TY - JOUR
T1 - Designing and conducting proof-of-concept chronic pain analgesic clinical trials
AU - Campbell, Claudia M.
AU - Gilron, Ian
AU - Doshi, Tina
AU - Raja, Srinivasa
N1 - Publisher Copyright:
© 2019 The Author(s).
PY - 2019/5/1
Y1 - 2019/5/1
N2 - 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.
AB - 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.
KW - ACTTION
KW - Clinical trial
KW - Pain
KW - Proof-of-concept
KW - Quantitative sensory testing, Pain testing, Personalized medicine
UR - http://www.scopus.com/inward/record.url?scp=85071142821&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85071142821&partnerID=8YFLogxK
U2 - 10.1097/PR9.0000000000000697
DO - 10.1097/PR9.0000000000000697
M3 - Article
C2 - 31583338
AN - SCOPUS:85071142821
SN - 2471-2531
VL - 4
JO - Pain Reports
JF - Pain Reports
IS - 3
M1 - e697
ER -