OBJECTIVES: Orphan drugs are pharmaceutical agents for rare diseases in which supply are usually shortage. Countries impose different policies to improve access to orphan drugs but lack of evidences to assess its impact. The aim of this study was to review and compare access to orphan drugs and orphan drugs policies across countries.

METHODS: Access to orphan drugs were compared using marketing authorization (availability) and reimbursement (affordability) status with orphan drugs designation status. United States(US), Japan, Australia, The Netherlands and Thailand were purposively selected based on the availability of an Orphan Drug Act (ODA) or an Orphan Drug List (ODL). Data were retrieved from official health authority database and websites from June 2017 to February 2018.

RESULTS: Number of orphan drugs in the ODL varied significantly with US ranked the highest in number of drugs listed in the ODL (464) followed by Australia(257), Japan(247), The Netherlands(103), and Thailand(71). Marketing authorization of drugs in ODL were 56.34%(Thailand), 70.43%(Australia), 91.59%(US), 94.17%(The Netherlands) and 97%(Japan). All drugs in ODL were fully reimbursed in the Netherlands as part of the orphan drug policy. Though, only some were fully reimbursed in Thailand and Australia (87.50% and 50.27% of the drug registered). Differences in tax credits for drugs in ODL as well as market exclusivity were found among these countries. Tax credits were provided only in USA and Japan while market exclusivity were different with 10 years in Japan and The Netherlands, 7 years in USA, 5 years in Australia and none in Thailand.

CONCLUSIONS: Having ODL does not guarantee the accessibility of orphan drugs. Countries should provide proper financial incentives including reimbursement of orphan drug to increase access to orphan drugs for rare disease patients.