OBJECTIVES: We aimed to understand the market access landscape for orphan drugs by comparing regulatory, health technology assessment (HTA), and reimbursement-related policies for rare disease and orphan drugs across European and Asia-Pacific (APAC) countries.

METHODS: We reviewed publicly available policy, legislative, and regulatory documents regarding rare diseases for nine European and APAC countries. Additionally, we examined documents describing HTA and reimbursement processes for rare disease and orphan drugs among the nine jurisdictions. Documents were sourced from government agency websites and supplemented with peer-reviewed literature from 2014–2023.

RESULTS: Among the countries we investigated, market authorization processes are the same for orphan drugs as for other drugs, although priority review procedures are available in France, Japan, China, and South Korea. However, HTA processes for orphan drugs varied across the different jurisdictions. Four countries do not have special procedures for orphan drugs, while four others have modified requirements. For example, orphan drugs in Japan, Germany, and France are exempt from HTA unless the economic burden exceeds pre-specified thresholds. Only England has a process specifically for orphan drugs, though it only pertains to those for ultra-rare diseases. Nearly all jurisdictions consider economic evaluations, budget impact analyses, and clinical evidence. Most do not have special reimbursement processes for orphan drugs, but many provide subsidies.

CONCLUSIONS: There is a wide variety of requirements and processes related to orphan drugs across European and APAC countries. In most countries, there is no difference in approach to market authorization between orphan drugs and standard drug assessments. Some countries have implemented modified HTA processes for orphan drugs and may provide subsidies. Developing policies and procedures specific for rare disease and orphan drugs may improve equitable access to treatments for patients with rare diseases.