In 2000, Taiwan promulgated the “Rare Diseases and Orphan Drug Act”, becoming the fifth nation in the world to introduce legislation specifically to protect rare disease patients, This Act is enacted for the prevention of the occurrence of rare diseases; for the early diagnosis of rare diseases; for the intensive care of rare disease patients; for assisting patients in gaining access to specific drugs for the treatment of rare diseases and special nutritional foods essential for the maintenance of life; and for promoting and ensuring the supply, manufacturing, research and development of such drugs and foods. According to the Act, the term “rare diseases” refers to diseases with prevalence lower than the standard announced by the central competent authority or with special circumstances, have been identified as rare diseases by the “Review Committee for Rare Diseases and Orphan Drugs” and as well as officially announced by the central competent authority. The prevalence rate of rare diseases in the current public notice is lower than 1 in 10,000.The rare disease policies in place in Taiwan are as follows:

1.Establishing a Comprehensive Medical Network for Rare Diseases
 (1) Review and Designate notifiable rare diseases, and establish a case reporting system:
The Ministry of Health and Welfare designates and publicly announces notifiable rare diseases in accordance with the “Rare Diseases and Orphan Drug Act”. The Ministry also developed a web-based reporting system for rare diseases and, to ensure the accurate reporting of rare diseases, all cases reported by medical personnel are reviewed by experts before being included into the database.

(2) Include rare diseases in the National Health Insurance program and in the categories of major illnesses and injuries:
Since 1994, Taiwan has had National Health Insurance which reimburses an insured person who has a disease (including rare diseases) during a valid insurance period in accordance with the “National Health Insurance Act”. Although the Act has stipulated that the object of insurance for medical treatment shall bear part of the cost of medical care, rare disease patients are free from such burden.

 (3) Subsidy for medical costs non-reimbursable by the National Health Insurance program:
To enhance medical care of those affected by rare diseases, according to Article 33 of the “ Rare Diseases and Orphan Drug Act” and the “Regulations Governing Subsidies for Medical and Healthcare Expenses for Rare Diseases”, the Health Promotion Administration, Ministry of Health and Welfare allocates a budget to subsidize the costs of diagnosis, treatments, and pharmaceuticals for rare diseases that are not included in the National Health Insurance. Items covered by the subsidy include domestic and international diagnostic tests, household medical facilities rental, nutritional counseling, emergency orphan drugs, treatments, etc. The maximum subsidy amount is limited to 80% of the expenses. However, expenses of patients from low-income or middle-low-income households, and expenses of emergency medicine and special nutrient foods essential for the maintenance of life are fully subsidized.

​(4) Subsidy and supply for special nutrient foods as well as emergency orphan drugs for rare diseases:
The government has established a logistics center to reserve and supply special nutrient foods essential for the maintenance of life for rare disease patients with congenital metabolic abnormalities, and emergency orphan drugs.

​(5) The HPA provides services and subsidies of prenatal diagnosis for high-risk pregnancies (with family history of genetic disorders), as well as congenital metabolic disorders screening for all newborns.

(6) Genetic counseling centers have been established in 14 medical centers to provide counseling, diagnosis and treatment services for hereditary diseases and rare diseases.

(7) In accordance with the provisions of the “Rare Diseases and Orphan Drug Act” and “Regulations Governing Healthcare Services for Rare Diseases and Rare Genetic Defects”, the entrusted professional institutions or groups will, upon receiving rare disease notifications and obtaining the consent of patients or their legal representative, dispatch professionals to visit and inform regarding the related impact of diseases, as well as to provide psychological support, childbirth care, care consultation and other services for patients and their families.

​2. Implementing Research, Education and Publicity Programs to Prevent Rare Diseases
​(1)  Programs related to domestic rare disease prevention and treatment can apply for subsidies in accordance with the “Regulations Governing the Incentives and Subsidies for Rare Disease Prevention and Treatment”. Programs eligible for subsidies include: (a) Fostering the necessary professional manpower for the research, diagnosis, testing and treatment of rare diseases. (b) Conducting academic research and clinical research related to prevention and treatment of rare diseases. (c) Purchasing special equipment for the research, diagnosis, testing and treatment of rare disease prevention and treatment. (d) Conducting academic exchange and advocacy related to prevention and treatment of rare diseases

(2) The HPA uses media resources to raise public awareness about rare diseases.

(3) The HPA subsidizes patient groups in holding disease prevention and patient support activities.