Biomarker Qualification - "Fit for Clinical Purpose"

Biomarker Qualification

Biomarker qualification is an official FDA evidence-based development process (see above links for details) that enables sponsors to tie an identified biomarker to a specific biological process(es) and associated clinical endpoint. This process essentially verifies that the biomarker is “fit-for-purpose” for the clinical application proposed. 

The following from the FDA's guidance details these goals:

The goals of the CDER Biomarker Qualification Program are to

  • Provide a framework for scientific development and regulatory acceptance of biomarkers for use in drug development
  • Facilitate integration of qualified biomarkers in the regulatory review process
  • Encourage the identification of new and emerging biomarkers for evaluation and utilization in regulatory decision-making
  • Support outreach to relevant external stakeholders to foster biomarker development

This FDA process for biomarker qualification allows the proposing entity to engage in an iterative data exchange process with the agency. To proceed along the biomarker qualification process, the biomarker sponsor defines the biomarker and its context of use; identifies the data types and quality requirements for the data needed to support qualification; and compiles and reviews the data with the FDA. If deficiencies are identified in the data, the sponsor is tasked with submitting additional data to thoroughly qualify the biomarker. This is a rigorous process that may require ongoing data submission and re-evaluation of the biomarker before the biomarker is officially "qualified." In addition, it should be noted that although the biomarker is being qualified on the basis of its relationship to changes in biology, the test and its ability to accurately and reproducibly measure the targeted change must also be considered in these submissions.

If a sponsor intends to qualify a biomarker, it is important to prepare documents for initial review with appropriate FDA personnel to ensure that there is mutual understanding on the types and quality of data required. Much of the data may exist, but it may also be necessary to acquire additional data from diverse sources and/or additional clinical studies. 

What makes a Qualified Marker



Qualification and Validation