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 more 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 set up a cross-committee task force on registries in 2014, comprising representatives from EMA scientific committees and working parties and experts from national competent authorities. EMA revised the task force's strategy and mandate in May 2017:

The task force has established links with health technology assessment bodies and payers and the European Commission.

Inventory of registries

EMA has set up an inventory of patient registries in the resources database of the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP). 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.

EMA also published a guidance document on how to search the ENCePP resources database for information about patient registries and on how to upload new registry details:

Stakeholder collaboration

To better understand the challenges and barriers to collaboration between stakeholders, EMA held a stakeholder workshop in October 2016. The Agency published a workshop report in February 2017:

It recommends activities for EMA to undertake, in cooperation with the cross-committee task force, to improve stakeholder collaboration and optimise the use of registries to support regulatory decisions. These include:

  • exploring mechanisms for regulators and marketing authorisation applicants to systematically consider where registry data may support benefit-risk evaluations throughout product lifecycles and to interact early with registry holders to determine data availability;
  • sharing and disseminating information on patient registries in specific disease areas;
  • recommending governance principles and standards for stakeholder interactions;
  • making recommendations on core data elements and quality standards acceptable for regulatory decision-making;
  • identifying registry holders' needs for methodological and technical guidance;
  • investigating what patient-reported outcomes to collect in registries;
  • exploring further measures to improve the sustainability of registries.

In 2017, EMA hosted two stakeholder workshops on patient registries in cystic fibrosis and in multiple sclerosis. Participants representing multiple stakeholder groups provided recommendations in each case on registry data elements, consents, governance, data sharing and interoperability. The reports, available in the related documents area of this page and on the event pages of the cystic fibrosis workshop and the multiple sclerosis workshop, may act as models for guiding use of patient registries in other disease areas.

As a next step, the task force will work with stakeholders to facilitate the development of implementation plans to support delivery of the recommendations.

The Agency welcomes feedback on the report's recommendations and interest from stakeholders in taking part in these activities.

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