation of Clinical Trials in Europe and the United States. Clin Pharmacol Ther. 2022;111(5):1093-1102. doi: 10.1002/cpt.2534.

Keire DA, Bream R, Wollein U, Schmaler-Ripcke J, Burchardt A, Conti M, et al. International Regulatory Collaboration on the Analysis of Nitrosamines in Metformin-Containing Medicines. AAPS J. 2022;24(3):56. doi: 10.1208/s12248-022-00702-4.

Klimek L, Agache I, Cooke E, Jutel M, Akdis CA, O'Hehir R. COVID-19 vaccines-The way forward. Allergy. 2022;77(1):15-16. doi: 10.1111/all.14995.

Hines PA, Agricola E, Llinares Garcia J, O'Dwyer L, Herold R. Therapeutic genome editing: regulatory horizons. Nat Rev Drug Discov. 2022;21(1):1-2. doi: 10.1038/d41573-021-00130-7.

Lasch F, Guizzaro L. Estimators for handling COVID-19-related intercurrent events with a hypothetical strategy. Pharm Stat. 2022;21(6):1258-1280. doi: 10.1002/pst.2244.

Smith EA, Horan WP, Demolle D, Schueler P, Fu DJ, Anderson AE, et al. Using Artificial Intelligence-based Methods to Address the Placebo Response in Clinical Trials. Innov Clin Neurosci. 2022;19(1-3):60-70.

Bakker E, Hendrikse NM, Ehmann F, van der Meer DS, Llinares Garcia J, Vetter T, et al. Biomarker Qualification at the European Medicines Agency: A Review of Biomarker Qualification Procedures From 2008 to 2020. Clin Pharmacol Ther. 2022;112(1):69-80. doi: 10.1002/cpt.2554.

Murphy A, Bere N, Vamvakas S, Mavris M. The Added Value of Patient Engagement in Early Dialogue at EMA: Scientific Advice as a Case Study. Front Med (Lausanne). 2022 20;8:811855. doi: 10.3389/fmed.2021.811855.

van Riet-Nales DA, van den Bemt B, van Bodegom D, Cerreta F, Dooley B, Eggenschwyler D, et al. Commentary on the EMA reflection paper on the pharmaceutical development of medicines for use in the older population. Br J Clin Pharmacol. 2022;88(4):1500-1514. doi: 10.1111/bcp.15207.

Oakley BFM, Loth E, Jones EJH, Chatham CH, Murphy DG. Advances in the identification and validation of autism biomarkers. Nat Rev Drug Discov. 2022;21(10):697-698. doi: 10.1038/d41573-022-00141-y.

Vaz A, Roldão Santos M, Gwaza L, Mezquita González E, Pajewska Lewandowska M, Azatyan S, et al. WHO collaborative registration procedure using stringent regulatory authorities' medicine evaluation: reliance in action? Expert Rev Clin Pharmacol. 2022;15(1):11-17. doi: 10.1080/17512433.2022.2037419.

Thanarajasingam G, Minasian LM, Bhatnagar V, Cavalli F, De Claro RA, Dueck AC, et al. Reaching beyond maximum grade: progress and future directions for modernising the assessment and reporting of adverse events in haematological malignancies. Lancet Haematol. 2022;9(5):e374-e384. doi: 10.1016/S2352-3026(22)00045-X.

Sellers JW, Mihaescu CM, Ayalew K, Kronstein PD, Yu B, Ning YM, et al. Descriptive Analysis of Good Clinical Practice Inspection Findings from U.S. Food and Drug Administration and European Medicines Agency. Ther Innov Regul Sci. 2022;56(5):753-764. doi: 10.1007/s43441-022-00417-w.

Rippin G, Ballarini N, Sanz H, Largent J, Quinten C, Pignatti F. A Review of Causal Inference for External Comparator Arm Studies. Drug Saf. 2022;45(8):815-837. doi: 10.1007/s40264-022-01206-y.

De Wilde B, Barry E, Fox E, Karres D, Kieran M, Manlay J, et al. The Critical Role of Academic Clinical Trials in Pediatric Cancer Drug Approvals: Design, Conduct, and Fit for Purpose Data for Positive Regulatory Decisions. J Clin Oncol. 2022;40(29):3456. doi: 10.1200/JCO.22.00033.

Pearson ADJ, Weiner SL, Adamson PC, Karres D, Reaman G, Rousseau R, et al. ACCELERATE - Five years accelerating cancer drug development for children and adolescents. Eur J Cancer. 2022;166:145-164. doi: 10.1016/j.ejca.2022.01.033.

