Malaysian Health Technology Assessment Section, Medical Development Division, Ministry of Health Malaysia, Putrajaya, Malaysia.
Int J Technol Assess Health Care. 2024 Sep 4;40(1):e35. doi: 10.1017/S026646232400031X.
Rare diseases (RD)-related policies have received significant attention due to the pressing medical requirements associated with these medical conditions and the substantial impact and treatments they may have on healthcare budgets. Nevertheless, policymakers frequently encounter difficulties in managing issues concerning resource allocation and prioritization within this population. Realizing the need to address such problems, this study was conducted to develop a framework based on the multicriteria decision analysis to improve RD reimbursement prioritization in Malaysia.
Primarily, a scoping review was performed to identify the methods and criteria used for the reimbursement of RD treatment, followed by strategic stakeholder engagement and a deliberative process on determining the best approach for the framework, including criteria identification, elicitation of weights, and a pilot assessment using the framework.
The findings reflected the priorities and perspectives of the stakeholders, which identified eight key criteria and their associated weights, namely effectiveness (19.6 percent), disease severity (15.6 percent), safety (14.2 percent), access to treatment (12.6 percent), economic consideration (12.2 percent), type of therapeutic treatment (11.5 percent), availability of alternatives (8.3 percent), and population group (6 percent).
In summary, the developed framework was well-accepted by the Rare Disease Committee, which will be applied as part of the committee deliberation for transparent and equitable decision making on fund allocation and reimbursement of orphan and RD treatment in Malaysia.
由于与这些病症相关的紧迫医疗需求以及它们对医疗预算可能产生的重大影响和治疗方法，罕见病（RD）相关政策受到了广泛关注。然而，政策制定者在管理这一人群中的资源分配和优先级问题时经常遇到困难。为了解决这些问题，本研究旨在制定一个基于多准则决策分析的框架，以改善马来西亚 RD 报销的优先级排序。
首先，进行了范围综述，以确定用于 RD 治疗报销的方法和标准，随后进行了战略利益相关者参与和关于确定框架最佳方法的审议过程，包括标准确定、权重 elicitation 和使用框架进行试点评估。
研究结果反映了利益相关者的优先事项和观点，确定了八个关键标准及其相关权重，即有效性（19.6%）、疾病严重程度（15.6%）、安全性（14.2%）、治疗可及性（12.6%）、经济考虑（12.2%）、治疗类型（11.5%）、替代品的可获得性（8.3%）和人群（6%）。
总之，该框架得到了罕见病委员会的广泛认可，将作为委员会审议的一部分，用于马来西亚孤儿和 RD 治疗的资金分配和报销的透明和公平决策。
