Biomarker Qualification: FDA Drafts Guidance

Regulatory NewsRegulatory News | 12 December 2018 |  By 

The US Food and Drug Administration (FDA) on Tuesday released draft guidance for industry discussing the recommended components of a biomarker development program, including how to determine the type and level of evidence sufficient to support qualification, and how these components interrelate to inform an evidentiary framework.

The draft comes as to date, FDA has qualified eight biomarkers and received 29 submissions. Still, as the draft notes, progress in biomarker qualification has been stymied by the lack of a clear, predictable and specific regulatory framework that lays out the type and level of evidence to support regulatory decision making.

With the current limitations in view, the draft opens with a section on the evidentiary framework that should be considered when determining the type and level of evidence necessary to support qualification of a biomarker, including: “(1) describing the drug development need, (2) defining the COU [context of use], (3) considering potential benefits if the biomarker is qualified for use, and (4) considering potential risks associated with the proposed use of the biomarker in a drug development program.”

The draft further explains what should be explained by sponsors in terms of a “needs assessment,” meaning why a biomarker is needed for drug development, COU, which should include the biomarker category and the proposed use in drug development, the “a clear and objective description” of a biomarker’s benefits and risks and how to determine the evidence that is scientifically sufficient to support a COU.

The draft also discusses analytical and statistical considerations, noting that because drug development decisions will be made based upon qualified biomarkers, any biomarker test used to measure the biomarker “should be robust, sensitive, and specific enough to support the decisions defined by the COU.” In addition, the goal of statistical analyses is to evaluate the degree and certainty of association between a biomarker and an outcome of interest, FDA says.

But FDA also makes clear that this draft guidance is just the first draft.

“Ultimately, FDA anticipates that a future revised draft guidance on this topic will meet the statutory requirement for draft guidance on a ‘conceptual framework describing appropriate standards and scientific approaches to  support the development of biomarkers’ described in section 3011(b)(1)(A) of the Cures Act and meet the commitment in section (1)(J)(6)(d) of the PDUFA VI goals letter related to publishing a draft guidance on ‘general evidentiary standards for biomarker qualification,’” the draft says.

Biomarker Qualification: Evidentiary Framework Guidance for Industry and FDA Staff


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