A Regulatory Perspective on Validation of Surrogate Endpoints

Abstract

The use of biomarkers as surrogate endpoints has had a dramatic and powerful impact on health care and the development and accessibility of drugs. For example, cholesterol is a biomarker used in many clinical trials of cardiovascular drugs such as “statins” where cholesterol reduction is used as a surrogate endpoint for reduced mortality. Although much progress had already been made over the past 20 years—especially in terms of the very real benefits surrogate endpoints have made to efficient medical product development and patient care—the industry faces a host of future challenges as the use of biomarkers and surrogate endpoints play a more integral role in the advancement of new medicines along the critical path of drug development. During the 41st DIA Annual Meeting in Washington, DC, Dr. Lawrence Lesko from the Office of Clinical Pharmacology at the Food and Drug Administration presented a discussion on the challenges associated with the validation of surrogate endpoints, highlighting progress to date as well as future objectives.