2014年,一场旨在帮助罕见病肌萎缩侧索硬化患者(也称“渐冻人”)的冰桶挑战赛活动在全球流行开来,让很多人了解到了“罕见病”这个概念。由于单种疾病患者人数较少,罕见病患者的用药也被称为“孤儿药”。长期以来,孤儿药一方面价格高且多数不在医保目录内,让患者用不起,另一方面研发难度大、投资回报低,也备受制药企业冷落。近年来各国政府正积极通过政策 In 2014, an ice-bucket challenge campaign to help patients with atypical ALS (also known as “getting cold”) became prevalent around the world and made many people aware of “rare disease” This concept. Due to the small number of patients with a single disease, the treatment of rare diseases is also known as “orphan medicine.” For a long time, orphan drugs on the one hand, the high prices and most are not in the health insurance directory, so that patients can not afford, on the other hand, research and development difficult, low return on investment, but also the pharmaceutical companies ignored. In recent years, governments of various countries are actively adopting policies