The Scottish Medicines Consortium (SMC) defines end-of-life medicines as drugs used for treating conditions that usually lead to death within three years using currently available treatments. Orphan medicines are drugs used for the treatment of very rare conditions and ultra-orphan drugs are used for the treatment of extremely rare conditions. The objective of this study is to determine the influence of unmet need on SMC health technology assessment (HTA) decisions for rare disease conditions.

The reimbursement data between 2004 and 2021 from SMC for rare disease conditions were included. These data were categorized based on the presence of an unmet need, that is, drugs considered under the orphan or ultra-orphan process, or those that fulfilled SMC end-of-life criteria. A chi square test was conducted to determine an association between the presence of an unmet need and the HTA decision. HTAs without a decision were excluded.

A total of 91 HTAs were included in the analysis of which, 57.1 percent (n = 52) were recommended, and 42.9 percent (n = 39) were not recommended. Out of the recommended reviews, 32.7 percent (n = 17) addressed an unmet need and 67.3 percent (n = 35) did not. Recommended drugs had positive clinical evidence and high cost-effectiveness or the submission of a patient access scheme while negative decisions were associated with lower or uncertain cost-effectiveness. The chi square test result showed no association between the presence of an unmet need and the HTA decision (p = 0.315).

Unmet need does not influence the SMC HTA decisions for rare disease conditions. Economic elements were the driving factors in the decision-making process.