基于欧洲药品管理局（2017-2021 年）批准的新型肿瘤药物的患者报告结局终点的标签评价综述。
A Review of Labeling Based on Patient-Reported Outcome Endpoints for New Oncology Drugs Approved by the European Medicines Agency (2017-2021).
机构信息
RTI Health Solutions, Research Triangle Park, NC, USA.
RTI Health Solutions, Manchester, England, UK.
出版信息
Value Health. 2023 Jun;26(6):893-901. doi: 10.1016/j.jval.2023.01.017. Epub 2023 Feb 4.
OBJECTIVE
A review of new oncology indications approved by the European Medicines Agency (EMA) for 2012-2016 showed that 33% of new drugs had labeling based on patient-reported outcomes (PROs). We reviewed labeling text based on PRO endpoints for new oncology indications approved during 2017-2021.
METHODS
New oncology drugs approved by EMA to treat indications of cancers during 2017-2021 were identified from the EMA website. PRO-related language reported in EMA summaries of product characteristics (SmPCs) were summarized and compared with similar findings reported for oncology indications approved during 2012-2016.
RESULTS
Review documents by the EMA during 2017-2021 were available for 49 new oncology drugs for 70 cancer indications. Submissions for 52 (74.3%) of the 70 indications included PRO data for EMA review. Of all submissions, 14 (20.0%) approvals contained PRO-related language in the SmPC. Broad concepts such as health-related quality of life were most common and found in 8 of 14 (57.1%) PRO-related labels.
CONCLUSION
PRO-related language appeared in SmPCs for 20% of all indications of new oncology drugs approved by EMA during 2017-2021 compared with approximately 33% of EMA approvals during 2012-2016. PRO-related labeling during the same periods showed a greater decline (from 47% to 27%) for indications of new oncology drugs that also included PRO data. One possible reason for this decline may be the increase in open-label studies from 62% between 2012 and 2016 to approximately 79% between 2017 and 2021.
目的
对 2012-2016 年欧洲药品管理局（EMA）批准的新肿瘤适应证进行审查后发现，33%的新药标签基于患者报告结局（PROs）。我们对 2017-2021 年批准的新肿瘤适应证的基于 PRO 终点的标签文本进行了审查。
方法
从 EMA 网站上确定了 2017-2021 年 EMA 批准用于治疗癌症适应证的新肿瘤药物。总结并比较了 EMA 产品特性摘要（SmPC）中报告的与 PRO 相关的语言，以及 2012-2016 年批准的肿瘤适应证的类似发现。
结果
2017-2021 年 EMA 审查的 70 种癌症适应证的 49 种新肿瘤药物的审查文件均可获得。70 个适应证中的 52 个（74.3%）提交的内容包括 EMA 审查的 PRO 数据。在所有提交的内容中，14 个（20.0%）批准的 SmPC 中包含与 PRO 相关的语言。最常见的是健康相关生活质量等广泛概念，在 14 个（57.1%）与 PRO 相关的标签中均有发现。
结论
与 2012-2016 年 EMA 批准的新肿瘤适应证中约 33%的 SmPC 相比，2017-2021 年 EMA 批准的所有新肿瘤适应证中，与 PRO 相关的语言出现在 SmPC 中的比例为 20%。在同一时期，对于同样包含 PRO 数据的新肿瘤药物适应证，与 PRO 相关的标签比例从 47%下降到 27%。出现这种下降的一个可能原因是，从 2012 年到 2016 年，开放标签研究从 62%增加到了 2017 年到 2021 年的约 79%。
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