The European Medicines Agency (EMA) is involved in and supports a number of research projects with academia, learned societies and research groups.
For more information on EMA's approach to contributing to research, see Support to research.
Many of the research projects that the Agency is involved in form part of the Innovative Medicines Initiative (IMI). This is Europe's largest public-private initiative, which aims to speed up the development of better and safer medicines for patients. IMI supports collaborative research projects and builds networks of industrial and academic experts in order to boost pharmaceutical innovation in Europe.
Table of contents
- Improving regulatory and methodological standards
- Bringing expertise together
- Helping to set the research agenda
- Improving medicines for children
- Investigating vaccines
- Addressing antimicrobial resistance
- Advising and observing projects
Strengthening the monitoring of benefits and risks
EMA coordinated the IMI project Pharmacoepidemiological Research on Outcomes of Therapeutics by a European Consortium (PROTECT) from September 2009 until June 2015. The project aimed to develop innovative methods to improve and strengthen the monitoring of the benefits and risks of medicines marketed in the European Union.
It concentrated on improving early and proactive signal detection, establishing a framework for pharmacoepidemiology studies, developing methods for continuous benefit-risk monitoring and enhancing data collection directly from patients.
EMA has produced a lay-language summary of PROTECT's key findings which are published in more detail in medical journals:
Further publications are expected to follow, including an overview of the project's impact on patient protection and life-cycle management of medicines.
EMA staff also contributed to:
- the development of the European Programme in Pharmacovigilance and Pharmacoepidemiology (EU2P), an online training and degree programme in pharmacoepidemiology and pharmacovigilance;
- the activities of the United States Department of Health's Agency for Healthcare Research and Quality;
The Agency is a key leader in the development and application of regulatory science, the areas of science that are used in the assessment of the quality, safety and efficacy of medicines throughout their life-span, as well as regulatory decision-making.
Regulatory science includes basic and applied biomedical sciences, including genetics, pharmacology, biostatistics, clinical-trial methodology and epidemiology, and social sciences such as decision sciences, risk assessment and communication sciences. It aims to contribute towards the development of standards and tools to be used in the regulation of medicines.
EMA is involved in the Council for International Organizations of Medical Sciences (CIOMS), an international, non-governmental, non-profit organisation established in 1949. CIOMS facilitates and promotes international activities in the field of biomedical sciences. The Agency takes part in:
- Working group IX: Practical considerations for development and application of a toolkit for medicinal-product risk management
- Working group X: Considerations for applying good meta-analysis practices to clinical safety data within the biopharmaceutical regulatory process
The concept of adaptive pathways foresees either an initial approval in a well-defined patient subgroup with a high medical need and subsequent widening of the indication to a larger patient population, or an early regulatory approval (e.g. conditional approval) which is prospectively planned, and where uncertainty is reduced through the collection of post-approval data on the medicine's use in patients. The EMA invited companies to participate in a pilot project on adaptive pathways, and published a framework to guide discussions on individual pilot studies. The pilot project built on earlier work with the Massachusetts Institute of Technology's Center for Biomedical Innovation (CBI) to investigate the feasibility of this approach.
The Agency is also leading Accelerated Development of Appropriate Patient Therapies: a Sustainable, Multi-stakeholder Approach from Research to Treatment-outcomes (ADAPT SMART), a project implemented under the IMI 2 programme. The project aims to establish a platform for coordinating activities related to 'Medicines Adaptive Pathways to Patients' and enable stakeholder dialogue in this field.
The Agency is working on a project to develop standard decision-making methodologies to make the assessment of the benefits and risks of medicines more consistent, more transparent and easier to audit. Until the end of 2011, this was carried out together with experts in decision theory from the London School of Economics and Political Science (LSE) and with the University of Groningen.
- Full details are available under benefit-risk methodology.
Addressing failure rates in medicine development
The Agency and the Netherlands Medicines Evaluation Board are working on a project to analyse the failure rate of innovative drugs in the current regulatory system, focusing on delivering evidence and credibility for regulatory reform and policy recommendations. The project forms part of the Escher project, which brings together experts from Utrecht University and partners from the pharmaceutical industry. This project aims to assess the extent to which the following aspects are associated with marketing-application failure:
- the design and different phases of the development plan;
- the clinical outcome;
- clinical relevance.
Improving risk communication on medicines
As part of its commitment to improving communication with its stakeholders on the benefits and risks of medicines, the Agency invited Dr Frederic Bouder, an independent expert from King's College London, to carry out a study looking into the expectations and attitudes of the Agency's stakeholders on its communication activities, which was finalised in 2011:
- Benefit / risk communication by the European Medicines Agency: a study of influential stakeholders' expectations and attitudes
Incorporating patient preferences into regulatory decision making
EMA collaborates with Myeloma UK and the University Medical Centre Groningen on a research project to understand the perspectives of myeloma patients on the benefits and risks of treatments and to evaluate how to capture individual preferences for consideration in regulatory decision making. Findings of the first stage of a survey of over 500 myeloma patients in the UK will be published later in 2016.
