Taiwan has been proactive in supporting rare disease patients through comprehensive policies. Since the 2000 passage of the Rare Disease Prevention and Drug Act, Taiwan has legally protected rare disease patients by integrating early diagnosis, treatment support, and orphan drug development. Orphan drugs are covered under Taiwan’s National Health Insurance (NHI), reducing financial burdens and ensuring better access to critical treatments. To further improve accessibility, the Taiwan Food and Drug Administration (TFDA) has introduced faster approval mechanisms, special importation programs, and regulatory updates to help patients receive life-saving medications more efficiently.
Expedited Approval for Orphan Drugs
To accelerate access to internationally approved orphan drugs, TFDA launched the Orphan Drug Recognition and Expedited Registration Pilot Program in July 2023. This initiative reduces the approval time from 300 to 240 days for drugs already authorized by the U.S. FDA, EMA, or Japan’s MHLW, allowing quicker market entry. The program streamlines applications by allowing simultaneous orphan drug recognition and market registration, ensuring that patients benefit from global medical advancements without unnecessary delays.
Key TFDA Initiatives Benefiting Rare Disease Patients
- Orphan Drug Listings: TFDA updates its orphan drug list regularly. As of December 2024, 140 orphan drugs have been officially recognized, ensuring more treatment options for patients.
- Recognition and Review: TFDA expedites the approval of internationally recognized orphan drugs, reducing waiting times for patients.
- Regulatory Updates: TFDA continues to regularly refine orphan drug policies.
- Transparency in Information: TFDA publishes orphan drug prescription guides and annual reports, offering essential data on drug use, safety monitoring, and treatment outcomes.
Conclusion
By accelerating approvals, enabling special imports, and continuously refining policies, TFDA is ensuring greater accessibility to orphan drugs. These efforts reflect Taiwan’s strong commitment to supporting rare disease patients, helping them receive timely and effective treatments.
