EMA Sets Up Two-Year Pilot Program on Patient Registries

The European Medicines Agency (EMA) has launched an initiative focused on existing patient registries, with the aim of seeing how they can be better used as a source of post-authorization data, and how they can help to establish new registries.

Background

EMA defines a registry as a system using observational methods over time to collect uniform data on a population defined by a particular disease, condition, or exposure. A disease registry includes patients based on diagnosis, while product registries include patients based on the treatment they receive.

Regulators, particularly the US Food and Drug Administration, may require drugmakers to establish a registry after a drug or device is approved in order to measure the safety or efficacy of the products in clinical practice.

But as the EMA points out, the current state of registries across a wide range of treatments and diseases has caused a variety of challenges, including a lack of sustainability and a lack of harmonized protocols and data structures. In addition, the registries at national or international levels maintained by physicians’ associations or national agencies are often not fully utilized, which may lead to duplicate efforts and inefficiencies.

EMA Initiative

The EMA's patient registry initiative will look into how to address current challenges faced by companies and regulators in regard to existing registries, as well as when to establish any new registries if they're necessary.

The initiative includes two components: a strategy on registries and a pilot to test whether the strategy better supports marketing authorization applicants/holders (MAAs/MAHs) to meet regulators’, and possibly other stakeholders', needs for data.

The strategy proposed starts from the stage where an advice or a request has been expressed pre- or post-authorization by a committee to an MAA/MAH of the need to collect additional data post-authorization, or an MAA/MAH has itself identified the potential need to collect data.

The approach includes five steps:

1. early dialogue with MAAs/MAHs

2. definition of data collection characteristics by or with the committee or working party: objectives, population, outcomes, any hypotheses to be tested; as appropriate, input from different stakeholders may be considered

3. identification of existing data sources that could fulfil the objectives, and evaluation of their adequacy by MAAs/MAHs in collaboration with regulatory authorities and data source custodians

4. need for data or information that is best addressed through a registry

5. a. amendment or addition to existing registry/registries

    b. definition of core components of a new registry

The MAA/MAH will be primarily responsible in identifying existing data sources that could be used, though EMA expects dialogue with the rapporteurs, Member States, other committees and working parties, such as the Patient & Consumers Working Party and the Health Care Professional Working Party.

Pilot

A two-year pilot phase, the start of which EMA says will be "dependent on the timing of the regulatory procedure for the products that will participate," will test whether this strategy better supports MAAs/MAHs in meeting regulators' needs for data and information.

"The aim of the pilot phase is not to make decisions about the need for a registry or to accelerate the granting of a market authorization but to test whether such a planned, collaborative approach is successful in facilitating robust data collection," EMA says.

The pilot will be coordinated by EMA in collaboration with the Cross-Committee Task Force on Registries.

The choice of the candidate diseases/products to be included in the pilot phase will be driven by the objectives of the pilot in terms of methodological tools and approach to be tested (e.g. existing registry or need for new registry, rare or common disease, registry with combined regulatory and health technology assessment purposes), as well as expressions of interest from MAAs/MAHs, such as those based on preliminary discussions in a scientific advice procedure or in a committee.

EMA Press Release

EMA Initiative for Patient Registries