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.