Officials from the European Medicines Agency (EMA) tout the benefits of patient registries to support regulatory decision making for orphan medicinal products in a recent journal article in Frontiers in Pharmacology.
Citing the increasing use of real-world evidence (RWE), including patient registries, the officials say that such sources offer “useful evidence at different stages of orphan medicinal products lifecycle, including during the development phase by providing information on disease natural history, its prevalence and incidence to contextualise pre-authorisation clinical studies, to support orphan designation initial and maintenance applications by demonstrating significant benefit versus existing treatments, but also to generate post-authorisation long term data on their effectiveness and safety profiles.”
The article offers examples of registries that have been used during the evaluation of various orphan drugs, including multiple advanced therapy medicinal products (ATMPs), and provides insights on how those data sources facilitated the agency’s decision making. For instance, they note the Committee for Medicinal Products for Human Use (CHMP) has successfully used the Cystic Fibrosis Foundation Patient Registry to expand therapy for certain cystic fibrosis subpopulations with a certain genetic marker…