Shivji R, Conocchia R, Korakianiti E, Jekerle V. Considerations for the chemistry, manufacturing and Controls (CMC) - quality package for COVID-19 vaccines- interim lessons learnt by the European medicines Agency (EMA). Vaccine. 2022;40(38):5539-5541. doi: 10.1016/j.vaccine.2022.06.058.

Slattery J, Quinten C, Candore G, Pinheiro L, Flynn R, Kurz X, et al. Ondansetron use in nausea and vomiting during pregnancy: A descriptive analysis of prescription patterns and patient characteristics in UK general practice. Br J Clin Pharmacol. 2022;88(10):4526-4539. doi: 10.1111/bcp.15370.

de Vries ST, Starokozhko V, Schellens IMM, Wijnans L, Enzmann H, Cavaleri M, et al. Attention for sex in COVID-19 trials: a review of regulatory dossiers. BMJ Glob Health. 2022;7(3):e008173. doi: 10.1136/bmjgh-2021-008173.

Hendrikse NM, Llinares Garcia J, Vetter T, Humphreys AJ, Ehmann F. Biomarkers in Medicines Development-From Discovery to Regulatory Qualification and Beyond. Front Med (Lausanne). 2022;9:878942. doi: 10.3389/fmed.2022.878942.

Karres D, Lesa G, Ligas F, Benchetrit S, Galluzzo S, Van Malderen K, et al. European regulatory strategy for supporting childhood cancer therapy developments. Eur J Cancer. 2022;177:25-29. doi: 10.1016/j.ejca.2022.09.025.

Plueschke K, Jonker C, Strassmann V, Kurz X. Collection of Data on Adverse Events Related to Medicinal Products: A Survey Among Registries in the ENCePP Resources Database. Drug Saf. 2022;45(7):747-754. doi: 10.1007/s40264-022-01188-x.

Renaud F, Mofenson LM, Bakker C, Dolk H, Leroy V, Namiba A, et al. Surveillance of ARV safety in pregnancy and breastfeeding: towards a new framework. J Int AIDS Soc. 2022;25(S2):e25922. doi: 10.1002/jia2.25922.

Michaleas S, Moreno Oliver A, Mueller-Berghaus J, Sarac SB, van der Elst ME, Müller-Egert S, et al. The European Medicines Agency review of sacituzumab govitecan for the treatment of triple-negative breast cancer. ESMO Open. 2022;7(3):100497. doi: 10.1016/j.esmoop.2022.100497.

Plueschke K, Flynn R, Hedenmalm K, Deli AC, Maciá-Martinez MA, García-Poza P, et al. Prescribing Patterns of Codeine and Alternative Medicines in Children in Europe. Drug Saf. 2022;45(10):1069-1081. doi: 10.1007/s40264-022-01214-y.

Rault O, Romeo B, Butlen-Ducuing F, Rari E, Benyamina A, Martelli C. Impact of cannabis use and its cessation on the dosage and the efficacy of antipsychotic drugs in in- and outpatients with schizophrenia taking medication: A systematic review and meta-analysis. J Psychiatr Res. 2022;156:713-721. doi: 10.1016/j.jpsychires.2022.11.012.

Pearson AD, Gaspar N, Janeway K, Campbell-Hewson Q, Lawlor ER, Copland C, et al. 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. Eur J Cancer. 2022;173:71-90. doi: 10.1016/j.ejca.2022.06.008.

Saesen R, Espinasse C, Pignatti F, Lacombe D. Advancing academia-driven treatment optimisation in oncology: Launch of the EMA Cancer Medicines Forum. Eur J Cancer. 2022;168:77-79. doi: 10.1016/j.ejca.2022.03.025.

Provansal C, Dooley D, Ziogas C. Pharmaceutical innovation sourcing. Nat Rev Drug Discov. 2022;21(9):627. doi: 10.1038/d41573-022-00125-y.

Paixão P, Silva N, Guerreiro RB, Blake K, Bonelli M, Morais JAG, et al. Evaluation of a Proposed Approach for the Determination of the Bioequivalence Acceptance Range for Narrow Therapeutic Index Drugs in the European Union. Pharmaceutics. 2022 Oct 31;14(11):2349. doi: 10.3390/pharmaceutics14112349.

Xie J, Prats-Uribe A, Gordillo-Marañón M, Strauss VY, Gill D, Prieto-Alhambra D. Genetic risk and incident venous thromboembolism in middle-aged and older adults following COVID-19 vaccination. J Thromb Haemost. 2022;20(12):2887-2895. doi: 10.1111/jth.15879.

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. 2022;13:924648. doi: 10.3389/fphar.2022.924648.

