目的:本系统文献综述旨在通过实施多标准决策分析(MCDA),探索将社会偏好纳入罕见病(RD)和孤儿药(OD)的健康技术评估(HTA)的全球经验。离散选择实验(DCE),和人员权衡(PTO)方法,在其他人中。
方法:2021年4月使用PubMed对文献进行了系统的搜索,科克伦,Embase,和Scopus数据库。审查阶段使用系统审查和荟萃分析方法的首选报告项目。最后,《促进卫生服务研究实施行动框架》被用来讨论在RD背景下实施这些文书的问题。
结果:共有33篇文章符合纳入标准。这些研究使用MCDA(n=17)测量了作为HTA一部分的RD和OD的社会偏好,DCE(n=8),和PTO(n=4),在其他方法中(n=4)。这些发现,患者和临床医生并没有根据稀有性优先考虑资金。只有OD显示成效及改善生活质量,公众才愿意拨款,将疾病严重程度作为相关因素,未满足的健康需求,和生活质量。相反,HTA机构专家更喜欢他们目前的方法,更加重视成本效益和证据质量,尽管他们对药物审查过程的公平性表示担忧。
结论:MCDA,PTO,和DCE是评估HTA对RD和OD的社会偏好的有用和透明的方法。然而,他们的方法论局限性,例如任意选择标准,主观评分方法,框架效果,加权适应,和价值计量模型,可能会使实施具有挑战性。
OBJECTIVE: This systematic literature review aimed to explore experiences worldwide of societal preferences integration into health technology assessments (HTAs) for rare diseases (RDs) and orphan drugs (ODs) through the implementation of multicriteria decision analysis (MCDA), discrete choice experiments (DCEs), and person trade-off (PTO) methods, among others.
METHODS: A systematic search of the literature was conducted in April 2021 using PubMed, Cochrane, Embase, and Scopus databases. The Preferred Reporting Items for Systematic Reviews and Meta-Analyses approach was used for the review phases. Finally, the Promoting Action on Research Implementation in Health Services framework was used to discuss the implementation of these instruments in the RD context.
RESULTS: A total of 33 articles met the inclusion criteria. The studies measured societal preferences for RD and OD as part of HTA using MCDA (n = 17), DCE (n = 8), and PTO (n = 4), among other methods (n = 4). These found that patients and clinicians do not prioritize funding based on rarity. The public is willing to allocate funds only if the OD demonstrates effectiveness and improves the quality of life, considering as relevant factors disease severity, unmet health needs, and quality of life. Conversely, HTA agency experts preferred their current approach, placing more weight on cost-effectiveness and evidence quality, even though they expressed concern about the fairness of the drug review process.
CONCLUSIONS: MCDA, PTO, and DCE are helpful and transparent methods for assessing societal preferences in HTA for RD and OD. However, their methodological limitations, such as arbitrary criteria selection, subjective scoring methods, framing effects, weighting adaptation, and value measurement models, could make implementation challenging.