Institute for Medical Technology Assessment, Erasmus University Rotterdam, P.O. Box 1738, 3000 DR, Rotterdam, The Netherlands.
Syreon Research Institute, Budapest, Hungary.
Pharmacoeconomics. 2021 Jul;39(7):771-788. doi: 10.1007/s40273-021-01010-z. Epub 2021 Apr 16.
The objective of this study was to develop guidance contributing to improved consistency and quality in economic evaluations of personalised medicine (PM), given current ambiguity about how to measure the value of PM as well as considerable variation in the methodology and reporting in economic evaluations of PM.
A targeted literature review of methodological papers was performed for an overview of modelling challenges in PM. Expert interviews were held to discuss best modelling practice. A systematic literature review of economic evaluations of PM was conducted to gain insight into current modelling practice. The findings were synthesised and used to develop a set of draft recommendations. The draft recommendations were discussed at a stakeholder workshop and subsequently finalised.
Twenty-two methodological papers were identified. Some argued that the challenges in modelling PM can be addressed within existing methodological frameworks, others disagreed. Eighteen experts were interviewed. They believed large uncertainty to be a key concern. Out of 195 economic evaluations of PM identified, 56% addressed none of the identified modelling challenges. A set of 23 recommendations was developed. Eight recommendations focus on the modelling of test-treatment pathways. The use of non-randomised controlled trial data is discouraged but several recommendations are provided in case randomised controlled trial data are unavailable. The parameterisation of structural uncertainty is recommended. Other recommendations consider perspective and discounting; premature survival data; additional value elements; patient and clinician compliance; and managed entry agreements.
This study provides a comprehensive list of recommendations to modellers of PM and to evaluators and reviewers of PM models.
本研究旨在提供指导，以提高个性化医学（PM）经济评估的一致性和质量，因为目前对于如何衡量 PM 的价值存在歧义，而且 PM 经济评估的方法和报告存在很大差异。
对方法学论文进行了有针对性的文献回顾，以概述 PM 建模挑战。进行了专家访谈，以讨论最佳建模实践。对 PM 经济评估进行了系统的文献回顾，以深入了解当前的建模实践。综合研究结果并提出了一系列建议草案。在利益相关者研讨会上讨论了这些建议草案，并随后定稿。
确定了 22 篇方法学论文。一些人认为可以在现有方法框架内解决 PM 建模中的挑战，而另一些人则不同意。对 18 名专家进行了访谈。他们认为存在很大的不确定性是一个关键问题。在确定的 195 项 PM 经济评估中，有 56%没有解决任何确定的建模挑战。制定了一套 23 项建议。有 8 项建议侧重于测试-治疗途径的建模。不鼓励使用非随机对照试验数据，但在没有随机对照试验数据的情况下提供了一些建议。建议参数化结构不确定性。其他建议考虑了视角和贴现率、过早的生存数据、附加价值元素、患者和临床医生的依从性以及管理准入协议。
本研究为 PM 建模者以及 PM 模型的评估者和审查者提供了一份全面的建议清单。
