Scientific Advice Office, Human Medicines Division, European Medicines Agency, Amsterdam, The Netherlands.
Pharmaceutical Quality Office, Human Medicines Division, European Medicines Agency, Amsterdam, The Netherlands.
Br J Clin Pharmacol. 2021 Jun;87(6):2459-2464. doi: 10.1111/bcp.14672. Epub 2020 Dec 11.
Scientific advice (SA) is an important tool offered by regulators to help developers generate robust evidence on a medicine's benefits and risks. Drawing on accumulated experience and looking at the SA provided by the European Medicines Agency in 2018 to advanced therapy medicinal products originally developed by public bodies, we discuss most commonly raised issues and the complexity and timings of the questions posed. Earlier and more frequent SA could help advanced therapy medicinal product developers to pre-empt delays at the marketing authorisation stage. Carefully addressing quality and nonclinical issues before entering the pivotal phase of development will clear the path for a smooth clinical development and successful marketing authorisation.
科学建议（SA）是监管机构提供的一种重要工具，可帮助开发者生成有关药物益处和风险的可靠证据。本文借鉴积累的经验，着眼于 2018 年欧洲药品管理局（EMA）为最初由公共机构开发的先进治疗药物产品提供的 SA，讨论了最常提出的问题以及提出问题的复杂性和时间安排。更早和更频繁的 SA 可以帮助先进治疗药物产品的开发者避免在上市许可阶段出现延迟。在进入开发的关键阶段之前，仔细解决质量和非临床问题将为顺利的临床开发和成功的上市许可铺平道路。
