Preclinical efficacy in therapeutic area guidelines from the US Food and Drug Administration and the European Medicines Agency: a cross-sectional study
Preclinical efficacy in therapeutic area guidelines from the US Food and Drug Administration and the European Medicines Agency: a cross-sectional study
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DOI：
10.1111/bph.14485
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发表时间：
2018-11-01
影响因子：
7.3
通讯作者：
Strech, Daniel
中科院分区：
文献类型：
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作者：
Langhof, Holger;Chin, William Wei Lim;Strech, Daniel
背景和结论EMA和FDA发布的治疗领域指南（TAG）为计划启动特定适应症的试验提供了指南。我们评估并比较了EMA和FDA所有可用TAG的临床前疗效指南。实验方法和FDA网站和数据库中搜索所有TAG。采用演绎和归纳相结合的方法对临床前有效性内容进行分析和聚类。确定了114个EMA和120个FDA TAG，涵盖126个适应症。我们的核心发现是，75%的EMA TAG和58%的FDA TAG没有提供任何临床前疗效指导。TAG的程度，性质和细节的guidance.CONCLUSIONS和IMPLICATIONS指导临床前疗效的一致，全面和明确的方式，仍然允许合理的偏差是一个重要的，但被忽视的方面，药物开发的透明度。这种透明度将有助于申办者设计临床前研究，并更有效地与监管机构进行谈判。
BACKGROUND AND PURPOSETherapeutic 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.EXPERIMENTAL APPROACHEMA 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.KEY RESULTSA 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.CONCLUSIONS AND IMPLICATIONSGuidance 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.