Patient registries may prove valuable in regulatory decisions, EMA study finds
Although patient registries can be heterogeneous in their quality and design, a new study in Drug Safety co-authored by an EMA official explains how patient registries could provide valuable data for regulatory decisions.
The authors explain how such registries can be used when randomized controlled trials are not feasible, such as with some rare diseases, or to meet postmarketing commitments.
“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,” the study says.
The eight authors, from the EMA, Queens Mary University of London, the University of Groningen in the Netherlands and the Dutch Medicines Evaluation Board, also explain how registries are currently underused.
Of 335 products recommended for approval by the EMA’s Committee for Medicinal Products for Human Use between 2005 and 2013, 31 registries were requested to fulfill a condition of the marketing authorization, but by December 2017, just ten had been completed.
To try to figure out what factors facilitate registry use, the authors synthesized observations and recommendations published in five reports arising from a multi-stakeholder EMA consultation and four disease-specific workshops conducted as part of the EMA’s Patient Registries Initiative.
The study explains how the absence or incompleteness of 10 different factors “greatly impeded registry use.”
Such factors include the use of common core data sets, common data coding terminologies, data access and sharing and data linkage capacity, among others.
“From a regulatory perspective, the ultimate requirement of patient registries is that they permit the conduct of high-quality studies that evaluate the safety and effectiveness of medicines. Recognising the benefits and challenges inherent in using observational data for medicines assessments, 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,” the study adds.
RAPS: First published in Regulatory Focus™ by the Regulatory Affairs Professionals Society, the largest global organization of and for those involved with the regulation of healthcare products. Click here for more information.
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