NIH Workshop: Discovery and Validation of Biomarkers

November 14, 2018 - November 15, 2018

Contact: Mary Ann Pelleymounter
Contact Number: 301-451-4551
Contact Email:

Marriott Wardman-Park
2660 Woodley Rd. NW
Washington, DC 20008

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The NIH Workshop: Discovery and Validation of Biomarkers to Develop Non-Addictive Therapeutics for Pain is focused on the discovery and validation of biomarkers and endpoints that will ultimately facilitate the development of safer therapies for people with pain.  It is part of the trans-NIH HEAL (Helping to End Addiction Long-term) initiative and is a collaborative effort between NINDS, NCI, NIAAA, NIBIB, NIDCR, NIDA, NIGMS, NINR, NCCIH and OBSSR.  The intent of the workshop is to:

  1. inform the community about current regulatory standards and guidelines for the development of biomarkers and endpoints,
  2. evaluate the state of the science in pain biomarker development
  3. explore potential scientific and collaborative approaches that could facilitate the discovery and validation of robust biomarkers and endpoints that would provide the tools necessary for the diagnosis and treatment of pain conditions.  


Related Resources

Event PresentationEvent ProgramAgenda


The intent of the workshop was to discuss challenges and opportunities in the development of biomarkers for pain.


The use of objective biomarkers and endpoints throughout the drug discovery and development process is critical because biomarkers and endpoints can help define pathophysiologic subsets of pain, evaluate target engagement of new drugs, and predict the analgesic efficacy of new drugs.

The Discovery and Validation of Biomarkers to Develop Non-Addictive Therapeutics for Pain workshop convened scientific leaders from academia, industry, government, and patient advocacy groups to discuss progress, challenges, gaps, and ideas focused on facilitating the development of biomarkers and endpoints for pain.

Summary of Meeting Discussion

Participants agreed that biomarkers, biomarker signatures, and better clinical endpoints have the potential to transform the landscape of non-addictive therapeutic development and treatment plan by providing tools that can: 1) predict treatment response, 2) indicate therapeutic target engagement, 3) provide prognostic indicators of transition to chronic pain, and 4) stratify patients into more homogenous cohorts in a clinical trial. Industry participants emphasized the importance of rigorously validated biomarkers as decision-making tools in clinical trials.

Participants provided guidance regarding the definitions and best practices for biomarker development.  It was agreed that the biopsychosocial dimensions of pain and variability across individuals should be incorporated in the development of pain biomarkers.  Further, composite or signature biomarkers and endpoints will likely continue to gain widespread acceptance due to the complexity of pain and its perception.  There are many examples of new and exciting biomarker detection technologies, particularly those that provide functional representations of neural circuits that predict transition to pain or reflect abnormal sensitivity.  Finally, ethical and societal considerations regarding the impact of imaging and other types of biomarkers were discussed.


Although there has been considerable progress in the development of biomarkers for pain, the field is still in its early stages.  Technical and scientific challenges in both the discovery and validation phases have slowed the advancement toward widespread clinical use.  However, advancements in detection technologies and “deconvolution” methods such as machine learning and statistical design have paved the way toward improved accuracy, precision and feasibility of biomarkers or endpoints for use in clinical trials and clinical practice.  In addition, the significant increase in funding and resources for biomarker development and non-addictive therapeutic development as a result of initiatives such as HEAL should stimulate research and technology development in this area of high unmet medical need.

Videocast: (Days 1 and 2)