2000 年至 2013 年在欧洲的罕见病药物的营销授权。
Marketing authorisation of orphan medicines in Europe from 2000 to 2013.
机构信息
European Medicines Agency, 30 Churchill Place, Canary Wharf, London E14 5 EU, UK.
Center for Medical Statistics, Informatics, and Intelligent Systems, Section for Medical Statistics, Medical University Vienna, Spitalgasse 23, 1090 Vienna, Austria.
出版信息
Drug Discov Today. 2018 Feb;23(2):424-433. doi: 10.1016/j.drudis.2017.10.012. Epub 2017 Oct 23.
An analysis was performed on a data set of 157 orphan designated medicines with an outcome for marketing authorisation application (MAA) between 2000 and 2013. The intention was to understand the factors associated with marketing authorisation success, the challenges developers face regarding orphan medicine development, and how scientific advice (SA) is used during development. The results demonstrated that orphan medicines have a lower success rate compared with non-orphan medicines and that determinants for marketing authorisation success were company size and compliance with SA. Compliance with SA could help orphan medicine developers overcome clinical development challenges.
对 2000 年至 2013 年间有营销授权申请（MAA）结果的 157 种孤儿指定药物数据集进行了分析。目的是了解与营销授权成功相关的因素、开发者在孤儿药物开发方面面临的挑战，以及如何在开发过程中使用科学建议（SA）。结果表明，孤儿药物的授权成功率低于非孤儿药物，而授权成功的决定因素是公司规模和对 SA 的遵守情况。遵守 SA 可以帮助孤儿药物开发者克服临床开发挑战。
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