Charité - University Medicine Berlin, QUEST - Center for Transforming Biomedical Research, Berlin Institute of Health (BIH), Berlin, Germany.
Institute for History, Ethics and Philosophy of Medicine, Hannover Medical School (MHH), Hannover, Germany.
Br J Pharmacol. 2018 Nov;175(22):4229-4238. doi: 10.1111/bph.14485. Epub 2018 Oct 1.
Therapeutic area guidelines (TAGs) published by the EMA and the FDA offer guidance in planning the launch of a trial in a certain indication. We assessed and compared the guidance on preclinical efficacy of all available TAGs from EMA and FDA.
EMA and FDA websites and databases were searched for all TAGs. A mixed deductive and inductive approach was applied to analyse and cluster content for preclinical efficacy.
A total of 114 EMA and 120 FDA TAGs were identified, covering 126 indications. Our core finding is that 75% of EMA TAGs and 58% from the FDA TAGs do not offer any guidance on preclinical efficacy. TAGs varied widely on the extent, nature and detail of guidance.
Guidance on preclinical efficacy in a consistent, comprehensive and explicit way that still allows for justified deviations is an important but neglected aspect of transparency for drug development. This transparency would help sponsors in designing preclinical studies and in negotiating more efficiently with regulators.
EMA 和 FDA 发布的治疗领域指南（TAG）为在特定适应症中规划试验启动提供了指导。我们评估并比较了 EMA 和 FDA 所有可用 TAG 中关于临床前疗效的指导。
搜索了 EMA 和 FDA 网站和数据库中的所有 TAG。应用混合演绎和归纳方法分析和聚类临床前疗效的内容。
共确定了 114 份 EMA 和 120 份 FDA TAG，涵盖 126 种适应症。我们的核心发现是，75%的 EMA TAG 和 58%的 FDA TAG 没有提供任何关于临床前疗效的指导。TAG 在指导的范围、性质和细节上差异很大。
以一致、全面和明确的方式提供临床前疗效指导，同时允许有合理的偏差，这是药物开发透明度的一个重要但被忽视的方面。这种透明度将有助于赞助商设计临床前研究，并更有效地与监管机构进行谈判。
