Getting by with a Little Help from Their Friends: FDA Using External Experts to Enhance Biomarker Qualification and Enable Precision Medicine

David E. Paul and Catherine Clements


Biomarkers are essential tools in expediting the development of new drugs, particularly Precision Medicines. While an innovator biopharmaceutical company often utilizes biomarkers to support developing their own drug, innovators may also seek to have biomarkers “qualified” by FDA. “Qualification” is an FDA determination that within an approved, specific context of use (COU) the qualified biomarker can be relied upon to have a specific use or interpretation to support drug development. That is, the biomarker will be publicly available for its COU to support any innovator’s applicable drug development program and application, without the need for FDA reviewers or the innovator to reconfirm the validity of the biomarker for its COU.
Recognizing the need to help accelerate biomarker qualification, Congress, in passing the recent 21st Century Cures Act, effectively statutorily codified FDA’s biomarker qualification process and included provisions for FDA to utilize external experts at the agency’s discretion. This paper argues the agency should follow historical precedent, fully utilizing an external expert consortium to conduct first substantive reviews and to make recommendations to FDA on biomarker submissions. We recommend the agency pilot this approach to explore further considerations such as application of its conflict of interest rules, process timelines, and costs involved.