How can biomarkers become surrogate endpoints?

AbstractAs there is an urgent need for careful planning of development schemes for new classes of molecularly targeted anticancer therapies, the use of biomarkers as surrogate endpoints in therapeutic trials was discussed by BDA delegates, representing the pharmaceutical industry, regulatory agencies, academia, and patient advocacy groups in a breakout session. The aim was the clarification of the role of surrogates in the conduct of clinical trials that serve as a basis for drug licensure or registration, especially in the setting of accelerated or conditional approval. The discussions focused on three questions: (a) how to validate biomarkers, (b) how biomarkers might be used as surrogate endpoints in small clinical trials, and (c) how a biomarker might be used in studies of agents other than the one for which it was validated. The deliberations of the group are discussed herein