Gao J J, Song P P, Tang W
Department of Surgery, Graduate School of Medicine, The University of Tokyo, Tokyo, Japan.
Drug Discov Ther. 2013 Jun;7(3):126-8.
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.
据估计，中国目前有超过1000万罕见病患者。由于缺乏有效药物以及针对这些疾病所带来的医疗费用的报销规定，大多数患者承受着巨大的身体痛苦和心理绝望。美国、日本和欧盟等其他国家过去的经验表明，立法是改善罕见病患者悲惨处境的关键一步。这些国家的罕见病法律法规规定了一系列激励孤儿药研发的措施，这显然促使这些药物蓬勃发展。立法还建立了药物报销制度以减轻患者的医疗负担。这些措施有效地保护了罕见病患者的权益。在中国，罕见病立法已开始引起当局的关注。预计将尽早制定相关法律法规，为中国的罕见病患者提供保障。
