RTI Health Solutions, Research Triangle Park, NC, USA.
RTI Health Solutions, Research Triangle Park, NC, USA.
Value Health. 2019 Feb;22(2):203-209. doi: 10.1016/j.jval.2018.09.2842.
To compare US Food and Drug Administration (FDA) and European Medicines Agency (EMA) labeling for evidence based on patient-reported outcomes (PROs) of new oncology treatments approved by both agencies.
Oncology drugs and indications approved between 2012 and 2016 by both the FDA and the EMA were identified. PRO-related language and analysis reported in US product labels and drug approval packages and EMA summaries of product characteristics were compared for each indication.
In total, 49 oncology drugs were approved for a total of 64 indications. Of the 64 indications, 45 (70.3%) included PRO data in either regulatory submission. No FDA PRO labeling was identified. PRO language was included in the summary of product characteristics for 21 (46.7%) of 45 indications. European Organisation for Research and Treatment of Cancer and Functional Assessment of Cancer Therapy measures were used frequently in submissions. FDA's comments suggest that aspects of study design (eg, open labels) or the validity of PRO measures was the primary reason for the lack of labeling based on PRO endpoints. Both agencies identified missing PRO data as problematic for interpretation.
During this time period, the FDA and the EMA used different evidentiary standards to assess PRO data from oncology studies, with the EMA more likely to accept data from open-label studies and broad concepts such as health-related quality of life. An understanding of the key differences between the agencies may guide sponsor PRO strategy when pursuing labeling. Patient-focused proximal concepts are more likely than distal concepts to receive positive reviews.
比较美国食品和药物管理局（FDA）和欧洲药品管理局（EMA）对经双方批准的新肿瘤治疗药物基于患者报告结局（PROs）的证据标签，以评估二者的差异。
确定了 FDA 和 EMA 在 2012 年至 2016 年间批准的肿瘤药物和适应证。比较了每个适应证的美国产品标签和药物批准文件包以及 EMA 产品特性摘要中报告的 PRO 相关语言和分析。
共有 49 种肿瘤药物被批准用于 64 种适应证。在这 64 种适应证中，有 45 种（70.3%）在监管申报中包含 PRO 数据。未发现 FDA 的 PRO 标签。21 种适应证（46.7%）的产品特性摘要中包含 PRO 语言。欧洲癌症研究与治疗组织和癌症治疗功能评估量表在提交材料中经常被使用。FDA 的意见表明，研究设计方面的某些因素（如开放标签）或 PRO 措施的有效性是未基于 PRO 终点进行标签的主要原因。两个机构都认为 PRO 数据缺失会影响对结果的解释。
在这段时间内，FDA 和 EMA 采用了不同的证据标准来评估肿瘤研究的 PRO 数据，EMA 更有可能接受来自开放标签研究和广泛概念（如健康相关生活质量）的数据。了解机构之间的关键差异可能有助于申办者在追求标签时制定 PRO 策略。与远端概念相比，以患者为中心的近端概念更有可能获得积极的评价。
