[co-author: Silvia López Arnao]
On 24 September 2020, the European Medicines Agency (“EMA”) published a draft Guideline on registry-based studies. The EMA’s intention is to enhance the use of registry-based studies as a source of real-world evidence (RWE). The guideline is primarily aimed at marketing authorization applicants and holders planning to conduct registry studies in the pre- or post-marketing authorization phase. A draft version of the guideline will be open for consultation until 31 December 2020.
On 24 September 2020, the European Medicines Agency (“EMA”) published a draft Guideline on registry-based studies. The document provides guidance on methodological, legal, and operational aspects of the use of registry-based studies. The guideline is primarily intended for marketing authorization applicants and holders planning to conduct registry studies in the pre- or post-marketing authorization phase.
The EMA Cross-Committee Task-Force on Registries was responsible for drafting the guideline, in close cooperation with the European Medicines Regulatory Network.
Patient registries are defined by the EMA as data collection systems on a group of people defined by a particular disease or condition that are used to conduct a registry-based study. They serve a pre-determined scientific, clinical, and/or public health purpose.
The guidance defines registry-based studies as an investigation of a research question using the infrastructure of a new or existing patient registry, or from a registry newly set-up for the study.
Registry-based studies as a source of RWE
In the draft guideline, the EMA focuses on registry-based studies as a tool to generate RWE. To illustrate this, the guideline provides examples where registry-based studies may be useful for the generation of RWE. The EMA recalls, in particular, that registry-based studies may help generate RWE for the following purposes:
- To supplement the evidence generated in a pre-authorisation phase. RWE derived from patient registries can support knowledge generated through pre-clinical studies and clinical trials. Examples of such evidence include information on standards of care for the disease, incidence and determinants of disease outcomes in clinical practice, and characteristics of the target population;
- To provide data sources or infrastructure for post-authorisation evidence generation. Patient registry-based studies can be data sources for randomised controlled trials (RCTs), post authorisation efficacy studies (PAES) or post-authorisation safety studies (PASS). These post-authorisation studies are particularly important for ATMPs;
- To evaluate the effects of medications received during pregnancy
Other regulatory considerations
The guideline also addresses other regulatory considerations concerning the establishment and management of patient registries. The document provides the requirements that must be respected when planning of a registry-based study. These include the identification of one or several suitable registries, the conclusion of a collaboration agreement with each registry, and the establishment of a database, a data extraction process, and quality control activities.
The EMA also expects applicants for marketing authorization and authorization holders to perform a “feasibility analysis” prior to writing the study protocol to guide its development and facilitate discussions with other stakeholders. The feasibility analysis will be performed in collaboration with registry holders and will include, among others, the general description of the registry, an analysis of the availability of the data elements required for the study, and an analysis of any potential information bias.
The guideline also offers recommendations in relation to the study protocol, study population, data collection, data quality management, data analysis, and data reporting. To support these recommendations, considerations on good practice for registries are included in the Annex to the document.
Open for consultation
Stakeholders will be required to send their comments on the draft guideline by 31 December 2020. For the submission of the proposals, an online form is available in the EMA’s webpage.
On 19 October 2020, the draft guideline will be presented to key stakeholders in a virtual workshop organised by the EMA.
After analysing and considering the feedback received, the EMA plans to publish the final version of the document in 2021.