FDA Shows No Letup with New Biomarker Guidance

Traditionally, the workplace pace slows as the holiday spirit overtakes us in December. Days are a little less productive. Employees take vacations. It’s a time usually reserved for office parties and long lunches.

Tell that to the U.S. Food and Drug Administration (FDA).

Yesterday (December 12), the agency released another clinical trial–related guidance—this one providing recommendations on general considerations to address when developing a biomarker for qualification under the Cures Act.

It will be interesting to see if this wave of guidances is the precursor to a surge in inspections or to some other shift in the agency’s focus in 2019.

The new guidance on “Biomarker Qualification: Evidentiary Framework” discusses the evidentiary framework that should be used to support biomarker qualification. Specifically, it describes needs assessment, context of use, and benefit-risk considerations, and how these considerations can relate to determining the type and level of evidence to support qualification of a biomarker. It also addresses general statistical and clinical considerations related to the correlation between the biomarker and the outcome of interest, as well as general analytical considerations related to the performance characteristics of the biomarker test.

Historically, biomarkers gained acceptance for use in drug development after evidence from scientific and medical communities accumulated over time, leading to the recognition of the role and value of the biomarker in decision-making, FDA says in the guidance. This evidence was considered as part of drug-specific development efforts, and there was no formal regulatory process to assess the broader utility of the biomarker independent from its use in a specific drug program.

“Even after the Center for Drug Evaluation and Research (CDER) established the legacy (pre-Cures Act) Biomarker Qualification Program in 2007, progress in the development of biomarkers and their application in drug development has been hampered by the lack of a clear, predictable, and specific regulatory framework for the evidence sufficient to support regulatory decision-making using biomarkers,” FDA says in the guidance.

FDA is accepting comments on the guidance (here’s where to submit).

Author: Michael Causey