Pain Research and Nuffield Division of Anaesthetics, Nuffield Department of Clinical Neurology, University of Oxford, The Churchill, Oxford OX3 7LE, UK.
Pain. 2013 Dec;154 Suppl 1:S77-S86. doi: 10.1016/j.pain.2013.03.024. Epub 2013 Mar 15.
There has been a tension between the needs of regulators and industry to demonstrate that interventions are effective and safe, and the needs of professionals to understand how well interventions will work for their patients, and patients to understand what might work for them as individuals. The custom has been to focus on statistical outcomes based on average results, but in-depth analysis based on outcomes obtained by individual patients demonstrates that few are average. Rather, a minority of patients achieve very large reductions in pain (responders), while the majority achieve little (nonresponders). Those who benefit in terms of pain also benefit in other areas, with improved sleep, fatigue, mood, function, quality of life, and ability to work. This changes how benefit and risk are seen; nonresponders should stop treatments that don't work and not, therefore, be exposed to risks, while responders have very large benefits to offset against rare but potentially serious harm. This alternative view, patient-centred and practice-orientated, has major implications for clinical practice, how and why we do clinical trials and how they are designed, how health economic evaluations are done, for decisions made by regulatory and other bodies, and for the theory and practice of evidence-based medicine.
监管机构和行业需要证明干预措施是有效和安全的，而专业人士需要了解干预措施对患者的效果如何，患者需要了解哪些措施对他们个人有效，这两者之间一直存在紧张关系。传统上，我们关注的是基于平均结果的统计结果，但基于个别患者获得的结果进行深入分析表明，很少有平均值。相反，只有少数患者的疼痛有很大程度的减轻（应答者），而大多数患者的疼痛减轻程度很小（无应答者）。那些在疼痛方面受益的人在其他方面也受益，睡眠、疲劳、情绪、功能、生活质量和工作能力得到改善。这改变了受益和风险的看法；无应答者应停止无效的治疗，因此不会面临风险，而应答者则可以获得很大的益处，以抵消罕见但潜在严重的伤害。这种以患者为中心、注重实践的观点对临床实践、我们进行临床试验的方式和原因以及临床试验的设计、如何进行健康经济评估、监管机构和其他机构的决策以及循证医学的理论和实践都有重大影响。
