On December 11, 2018, the Food and Drug Administrative (“FDA”) issued a draft guidance for comment entitled, “Biomarker Qualification: Evidentiary Framework” (the “Guidance”).  The Guidance provides insight regarding standards for biomarker qualification under the 21^st Century Cures Act (“Cures Act”). ^[1]

FDA defines the term “biomarker” as a “characteristic that is measured as an indicator of normal biological processes, pathogenic processes, or responses to an exposure or intervention, including therapeutic interventions.”^[2] There are various types of biomarkers including, but not limited to: molecular – (i.e. blood glucose); radiographic (i.e. the size of a tumor); and physiologic (i.e. blood pressure), and each of these biomarkers fall into various categories, all of which are regulated by FDA.^[3] The term “biomarker qualification” is defined as “a conclusion, based on a formal regulatory process that within the stated context of use, can be relied upon to have a specific interpretation and application in medical product development and regulatory review.”^[4] Importantly, once a biomarker is qualified for a particular context of use, it becomes publically available and can be applied in any drug development program for that qualified context of use.^[5]

The Guidance discusses the evidentiary framework for supporting biomarker qualification, including needs assessments; context of use; and benefit-risk considerations, and how these considerations can relate to determine the type and level of evidence required to support the qualification of a biomarker. Additionally, the Guidance addresses general statistical and clinical considerations related to the correlation between the biomarker and outcome of interest, and general analytical considerations related to performance characteristics of the biomarker test.

Ultimately, the success of the Guidance in advancing biomarker qualification will turn on its contents and stakeholder input.  The Agency has asked for comments on the Guidance by February 9, 2019, to ensure that comments can be fully considered before the Guidance is finalized, although comments may be submitted on FDA guidance at any time. The formal announcement about the draft Guidance issued by FDA is available here.

[1] The Cures Act, which was enacted in December 2016, added a new section 507 to the Food, Drug, and Cosmetic Act (“FDCA”) called “Qualification of Drug Development Tools” (“Section 507”), which includes transparency provisions that apply to requestors’ submissions, and FDA’s written determinations.

[2] Amur, Shashi Ph.D., “Biomarker Terminology: Speaking the same Language,” FDA, available at https://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/ucm533161.pdf.

[3] Id.

[4] Id.

[5] Id.