Randomized clinical trials are critical for evaluating the safety and efficacy of interventions in oncology and informing regulatory decisions, practice guidelines, and health policy. Patient-reported outcomes (PROs) are increasingly used in randomized trials to reflect the impact of receiving cancer therapies from the patient perspective and can inform evaluations of interventions by providing evidence that cannot be obtained or deduced from clinicians’ reports or from other biomedical measures. This commentary focuses on how PROs add value to clinical trials by representing the patient voice. We employed two previously published descriptive frameworks (addressing how PROs are used in clinical trials and how PROs have an impact, respectively) and selected nine clinical trial publications that illustrate the value of PROs according to the framework categories. These include three trials where PROs were a primary trial endpoint, three trials where PROs as secondary endpoints supported the primary endpoint, and three trials where PROs as secondary endpoints contrast the primary endpoint findings in clinically important ways. The nine examples illustrate that PROs add valuable data to the care and treatment context by informing future patients about how they may feel and function on different treatments and by providing clinicians with evidence to support changes to clinical practice and shared decision-making. Beyond the patient and clinician, PROs can enable administrators to consider the cost-effectiveness of implementing new interventions, and contribute vital information to policy-makers, health technology assessors, and regulators. These examples provide a strong case for the wider implementation of PROs in cancer trials.

中文翻译：

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倾听患者的声音为癌症临床试验增加价值

随机临床试验对于评估肿瘤学干预措施的安全性和有效性以及为监管决策、实践指南和卫生政策提供信息至关重要。患者报告的结果（PRO）越来越多地用于随机试验，以从患者的角度反映接受癌症治疗的影响，并可以通过提供无法从临床医生报告或其他生物医学措施中获得或推断的证据来为干预措施的评估提供信息。本评论重点关注 PRO 如何通过代表患者的声音为临床试验增加价值。我们采用了两个先前发布的描述性框架（分别说明 PRO 在临床试验中的使用方式以及 PRO 如何产生影响），并根据框架类别选择了九份临床试验出版物来说明 PRO 的价值。其中包括三项以 PRO 为主要试验终点的试验、三项以 PRO 作为次要终点支持主要终点的试验、以及三项以 PRO 作为次要终点以临床重要方式对比主要终点结果的试验。这九个例子表明，PRO 通过告知未来患者他们在不同治疗中的感受和功能，以及为临床医生提供证据来支持临床实践和共同决策的改变，为护理和治疗环境添加了有价值的数据。除了患者和临床医生之外，PRO 还可以使管理者考虑实施新干预措施的成本效益，并为政策制定者、卫生技术评估者和监管者提供重要信息。这些例子为在癌症试验中更广泛地实施 PRO 提供了强有力的案例。