Voluntary exploratory data submissions to the US FDA and the EMA: experience and impact
Voluntary exploratory data submissions to the US FDA and the EMA: experience and impact
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DOI：
10.1038/nrd3116
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发表时间：
2010-06-01
影响因子：
120.1
通讯作者：
Zineh, Issam
中科院分区：
文献类型：
--
作者：
Goodsaid, Federico M.;Amur, Shashi;Zineh, Issam
疾病过程的潜在机制中的异质性和药物反应的患者间变异性是药物开发中的主要挑战。为了应对这些挑战，基于一系列平台的生物标志物策略，如微阵列基因表达技术，越来越多地被应用于阐明这些变异性来源，从而可能提高药物开发的成功率。为了提高监管机构和申办者对此类生物标志物数据的监管意义的理解，美国食品药品监督管理局于2004年启动了一项计划，允许申办者自愿提交探索性基因组数据，而不会立即产生监管影响。在这篇文章中，从该计划的前5年选择的案例研究-这是现在被称为自愿探索性数据提交计划，并涉及与欧洲药品管理局的合作-进行了讨论，并强调了一般的经验教训。
Heterogeneity in the underlying mechanisms of disease processes and inter-patient variability in drug responses are major challenges in drug development. To address these challenges, biomarker strategies based on a range of platforms, such as microarray gene-expression technologies, are increasingly being applied to elucidate these sources of variability and thereby potentially increase drug development success rates. With the aim of enhancing understanding of the regulatory significance of such biomarker data by regulators and sponsors, the US Food and Drug Administration initiated a programme in 2004 to allow sponsors to submit exploratory genomic data voluntarily, without immediate regulatory impact. In this article, a selection of case studies from the first 5 years of this programme - which is now known as the voluntary exploratory data submission programme, and also involves collaboration with the European Medicines Agency - are discussed, and general lessons are highlighted.