The research project follows a pilot study EMA conducted in 2015 on how the collection of individual patient preferences can inform regulatory reviews. EMA published the findings of the pilot project in a medical journal in 2016.
The Agency plays a key role in forming and managing networks, bringing together expertise and enabling knowledge sharing. It has launched and manages two major such networks:
- European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP), a network of excellence in collaboration with European academics to improve the independence, transparency and research standards in the field of post-authorisation research, particularly observational research. Its goal is to further strengthen the post-authorisation monitoring of medicines in Europe by facilitating the conduct of multi-centre, independent, post-authorisation studies focusing on safety and on the benefit-risk balance, using available expertise and research experience across the EU;
- European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA), a network of research networks, investigators and centres with recognised expertise in performing clinical studies in children. Enpr-EMA aims to foster high-quality ethical research on the quality, safety and efficacy of medicines to be used in children. It does this through networking and stakeholder collaboration with members from within and outside the European Union (EU).
The Agency also took part in Patient Registries Initiative (PARENT) Joint Action, which aimed to support EU Member States in developing comparable and coherent patient registries in fields of identified importance such as chronic diseases, rare diseases and medical technology. It intended to rationalise and harmonise the development and governance of patient registries, enabling analyses of secondary data for public-health and research purposes. The project, which was partly funded by the European Commission, ran between May 2012-2015.
The Agency played an important role in setting the research agenda under the European Commission's Seventh Framework programme (FP7) funding tool, which ran from 2007 until 2013. The programme provided funding from a number of health-related topics such as infectious diseases, personalised medicines and public heath. EMA provided recommendations on the types of research to be funded.
The Agency's Paediatric Committee (PDCO) also provided recommendations for FP7 funding of studies into off-patent medicines, with a view to the submission of an application for a paediatric-use marketing authorisation. Preparations are currently underway for the European Commission's current funding programme Horizon 2020. For more information, see Priority list of off-patent medicines.
The Agency is involved in two work packages of the Global Research in Paediatrics – Network of Excellence (GRIP) project, an FP7-funded project that aims to improve the health of children globally, by stimulating and facilitating the development and safe use of paediatric medicines:
- Work package 1: Joint paediatric clinical pharmacology international educational program, an educational module developing a masters-level academic course in paediatric clinical pharmacology;
- Work package 3: Research tools to facilitate interoperability in paediatric research, including the validation of biomarkers and outcome measures.
Representatives of the Agency lead or take part in a number of projects looking at the development, benefits and risks of vaccines. These include:
- European Network of Vaccine Research and Development (TRANSVAC);
- Global Vaccine Safety Initiative;
- Vaccine Adverse Event Surveillance and Communication (VAESCO);
- Accelerated development of vaccine benefit-risk collaboration in Europe (ADVANCE).
For more information on the ADVANCE project, see the ADVANCE website.
The Agency is concerned about the development of antimicrobial resistance, particularly resistance to antibiotics. It works in collaboration with its European Union (EU) and international partners in a number of initiatives aiming to limit the development of resistance. These include:
- Transatlantic Task Force on Antimicrobial Resistance (TATFAR);
- European Centre for Disease Prevention and Control (ECDC);
- European Food Safety Authority (EFSA);
- ReAct - Action on Antibiotic Resistance.
Since 2010, the Agency has also been leading a project collecting information on the sale of veterinary antimicrobials across the EU since 2010 called the European Surveillance of Veterinary Antimicrobial Consumption.
The Agency has an advisory role in a number of projects. These include:
- Advanced Immunization Technologies (ADITEC);
- Alzheimer's Association Research Roundtable;
- Advances in Small Trials dEsign for Regulatory Innovation and eXcellence (Asterix);
- Core Outcome Measures in Effectiveness Trials Initiative (COMET);
- Electronic Health Record for Clinical Research (EHR4CR);
- European Autism Interventions - A Multicentre Study for Developing New Medications (EU-AIMS);
- European Medicines Research Training Network (EMTRAIN);
- European Patients' Academy on Therapeutic Innovation (EUPATI);
- Innovative methodology for Small Populations Research (INSPIRE);
- Integrated design analysis small population trials (IDEAL);
- Integrating Bioinformatics and Chemoinformatics Approaches for the Development of Expert Systems Allowing the In Silico Prediction of Toxicities (eTOX);
- Multiple Sclerosis Outcome Assessments Consortium (MSOAC);
- Prediction of Cognitive Properties of New Drug Candidates for Neurodegenerative Diseases in Early Clinical Development (PharmaCog);
- PROactive COPD;
- Safer and Faster Evidence-Based Translation (SAFE-T);
- Scalable, Standard-based Interoperability Framework for Sustainable Proactive Post-market Safety Studies (SALUS);
- Surrogate Markers for Micro- and Macro-Vascular Hard Endpoints for Innovative Diabetes Tools (SUMMIT)
- Unbiased Biomarkers in Prediction of Respiratory Disease Outcomes (BIOPRED).
The Agency also acts as an observer for projects, including: