谁来投入罕见病研究和药物研发?埃博拉疫情暴发带给我们的警示

Who is responsible for the rare disease research? Alert posed by Ebola Viral Disease outbreak

2014年西非暴发了历年来最严重的埃博拉疫情,已引起世界各国的高度关注.虽然埃博拉病毒被发现已有近40年,但目前却还没有批准上市的有效药物和疫苗.直至2014年,在本次疫情的威胁下,少数国家才启动了埃博拉病毒病(EVD)药物和疫苗的临床试验,其安全性和有效性还有待证明.造成这一困境的主要原因之一是EVD作为一种罕见病,市场需求小,研发投入得不到回报,很少有制药公司涉足,类似情况普遍存在于肌萎缩侧索硬化等罕见病.本文分析了我国罕见病的需求和研究现状,建议在国家层面支持罕见病相关基础和临床研究,并发挥政府的市场引导作用,通过多种措施提高我国罕见病的诊疗水平,推动罕见病医药产业的发展.

The outbreak of Ebola Virus Disease（EVD） in West Africa of 2014 is the worst so far. Although it has been almost 40 years since EVD was identified in 1976, efficacious vaccines or therapeutics for Ebola are still not available. Clinical trials of Ebola vaccines and drugs were launched in 2014; their efficacy and safety remain to be demonstrated. EVD is a rare disease. Due to their limited market potential, the pharmaceutical industry traditionally has little motivation in investing in the drug development of rare diseases such as EVD and amyotrophic lateral sclerosis. In this review we analyze the current research status of rare diseases in China, and make specific proposals on how to promote the development of rare disease research and to attract the interest of pharmaceutical industries through governmental policy support.