EMA study outlines how patient registries may prove valuable in regulatory decisions

Patient registries can be heterogeneous in their quality and design, however a new study in Drug Safety co-authored by a European Medicines Agency (EMA) official outlines how patient registries may provide valuable data for regulatory decisions. Such registries may be used when randomised controlled trials are not feasible, for example with some rare diseases, or to meet post-marketing commitments.

The eight authors, who are from the EMA, Queens Mary University of London, the Netherlands University of Groningen and the Dutch Medicines Evaluation board, discuss how registries are currently underused.

The study states that “for products granted conditional marketing approval, registry studies may provide post-authorisation data to fulfil regulator-imposed specific obligations to confirm safety and/or effectiveness, as is the case with the recently authorised chimeric antigen receptor (CAR) T-cell products, tiagenlecleucel and axicabtagene ciloleucel”.

Between 2005 and 2013, of 335 products recommended for approval by EMA’s Committee for Medicinal Products for Human Use, 31 registries were requested to fulfill a condition of the marketing authorisation. However, by December 2017 only 10 had been completed. The study explains how the absence or incompleteness of several different factors such as the use of common core data sets, common data coding terminologies, data access and data linkage greatly impeded registry use.

The study adds that, in 2019, “EMA will publish methodological and operational advice on handling registry data in post-authorisation studies, taking into account responses to its open consultation on a preliminary discussion paper”.

To read the study from Drug Safety on patient registries: an underused resource for medicines evaluation, please click here.