FOM University of Applied Science for Economics & Management, Essen, Germany.
Children's Hospital, University Hospital, Cologne, Germany.
J Evid Based Med. 2019 Feb;12(1):9-15. doi: 10.1111/jebm.12340. Epub 2019 Jan 30.
Since January 2011, the Federal Joint Committee (FJC) conducts early benefit assessments (EBA) of newly approved pharmaceutical drugs compared to appropriate standard therapies. The FJC commissions the Institute for Quality and Efficiency in Healthcare (IQEH) to prepare preliminary reports. We aimed to evaluate the extent, impact, and reason for different judgments on added benefit of both institutions.
We searched EBA data on the FJC website and included completed procedures from 2011 to 2017. We conducted a quantitative analysis of the difference between FJC and IQEH on divergent judgments, a quantitative analysis of the impact of EBA on market withdrawal, and a qualitative analysis to identify potential factors contributing to divergent judgments.
FJC rated an added benefit in 30% (139 of 457) and IQEH in 22% (101 of 457) matching research questions (P = 0.004). In the aftermath of EBA, 28 pharmaceutical drugs were withdrawn from the German market. We identified three potential factors that might have contributed to the divergent judgments. IQEH used a unique threshold concept to define the rating, FJC conducted additional public hearings, and FJC showed more flexibility with adherence to stringent criteria and interpretation of results.
FJC and IQEH differed significantly in their early benefit assessment. In response to negative EBA decisions, pharmaceutical companies withdrew a considerable number of medicines from the German market. The present work uncovers the subjectivity and possible variance inherent in benefit assessment, as the two institutions observe the same rules of procedure.
自 2011 年 1 月以来，联邦联合委员会（FJC）对新批准的药物与适当的标准疗法进行早期效益评估（EBA）。FJC 委托医疗质量和效率研究所（IQEH）编写初步报告。我们旨在评估两个机构对附加效益的不同判断的程度、影响和原因。
我们在 FJC 网站上搜索 EBA 数据，并纳入了 2011 年至 2017 年完成的程序。我们对 FJC 和 IQEH 之间在分歧判断上的差异进行了定量分析，对 EBA 对市场撤出的影响进行了定量分析，并进行了定性分析，以确定导致分歧判断的潜在因素。
FJC 对 30%（457 项中的 139 项）和 IQEH 对 22%（457 项中的 101 项）的附加效益进行了评估，与研究问题相匹配（P=0.004）。在 EBA 之后，28 种药物从德国市场撤出。我们确定了三个可能导致分歧判断的潜在因素。IQEH 使用了一个独特的阈值概念来定义评估，FJC 进行了额外的公开听证会，并且 FJC 在遵守严格的标准和解释结果方面表现出更大的灵活性。
FJC 和 IQEH 在早期效益评估方面存在显著差异。由于 EBA 的负面决定，制药公司从德国市场撤出了相当数量的药品。本工作揭示了效益评估中固有的主观性和可能的差异，因为两个机构都遵守相同的程序规则。
