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  1. Biomarker Qualification Program

Pathways for Biomarker Integration in Drug Development

Pathways for Using Biomarkers in Drug Development


Pathways for Using Biomarkers in Drug Development

Transcript | Slides (PDF - 1.2MB)


What are the regulatory pathways used in the Center for Drug Evaluation and Research (CDER) to integrate biomarkers into drug development?

In CDER, new biomarkers may be integrated into drug development through two review pathways: (1) the drug approval process and (2) the Biomarker Qualification Program.

The most common pathway to integrate biomarkers into drug development is using them within the context of a specific drug development program. Drug developers may choose to use biomarkers, established or novel, as part of clinical trials to answer questions pertinent to a particular drug. For novel biomarkers, the drug developer is responsible for all aspects of the biomarker’s development. If new information suggests that the biomarker may have use in other drug development programs, then CDER may include the use of the biomarker in Guidance or approved product labeling.

Qualification through the Biomarker Qualification Program (BQP) is for biomarkers that may be used in multiple drug development programs. Once qualified, a biomarker can be used under its qualified context of use (COU) during the development of any candidate drug. Details on the biomarker qualification process can be found at:

These pathways do not exist in isolation, and many times parallel efforts to develop a biomarker for regulatory use are underway within or between these pathways. These pathways share common core concepts, are data-driven, and involve regulatory assessment and outcomes based on the available data.

What is the difference between CDER’s and the Center for Devices and Radiological Health’s (CDRH’s) qualification programs for biomarkers?

CDRH has a Medical Device Development Tools Program that qualifies biomarker tests for use in medical device development, while CDER’s BQP qualifies biomarkers for use in drug development.

Are there any additional pathways for having a discussion about biomarkers with CDER?

Yes. Critical Path Innovation Meetings (CPIMs) are an opportunity for CDER to discuss a methodology or technology proposed by a meeting requestor and for CDER to engage in a general scientific discussion of how the methodology or technology might enhance drug development. Through CPIMs, CDER may enhance its familiarity with prospective innovations in drug development, broadening its regulatory perspective. The discussions and background information submitted through the CPIM are drug product–independent and nonbinding on both FDA and CPIM requestors. Potential topics for a CPIM may include, but are not limited to,

  • Biomarkers in the early phase of development and not yet ready for the BQP
  • Clinical outcome assessments in the early phase of development and not yet ready for the Clinical Outcome Assessment Qualification Program
  • Natural history study designs and implementation
  • Emerging technologies or new uses of existing technologies
  • Innovative conceptual approaches to clinical trial design and analysis

What is a Letter of Support (LOS)?

An LOS briefly describes CDER’s thoughts on the potential value of a biomarker and encourages its further development. FDA may issue an LOS when a requestor, submits a package of supporting information about a promising biomarker that has not yet been accepted into the BQP. The letter issued by FDA does not suggest qualification of a biomarker or, its analytics, nor does it confirm the proposed context of use. It is meant to enhance the visibility of the biomarker, encourage data sharing, and stimulate additional studies.

Contact CDER-BiomarkerQualificationProgram@fda.hhs.gov for more information

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