Assessment of PRO Label Claims Granted by the FDA as Compared to the EMA (2006-2010)
Assessment of PRO Label Claims Granted by the FDA as Compared to the EMA (2006-2010)
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DOI：
10.1016/j.jval.2013.08.2293
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发表时间：
2013-12-01
期刊：
影响因子：
4.5
通讯作者：
Gnanasakthy, Ari
中科院分区：
文献类型：
--
作者：
DeMuro, Carla;Clark, Marci;Gnanasakthy, Ari
背景资料：美国食品药品监督管理局（FDA）提供了使用患者报告结局（PRO）支持标签声明的正式指南，而欧洲药品管理局（EMA）在与健康相关生活质量相关的反思文件中提供了见解，而不是正式指南。目的：审查了2006年至2010年期间批准的新分子实体和生物许可申请的PRO标签声明，以评价FDA和EMA批准的PRO标签声明的差异和差异，并强调各机构接受PRO声明的趋势。方法：确定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，比较了同一公司对同一产品的任何PRO标签声明。结果：两个机构共批准了75种产品。其中，35（47%）至少有一个EMA批准的PRO标签声明，而FDA批准的只有14（19%）。大多数FDA认证的索赔集中在症状上;然而，EMA批准的索赔更可能包括更高阶的概念。只有少数人（约12%）获得了相同的标签声明。尽管这两个机构之间存在不一致，但FDA和EMA均批准了PRO标签声明，但标签声明类型相似。结论：EMA比FDA更有可能批准PRO声明和高阶结构。在宏观层面上，两个机构批准的索赔之间似乎不太一致。然而，在仔细检查，似乎有更大的一致性比以前认识到，这可能是有益的，在制定未来的PRO战略。进一步研究，以建立跨机构的战略一致性可能是有益的。
Background: 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. Objectives: 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. Methods: 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. Results: 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 (similar to 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. Conclusions: 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.