Department of Health Services Research and Policy, London School of Hygiene and Tropical Medicine, London, England, UK.
Department of Health Services Research and Policy, London School of Hygiene and Tropical Medicine, London, England, UK.
Value Health. 2024 Dec;27(12):1662-1670. doi: 10.1016/j.jval.2024.07.012. Epub 2024 Jul 31.
Evidence on reappraisals of health technologies in Germany is limited, and for rare disease treatments (RDTs), the Federal Joint Committee follows different processes (limited or regular), depending on whether an annual revenue threshold has been exceeded. Our objective is to better understand (re)appraisal processes and their outcomes for RDTs in Germany.
We analyzed appraisal documents of 55 RDT indications for which an initial appraisal and a reappraisal were conducted between 2011 and 2023. We extracted information for the type of evidence, the risk of bias, the availability of additional evidence, and the change in the maturity of survival data as proxies for evidence quality. Specifically, we reviewed the reasons for conducting reappraisals, examined how evidence quality and the clinical benefit rating (CBR) differed between initial appraisals and reappraisals, and explored the association between evidence quality and (1) the CBR and (2) the change in the CBR after reappraisal.
Most reappraisals were conducted because the annual revenue threshold was exceeded or the initial appraisal resolution was time limited. Almost all initial appraisals used the limited process, whereas the majority of reappraisals used the regular process. The CBR increased in only 9 and decreased in 21 of 55 reappraisals. There was some evidence that reappraisals with an accepted randomized controlled trial were significantly more likely to achieve a higher CBR.
Findings confirmed that reasons and processes for conducting reappraisals of RDTs in Germany differ. Further, high CBRs in reappraisals were not common and evidence quality in initial appraisals and reappraisals was limited.
德国关于卫生技术再评价的证据有限，对于罕见病治疗（RDT），联邦联合委员会根据是否超过年度收入门槛，采用不同的程序（有限或常规）。我们的目标是更好地了解德国 RDT 的（再）评价过程及其结果。
我们分析了 2011 年至 2023 年期间对 55 种 RDT 适应证进行初始评估和再评估的评估文件。我们提取了证据类型、偏倚风险、额外证据的可用性以及生存数据成熟度变化等证据质量的替代指标信息。具体来说，我们审查了进行再评估的原因，检查了初始评估和再评估之间证据质量和临床获益评分（CBR）的差异，并探讨了证据质量与（1）CBR 和（2）再评估后 CBR 变化之间的关系。
大多数再评估是因为年度收入门槛被超过或初始评估的决议是有时限的。几乎所有的初始评估都使用了有限的程序，而大多数的再评估都使用了常规的程序。在 55 次再评估中，只有 9 次 CBR 增加，21 次 CBR 降低。有一些证据表明，接受随机对照试验的再评估更有可能获得更高的 CBR。
研究结果证实，德国对 RDT 进行再评估的原因和程序不同。此外，再评估中 CBR 较高的情况并不常见，初始评估和再评估中的证据质量有限。
