Table 2_From clinical trials to informing clinical decision-making: a review of patient-reported outcomes in nononcology medicines approved by the European Medicines Agency (2018–2022).docx

IntroductionInformation about a medicine published in the Summary of Product Characteristics (SmPC) and the product’s package leaflet by the European Medicines Agency (EMA) is key to communicate its value to prescribers and patients. The aim of this study was to examine the inclusion of statements related to patient-reported outcomes (PROs) in these documents to communicate patients’ perspectives and experiences of new nononcology medicines.
MethodsNononcology therapeutic indications recommended for approval by the EMA between 2018–2022 were identified. The Public Assessment Report(s) (PAR), SmPC, and package leaflet published for each indication were examined. Information about the indication and characteristics relating to how the PROs were assessed in confirmatory studies was extracted.
ResultsMost nononcology therapeutic indications (n = 98/140, 70%) contained PRO trial data but less than 50% (n = 64/140, 46%) had PRO-related statements in the SmPC and/or package leaflet. Most statements described treatment benefit (n = 60/64, 94%). Statements were most likely to be included in the SmPC and/or package leaflet if supported by at least 1 randomized controlled trial (n = 52/71, 73%), the endpoint assessed patient-reported symptoms or symptom burden (n = 56/71, 79%), and/or the PRO(s) were assessed as a primary endpoint (n = 24/24, 100%).
DiscussionAlthough trial data pertaining to PROs are reviewed when evaluating nononcology drugs, shortfalls persist in the inclusion of PROs when describing treatment benefit in critical documents used to inform treatment decision-making.

引言
欧洲药品管理局（European Medicines Agency，EMA）发布的产品特性总结（Summary of Product Characteristics，SmPC）与药品说明书（package leaflet）中的信息，是向处方医师与患者传递药品价值的核心载体。本研究旨在考察上述两类文件中是否纳入与患者报告结局（patient-reported outcomes，PROs）相关的表述，以传递新型非抗肿瘤药物的患者视角与使用体验。

方法
本研究筛选确定了2018至2022年间欧洲药品管理局建议批准的非抗肿瘤治疗适应证。针对每一项适应证所发布的公共评估报告（Public Assessment Report，PAR）、产品特性总结及药品说明书均纳入考察范围，并提取与该适应证相关的信息，以及验证性临床试验中患者报告结局的评估特征。

结果
多数非抗肿瘤治疗适应证（n=98/140，70%）纳入了患者报告结局的试验数据，但仅不到半数（n=64/140，46%）在产品特性总结和/或药品说明书中包含与患者报告结局相关的表述。其中绝大多数表述用于说明治疗获益（n=60/64，94%）。若相关表述至少有1项随机对照试验作为支持依据（n=52/71，73%）、评估终点为患者报告症状或症状负荷（n=56/71，79%）、且/或患者报告结局被设为主要评估终点（n=24/24，100%），则其被纳入产品特性总结和/或药品说明书的概率显著更高。

讨论
尽管在评估非抗肿瘤药物时会审查与患者报告结局相关的试验数据，但在用于指导临床治疗决策的关键文件中，纳入患者报告结局相关内容的情况仍存在明显不足。