OBJECTIVES : To gather insights into reimbursement rates and timelines for orphan drugs appraised by the National Centre for Pharmacoeconomics (NCPE).

METHODS : The European Commission's Community Register of orphan medicinal products for human use was cross-checked against appraisals on the NCPE website to identify orphan drugs that have been evaluated by the NCPE. The Health Services Executive (HSE) website was also searched for reimbursed drugs and cross-checked with NCPE assessments. The data from the identified evaluations were extracted and analysed in Excel.

RESULTS : Between 2010 and 2019, 86 appraisals of 70 drugs were conducted by the NCPE. The majority were for non-cancer drugs (n=47, 54.7%) and in adults (n=58; 67.4%). Only 7 (8.1%) did not require a full pharmacoeconomic evaluation. In 18 (20.9%) instances the manufacturer did not provide a full pharmacoeconomic submission as requested. Of 68 completed appraisals, only 5 (4.7%) were positive recommendations, of which at least two required confidential price negotiations. The HSE approved another 34 drugs after confidential price negotiations (at least 22; 64.7%), the majority being cancer drugs (n=19; 55.9%). Average time from completion of the reimbursement process (either from the end of the Rapid Review or full pharmacoeconomic evaluation, whichever was the last assessment step) was 337 days (SD = 219) for cancer drugs, and 449 days (SD = 282) for non-cancer drugs. 8 drugs did not complete a full pharmacoeconomic evaluation, but was recommended by the HSE, on average after 495 days (SD = 330).

CONCLUSIONS : Orphan drugs are extremely unlikely to be recommended by the NCPE, but are more likely to be recommended by the HSE. Manufacturers making submissions to Ireland should expect to discuss commercial discounts to secure reimbursement. Low positive recommendation rates by the NCPE, requirements for price negotiations and time to positive reimbursement with the HSE may deter manufacturers from making orphan drug submissions.