Dworkin Robert H, Evans Scott R, Mbowe Omar, McDermott Michael P
Departments of Anesthesiology and Perioperative Medicine, Neurology, and Psychiatry, and Center for Health + Technology, University of Rochester School of Medicine and Dentistry, Rochester, NY, USA.
Department of Biostatistics and Bioinformatics and the Biostatistics Center, George, Washington University, Washington DC, USA.
Pain Rep. 2020 Dec 18;6(1):e863. doi: 10.1097/PR9.0000000000000863. eCollection 2021 Jan-Feb.
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.
本文概述了疼痛治疗临床试验的基本统计原则。讨论了与研究疗效和安全性的2期概念验证及3期确证性随机试验相关的统计考量，包括：（1）研究设计；（2）终点指标与分析；（3）样本量确定与统计效能；（4）缺失数据与试验估计值；（5）数据监测与期中分析；以及（6）结果解读。尽管重点强调了药物治疗的临床试验，但这些试验所提出的关键问题也直接适用于其他类型治疗的临床试验，包括生物制品、器械、非药物疗法（如物理治疗和认知行为疗法）以及补充和综合健康干预措施。
