The impact of the rare disease and Orphan Drug Act in Taiwan   全文下載

The impact of the rare disease and Orphan Drug Act in Taiwan

"The Rare Disease and Orphan Drug Act (the Act) was enacted in 2000 in Taiwan for the facilitation of the research,development, and accessibility of orphan drugs and special nutritional foods; for the prevention and early diagnosis ofrare diseases; and for providing intensive care for patients with rare diseases. The aim was to investigate the impact ofthe Act on the availability and use of orphan drugs in Taiwan in the hope of identifying the remaining challenges andpossible solutions to assist future policy making, which may be applicable in other countries as well. The informationand statistics for rare diseases and orphan drugs retrieved from the official annual reports and documents were analyzed.There were 225 diseases recognized as rare diseases, and one-third (75/225) of them were congenital metabolic disorders.Among the 110 designated orphan drugs that could apply for listing in the National Health Insurance (NHI) PharmaceuticalBenefits and Reimbursement Scheme, approximately half (62/110) of them were granted marketing authorization.While the NHI program compulsory for all citizens increased patient accessibility to orphan drugs, the rapidlyincreasing economic burden became an urgent issue for the government. Emerging gene therapies may be the solution tounmet medical needs and also a financial obstacle to tackle. The Act increased the availability of orphan drugs while theNHI system facilitated patient access, which benefited many patients with rare diseases in Taiwan. However, the soaringeconomic burden was noticed and was anticipated to aggravate. More communication and cooperation betweenstakeholders is critical in finding solutions for the long-term sustainability of the NHI system."