Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, 20993, MD, USA.
Neurotherapeutics. 2020 Jul;17(3):770-773. doi: 10.1007/s13311-020-00906-6.
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，药物疗效证明的监管标准是充分且良好对照临床试验中临床获益的证据。了解疾病的自然史以及治疗预期如何改变其进程，并从相关利益相关者（如患者、护理人员和临床医生）收集意见，对于理解如何在临床试验中衡量临床获益至关重要。尽管疼痛强度一直是疼痛临床试验的主要结果测量指标，但一系列从多个角度评估临床结果的措施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，以及哪种或哪种组合的结果评估可能需要提供更全面的患者体验视图。在药物产品开发的早期阶段，与监管机构进行接触，讨论潜在的临床试验设计和结果测量策略方法是一个重要机会。
