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  6. Focus Area: Biomarkers
  1. Focus Areas of Regulatory Science Report

Focus Area: Biomarkers

FDA scientist Anjuli Jain conducts an experiment validating nonclinical biomarker models for traumatic brain injury. This research could help establish a regulatory pathway for flexible EEG technologies to rapidly detect brain injury during emergencies.
Apolipoprotein D, a protein mainly produced in the brain and testes. A biomarker of androgen insensitivity syndrome. 3d rendering.

Importance to FDA

Biomarkers are characteristics that are objectively measured as indicators of health, disease, or a response to an exposure or intervention, including therapeutic interventions. Biomarkers are useful to medical product developers and FDA for uses such as:

  • Identification of a biomarker that may lead to improved diagnosis of a disease or predict future disease severity or outcomes. Examples include blood pressure measurements as an indicator of cardiovascular risks, or measurements of blood sugar in diabetes.
  • Identification of biomarkers that may lead to improved understanding of the impact to tobacco products.
  • Biomarkers can be used to identify and evaluate the effectiveness of medical or veterinary treatments or devices, monitor the safety of a therapy, and evaluates whether a treatment is having the desired effect on the human or animal body.
  • Biomarkers can play a critical role in bridging non-clinical results to clinical research and identifying patient populations susceptible to adverse effects of or those responsive to medical treatments (also known as Precision Medicine.)

FDA and medical product developers are interested in developing novel biomarkers to use in product development and/or evaluation to improve accuracy and efficiency of clinical trials. FDA has a biomarker qualification program for new biomarkers aimed to enable understanding of how a biomarker may be applied in a specific context of use. Qualified biomarkers have the potential to provide valuable information that may promote innovation and reduce uncertainty in regulatory decisions during drug development. When a biomarker is qualified, it means that the biomarker has undergone a formal regulatory process to ensure that FDA can rely on it to have a specific interpretation and application in medical product development and regulatory review, within the stated context of use. It is important to note that the biomarker can be qualified and not the biomarker measurement method.

The Biomarkers, EndpointS, and other Tools resource is a living document developed jointly by FDA and the National Institutes of Health (NIH) to promote consistent use of biomarker terms and concepts, and thereby advance biomarker science. This resource clarifies terminology and uses of biomarkers and endpoints as they pertain to the progression from basic biomedical research to medical product development to clinical care.


FDA research is advancing the development of novel biomarkers:

  • Identifying biomarkers of tobacco exposure such as 4-(methylnitrosamino)-1-(3-pyridyl)-1-butanol (NNAL) that are informative for assessing potential health risks of tobacco products in the review of pre-market and modified risk tobacco product applications.
  • Identifying and evaluating immune correlates of protection for many pathogens of public health concern. Immune correlates of protection serve as a biomarker of effectiveness for vaccines.
  • Identifying more sensitive markers of drug-induced cardiac (heart) injury and liver injury, as well as developing in vitro and in vivo tests for assessment of treatment-induced genetic damage.
  • Exploring biomarkers to assess patient reactions to metal implants and susceptibility to hypersensitive and chronic immune responses to promote device safety.
  • Collaborating in a number of studies, including but not limited to, research in diagnostic biomarkers of traumatic brain injury and kidney injury, and biomarkers of tobacco exposure and potential harm as documented in the FDA-NIH Population Assessment of Tobacco and Health Study.
  • Analyzing new measures of tumor response and progression developed specifically for cancer immunotherapies (e.g., iRECIST) through pooled analyses of oncology clinical trial data submitted to FDA. These new measures of tumor response were designed to capture atypical responses seen with immunotherapies (e.g., an initial increase in tumor size followed by a clinically important reduction in tumor burden or initial reduction in tumor size with the appearance of new lesions that subsequently regress), but there is limited evidence to support whether the new measures better assess patient outcomes.
  • FDA qualified total kidney volume (TKV) as a prognostic biomarker to predict the rate of loss of kidney function in the orphan disease autosomal dominant polycystic kidney disease. Subsequently, the clinical review division accepted TKV as a reasonably likely surrogate endpoint for drug approval under accelerated approval thereby substantially shortening the required duration and size of Phase 3 trials.




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