Abstract：
Many new drugs have been approved over the past decade for rare or orphan diseases. The passage of the Orphan Drug Act (ODA) in 1983 has provided key economic and regulatory incentives to provide medicines for patients who are suffering from rare diseases that may not be commercially attractive for research and development. We have analyzed 497 novel drugs approved from 2010 - June 13, 2022, of which 220 were given orphan designation status. We discuss trends over this time period, potential risks for long development times, and provide example case studies of successful development and launch of novel drugs for rare diseases.
摘要：
在过去的十年中，许多新药已被批准用于治疗罕见或孤儿疾病。1983年通过的《孤儿药法案》（ODA）提供了关键的经济和监管激励措施，为患有罕见疾病的患者提供药物，这些疾病可能对研发没有商业吸引力。我们分析了2010年至2022年6月13日批准的497种新药，其中220种获得了孤儿称号。我们讨论了这段时间的趋势，长期开发的潜在风险，并提供成功开发和推出罕见疾病新药的案例研究。