Li X, Burn E, Duarte-Salles T, Yin C, Reich C, Delmestri A, et al. Comparative risk of thrombosis with thrombocytopenia syndrome or thromboembolic events associated with different covid-19 vaccines: international network cohort study from five European countries and the US. BMJ. 2022;379:e071594. doi: 10.1136/bmj-2022-071594.

Palmi Reig V, Durvy B, Harvey Allchurch M. EU Mobilizes Cooperation with the African Medicines Agency. DIA Global Forum 2022;14 (7)

Mintzes B, Reynolds E, Bahri P, Perry LT, Bhasale AL, Morrow RL, et al. How do safety warnings on medicines affect prescribing? Expert Opin Drug Saf. 2022 Oct;21(10):1269-1273. doi: 10.1080/14740338.2022.2134342.

Pearson ADJ, de Rojas T, Karres D, Reaman G, Scobie N, Fox E, et al. ACCELERATE Paediatric Strategy Forums: an advance for oncological drug development? Lancet Oncol. 2022;23(11):1354-1357. doi: 10.1016/S1470-2045(22)00619-2.

Pearson AD, Allen C, Fangusaro J, Hutter C, Witt O, Weiner S, et al. Paediatric Strategy Forum for medicinal product development in mitogen-activated protein kinase pathway inhibitors: ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration. Eur J Cancer. 2022;177:120-142. doi: 10.1016/j.ejca.2022.09.036.

Bergman E, Sherwood K, Forslund M, Arlett P, Westman G. A natural language processing approach towards harmonisation of European medicinal product information. PLoS One. 2022;17(10):e0275386. doi: 10.1371/journal.pone.0275386.

Pinheiro LC, Kurz X. Artificial intelligence in pharmacovigilance: A regulatory perspective on explainability. Pharmacoepidemiol Drug Saf. 2022;31(12):1308-1310. doi: 10.1002/pds.5524.

Buoninfante A, Andeweg A, Baker AT, Borad M, Crawford N, Dogné JM, et al. Understanding thrombosis with thrombocytopenia syndrome after COVID-19 vaccination. NPJ Vaccines. 2022;7(1):141. doi: 10.1038/s41541-022-00569-8.

Vignali V, Hines PA, Cruz AG, Ziętek B, Herold R. Health horizons: Future trends and technologies from the European Medicines Agency's horizon scanning collaborations. Front Med (Lausanne). 2022;9:1064003. doi: 10.3389/fmed.2022.1064003.

Minichmayr IK, Ravenstijn P, van der Graaf PH, Vamvakas S. Therapeutic Innovations in Neuroscience: What's New on the Horizon? Clin Pharmacol Ther. 2022;111(4):715-717. doi: 10.1002/cpt.2547.

Bronzwaer S, Catchpole M, de Coen W, Dingwall Z, Fabbri K, Foltz C, et al. One Health collaboration with and among EU Agencies - Bridging research and policy. One Health. 2022;15:100464. doi: 10.1016/j.onehlt.2022.100464.

Epps C, Bax R, Croker A, Green D, Gropman A, Klein AV, et al. Global Regulatory and Public Health Initiatives to Advance Pediatric Drug Development for Rare Diseases. Ther Innov Regul Sci. 2022;56(6):964-975. doi: 10.1007/s43441-022-00409-w.

Fuggle N, Bere N, Bruyère O, Rosa MM, Prieto Yerro MC, Dennison E, et al. Management of hand osteoarthritis: from an US evidence-based medicine guideline to a European patient-centric approach. Aging Clin Exp Res. 2022;34(9):1985-1995. doi: 10.1007/s40520-022-02176-y.

Saint-Raymond A, Valentin M, Nakashima N, Orphanos N, Santos G, Balkamos G, et al. Reliance is key to effective access and oversight of medical products in case of public health emergencies. Expert Rev Clin Pharmacol. 2022;15(7):805-810. doi: 10.1080/17512433.2022.2088503.

Wang SV, Pottegård A, Crown W, Arlett P, Ashcroft DM, Benchimol EI, et al. HARmonized Protocol Template to Enhance Reproducibility of Hypothesis Evaluating Real-World Evidence Studies on Treatment Effects: A Good Practices Report of a Joint ISPE/ISPOR Task Force. Value Health. 2022;25(10):1663-1672. doi: 10.1016/j.jval.2022.09.001.

Szepessy E, Arlett P, Sweeney F, Broich K, Rys AJ. COVID-19 kick-starts a new era for clinical trials and pandemic preparedness in Europe. Lancet Infect Dis. 2022;22(3):315-316. doi: 10.1016/S1473-3099(22)00061-5.

Cooke E. Preparing Europe for future health threats and crises ─ the European Medicines Agency; ensuring safe and effective medicines and medical devices. Euro Surveill. 2022;27(42):2200798. doi: 10.2807/1560-7917.ES.2022.27.42.2200798.

