Designing and conducting proof-of-concept chronic pain analgesic clinical trials

Claudia M. Campbell, Ian Gilron, Tina Doshi, Srinivasa Raja

Research output: Contribution to journalArticlepeer-review

4 Scopus citations


Introduction:The evolution of pain treatment is dependent on successful development and testing of interventions. Proof-of-concept (POC) studies bridge the gap between identification of a novel target and evaluation of the candidate intervention's efficacy within a pain model or the intended clinical pain population.Methods:This narrative review describes and evaluates clinical trial phases, specific POC pain trials, and approaches to patient profiling.Results:We describe common POC trial designs and their value and challenges, a mechanism-based approach, and statistical issues for consideration.Conclusion:Proof-of-concept trials provide initial evidence for target use in a specific population, the most appropriate dosing strategy, and duration of treatment. A significant goal in designing an informative and efficient POC study is to ensure that the study is safe and sufficiently sensitive to detect a preliminary efficacy signal (ie, a potentially valuable therapy). Proof-of-concept studies help avoid resources wasted on targets/molecules that are not likely to succeed. As such, the design of a successful POC trial requires careful consideration of the research objective, patient population, the particular intervention, and outcome(s) of interest. These trials provide the basis for future, larger-scale studies confirming efficacy, tolerability, side effects, and other associated risks.

Original languageEnglish (US)
Article numbere697
JournalPain Reports
Issue number3
StatePublished - May 1 2019


  • Clinical trial
  • Pain
  • Proof-of-concept
  • Quantitative sensory testing, Pain testing, Personalized medicine

ASJC Scopus subject areas

  • Anesthesiology and Pain Medicine


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