Draft guidance on registry-based studies

The European Medicines Agency (EMA) has addressed the used of registry-based studies in pre-and post- authorisation phases for medicines.

The new draft guidance aims to optimise the use of registry-based studies as a source of real-world evidence. The guideline’s primary focus is on the use of patient registries for studies by marketing authorisation applicants and holders (MAAs/MAHs). The guideline’s scope includes disease and condition registries, such as those for patients with a particular disease or disease characteristic, a condition such as pregnancy, or molecular or genomic features.

In the post-authorisation phase, patient registry-based studies can be useful for both safety and efficacy studies. Registry-based post-authorisation studies can help with assessment of effectiveness of adapted drug dosing schemes. They can also aid understanding of the effectiveness and safety of medicinal products in a broader clinical disease-related context and a more heterogeneous patient population.

Registries are important for patients with rare diseases who may receive advanced therapy medicinal products (ATMPs) such as gene therapy, since approval of these products is often based on small patient populations. According to the guidance, post-authorisation activity planning should begin in the pre-authorisation phase for ATMPs.

For pre-authorisation studies, observational evidence from patient registries can be helpful since the registries can supply information about population characteristics which can help to identify valid surrogate endpoints.

Also included is guidance on study protocol, population, and data management. The guideline also includes an annex detailing general considerations on constructing and using patient registries.

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