OBJECTIVES: This study aimed to analyse health technology appraisal (HTA) outcomes made by the National Centre of Pharmacoeconomics (NCPE) in 2023 and identify any impact that orphan status might have on reimbursement success. It builds on a similar analysis of the year 2022 conducted by the authors and presented at ISPOR US (HTA92).

METHODS: Rapid reviews and full HTAs published by NCPE in 2023 were reviewed, with data extracted regarding appraisal type and decision. Results were tabulated and descriptive statistics were compiled. Drugs were classified as orphan based on the list of authorised pharmaceuticals with orphan designations obtained from the European Medicines Agency (EMA) website.

RESULTS: A total of 108 submissions (86 non-orphan and 22 orphan) were made in Ireland in 2023. Of these, 76 were Rapid Reviews and 32 were full HTAs. Of all treatments assessed, 5 (15.6%) that underwent a full HTA and 1 (2.6%) that underwent a Rapid Review were reimbursed. Of the 75 drugs which were not reimbursed at Rapid Review, 43 (57.3%) were recommended for a full HTA.

1 of the 22 (4.5%) orphan drugs reviewed in 2023 was successfully reimbursed after a full HTA, compared to 3 in 2022 (2 through the Rapid Review process). 8 (7%) non-orphan drugs were reimbursed in 2023: 2 via the Rapid Review process and 6 after full HTAs.

CONCLUSIONS: Overall, only 6.5% of NCPE submissions in 2023 received positive reimbursement decisions. Orphan drugs were reimbursed at a lower rate than non-orphan drugs regardless of reimbursement route. Notably, the Irish HTA process is not tailored to the reimbursement of orphan drugs. Patients suffering from rare diseases in Ireland may face setbacks as a result of the adoption of a single, non-tailored approach to assessing drugs.