Abstract
BACKGROUND: In Ireland, health technology assessment (HTA) submissions for orphan drugs or drugs for rare diseases have increased in recent years but have not been explicitly analysed. All evaluations are conducted by the National Centre for Pharmacoeconomics (NCPE). OBJECTIVES: The objectives of this study were to ascertain the number of orphan drug submissions to the NCPE and determine how these drugs proceeded through the NCPE critical evaluation process compared with non-orphan drug submissions. METHODS: This was a retrospective analysis of applicant rapid review submissions made to the NCPE from January 2012 to December 2017 inclusive. Drugs were categorised according to the following definitions: orphan (non-cancer) drug, orphan (cancer) drug and ultra-orphan drug. In each of the three categories, the outcome of rapid review appraisal, and where relevant, the outcome of the subsequent HTA was recorded. RESULTS: During the period of study, 280 rapid review submissions were made to the NCPE, of which 21 were for orphan (non-cancer) drugs, 24 were for orphan (cancer) and ten were for ultra-orphan drugs. After rapid review, 44%, 78% and 100% of orphan (non-cancer) drugs, orphan (cancer) drugs and ultra-orphan products, respectively, were recommended for full HTA. When the outcome of the rapid review process was compared between orphan drugs and non-orphan drugs, a statistically significant difference was detected in the proportion of rapid reviews for which the outcome was 'HTA recommended' (Pearson's Chi-squared test; p = 0.04). CONCLUSIONS: The number of submissions to the NCPE for orphan drugs has increased in recent years. The rapid review and HTA process in Ireland plays a role in supporting the reimbursement decision-making process for orphan drugs in a similar manner to the process established for non-orphan drugs. However, the outcome of the reimbursement process for orphan drugs versus non-orphan drugs (in terms of access for patients) has yet to be quantified.