Patient-Reported Outcomes, RTI Health Solutions, Research Triangle Park, NC, USA.
Value Health. 2013 Dec;16(8):1150-5. doi: 10.1016/j.jval.2013.08.2293. Epub 2013 Oct 17.
The US Food and Drug Administration (FDA) provides formal guidance for the use of patient-reported outcomes (PROs) in support of labeling claims, whereas the European Medicines Agency (EMA) offers insight in a reflection paper relating to health-related quality of life in lieu of formal guidance.
PRO label claims granted for new molecular entities and biologic license applications from 2006 through 2010 were reviewed to evaluate consistencies and discrepancies in PRO label claims granted by the FDA and the EMA and to highlight trends in the acceptance of PRO claims across agencies.
Products approved by both the FDA and the EMA were identified. By using US Drug Approval Packages and European Public Assessment Reports packages, any PRO label claims made for the same product by the same company were compared.
Both agencies approved a total of 75 products. Of these, 35 (47%) had at least one EMA-granted PRO label claim compared with 14 (19%) by the FDA. Most FDA-grated claims focused on symptoms; however, EMA-granted claims were more likely to include higher order concepts. Few (~12%) were granted the same label claims. Despite this discordance between the two agencies, where PRO label claims were granted by both the FDA and the EMA, there was similarity in the type of label claim.
The EMA is more likely than the FDA to grant PRO claims and for higher order constructs. On a macro level, there appears to be poor concordance between claims granted by both agencies. On close examination, however, there appears to be greater concordance than previously recognized, which may be instructive in formulating future PRO strategies. Further research to create strategic alignment across agencies may be beneficial.
美国食品和药物管理局（FDA）提供了使用患者报告结果（PROs）的正式指导，以支持标签声明，而欧洲药品管理局（EMA）则在一份关于健康相关生活质量的反思文件中提供了见解，而没有提供正式指导。
审查了 2006 年至 2010 年期间新分子实体和生物许可申请获得的 PRO 标签声明，以评估 FDA 和 EMA 授予的 PRO 标签声明的一致性和差异，并强调各机构对 PRO 声明的接受趋势。
确定了同时获得 FDA 和 EMA 批准的产品。通过使用美国药物批准包和欧洲公共评估报告包，比较了同一公司为同一产品提出的任何 PRO 标签声明。
两个机构共批准了 75 种产品。其中，35 种（47%）至少有一种 EMA 授予的 PRO 标签声明，而 FDA 授予的则为 14 种（19%）。大多数 FDA 授予的声明都集中在症状上；然而，EMA 授予的声明更有可能包括更高阶的概念。只有少数（约 12%）获得了相同的标签声明。尽管这两个机构之间存在这种不一致性，但 PRO 标签声明同时获得了 FDA 和 EMA 的批准，它们的标签声明类型仍具有相似性。
EMA 比 FDA 更有可能授予 PRO 声明和更高阶的概念。在宏观层面上，两个机构授予的声明之间似乎存在较差的一致性。然而，经过仔细审查，似乎存在比以前认识到的更高的一致性，这可能对制定未来的 PRO 策略具有指导意义。进一步的研究以在各机构之间建立战略一致性可能是有益的。