Kochhar S, Barreira D, Beattie P, Cavaleri M, Cravioto A, Frick MW, et al. Building the concept for WHO Evidence Considerations for Vaccine Policy (ECVP): Tuberculosis vaccines intended for adults and adolescents as a test case. Vaccine. 2022;40(12):1681-1690. doi: 10.1016/j.vaccine.2021.10.062.

McAllister M, Flanagan T, Cole S, Abend A, Kotzagiorgis E, Limberg J, et al. Developing Clinically Relevant Dissolution Specifications (CRDSs) for Oral Drug Products: Virtual Webinar Series. Pharmaceutics. 2022;14(5):1010. doi: 10.3390/pharmaceutics14051010.

de Jong VMT, Rousset RZ, Antonio-Villa NE, Buenen AG, Van Calster B, Bello-Chavolla OY, et al. Clinical prediction models for mortality in patients with covid-19: external validation and individual participant data meta-analysis. BMJ. 2022;378:e069881. doi: 10.1136/bmj-2021-069881.

Selker HP, Cohen T, D'Agostino RB, Dere WH, Ghaemi SN, Honig PK, et al. A Useful and Sustainable Role for N-of-1 Trials in the Healthcare Ecosystem. Clin Pharmacol Ther. 2022;112(2):224-232. doi: 10.1002/cpt.2425.

Candore G, Monzon S, Slattery J, Piccolo L, Postigo R, Xurz X, et al. The Impact of Mandatory Reporting of Non-Serious Safety Reports to EudraVigilance on the Detection of Adverse Reactions. Drug Saf. 2022;45(1):83-95. doi: 10.1007/s40264-021-01137-0.

Paixão P, Guerreiro RB, Silva N, Blake K, Bonelli M, Morais JAG, et al. A Proposed Approach for the Determination of the Bioequivalence Acceptance Range for Narrow Therapeutic Index Drugs in the European Union. Clin Pharmacol Ther. 2022;111(2):470-476. doi: 10.1002/cpt.2451.

Pignatti F, Wilking U, Postmus D, Wilking N, Delgado J, Bergh J. The value of anticancer drugs - a regulatory view. Nat Rev Clin Oncol. 2022;19(3):207-215. doi: 10.1038/s41571-021-00584-z.

Flynn R, Plueschke K, Quinten C, Strassmann V, Duijnhoven RG, Gordillo-Marañon M, et al. Marketing Authorization Applications Made to the European Medicines Agency in 2018-2019: What was the Contribution of Real-World Evidence? Clin Pharmacol Ther. 2022;111(1):90-97. doi: 10.1002/cpt.2461.

Pearson AD, Rossig C, Mackall C, Shah NN, Baruchel A, Reaman G, et al. 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. Eur J Cancer. 2022;160:112-133. doi: 10.1016/j.ejca.2021.10.016.

Arlett P, Kjaer J, Broich K, Cooke E. Real-World Evidence in EU Medicines Regulation: Enabling Use and Establishing Value. Clin Pharmacol Ther. 2022;111(1):21-23. doi: 10.1002/cpt.2479.

Liu Q, Schwartz JB, Slattum PW, Lau SWJ, Guinn D, Madabushi R, et al. Roadmap to 2030 for Drug Evaluation in Older Adults. Clin Pharmacol Ther. 2022;112(2):210-223. doi: 10.1002/cpt.2452.

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. Stat Biopharm Res. 2022;15(2):386-399. doi: 10.1080/19466315.2022.2055633.

2021

Januskiene J, Segec A, Slattery J, Genov G, Plueschke K, Kurz X, et al. What are the patients' and health care professionals' understanding and behaviors towards adverse drug reaction reporting and additional monitoring? Pharmacoepidemiol Drug Saf. 2021;30(3):334-341. doi: 10.1002/pds.5162.

Guizzaro L, Pétavy F, Ristl R, Gallo C. The Use of a Variable Representing Compliance Improves Accuracy of Estimation of the Effect of Treatment Allocation Regardless of Discontinuation in Trials with Incomplete Follow-up. Stat Biopharm Res. 2021;13(1):119-127. doi: 10.1080/19466315.2020.1736141.

Mitra A, Suarez-Sharp S, Pepin XJH, Flanagan T, Zhao Y, Kotzagiorgis E, et al. Applications of Physiologically Based Biopharmaceutics Modeling (PBBM) to Support Drug Product Quality: A Workshop Summary Report. J Pharm Sci. 2021;110(2):594-609. doi: 10.1016/j.xphs.2020.10.059.

