OBJECTIVES: Drugs granted orphan status typically receive additional support and incentives during the health technology assessment (HTA) process. Orphan designated is granted by the European Medicines Agency (EMA) and subsequently recognised by all EU member states, and may be granted separately by non-EU countries. In this study we examined the relationship between orphan drug status and reimbursement outcome, and looked at differences between the likelihood of reimbursement in EU and non-EU countries.

METHODS: All HTA recommendations published in 2023 for eight countries (EU: France, Ireland, Spain, Sweden; non-EU: England, Scotland, Australia, Canada) were reviewed, with data extracted regarding HTA appraisal type, reimbursement decision, and EMA orphan-designation status. Peason’s chi-squared tests were used to examine the association between orphan drug status and positive reimbursement decisions across the eight countries, and to further determine whether there were differences in the likelihood of successful reimbursement recommendation between EU and non-EU countries. Statistical significance was evaluated at a level of p<0.05.

RESULTS: In total, 680 appraisals were conducted across the eight countries, of which 156 (23%) were for orphan-designated medicines. Statistically significant differences were observed between EU and non-EU countries in the proportion of positive reimbursement recommendations for all products (Χ² = 24.63, p < 0.001) and orphan-designated medicines (Χ² = 5.25, p = 0.022). Overall, non-EU countries were more likely to give positive reimbursement recommendations. No association was found between orphan drug status and reimbursement decision (Χ² = 0.296, p = 0.586).

CONCLUSIONS: Our findings show that there is a higher probability of reimbursement success for medicinal products (both orphan and non-orphan designated) in non-EU countries compared to EU countries. Further research is required to identify driving factors behind the observed heterogeneity.