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.
Reference Type
Journal Article
Periodical Full
Pain reports
Publication Year
2021
Publication Date
Jan
Volume
6
Issue
1
Other Pages
e863
Publisher
Wolters Kluwer
Place of Publication
United States
ISSN/ISBN
2471-2531
Document Object Index
10.1097/PR9.0000000000000863
URL
https://www.ncbi.nlm.nih.gov/pubmed/33521483
PMID
33521483