Verhagen H, Alonso-Andicoberry C, Assunção R, Cavaliere F, Eneroth H, Hoekstra J, et al. Risk-benefit in food safety and nutrition - Outcome of the 2019 Parma Summer School. Food Res Int. 2021;141:110073. doi: 10.1016/j.foodres.2020.110073.

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. 2021;30(3):350-359. doi: 10.1002/pds.5174.

Bhasale AL, Sarpatwari A, De Bruin ML, Lexchin J, Lopert R, Bahri P, et al. 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. 2021;109(6):1424-1442. doi: 10.1002/cpt.2010.

Arlett P, Kurz X, Soltys K, Blum MD. International Collaboration in Real-World Evidence Generation for Direct Acting Oral Anti-Coagulants. Clin Pharmacol Ther. 2021;109(2):299-301. doi: 10.1002/cpt.1999.

Cocoros NM, Arlett P, Dreyer NA, Ishiguro C, Iyasu S, Sturkenboom M, et al. The Certainty Framework for Assessing Real-World Data in Studies of Medical Product Safety and Effectiveness. Clin Pharmacol Ther. 2021;109(5):1189-1196. doi: 10.1002/cpt.2045.

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. 2021;44(2):193-209. doi: 10.1007/s40264-020-01005-3.

Guizzaro L, Drosos S, Kihlbom U, Pignatti F. Ethical Aspects of Regulating Oncology Products. Recent Results Cancer Res. 2021;218:119-134. doi: 10.1007/978-3-030-63749-1_9.

Wang SV, Pinheiro S, Hua W, Arlett P, Uyama Y, Berlin JA, et al. STaRT-RWE: structured template for planning and reporting on the implementation of real world evidence studies. BMJ. 2021;372:m4856. doi: 10.1136/bmj.m4856.

Butler D, Vucic K, Straus S, Cupelli A, Micallef B, Serracino-Inglott A, et al. Regulatory experience of handling Risk Management Plans (RMPs) for medicinal products in the EU. Expert Opin Drug Saf. 2021;20(7):815-826. doi: 10.1080/14740338.2021.1909569.

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. 2021;20(2):83-84. doi: 10.1038/d41573-020-00168-z.

Soumyanarayanan U, Choong M, Leong J, Lumpkin MM, Rasi G, Skerritt JH, et al. The COVID-19 crisis as an opportunity to strengthen global regulatory coordination for sustained enhanced access to diagnostics and therapeutics. Clin Transl Sci. 2021;14(3):777-780. doi: 10.1111/cts.12954.

Whomsley R, Palmi Reig V, Hidalgo-Simon A. Environmental risk assessment of advanced therapies containing genetically modified organisms in the EU. Br J Clin Pharmacol. 2021;87(6):2450-2458. doi: 10.1111/bcp.14781.

Hedenmalm K, Pacurariu A, Slattery J, Kurz X, Candore G, Flynn R. Is There an Increased Risk of Hepatotoxicity with Metamizole? A Comparative Cohort Study in Incident Users. Drug Saf. 2021;44(9):973-985. doi: 10.1007/s40264-021-01087-7.

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. 2021;87(6):2459-2464. doi: 10.1111/bcp.14672.

Jaksa A, Wu J, Jónsson P, Eichler HG, Vititoe S, Gatto NM. Organized structure of real-world evidence best practices: moving from fragmented recommendations to comprehensive guidance. J Comp Eff Res. 2021;10(9):711-731. doi: 10.2217/cer-2020-0228.

Eichler HG, Pignatti F, Schwarzer-Daum B, Hidalgo-Simon A, Eichler I, Arlett P, et al. Randomized Controlled Trials Versus Real World Evidence: Neither Magic Nor Myth. Clin Pharmacol Ther. 2021;109(5):1212-1218. doi: 10.1002/cpt.2083.

Tzogani K, Penttilä K, Lähteenvuo J, Lapveteläinen T, Lopez Anglada L, Prieto C, et al. EMA Review of Belantamab Mafodotin (Blenrep) for the Treatment of Adult Patients with Relapsed/Refractory Multiple Myeloma. Oncologist. 2021;26(1):70-76. doi: 10.1002/onco.13592.

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. 2021;20(4):245-246. doi: 10.1038/d41573-020-00205-x.

Starokozhko V, Kallio M, Kumlin Howell Å, Mäkinen Salmi A, Andrew-Nielsen G, Goldammer M, et al. Strengthening regulatory science in academia: STARS, an EU initiative to bridge the translational gap. Drug Discov Today. 2021;26(2):283-288. doi: 10.1016/j.drudis.2020.10.017.

Doerr P, Valentin M, Nakashima N, Orphanos N, Santos G, Balkamos G, et al. Reliance: a smarter way of regulating medical products - The IPRP survey. Expert Rev Clin Pharmacol. 2021;14(2):173-177. doi: 10.1080/17512433.2021.1865798.

