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Overseas Experience

Many countries had laid down clear definitions for rare diseases since the 1980s. They passed clear legislations and formulated evidence-based long-term policy, which enabled rare diseases patients to have proper treatment and timely care, in such areas as early approval of orphan drugs, subsidy and more.

US, EU and neighbouring jurisdictions, such as Singapore, Japan, Australia, Taiwan and Korea have adopted the following rare diseases policies: (1) Defining rare diseases; (2) Formulating supportive policy and measures for rare disease patients; (3) Establishing rare diseases database; (4) Requiring insurance schemes to cover the expenses of rare diseases treatment, and (5) establishing legal framework to encourage pharmaceutical companies to develop drugs for rare diseases.

In 2000, Taiwan passed the Rare Disease Control and Orphan Drug Act. Then, regulations such as the Enforcement Rules of the Rare Diseases and the Orphan Drugs Act were also implemented to meet with the challenges of rare diseases. According to the People with Disabilities Rights Protection Act, rare diseases patients are entitled to enjoy the welfare, support and protection as a person with disabilities. As at 2013, 197 diseases were identified as rare diseases with a population of more than 4,000 patients in Taiwan. 78 drugs have been classified as orphan drugs and more than 1,000 patients are receiving the treatment. Through the National Health Insurance Scheme, low-income families would be fully subsidised with medical treatment, drugs and special nutrient products. Other families would get a subsidy to cover 80% of the expenses.

In 2003, Korea passed a legislation that defined rare diseases. Korea also issued a guideline on rare diseases, providing drug subsidy and funding a national survey on rare diseases. At present, there are 500,000[1] rare disease patients in Korea. As the Korean National Health Insurance (NHI) covers rare disease cases, patients only need to pay for 10% of their medical expenses; the rest would be covered by NHI. People with low income would enjoy full coverage by NHI. In early 2014, the launch of a USD540 million post-genome project[2] was announced to develop and commercialise new genomic technologies.

Although there is no official definition of rare diseases in Mainland China, the Chinese National Birth Defects Monitoring Network[3] had been established since 1986 to monitor rare diseases, including relevant prevention, diagnosis and research works. The government also drafted the Provisions for New Drugs Approval giving priority status to rare diseases-related new drugs; established Hemophilia Home of China – a patient group; adopted the Action Plan of China to Increase the Quality of Newborns and Reduce Birth Defects and Deformity; classified birth defects as non-genetic diseases and genetic diseases; established Rare Disease Relief Public Fund and Shandong Province Rare Disease Association. In addition, the National Natural Science Foundation of China has allocated additional resources in support of relevant research correspondingly every year. Regarding drug control, the foundation set up a negotiation mechanism on orphan drug prices. It also suggested using the entry system of orphan drug market to encourage its research and development to improve diagnosis, with reference to experiences from other countries and regions.