Discovery and validation of biomarkers to aid the development of safe and effective pain therapeutics: challenges and opportunities

Nat Rev Neurol. 2020 Jul;16(7):381-400. doi: 10.1038/s41582-020-0362-2. Epub 2020 Jun 15.


Pain medication plays an important role in the treatment of acute and chronic pain conditions, but some drugs, opioids in particular, have been overprescribed or prescribed without adequate safeguards, leading to an alarming rise in medication-related overdose deaths. The NIH Helping to End Addiction Long-term (HEAL) Initiative is a trans-agency effort to provide scientific solutions to stem the opioid crisis. One component of the initiative is to support biomarker discovery and rigorous validation in collaboration with industry leaders to accelerate high-quality clinical research into neurotherapeutics and pain. The use of objective biomarkers and clinical trial end points throughout the drug discovery and development process is crucial to help define pathophysiological subsets of pain, evaluate target engagement of new drugs and predict the analgesic efficacy of new drugs. In 2018, the NIH-led 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 to facilitate the development of biomarkers and end points for pain. The outcomes of this workshop are outlined in this Consensus Statement.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Analgesics, Opioid / adverse effects
  • Biomarkers / blood
  • Chronic Pain / blood*
  • Chronic Pain / diagnostic imaging*
  • Chronic Pain / genetics
  • Chronic Pain / therapy
  • Education / methods
  • Education / trends
  • Humans
  • National Institutes of Health (U.S.) / trends*
  • Neuroimaging / methods
  • Opioid Epidemic / prevention & control
  • Opioid Epidemic / trends
  • Opioid-Related Disorders / blood
  • Opioid-Related Disorders / diagnostic imaging
  • Opioid-Related Disorders / genetics
  • Opioid-Related Disorders / therapy
  • Pain Management / methods*
  • Pain Management / trends*
  • Treatment Outcome
  • United States


  • Analgesics, Opioid
  • Biomarkers