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首页> 外文期刊>Rheumatology >Are head-to-head trials of biologics needed? The role of value of information methods in arthritis research.
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Are head-to-head trials of biologics needed? The role of value of information methods in arthritis research.

机译:是否需要进行生物制剂的面对面试验?信息方法的价值在关节炎研究中的作用。

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摘要

Reimbursement decisions are typically based on cost-effectiveness analyses. While a cost-effectiveness analysis can identify the optimum strategy, there is usually some degree of uncertainty around this decision. Sources of uncertainty include statistical sampling error in treatment efficacy measures, underlying baseline risk, utility measures and costs, as well as uncertainty in the structure of the model. The optimal strategy is therefore only optimal on average, and a decision to adopt this strategy might still be the wrong decision if all uncertainty could be eliminated. This means that there is a quantifiable expected (average) loss attaching to decisions made under uncertainty, and hence a value in collecting information to reduce that uncertainty. Value of information (VOI) analyses can be used to provide guidance on whether more research would be cost-effective, which particular model inputs (parameters) have the most bearing on decision uncertainty, and can also help with the design and sample size of further research. Here, we introduce the key concepts in VOI analyses, and highlight the inputs required to calculate it. The adoption of the new biologic treatments for RA and PsA tends to be based on placebo-controlled trials. We discuss the possible role of VOI analyses in deciding whether head-to-head comparisons of the biologic therapies should be carried out, illustrating with examples from other fields. We emphasize the need for a model of the natural history of RA and PsA, which reflects a consensus view.
机译:报销决策通常基于成本效益分析。虽然成本效益分析可以确定最佳策略,但此决策通常存在一定程度的不确定性。不确定性的来源包括治疗功效度量,潜在基准风险,效用度量和成本的统计抽样误差,以及模型结构的不确定性。因此,最优策略仅是平均最优值,并且如果可以消除所有不确定性,则采用该策略的决策可能仍然是错误的决策。这意味着在不确定性条件下做出的决策中存在可量化的预期(平均)损失,因此在收集信息以减少不确定性方面具有价值。信息价值(VOI)分析可用于为更多的研究是否具有成本效益提供指导,哪些特定模型输入(参数)对决策不确定性的影响最大,并且还可以帮助进一步研究设计和样本量研究。在这里,我们介绍了VOI分析中的关键概念,并强调了计算所需的输入。 RA和PsA的新生物疗法的采用往往基于安慰剂对照试验。我们讨论了VOI分析在决定是否应该进行生物学治疗的头对头比较中可能发挥的作用,并举例说明了其他领域。我们强调需要建立RA和PsA的自然史模型,以反映共识。

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