Health Economics, National Institute for Health Research, Tehran University of Medical Sciences, Tehran, Iran.
Health Policy, National Institute for Health Research and Health Equity Research Center (HERC), Tehran University of Medical Sciences, Tehran, Iran.
Int J Technol Assess Health Care. 2022 Jun 22;38(1):e59. doi: 10.1017/S0266462322000393.
This study aimed to identify different criteria for priority setting of rare diseases to help policy makers in making evidence-informed decisions.
A scoping review was conducted to comprehensively examine the existing various methods and criteria for prioritizing orphan drugs and rare diseases. We performed searching in Scopus, PubMed, Embase, and websites of health technology assessment (HTA) agencies, 2000-21, and data were extracted.
From the 1,580 identified publications, eleven articles were included. Multicriteria decision analysis was the most frequent method (seven out of eleven studies) used for priority setting. The extracted criteria for priority setting of orphan products were analyzed based on six main categories as follows: health outcomes and clinical implications (six subsets which showed clinical implications), economic aspects (four subsets that indicated the economic effects of orphan drugs and rare diseases), disease and population characteristics (six subsets that included the characteristics of the rare diseases), therapeutic alternatives and uniqueness of orphan technologies (two subsets which discussed the alternatives and uniqueness of orphan technologies), evidence (three subsets which regarded the quality and availability of evidence), and other criteria (three subsets dealing with social and organizational criteria). Cost-effectiveness, budget impact, and disease severity were the most frequent criteria in the studies.
Because of the high price of orphan drugs and limitations of using HTA for reimbursement of them, it is critical to explore them by precise technical methods like multiple criteria decision making in priority setting.
本研究旨在确定罕见病优先排序的不同标准，以帮助政策制定者做出基于证据的决策。
进行了范围综述，以全面考察优先考虑孤儿药和罕见病的现有各种方法和标准。我们在 Scopus、PubMed、Embase 和卫生技术评估 (HTA) 机构的网站上进行了搜索，时间范围为 2000-21 年，并提取了数据。
从 1580 篇已确定的出版物中，有 11 篇文章被纳入。多标准决策分析是最常用的方法 (11 项研究中有 7 项) 用于确定优先级。提取的孤儿产品优先排序标准基于以下六个主要类别进行分析：健康结果和临床意义 (六个子集，显示临床意义)、经济方面 (四个子集，表明孤儿药物和罕见病的经济影响)、疾病和人群特征 (六个子集，包括罕见病的特征)、治疗替代方案和孤儿技术的独特性 (两个子集，讨论孤儿技术的替代方案和独特性)、证据 (三个子集，涉及证据的质量和可用性) 和其他标准 (三个子集涉及社会和组织标准)。成本效益、预算影响和疾病严重程度是研究中最常见的标准。
由于孤儿药价格高昂，以及使用 HTA 来报销它们的限制，因此通过多标准决策等精确技术方法在优先级设置中探索它们至关重要。
