参考资料：https://blog.petrieflom.law.harvard.edu/2017/09/25/orphan-drugs-designations-and-approvals-have-something-to-say-about-risks/ https://www.fda.gov/industry/designating-orphan-product-drugs-and-biological-products/orphan-drug-designation-disease-considerations https://www.toutiao.com/a68076...
2023 年 11 月 14 日 Daniel Tobias Michaeli 等人在《The European Journal of Health Economics》发布的“Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy”,文章回顾了 FDA 针对药物研发的五种特殊认定,涉及其安全性、疗效/...
求大神出来指点一下
Orphan drug designations and approvals: Gives financial incentives to sponsors for manufacturing drugs that treat rare (orphan) diseases. In the US, an orphan disease is defined as one that affects fewer than 200,000 individuals. One example isDupixent(dupilumab), designated orphan status in 2024...
[1]. 奥来恩专家解读:FDA孤儿药认定-新药研发与审评的加速通道系列之五。https://mp.weixin.qq.com/s/4OAmFQ6TSNsdsq8On7swgg [2]. FDA Website: Search Orphan Drug Designations and Approvals. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/listResult.cfm ...
2023 年 11 月 14 日 Daniel Tobias Michaeli 等人在《The European Journal of Health Economics》发布的“Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy”,文章回顾了 FDA 针对药物研发的五种特殊认定,涉及其安全性、疗效/...
HP1 ANALYSIS OF THE ORPHAN DRUG DESIGNATIONS AND APPROVALS GRANTED BY THE EMEA AND THE USA FDA BETWEEN 2000 AND 2007 - Value in Healthdoi:10.1016/S1098-3015(10)66236-5E Seoane-VazquezR Rodriguez-MonguioValue in Health
2023 年 11 月 14 日 Daniel Tobias Michaeli 等人在《The European Journal of Health Economics》发布的“Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy”,文章回顾了 FDA 针对药物研发的五种特殊认定,涉及其安全性、疗效/...
[4] Search Orphan Drug Designations and Approvals. Retrieved March 21, 2020, from https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=730320 [5] Elezanumab. Retrieved March 21, 2020, from https://www.abbvie.com/our-science/pipeline/abt-555.html ...
近日,吉美瑞生再生医学集团(Regend Therapeutics)宣布,美国食品药品监督管理局(FDA)已授予其气道基底层干细胞产品Pulmovinci孤儿药认定(Orphan Drug Designation, ODD),用于治疗特发性肺纤维化(Idiopathic Pulmonary Fibrosis,IPF)。与此同时,其最...