Biomarker Guidances and Reference Materials

On this page:

• Guidance Documents
• Recent FDA-Cosponsored Workshops and Events
• Biomarker Materials and Informational Websites
• Biomarker Survey

Guidance Documents

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.

For more information on the biomarker qualification process described in the 21st Century Cures Act please see:

•  21st Century Cures Act: Qualification of DDT,  
• Resources for Biomarker Requestors: Get Started With Your Submission
• Biomarker FAQ’s
• BEST Glossary
• Considerations for Use of Histopathology and Its Associated Methodologies to Support Biomarker Qualification Guidance for Industry (PDF - 126 KB): Describes scientific standards recommended for nonclinical studies using histopathology to support biomarker qualification.

Recent FDA-Cosponsored Workshops and Events

• Biomarker-driven Drug Development for Allergic Diseases and Asthma, February 2024
• Endpoints and Trial Designs to Advance Drug Development in Kidney Transplantation, November 2023
• Use of Biomarkers for Diagnosing and Assessing Treatment Response in Noncirrhotic NASH Trials, September 2023
• Identification of Concepts and Terminology for Multi-Component Biomarkers, March 2022
• Scientific and Regulatory Considerations for the Analytical Validation of Assays Used in the Qualification of Biomarkers in Biological Matrices, June 2019
• Defining an Evidentiary Criteria Framework for Surrogate Endpoint Qualification, July 2018
• Scientific and Regulatory Considerations for the Analytical Validation of Assays Used in the Qualification of Biomarkers in Biological Matrices, June 2017
• DIA/FDA Statistics 2016 Forum, April 2016
• Developing an Evidentiary Standards Framework for Safety Biomarkers Qualification Workshop, April 2016
• Advancing the Development of Biomarkers in Traumatic Brain Injury, March 2016
• Collaboratively Building a Foundation for FDA Biomarker Qualification, December 2015
• Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: An IOM Workshop, October 2015
• Facilitating Biomarker Development and Qualification: Strategies for Prioritization, Data-sharing, and Stakeholder Collaboration, October 2015
• Evidentiary Considerations for Integration of Biomarkers in Drug Development Symposium, August 2015
• Advancing the Use of Biomarkers and Pharmacogenomics in Drug Development, September 2014

Biomarker Materials and Informational Websites

• Biomarker Qualification Program
• The BEST (Biomarkers, EndpointS, and other Tools) Resource: Harmonizing Biomarker Terminology (PDF)
• BEST (Biomarkers, EndpointS, and other Tools) Resource: Glossary
• Resources for Biomarker Requestors
• FDA: Letter of Support Initiative
• FDA: Critical Path Innovation Meeting
• FDA: Medical Product Development Tools
• European Medicines Agency: Qualification of Novel Methodologies for Medicine Development
• Critical Path Institute (C-Path)

Biomarker Survey (Published February 13, 2015)

Identifying Potential Biomarkers for Qualification and Describing Contexts of Use to Address Areas Important to Drug Development: Survey Results

Contact us at: CDER-BiomarkerQualificationProgram@fda.hhs.gov