The initiative for patient registries, launched in September 2015, explores ways of expanding the use of patient registries by introducing and supporting a systematic and standardised approach to their contribution to the benefit-risk evaluation of medicines within the European Economic Area.
Regulators and pharmaceutical companies currently face a number of challenges in using existing registries or establishing new ones, including a lack of:
- coordination between ongoing initiatives at national and international levels;
- harmonised protocols, scientific methods and data structures;
- data sharing and transparency;
- sustainability.
These factors have led to inefficiency and a duplication of efforts. To address the problems, the EMA initiative seeks to create a European Union-wide framework on patient registries, facilitating collaboration between:
- registry coordinators, such as physicians' associations, patients' associations, academic institutions or national agencies responsible for overseeing healthcare services;
- potential users of registry data, such as medicines regulators and pharmaceutical companies.
To support the initiative, EMA has set up a cross-committee task force on registries, comprising representatives from EMA scientific committees and working parties and experts from national competent authorities. EMA has published the task force's strategy and mandate:
Guideline on registry-based studies
Guidance is available on the methodological, regulatory and operational aspects involved in using registry-based studies to support regulatory decision-making:
The guideline aims to help with defining study populations and designing study protocols. It provides guidance on data collection, data quality management and data analysis to achieve higher quality evidence.
It focuses on studies involving disease registries or condition registries to evaluate the benefit-risk of medicines prescribed to or consumed by patients. It explains the methodological differences between these types of studies and registries.
It also contains an annex with good practices in the establishment and management of patient registries and their use for other possible regulatory purposes.
EMA's Cross-Committee Task Force on Registries and the CHMP agreed the final guideline following a public consultation.
Inventory of registries
EMA provides an inventory of patient registries in the Catalogue of RWD sources as a part of the HMA-EMA Catalogues of real-world data sources and studies. The inventory aims to facilitate the interaction between stakeholders and existing patient registries.
EMA encourages patient registry owners whose registries are not listed in the inventory to add their registries to the database.
Further information on the HMA-EMA Catalogues of real-world data sources and studies with useful videos, materials and questions and answers can be found on the support page.
Stakeholder collaboration and workshop reports
EMA held a stakeholder workshop to better understand the barriers and facilitators to collaboration between stakeholders. The workshop report which provides recommendations on actions to improve stakeholder collaboration and optimise the use of registries to support regulatory decision-making:
EMA also held disease-specific workshops where participants provided recommendations on the use of registries in these disease areas, including on core data elements, consents, governance, data sharing and interoperability. The workshop reports may act as models for guiding use of patient registries in other disease areas:
- Joint Heads of Medicines Agencies (HMA)/European Medicines Agency (EMA) Multistakeholder workshop on Patient Registries (12-13/02/2024)
- Workshop on the role of registries in the monitoring of cancer therapies based on genetic and molecular features (29/11/2019)
- Haemophilia registries workshop (08/06/2018)
- Chimeric antigen receptor (CAR) T-cell therapy registries workshop (09/02/2018)
- Multiple sclerosis workshop - Registries initiative (07/07/2017)
- Cystic fibrosis workshop - Registries initiative (14/06/2017)
EMA has provided qualification opinions and letters of support on several registries, describing the contexts in which EMA considers the use of these registry data suitable for regulatory purposes:
- Opinions and letters of support on the qualification of novel methodologies for medicine development
The task force welcomes the opportunity to work with stakeholders in facilitating the development of implementation plans to support the delivery of the workshop recommendations.
It also welcomes feedback on these recommendations and interest from stakeholders in taking part in activities facilitating the use of registries for regulatory decision-making.