Europe – Study Finds Extent of Unvalidated Surrogate Endpoint Use in Expedited Approvals ‘Concerning’

Research published this week in PLOS Medicine raises concerns about the use of surrogate endpoints to support the marketing authorizations of products assessed via the European Medicines Agency’s (EMA) conditional marketing authorisation (CMA) and accelerated assessment (AA) pathways.

The use of the CMA and AA pathways, which is restricted to situations of “unmet medical need” and/or “in the interests of public health,” are often based on incomplete benefit-risk assessment data and may have post-authorization conditions or restrictions to ensure the products live up to early data.

The study evaluated 33 pivotal trials for CMA and 58 trials for products using the AA pathway between January 2011 and December 2018, with the researchers finding that most of the marketing authorizations were based on pivotal trials that reported unvalidated surrogate endpoints…