Patient registries

Patient registries are organised systems that use observational methods to collect uniform data on a population defined by a particular disease, condition or exposure, and that is followed over time. Patient registries can play an important role in monitoring the safety of medicines. The European Medicines Agency (EMA) has set up an initiative to make better use of existing registries and facilitate the establishment of high-quality new registries if none provide an adequate source of post-authorisation data for regulatory decision-making.

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:

• Patient Registry Initiative- Strategy and Mandate of the Cross-Committee Task Force

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:

• Guideline on registry-based studies

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:

• Report - Patient registries workshop

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)
  • Report - Joint HMA/EMA multi-stakeholder workshop on Patient Registries
• Workshop on the role of registries in the monitoring of cancer therapies based on genetic and molecular features (29/11/2019)
  • Report of the workshop on the use of registries in the monitoring of cancer therapies based on tumours’ genetic and molecular features - 29 November 2019
• Haemophilia registries workshop (08/06/2018)
  • Report on Haemophilia Registries Workshop
• Chimeric antigen receptor (CAR) T-cell therapy registries workshop (09/02/2018)
  • Report - CAR-T cell therapy registries workshop
• Multiple sclerosis workshop - Registries initiative (07/07/2017)
  • Report on multiple sclerosis registries
• Cystic fibrosis workshop - Registries initiative (14/06/2017)
  • Report on cystic fibrosis registries

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.