A new study has raised questions about the European Medicines Agency’s (EMA’s) expedited approval pathways for new drugs.
The study published in PLoS Medicine found that the majority of marketing authorisations granted through two expedited assessment pathways in Europe are based on non-validated surrogate endpoints rather than clinical outcomes.
The authors used European Public Assessment Reports (EPARs) to identify the primary endpoints in the pivotal trials supporting products authorised through conditional marketing authorisation (CMA) or accelerated assessments (AA) pathways from 2011 through 2018.
Most of the expedited approvals studied (46/51, 90%) were based on surrogate endpoints, none of which has been shown to reliably predict clinical outcomes.
Among a total of 49 products with surrogate endpoints reported, most were rated as being reasonably likely (n=30, 63%) or of having biological plausibility (n=45, 94%) to predict clinical outcomes.
“The extensive use of non-validated surrogate endpoints is concerning because the likelihood that treatment will provide the intended clinical benefit is unknown,” the authors say. They say, for products supported by non-validated surrogate endpoints, post-authorisation measures to confirm clinical benefit should be imposed by the regulator on the marketing authorisation holders.