DRW Monthly
March 2007
Issue No. 4

Taiwan Establishes Successful Orphan Drug Program

Taiwan's Rare Disease and Orphan Drug Act was enacted in 2000 following a period of negotiation between the Department of Health's Bureau of Pharmaceutical Affairs, the Legislative Yuan, Taiwan's central legislative body, and the Taiwan Foundation for Rare Disorders (TDRD), an advocacy organization. Each of these entities drafted their own version of the law that was subsequently consolidated into the 36 articles comprising the Act. The Act incorporates the objectives of all three bodies including the prevention and treatment of rare diseases; the early diagnosis of these diseases; intensive care of rare disease patients; subsidized assistance for patients requiring access to specific drugs for treatment as well as special nutrient foods; promotion of orphan drug research and development; establishment of manufacturing facilities to ensure the supply of these pharmaceuticals and special nutrient foods; and cooperation with international rare disease organizations.(1) In addition, the Department of Health, the central competent authority designated by the Act, is directed to establish a Committee for the Review and Examination of Rare Diseases and Orphan Drugs. This Committee, consisting of government organization representatives, scholars and specialists in medical affairs, and impartial citizens where at least two-thirds of the members must be scholars and specialists in medical affairs(2), is well positioned to ensure that the objectives of the Act be implemented.

Insofar as drug approval requirements are concerned, pharmaceuticals designated as orphan drugs are not required to undergo clinical trials to be approved for use in Taiwan if the drug has already been approved by the US Food and Drug Administration (FDA). If, however, the drug has not received FDA approval, local clinical trials will be required. Drug applications can only be submitted by a subsidiary of the manufacturer based in Taiwan or by a local Taiwanese agent such as a local distributor, local company office or independent third party. The application review process takes 6-10 months to complete.(3) Another noteworthy article delineated in the Act is the 10-year marketing exclusivity period granted to approved orphan drugs during which time the Department of Health will not accept registration applications for any similar drugs.

Although the patient's ability to obtain rapid access to orphan drugs is still an issue in Taiwan, the government on the whole has been very generous to individuals with officially designated rare diseases as laid down in the Rare Disease Prevention and Medicine Law. Following a five day review of a patient's diagnosis report, the Bureau of National Health Insurance grants full financial reimbursement for medication. Since its establishment in 1999, the TFRD has also been an extremely vocal advocate for patients with rare diseases. Through its unrelenting efforts to ensure that patients received adequate legal representation as well as health care benefits, the TFRD has enabled Taiwanese citizens suffering from a rare disease, which afflicts one out of every 10,000 or less individuals(4), to significantly improve their chances of survival. Patients can now apply to the Department of Health's Bureau of Health Promotion for reimbursement through their doctors or medical institutions using a standardized rare disorders report sheet accompanied by medical literature that documents the disease.

Related Resources