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Regulatory Focus™ > News Articles > FDA Offers Biomarker Qualification Case Studies

FDA Offers Biomarker Qualification Case Studies

Posted 14 June 2017 | By Zachary Brennan 

FDA Offers Biomarker Qualification Case Studies

As part of an educational series on qualifying biomarkers for use in drug development, the US Food and Drug Administration (FDA) has released two fictitious examples of how biomarkers can improve the drug development process and how FDA works with researchers, pharmaceutical companies and patient advocates to qualify biomarkers.

One follows a researcher who discovers a promising biomarker of drug-induced kidney injury in rats and explores how he partnered with researchers and engaged with FDA early in the biomarker development process to get the data to support biomarker qualification, while the other deals with a patient advocate who works with other patient advocates, researchers and FDA to qualify a biomarker that will make clinical trials on new osteoporosis drugs more efficient. 


FDA defines a biomarker as a physiologic, molecular, histologic or radiographic characteristic or measurement that is “an indicator of normal biological processes, pathologic processes, or responses to an exposure or intervention, including therapeutic interventions.”

Measured in patients before treatment, biomarkers can be used to select patients for a clinical trial and for dose selection. And changes in biomarkers following treatment may predict or identify safety problems related to a candidate drug, reveal a pharmacological activity or indicate clinical benefit.

FDA notes seven different categories of biomarkers:

  • Diagnostic biomarker — “used to detect or confirm presence of a disease or condition of interest or to identify individuals with a subtype of the disease.”
  • Monitoring biomarker — “measured serially for assessing status of a disease or medical condition or for evidence of exposure to (or effect of) a medical product or an environmental agent.”
  • Pharmacodynamic/response biomarker— “used to show that a biological response has occurred in an individual who has been exposed to a medical product or an environmental agent.”
  • Predictive biomarker — “used to identify individuals who are more likely than similar individuals without the biomarker to experience a favorable or unfavorable effect from exposure to a medical product or an environmental agent.”
  • Prognostic biomarker — “used to identify likelihood of a clinical event, disease recurrence or progression in patients who have the disease or medical condition of interest.”
  • Safety Biomarker — “measured before or after an exposure to a medical product or an environmental agent to indicate the likelihood, presence, or extent of toxicity as an adverse effect.”
  • Susceptibility/risk biomarker — “indicates the potential for developing a disease or medical condition in an individual who does not currently have clinically apparent disease or the medical condition.”

Case Studies

The examples from FDA break down the two pathways for biomarker integration in drug development through CDER, critical path innovation meetings and outcomes, and other details intended to help companies better understand the process.

They also aim to help further an understanding of the validation studies necessary to support the qualification of a biomarker and the collaborative efforts and benefits of biomarker qualification.

CDER Biomarker Qualification Case Studies

Biomarker Qualification Program (BQP) Education and Training

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