OBJECTIVES: To analyse the relationship between orphan drug status and successful reimbursement in Ireland, Sweden, France, and Canada. Also, to investigate the impact having an orphan drug policy has on reimbursement decisions.

METHODS: A list of approved medications with current orphan designations was compiled from the European Medicines Agency (EMA) website. Information about reimbursement status was taken from the websites of national reimbursement or Health Technology Assessment (HTA) agencies, along with information regarding the orphan drug policy associated with each national reimbursement framework.

RESULTS: France and Sweden both have an orphan drug policy. Only 62 reimbursement decisions were made in Sweden in 2022, 10 of which were for orphan drugs (16%) and 52 for non-orphan drugs (84%). In Sweden, 9 orphan drugs (90%) achieved either full or limited reimbursement; the same figure for non-orphan drugs w (83%) achieved this.

France published 230 reimbursement decisions in 2022. Two analyses were conducted: one including the 24 early access submissions and one excluding them. Excluding early access submissions, 12 of 15 submissions made for orphan drugs (80%) were successful, compared to 154 (81%) for non-orphan drugs. Including early access programmes, this figure reached 19 (86%) for orphan drugs and 171 (82%) for non-orphan drugs.

Neither Ireland nor Canada have an orphan drug policy. 42 decisions were made in Ireland: 3 of 14 orphan drugs (21%) and 9 of 28 non-orphan drugs were successful. In Canada, 83 decisions were made, with 10 of 13 (77%) orphan drugs and 56 of 70 (80%) non-orphan drugs receiving a positive decision.

CONCLUSIONS: There is only a small difference in orphan drug reimbursement success rates between countries which incorporate an orphan drug policy into their HTA frameworks and those who do not. Regardless, implementing orphan drug policies would help avoid negative consequences for innovation, development, and access to orphan drugs.