Sensible approaches for reducing clinical trial costs
Sensible approaches for reducing clinical trial costs
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DOI：
10.1177/1740774507087551
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发表时间：
2008-01-01
期刊：
影响因子：
2.7
通讯作者：
Diaz, Rafael
中科院分区：
文献类型：
--
作者：
Eisenstein, Eric L.;Collins, Rory;Diaz, Rafael
背景在过去的十年中，每年用于生物医学研究的资金增加了一倍以上，而新的分子实体批准却下降了三分之一。目的评估大规模临床试验中常用的实践的价值，并确定在不影响科学有效性的情况下可以降低成本的领域。一个专家小组建议对大型试验的设计和操作进行可能的修改，以最大限度地发挥其价值（成本与科学效益的权衡）。在定量阶段，使用大型试验经济模型来评估这些建议的财务影响。我们最初的慢性病试验设计包括在1000个地点随机分配的20，000名患者。每个研究中心分配24次监查访视，每个患者研究中心支付10，000美元。病例报告表（CRF）是60页长，试验持续时间被假定为48 months.Results的初始试验设计的总成本为4.21亿美元（2007年美元）。根据专家小组的建议，我们改变了研究持续时间、CRF长度、研究中心数量、电子数据采集（EDC）和研究中心管理组件，以确定其对总试验成本的单独和综合影响。EDC的使用和改良的研究中心管理实践与总试验成本的显著降低相关。当所有五个试验组成部分的减少结合在一个精简的制药工业设计中时，总试验成本降低了59%。当我们假设一个更精简的试验设计比通常被认为是在过去的监管提交，有一个90%的减少，在总的试验costs.Conclusion我们的研究结果表明，这是可能的，以大幅降低成本的大规模临床试验，而不影响其结果的科学有效性。如果实施，我们的建议每年可以节省数十亿美元用于额外的临床研究。应在临床试验的背景下进行研究，以完善这些发现。
Background Over the past decade, annual funding for biomedical research has more than doubled while new molecular entity approvals have declined by one third.Objective To assess the value of practices commonly employed in the conduct of large-scale clinical trials, and to identify areas where costs could be reduced without compromising scientific validity.Methods In the qualitative phase of the study, an expert panel recommended potential modifications of mega-trial designs and operations in order to maximize their value (cost versus scientific benefit tradeoff). In the quantitative phase, a mega-trial economic model was used to assess the financial implications of these recommendations. Our initial chronic disease trial design included 20,000 patients randomized at 1000 sites. Each site was assigned 24 monitoring visits and a $10,000 per patient site payment. The case report form (CRF) was 60 pages long, and trial duration was assumed to be 48 months.Results The total costs of the initial trial design were $421 million ($US 2007). Following the expert panel's recommendations, we varied study duration, CRF length, number of sites, electronic data capture (EDC), and site management components to determine their individual and combined effects upon total trial costs. The use of EDC and modified site management practices were associated with significant reductions in total trial costs. When reductions in all five trial components were combined in a streamlined pharmaceutical industry design, a 59% reduction in total trial costs resulted. When we assumed an even more streamlined trial design than has typically been considered for regulatory submissions in the past, there was a 90% reduction in total trial costs.Conclusion Our results suggest that it is possible to reduce substantially the cost of large-scale clinical trials without compromising the scientific validity of their results. If implemented, our recommendations could free billions of dollars annually for additional clinical studies. Research in the setting of clinical trials should be conducted to refine these findings.