We reviewed the health technology assessment (HTA) guidelines for therapies targeting orphan conditions in four countries/regions in Asia.

A pragmatic literature search was conducted to identify and review key documents outlining reimbursement, pricing, and coverage policies in China, Taiwan, Korea, and Japan.

Therapies for rare diseases in Japan and those for ultra-rare diseases in Korea are exempt from cost-effectiveness evaluations. Taiwan provides full financial coverage for rare disease therapies. China has no special considerations for rare diseases. Drugs included in the medical insurance list are reimbursed at varying levels depending on the “class” of the listing. Unlike prior variations at provincial levels for coverage of off-the-list drugs, new national policy has introduced consistency in coverage.

Access and reimbursement processes vary between markets in Asia. New HTA guidelines in Japan allow for easier access to therapies targeting rare diseases by eliminating cost-effectiveness analysis for price determination. On the other hand, a value dossier including an economic evaluation is necessary for rare diseases in Korea. However, manufacturers can provide risk-sharing schemes for rare diseases. China has not yet introduced any specific evaluations or reimbursement criteria for therapies targeting rare diseases. Policies for rare diseases are evolving rapidly to improve access and affordability.