Hurry Up and Wait: Timelines and Takeaways from the Biomarker Qualification Program

Area of Science:

• Biomarker Development
• Regulatory Science
• Drug Development Tools

Background:

• The Biomarker Qualification Program (BQP) was established in 2016 to facilitate the development of novel biomarkers for regulatory use.
• This study evaluates eight years of BQP experience to assess its effectiveness in qualifying new biomarkers.

Purpose of the Study:

• To assess the effectiveness of the Biomarker Qualification Program (BQP) in facilitating the qualification of novel biomarkers.
• To identify trends and challenges in the BQP process, particularly for surrogate endpoint biomarkers.

Main Methods:

• Data on accepted biomarker qualification projects were collected from the FDA's Drug Development Tool Qualification Project Search database.
• Characteristics and submission dates of 61 accepted projects were analyzed.

Main Results:

• Safety, Diagnostic, and PD Response biomarkers were most common; molecular and radiologic/imaging methods predominated.
• Half of projects stalled at the Letter of Intent (LOI) stage, with only eight biomarkers fully qualified.
• Review timelines frequently exceeded targets, and Qualification Plan (QP) development was lengthy, especially for surrogate endpoints.

Conclusions:

• The BQP supports certain biomarker types but has limited utility for novel surrogate endpoints due to extended development timelines.
• The program may not be optimally suited for advancing novel response biomarkers.
• A dedicated program could better support the development of novel surrogate measures for broader drug development applications.