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

Servais L, Camino E, Clement A, McDonald CM, Lukawy J, Lowes LP, et al. First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases. Digit Biomark. 2021;5(2):183-190. doi: 10.1159/000517411.

Araujo-Fernandez I, Delgado J, Moscetti L, Sarac SB, Zander H, Mueller-Egert S, et al. The European Medicines Agency review of the initial application of atezolizumab and the role of PD-L1 expression as biomarker for checkpoint inhibitors. ESMO Open. 2021;6(1):100008. doi: 10.1016/j.esmoop.2020.100008.

Nooney J, Thor S, de Vries C, Clements J, Sahin L, Hua W, et al. Assuring Access to Safe Medicines in Pregnancy and Breastfeeding. Clin Pharmacol Ther. 2021;110(4):941-945. doi: 10.1002/cpt.2212.

Delgado J, Josephson F, Camarero J, Garcia-Ochoa B, Lopez-Anglada L, Prieto-Fernandez C, et al. EMA Review of Acalabrutinib for the Treatment of Adult Patients with Chronic Lymphocytic Leukemia. Oncologist. 2021;26(3):242-249. doi: 10.1002/onco.13685.

Bakker E, Mol PGM, Nabais J, Vetter T, Kretzler M, Nolan JJ, et al. Perspectives on a Way Forward to Implementation of Precision Medicine in Patients With Diabetic Kidney Disease; Results of a Stakeholder Consensus-Building Meeting. Front Pharmacol. 2021;12:662642. doi: 10.3389/fphar.2021.662642.

Trullas A, Delgado J, Koenig J, Fuerstenau U, Dedorath J, Hausmann S, et al. 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;6(1):100031. doi: 10.1016/j.esmoop.2020.100031.

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. 2021;83:6-7. doi: 10.1016/j.ejim.2020.11.019.

Lasch F, Guizzaro L, Aguirre Dávila L, Müller-Vahl K, Koch A. Potential impact of COVID-19 on ongoing clinical trials: a simulation study with the neurological Yale Global Tic Severity Scale based on the CANNA-TICS study. Pharm Stat. 2021;20(3):675-691. doi: 10.1002/pst.2100.

Eichler HG, Adams R, Andreassen E, Arlett P, van de Casteele M, Chapman SJ, et al. 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;37(1):e83. doi: 10.1017/S026646232100057X.

Delgado J, Pean E, Melchiorri D, Migali C, Josephson F, Enzmann H, et al. The European Medicines Agency review of entrectinib for the treatment of adult or paediatric patients with solid tumours who have a neurotrophic tyrosine receptor kinase gene fusions and adult patients with non-small-cell lung cancer harbouring ROS1 rearrangements. ESMO Open. 2021;6(2):100087. doi: 10.1016/j.esmoop.2021.100087.

de la Casa-Resino I, Empl MT, Villa S, Kolar B, Fabrega J, Lillicrap AD, et al. Environmental risk assessment of veterinary medicinal products intended for use in aquaculture in Europe: the need for developing a harmonised approach. Environ Sci Eur. 2021;33:84. doi: 10.1186/s12302-021-00509-8.

Siapkara A, Fracasso C, Egger GF, Rizzari C, Trasorras CS, Athanasiou D, et al. 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. Arch Dis Child. 2021;106(12):1149-1154. doi: 10.1136/archdischild-2020-321433.

Cavaleri M, Enzmann H, Straus S, Cooke E. The European Medicines Agency's EU conditional marketing authorisations for COVID-19 vaccines. Lancet. 2021;397(10272):355-357. doi: 10.1016/S0140-6736(21)00085-4.

Reginster JY, Beaudart C, Al-Daghri N, Avouac B, Bauer J, Bere N, et al. 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. 2021;33(1):3-17. doi: 10.1007/s40520-020-01663-4.

Tomeo F, Mariz S, Brunetta AL, Stoyanova-Beninska V, Penttila K, Magrelli A. Haemophilia, state of the art and new therapeutic opportunities, a regulatory perspective. Br J Clin Pharmacol. 2021;87(11):4183-4196. doi: 10.1111/bcp.14838.

Delgado J, Vleminckx C, Sarac S, Sosa A, Bergh J, Giuliani R, et al. The EMA review of trastuzumab emtansine (T-DM1) for the adjuvant treatment of adult patients with HER2-positive early breast cancer. ESMO Open. 2021;6(2):100074. doi: 10.1016/j.esmoop.2021.100074.

Manolis E, Musuamba FT, Karlsson KE. The European Medicines Agency Experience With Pediatric Dose Selection. J Clin Pharmacol. 2021;61 Suppl 1:S22-S27. doi: 10.1002/jcph.1863.

