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Qual Life Res. 2013 Apr;22(3):475-83. doi: 10.1007/s11136-012-0175-x. Epub 2012 Apr 17.
Interpretation guidelines are needed for patient-reported outcome (PRO) measures' change scores to evaluate efficacy of an intervention and to communicate PRO results to regulators, patients, physicians, and providers. The 2009 Food and Drug Administration (FDA) Guidance for Industry Patient-Reported Outcomes (PRO) Measures: Use in Medical Product Development to Support Labeling Claims (hereafter referred to as the final FDA PRO Guidance) provides some recommendations for the interpretation of change in PRO scores as evidence of treatment efficacy.
This article reviews the evolution of the methods and the terminology used to describe and aid in the communication of meaningful PRO change score thresholds.
Anchor- and distribution-based methods have played important roles, and the FDA has recently stressed the importance of cross-sectional patient global assessments of concept as anchor-based methods for estimation of the responder definition, which describes an individual-level treatment benefit. The final FDA PRO Guidance proposes the cumulative distribution function (CDF) of responses as a useful method to depict the effect of treatments across the study population.
While CDFs serve an important role, they should not be a replacement for the careful investigation of a PRO's relevant responder definition using anchor-based methods and providing stakeholders with a relevant threshold for the interpretation of change over time.
需要为患者报告的结局（PRO）测量的变化分数提供解释指南，以评估干预措施的疗效，并向监管机构、患者、医生和提供者传达 PRO 结果。2009 年食品和药物管理局（FDA）关于行业患者报告的结局（PRO）措施的指南：在医疗产品开发中使用以支持标签声明（以下简称最终 FDA PRO 指南）提供了一些关于 PRO 评分变化解释为治疗效果证据的建议。
本文回顾了用于描述和帮助传达有意义的 PRO 变化评分阈值的方法和术语的演变。
基于锚点和分布的方法发挥了重要作用，FDA 最近强调了概念的横向患者整体评估作为基于锚点的方法对反应者定义估计的重要性，反应者定义描述了个体治疗获益。最终 FDA PRO 指南提出了响应的累积分布函数（CDF）作为一种有用的方法来描绘治疗对研究人群的影响。
虽然 CDF 具有重要作用，但它们不应该替代使用基于锚点的方法对 PRO 的相关反应者定义进行仔细调查，并为利益相关者提供随时间变化的解释的相关阈值。
