Study: How Dependable Are Surrogate Endpoints?

Drugmakers are all familiar with the expedited programs offered by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) to help accelerate drug approval. These programs are especially helpful for companies developing drugs for rare diseases, for which there are few, if any, effective treatment options available.

Because these programs operate on a tighter timeline, regulators might accept surrogate endpoints — substitutes for clinical endpoints that are expected to predict the effect of the therapy.