Biomarker Guidances and Reference Materials

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 definitions. JAMA. 2016;315(11):1107-1108. http://jama.jamanetwork.com/article.aspx?articleID=2503184disclaimer icon.

Duke-Margolis Center for Health Policy. Facilitating Biomarker Development: Strategies for Scientific Communication, Pathway Prioritization, Data-Sharing, and Stakeholder Collaboration. 2016. https://custom.cvent.com/00BF8C9066844371A697530EA9BB54B7/files/a5a2d54337f249f897211ee04058c4f3.pdfdisclaimer icon

Amur S, LaVange L, Zineh I, Buckman-Garner S, Woodcock J. Biomarker qualification: Toward a multiple stakeholder framework for biomarker development, regulatory acceptance, and utilization. Clin Pharmacol Ther. 2015;98(1):34-46. http://www.ncbi.nlm.nih.gov/pubmed/25868461disclaimer icon

Amur SG, Sanyal S, Chakravarty AG, et al. Building a roadmap to biomarker qualification: Challenges and opportunities. Biomark Med. 2015;9(11):1095-1105. http://www.ncbi.nlm.nih.gov/pubmed/26526897disclaimer icon.

Hausner EA, Hicks KA, Leighton JK, Szarfman A, Thompson AM, Harlow P. Qualification of cardiac troponins for nonclinical use: A regulatory perspective. Regul Toxicol Pharmacol. 2013;67(1):108-114. http://www.ncbi.nlm.nih.gov/pubmed/23876748disclaimer icon.

Goodsaid F, Mattes WB, eds. The Path from Biomarker Discovery to Regulatory Qualification. 1st ed. Cambridge, MA: Academic Press, 2013. http://www.sciencedirect.com/science/book/9780123914965disclaimer icon.

Woodcock J, Buckman S, Goodsaid F, Walton MK, Zineh I. Qualifying biomarkers for use in drug development: A U.S. Food and Drug Administration overview. Expert Opin Med Diagn. 2011;5(5):369-374. http://www.ncbi.nlm.nih.gov/pubmed/23484625disclaimer icon.

Dieterle F, Sistare F, Goodsaid F, et al. Renal biomarker qualification submission: A dialog between the FDA-EMEA and Predictive Safety Testing Consortium. Nat Biotechnol. 2010;28(5):455-462. http://www.ncbi.nlm.nih.gov/pubmed/20458315disclaimer icon.

Goodsaid F, Papaluca M. Evolution of biomarker qualification at the health authorities. Nat Biotechnol. 2010;28(5):441-443. http://www.nature.com/nbt/journal/v28/n5/full/nbt0510-441.htmldisclaimer icon.

Throckmorton DC. How a U.S. regulator can encourage new science. Clin Pharmacol Ther. 2009;86(5):471-472. http://www.ncbi.nlm.nih.gov/pubmed/19844222disclaimer icon.

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: 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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