Hidalgo-Simon A, Fibbe WE. Advanced therapies are ready to take centre stage: Academia's involvement with regulation needs to raise its game. Br J Clin Pharmacol. 2021;87(6):2412-2413. doi: 10.1111/bcp.14863.

Delgado J, Zienowicz M, van Hennik PB, Moreau A, Gisselbrecht C, Enzmann H, et al. EMA Review of Isatuximab in Combination with Pomalidomide and Dexamethasone for the Treatment of Adult Patients with Relapsed and Refractory Multiple Myeloma. Oncologist. 2021;26(11):983-987. doi: 10.1002/onco.13892.

Trullas-Jimeno A, Delgado J, Garcia-Ochoa B, Wang I, Sancho-Lopez A, Payares-Herrera C, et al. The EMA assessment of avapritinib in the treatment of gastrointestinal stromal tumours harbouring the PDGFRA D842V mutation. ESMO Open. 2021;6(3):100159. doi: 10.1016/j.esmoop.2021.100159.

Smith MY, Bahri P, Gaudino JA, Moreira RS, Danyluk GM, Palevsky SL. The role of epidemiologists in communicating SARS-CoV-2 evidence: a call for adopting standards. Int J Epidemiol. 2021;50(5):1410-1415. doi: 10.1093/ije/dyab128.

Trullas A, Delgado J, Genazzani A, Mueller-Berghaus J, Migali C, Müller-Egert S, et al. 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;6(3):100145. doi: 10.1016/j.esmoop.2021.100145.

Correia Pinheiro L, Giezen TJ, Wolff-Holz E, Weise M, Laslop A, Hidalgo-Simon A. Identifiability of Biologicals: An Analysis Using EudraVigilance, the European Union's Database of Reports of Suspected Adverse Drug Reactions. Clin Pharmacol Ther. 2021;110(5):1311-1317. doi: 10.1002/cpt.2411.

Ruepp R, Frötschl R, Bream R, Filancia M, Girard T, Spinei A, et al. The EU Response to the Presence of Nitrosamine Impurities in Medicines. Front Med (Lausanne). 2021;8:782536. doi: 10.3389/fmed.2021.782536.

Mulder J, van Rossum T, Mariz S, Magrelli A, de Boer A, Pasmooij AMG, et al. Orphan Medicinal Products for the Treatment of Pancreatic Cancer: Lessons Learned From Two Decades of Orphan Designation. Front Oncol. 2021;11:809035. doi: 10.3389/fonc.2021.809035.

Del Seppia I, Schalansky J, Claassen I. From collection to connection – the EMA veterinary data strategy. Regulatory Rapporteur 2021;18(6):18-22.

Perrone F, Di Liello R, Gargiulo P, Arenare L, Guizzaro L, Chiodini P, et al. The opportunity of patient-journey studies for academic clinical research in oncology. BMJ Open. 2021;11(9):e052871. doi: 10.1136/bmjopen-2021-052871.

Delgado J, Voltz C, Stain M, Lapveteläinen T, Urach S, Lähteenvuo J, et al. The European Medicines Agency Review of Crizanlizumab for the Prevention of Recurrent Vaso-Occlusive Crises in Patients With Sickle Cell Disease. Hemasphere. 2021;5(7):e604. doi: 10.1097/HS9.0000000000000604.

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;44(11):1193-1208. doi: 10.1007/s40264-021-01111-w.

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;8:698534. doi: 10.3389/fmed.2021.698534.

Delgado J, Voltz C, Stain M, Balkowiec-Iskra E, Mueller B, Wernsperger J, et al. The European Medicines Agency Review of Luspatercept for the Treatment of Adult Patients With Transfusion-dependent Anemia Caused by Low-risk Myelodysplastic Syndromes With Ring Sideroblasts or Beta-thalassemia. Hemasphere. 2021;5(8):e616. doi: 10.1097/HS9.0000000000000616.

Butlen-Ducuing F, Balkowiec-Iskra E, Dalla C, Slattery DA, Ferretti MT, Kokras N, et al. Implications of sex-related differences in central nervous system disorders for drug research and development. Nat Rev Drug Discov. 2021;20(12):881-882. doi: 10.1038/d41573-021-00115-6.

Hidalgo-Simon A, Botgros R, Cochino E. Authorization of vaccines in the European Union. Mol Front J. 2021;5(01n02):58-65. doi: 10.1142/S2529732521400034.

Orellana García LP, Ehmann F, Hines PA, Ritzhaupt A, Brand A. Biomarker and Companion Diagnostics-A Review of Medicinal Products Approved by the European Medicines Agency. Front Med (Lausanne). 2021;8:753187. doi: 10.3389/fmed.2021.753187.

