25 August 2022 - Officials from the 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, 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.”

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