In the USA, the regulatory standard for demonstration of efficacy of a drug is evidence of clinical benefit from adequate and well-controlled clinical trials. Understanding the natural history of disease and how treatment is expected to alter its course, and gathering input from relevant stakeholders, such as patients, caregivers, and clinicians, is essential to understand the best way to measure clinical benefit in a clinical trial. Though pain intensity has been the primary outcome measure in clinical trials for pain, an array of measures assessing clinical outcomes from multiple perspectives can allow for more comprehensive interpretation of how a treatment affects patients’ lives. Careful consideration should be given to how pain affects the feeling and functioning of each distinct patient population and which outcome assessment, or combination of outcome assessments, may be necessary to provide a more comprehensive view of the patient experience. The early stages of medical product development are an important opportunity to engage with regulatory agencies to discuss potential approaches to clinical trial design and outcome measurement strategies.

在美国，证明药物疗效的监管标准是充分和控制良好的临床试验临床获益的证据。了解疾病的自然史以及治疗预期如何改变其病程，并收集患者、护理人员和临床医生等相关利益相关者的意见，对于了解在临床试验中衡量临床获益的最佳方法至关重要。尽管疼痛强度一直是疼痛临床试验的主要结果衡量标准，但从多个角度评估临床结果的一系列衡量标准可以更全面地解释治疗如何影响患者的生活。应仔细考虑疼痛如何影响每个不同患者群体的感觉和功能，以及哪些结果评估，或结果评估的组合，可能需要提供更全面的患者体验视图。医疗产品开发的早期阶段是与监管机构合作讨论临床试验设计和结果测量策略的潜在方法的重要机会。