Drug Discov Ther. 2013;7(3):126-128. (DOI: 10.5582/ddt.2013.v7.3.126)

Rare disease patients in China anticipate the sunlight of legislation.

Gao JJ, Song PP, Tang W


It is estimated that there are over ten million rare disease patients in China currently. Due to a lack of effective drugs and reimbursement regulations for medical expenses the diseases bring most patients enormous physical suffering and psychological despair. Past experience in other countries such as the United States, Japan, and the European Union have shown that legislation is the critical step to improve the miserable situation of rare disease patients. Laws and regulations for rare diseases in these countries prescribe a series of incentives for research and development of orphan drugs which turn out to obviously allow these drugs to flourish. Legislation has also established a drug reimbursement system to reduce the medical burden of the patients. These measures effectively protect the rights and interests of patients with rare diseases. In China, legislation for rare diseases has begun to attract the attention of authorities. It is anticipated that relevant laws and regulations will be established as early as possible to provide safeguards for rare disease patients in China.

KEYWORDS: Rare disease, orphan drugs, laws and regulations, medical reimbursement

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