The Biomarker Qualification Program was established to support the Center for Drug Evaluation and Research’s (CDER's) work with external stakeholders to develop biomarkers that aid in the drug development process. Through the FDA’s Biomarker Qualification Program, you may request regulatory qualification of a biomarker for a particular context of use in drug development.
Biomarkers can be used in a variety of settings, including basic research, drug development, and clinical practice. The Biomarker Qualification Program focuses on biomarkers used in drug development. Once a biomarker is qualified, it can be used in any drug development program under the context for which it obtained qualification.
The Biomarker Qualification Program is one of the Drug Development Tools (DDT) Qualification Programs created by CDER to provide a framework for development and regulatory acceptance of scientific tools for use in drug development programs.
The goals of the CDER Biomarker Qualification Program are to:
- Provide a framework for development and regulatory acceptance of biomarkers for use in drug development
- Facilitate integration of qualified biomarkers into the regulatory review process
- Encourage the identification of new and emerging biomarkers for evaluation and use in regulatory decision making
- Support outreach to stakeholders to encourage biomarker development
A biomarker is a defined characteristic that is measured as an indicator of normal biological processes, pathogenic processes, or responses to an exposure or intervention, including therapeutic interventions.
For clarifications of important definitions and explanations of some of the relationships among terms, review the BEST (Biomarkers, EndpointS, and other Tools) Resource and the BEST Resource Fact Sheet.
Biomarker Terminology: Speaking the Same Language
Biomarkers can provide valuable information at various stages of drug development. They have the potential to:
- Reduce the time required to complete clinical trials
- Reduce the sample sizes required to achieve statistical significance
- Provide valuable information that could reduce uncertainty in regulatory decisions
Why qualify a biomarker through the CDER Biomarker Qualification Program?
A qualified biomarker can be used in multiple drug development programs without a need for CDER to reconfirm the suitability of the biomarker’s qualified context of use and has the potential to advance public health by streamlining the drug development paradigm.
What is the process to qualify a biomarker?
The biomarker qualification process consists of three stages: (1) Initiation, (2) Consultation and Advice, and (3) Review.
Additional resources for submitting a biomarker for qualification:
- Biomarker Qualification Context of Use—Explains the context of use statement and provides sample statements.
- Biomarker Qualification Submission Resources by Stage—Includes the resources needed for submitting a biomarker, including templates for submission documents, and an example context of use.
- Biomarker Qualification Submissions Checklist– Lists the content that should be included in the submission documents for each stage of the qualification process.
- Biomarker Qualification FAQ—Provides responses to some frequently asked questions about the program.
- Biomarker Qualification Contacts and Submitting Procedures—Provides program contact information, including the mailing address and format requirements for the submission documents.
- Biomarker Qualification Submission Numbers by Stage—List of the current number of Drug Development Tools Qualification Projects by stage including the Biomarker Qualification Program submissions.
- Biomarker Qualification Submissions—Lists the submissions that are currently in the qualification process. Submissions listed on the page are only for those that submitters have agreed to make public.
- Guidance for Industry and FDA Staff: Qualification Process for Drug Development Tools—Describes the process for qualifying drug development tools intended for potential use, over time, in multiple drug development programs.
|General Area||Submitter||Biomarker(s) Qualified for Specific Contexts of Use||Issuance Date with Link to Specific Guidance||Guidance Type||Supporting Information|
Predictive Safety and Testing Consortium (PSTC), Nephrotoxicity Working Group (NWG)
Urinary biomarkers: Albumin, β2- Microglobulin, Clusterin, Cystatin C, KIM-1, Total Protein, and Trefoil factor-3
International Life Sciences Institute (ILSI)/ Health and Environmental Sciences Institute (HESI), Nephrotoxicity Working Group
Urinary biomarkers: Clusterin, Renal Papillary Antigen (RPA-1)
PJ O'Brien, WJ Reagan, MJ York and MC Jacobsen
Serum/plasma biomarkers: Cardiac troponins T (cTnT) and I (cTnI)
|Clinical||Mycoses Study Group||Serum/bronchoalveolar lavage fluid biomarker: Galactomannan||11/14/2015 Patient selection biomarker for enrollment in Invasive Aspergillosis (IA) clinical trials||Final|
|Clinical||Chronic Obstructive Pulmonary Disease (COPD) Biomarker Qualification Consortium (CBQC)||Plasma biomarker: Fibrinogen||9/14/2016 Prognostic biomarker for enrichment of clinical trials in Chronic Obstructive Pulmonary Disease (COPD)||Final|
|Clinical||Polycystic Kidney Disease Outcomes Consortium||Imaging Biomarker: Total Kidney Volume (TKV)||9/15/2016 Prognostic biomarker for enrichment of clinical trials in Autosomal Dominant Polycystic Kidney Disease.||Final||Reviews|
*Qualification determinations issued prior to publication of Guidance for Industry: Qualification Process for Drug Development Tools