Last September, the EMA published its draft Guideline on registry-based studies (“Guideline”). This draft Guideline is currently open for public consultation: stakeholders may post comments until December 31, 2020.

A registry-based study is an investigation of a research question using data from a new or existing patient registry for patient recruitment and data collection. Patient registries are data collection systems for data on a group of people with a particular disease or condition; a registry is established for a specific purpose and used to conduct a registry-based study. Registries contain a lot of information on diseases, patients, standards of care, use of medicines, devices, and results of care and have already been the subject of initiatives carried out by the EMA since September 2015 (with regard to the initiative for patient registries launched in November 2018, please also refer to our article “Registri di malattia dei pazienti: potenzialità di utilizzo a fini regolatori”).

A registry-based study may be a clinical study or a non-interventional study. Studies based on patient registries provide real evidence to complement the results obtained from clinical trials and pre-clinical studies and to improve knowledge about the risks and benefits associated with the daily use of medicines.

The Guideline provides regulatory requirements and recommendations on key methodological aspects for the use of patient registries by marketing authorization applicants and holders in order to carry out studies, including the following:

• planning a registry-based study requires identifying one suitable registry or multiple suitable registries and obtaining agreement to participate from each registry, as well as from each individual center if no central registry coordination exists, potentially selecting a third party to be involved in the study and set up a database, and establishing a data extraction process and quality control practices (par. 3.3 of the Guideline);
• the study protocol should describe how the registry infrastructure and population will be used to address the scientific question at stake, how the study will be conducted, and how the validity of the results will be ensured (par. 3.4 of the Guideline).

The final version of Guideline will reflect stakeholders’ comments and will be published in 2021.