Focus On Biomarkers Research

Photo of a neurological response to a sensory stimulus.

NINDS Program Description

A biomarker is a defined characteristic that is used as an indicator of normal biological processes, pathogenic processes or responses to a therapeutic intervention.  Biomarkers are recognized as essential tools for the development of therapeutics, allowing improved and better informed clinical trial design through indicators of target engagement and those that enable patient stratification.  In addition, biomarkers can facilitate the evaluation of therapeutic intervention on disease progression or recurrence. Although biomarker discovery is a very active research area, few biomarkers have been sufficiently validated for use in clinical practice or clinical trials.

The NINDS biomarker program is focused on improving the quality and efficiency of neurotherapeutic clinical research toward Phase II and beyond by supporting rigorous biomarker validation.  To achieve this goal, the program will: 1) promote rigorous biomarker validation through cooperative agreement funding opportunities, 2) centralize information about existing NINDS and NIH biomarker sample and data repository resources and 3) facilitate the development of future resources focused on bridging the gaps in the biomarker development pipeline.

Biomarkers Development Process

Biomarker Development Process diagram

  • Only 13% of new drug approvals in 2017 were in neurology
  • Selection biomarkers improve the probability of advancement from Phase II-NDAby 18-21%
  • Only 5% of biomarker publications resulted in patented biomarkers in 2011

Resources and Tools

Brings together diverse partners around a common mission to develop promising biomarkers in order to help accelerate the delivery of successful new technologies, medicines and therapies for prevention, early detection, diagnosis and treatment of disease.

A searchable catalogue of nearly 500 profiles on research consortia. Includes information on mission, structure, data sharing, partners, ways to connect, and more.

Provides draft guidance to industry on enrichment strategies that can be used in clinical trials intended to support effectiveness and safety claims in new drug applications (NDAs) and biologics license applications (BLAs).

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. Developed by FDA and NIH to promote consistent use of biomarker terms and concepts, and thereby advance biomarker science.