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Papers of the Week

Papers: 30 Jan 2021 - 5 Feb 2021

Pharmacology/Drug Development

2021 Jan-Feb

Pain Rep



Essential statistical principles of clinical trials of pain treatments.


Dworkin RH, Evans SR, Mbowe O, McDermott MP
Pain Rep. 2021 Jan-Feb; 6(1):e863.
PMID: 33521483.


This article presents an overview of fundamental statistical principles of clinical trials of pain treatments. Statistical considerations relevant to phase 2 proof of concept and phase 3 confirmatory randomized trials investigating efficacy and safety are discussed, including (1) research design; (2) endpoints and analyses; (3) sample size determination and statistical power; (4) missing data and trial estimands; (5) data monitoring and interim analyses; and (6) interpretation of results. Although clinical trials of pharmacologic treatments are emphasized, the key issues raised by these trials are also directly applicable to clinical trials of other types of treatments, including biologics, devices, nonpharmacologic therapies (eg, physical therapy and cognitive-behavior therapy), and complementary and integrative health interventions.