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罕见病一般指患病人数占总人数的比例在0.65％-1％的疾病或病变。长期以来,罕见病一直是医学界的一个难题。世界各国的医务工作者在临床上总会遇到这类情况:许多对人类健康危害很大的罕见疾病却苦于无药可治。究其原因,主要是因为罕见病患病人数较少,此类治疗药的市场不大,商业价值有限,制药厂商不愿投资开发或生产。在WHO公布的近5000种罕见疾病中,由于尚未开发出有效的药物,估计大多数未得到有效的治疗。据统计,在美国人中,患有这类疾病的人可能达到近10％。自20世纪80年代以来,罕见病治疗药物（简称罕用药）的开发问题日益引起社会关注,美国、日本、澳大利亚、欧洲等一些发达国家和地区相继实施了特殊政策,以促进本国或本地区罕用药的开发。展望未来,开发罕用药物前景看好,有可能发掘出市场热销的品种。
Rare disease generally refers to the number of patients in the total number of the proportion of 0.65% -1% of the disease or disease. For a long time, rare diseases have always been a problem for the medical profession. In the clinic, medical workers in all countries in the world often encounter such situations: Many rare diseases that are very harmful to human health suffer from no cure. The reason is mainly due to the small number of rare patients, the market for such therapeutic drugs is not, the commercial value is limited, pharmaceutical companies are reluctant to invest in the development or production. Of the nearly 5,000 rare diseases reported by the WHO, most of them are estimated to have no effective treatment because no effective drugs have been developed. According to statistics, among Americans, people with such diseases may reach nearly 10%. Since the 1980s, the development of rare medical treatments has drawn increasing public concern. Some developed countries such as the United States, Japan, Australia, and Europe have successively implemented special policies to promote the development of their own country or region. Medication development. Looking to the future, there is a good prospect for the development of rare drugs and it is possible to find varieties that are hot in the market.