Department of Neuroscience, Functional Pharmacology, University of Uppsala, Uppsala, Sweden.
Department of Immunology, Genetics and Pathology, Medical Genetics and Genomics, University of Uppsala, Uppsala, Sweden.
Trends Pharmacol Sci. 2018 Jun;39(6):525-535. doi: 10.1016/j.tips.2018.03.003.
High levels of productivity, with an increasing number of approvals for new molecular entities (NMEs) by the FDA during the past decade, have coincided with the emergence of innovative drugs for treatments of rare diseases that have utilized the FDA orphan drug program. Since 2000, NMEs with orphan designation encompass a significant portion of approved drugs and constitute about 80% of the approved drugs that have established novel human genome-encoded products in recent years. Biological approvals are also expanding, with 40% of the approved biological agents having orphan designation. This trend illustrates a pivot within the pharmaceutical industry: from research programs that focus on canonical blockbuster indications and targets, towards the establishment of new treatments for rare and difficult to treat diseases.
高生产力水平，过去十年中 FDA 批准的新分子实体 (NME) 数量不断增加，与利用 FDA 孤儿药计划治疗罕见病的创新药物的出现相吻合。自 2000 年以来，具有孤儿药指定的 NME 占据了已批准药物的重要部分，并且约占近年来已建立新颖人类基因组编码产品的已批准药物的 80%。生物批准也在扩大，其中 40%的已批准生物制剂具有孤儿药指定。这一趋势说明了制药行业的一个转变：从专注于经典重磅适应症和靶点的研究计划，转向为罕见和难以治疗的疾病建立新的治疗方法。