Cavaleri M, Sweeney F, Gonzalez-Quevedo R, Carr M. Shaping EU medicines regulation in the post COVID-19 era. Lancet Reg Health Eur. 2021;9:100192. doi: 10.1016/j.lanepe.2021.100192.

Abou-El-Enein M, Angelis A, Appelbaum FR, Andrews NC, Bates SE, Bierman AS, et al. Evidence generation and reproducibility in cell and gene therapy research: A call to action. Mol Ther Methods Clin Dev. 2021;22:11-14. doi: 10.1016/j.omtm.2021.06.012.

Pearson ADJ, Barry E, Mossé YP, Ligas F, Bird N, de Rojas T, et al. Second Paediatric Strategy Forum for anaplastic lymphoma kinase (ALK) inhibition in paediatric malignancies: ACCELERATE in collaboration with the European Medicines Agency with the participation of the Food and Drug Administration. Eur J Cancer. 2021;157:198-213. doi: 10.1016/j.ejca.2021.08.022.

Sheean ME, Naumann-Winter F, Capovilla G, Kalland ME, Malikova E, Mariz S, et al. Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit-The EU Regulator's Perspective. Front Med (Lausanne). 2021;8:744625. doi: 10.3389/fmed.2021.744625.

Karres D, Reaman G, Ligas F, Lesa G, McCune S, Malli S, et al. Common Commentary on Paediatric Oncology Drug Development Published: Another Step in Optimising Global Regulatory Coordination of Paediatric Development Plans. Ther Innov Regul Sci. 2021;55(6):1109-1110. doi: 10.1007/s43441-021-00339-z.

Delgado J, Papadouli I, Sarac SB, Moreau A, Hovgaard D, Gisselbrecht C, et al. The European Medicines Agency Review of Tafasitamab in Combination With Lenalidomide for the Treatment of Adult Patients With Relapsed/Refractory Diffuse Large B-cell Lymphoma. Hemasphere. 2021;5(12):e666. doi: 10.1097/HS9.0000000000000666.

Mulder J, Verjans R, Verbaanderd C, Pean E, Weemers J, Leufkens HGM, et al. Extension of Indication for Authorised Oncology Products in the European Union: A Joint Effort of Multiple Stakeholders. Front Med (Lausanne). 2021;8:790782. doi: 10.3389/fmed.2021.790782.

Moore KA, Ostrowsky JT, Kraigsley AM, Mehr AJ, Bresee JS, Friede MH, et al. A Research and Development (R&D) roadmap for influenza vaccines: Looking toward the future. Vaccine. 2021;39(45):6573-6584. doi: 10.1016/j.vaccine.2021.08.010.

Stephenson D, Badawy R, Mathur S, Tome M, Rochester L. Digital Progression Biomarkers as Novel Endpoints in Clinical Trials: A Multistakeholder Perspective. J Parkinsons Dis. 2021;11(s1):S103-S109. doi: 10.3233/JPD-202428.

Cavaller-Bellaubi M, Faulkner SD, Teixeira B, Boudes M, Molero E, Brooke N, et al. Sustaining Meaningful Patient Engagement Across the Lifecycle of Medicines: A Roadmap for Action. Ther Innov Regul Sci. 2021;55(5):936-953. doi: 10.1007/s43441-021-00282-z.

Musuamba FT, Skottheim Rusten I, Lesage R, Russo G, Bursi R, Emili L, et al. Scientific and regulatory evaluation of mechanistic in silico drug and disease models in drug development: Building model credibility. CPT Pharmacometrics Syst Pharmacol. 2021;10(8):804-825. doi: 10.1002/psp4.12669.

Vandeputte J, Saville M, Cavaleri M, Friede M, Hacker A, Mueller SO, et al. IABS/CEPI platform technology webinar: Is it possible to reduce the vaccine development time? Biologicals. 2021;71:55-60. doi: 10.1016/j.biologicals.2021.04.005.

Alteri E. Benznidazole in Chagas disease study: do the data justify progression to phase 3? Lancet Infect Dis. 2021 Aug;21(8):1066-1067. doi: 10.1016/S1473-3099(21)00320-0.

Tang BH, Guan Z, Allegaert K, Wu YE, Manolis E, Leroux S, et al. Drug Clearance in Neonates: A Combination of Population Pharmacokinetic Modelling and Machine Learning Approaches to Improve Individual Prediction. Clin Pharmacokinet. 2021;60(11):1435-1448. doi: 10.1007/s40262-021-01033-x.

2026

2025

2024

2023

2022

2021

Related information

Related documents

Share this page