School of Social and Community Medicine, University of Bristol, Canynge Hall, Whatley Road, Bristol, UK.
Rheumatology (Oxford). 2011 Sep;50 Suppl 4:iv19-25. doi: 10.1093/rheumatology/ker242.
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.
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，但该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效果测量中的统计抽样误差、潜在的基线风险、效用测量和成本，以及模型结构的不确定性。因此，最佳策略只是平均最优，如果能够消除所有不确定性，那么采用该策略可能仍然是错误的决策。这意味着在不确定情况下做出的决策会带来可量化的预期（平均）损失，因此收集信息以降低不确定性是有价值的。信息价值（VOI）分析可用于提供指导，以确定更多的研究是否具有成本效益，哪些特定的模型输入（参数）对决策不确定性影响最大，并有助于进一步研究的设计和样本量。在这里，我们介绍 VOI 分析的关键概念，并强调计算它所需的输入。新的生物疗法在 RA 和 PsA 中的应用往往基于安慰剂对照试验。我们讨论了在决定是否应该进行生物疗法的头对头比较时使用 VOI 分析的可能作用，并用其他领域的例子来说明。我们强调需要建立一个反映共识观点的 RA 和 PsA 自然史模型。
