Biomarker Guidances and Reference Materials

Below is a suggested template that submitters may modify to accommodate their submission data, as needed:

Guidance Documents

Qualification Process for Drug Development Tools (PDF - 499 KB): Describes the process for qualifying drug development tools intended for potential use, over time, in multiple drug development programs.

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.

FDA-Cosponsored Workshops and Events

DIA/FDA Statistics 2016 Forumdisclaimer icon, April 2016

Developing an Evidentiary Standards Framework for Safety Biomarkers Qualification Workshopdisclaimer icon, April 2016

Advancing the Development of Biomarkers in Traumatic Brain Injury, March 2016

Collaboratively Building a Foundation for FDA Biomarker Qualificationdisclaimer icon, December 2015

Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: An IOM Workshopdisclaimer icon, October 2015

Facilitating Biomarker Development and Qualification: Strategies for Prioritization, Data-sharing, and Stakeholder Collaborationdisclaimer icon, October 2015

Evidentiary Considerations for Integration of Biomarkers in Drug Development Symposiumdisclaimer icon, August 2015

Advancing the Use of Biomarkers and Pharmacogenomics in Drug Developmentdisclaimer icon, September 2014

Presentation Slides

Amur S. Biomarker Qualification at CDER, FDA. Advancing the Development of Biomarkers in Traumatic Brain Injury; March 3, 2016; Silver Spring, MD.

Amur S. Biomarker Qualification. Osteoporosis Drug Development Moving Forward: Public Workshop; November 4, 2015; Silver Spring, MD.

Amur S. Opportunities to Develop Meaningful Biomarkersdisclaimer icon. IOM Workshop, October 2015.

Woodcock J. Keynote Addressdisclaimer icon. M-CERSI Symposium; August 21, 2015; Baltimore, MD.

Amur S. FDA’s Efforts to Encourage Biomarker Development and Qualificationdisclaimer icon. M-CERSI Symposium; August 21, 2015; Baltimore, MD.

Buckman-Garner S. Biomarker-Based Enrichment of Clinical Study Populationsdisclaimer icon. M-CERSI Symposium; August 21, 2015; Baltimore, MD.

Amur S. Biomarker Qualification at CDER/FDAdisclaimer icon. EMA-FDA Webinar, May 23, 2013.

Parekh A. Drug Development Tool Qualification at CDER/FDAdisclaimer icon. EMA-FDA Webinar, May 23, 2013.

Select Publications

Robb MA, McInnes PM, Califf RM. Biomarkers and surrogate endpoints: Developing common terminology and definitionsdisclaimer icon. JAMA. 2016;315(11):1107-1108..

Duke-Margolis Center for Health Policy. Facilitating Biomarker Development: Strategies for Scientific Communication, Pathway Prioritization, Data-Sharing, and Stakeholder Collaborationdisclaimer icon. 2016.

Amur S, LaVange L, Zineh I, Buckman-Garner S, Woodcock J. Biomarker qualification: Toward a multiple stakeholder framework for biomarker development, regulatory acceptance, and utilizationdisclaimer icon. Clin Pharmacol Ther. 2015;98(1):34-46.

Amur SG, Sanyal S, Chakravarty AG, et al. Building a roadmap to biomarker qualification: Challenges and opportunitiesdisclaimer icon. Biomark Med. 2015;9(11):1095-1105.

Hausner EA, Hicks KA, Leighton JK, Szarfman A, Thompson AM, Harlow P. Qualification of cardiac troponins for nonclinical use: A regulatory perspectivedisclaimer icon. Regul Toxicol Pharmacol. 2013;67(1):108-114.

Goodsaid F, Mattes WB, eds. The Path from Biomarker Discovery to Regulatory Qualificationdisclaimer icon. 1st ed. Cambridge, MA: Academic Press, 2013.

Woodcock J, Buckman S, Goodsaid F, Walton MK, Zineh I. Qualifying biomarkers for use in drug development: A U.S. Food and Drug Administration overviewdisclaimer icon. Expert Opin Med Diagn. 2011;5(5):369-374.

Dieterle F, Sistare F, Goodsaid F, et al. Renal biomarker qualification submission: A dialog between the FDA-EMEA and Predictive Safety Testing Consortiumdisclaimer icon. Nat Biotechnol. 2010;28(5):455-462.

Goodsaid F, Papaluca M. Evolution of biomarker qualification at the health authoritiesdisclaimer icon. Nat Biotechnol. 2010;28(5):441-443.

Throckmorton DC. How a U.S. regulator can encourage new sciencedisclaimer icon. Clin Pharmacol Ther. 2009;86(5):471-472.

Biomarker Materials and Informational Websites

The BEST (Biomarkers, EndpointS, and other Tools) Resource: Harmonizing Biomarker Terminology (PDF)

BEST (Biomarkers, EndpointS, and other Tools) Resource: Glossarydisclaimer icon

Food and Drug Administration: FDA Case Study: Biomarker Qualification – Developing and Obtaining Regulatory Acceptance of a New Biomarker (PDF - 944KB)

Food and Drug Administration: Letter of Support Initiative

Food and Drug Administration: Critical Path Innovation Meeting

Food and Drug Administration: Medical Product Development Tools

Food and Drug Administration: Biomarkers Used as Outcomes in Development of FDA-Approved Therapeutics (October 2007- December 2015)

European Medicines Agency: Qualification of Novel Methodologies for Medicine Developmentdisclaimer icon

Critical Path Institute (C-Path)disclaimer icon

Biomarker Survey

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


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