Flight Laura, Brennan Alan, Chick Stephen E, Forster Martin, Julious Steven, Tharmanathan Puvan
Sheffield Centre for Health and Related Research (SCHARR), School of Medicine and Population Health, University of Sheffield, Regent Court, 30 Regent Street, Sheffield, S1 4DA, UK.
INSEAD, Technology and Operations Management Area, Fontainebleau, France.
BMC Med Res Methodol. 2025 Jun 5;25(1):153. doi: 10.1186/s12874-025-02566-6.
Value-adaptive designs for clinical trials are a novel set of emerging methods for delivering greater value for clinical research. There is increasing interest in using them within publicly funded health systems. A value-adaptive design permits 'in progress' changes to be made to the trial according to criteria which reflect its overall value to the healthcare system, including the cost-effectiveness of the technologies under investigation, the cost of running the trial and the total health benefit delivered to patients. These trial designs offer the potential to explicitly balance the costs and benefits of adaptive clinical trials with the health economic benefits expected for populations that are affected by any subsequent health technology adoption decisions. They may also improve the expected value of learning from the budget that is spent within a trial.
This paper introduces value-adaptive designs for publicly funded clinical trials. It discusses the idea of delivering 'value for money' in health technology assessment, what is meant by being 'value-adaptive' and the key features that characterise these designs. The methodology behind one kind of value-adaptive design - the value-based sequential model of a two-armed clinical trial proposed by Chick et al. (2017) - is described and illustrated using three retrospective case studies from the United Kingdom. The paper concludes by reviewing a range of perspectives provided by stakeholders, together with our own thoughts, on the practical opportunities and changes required for implementing a value-adaptive approach.
Value-adaptive clinical trial designs offer the potential to align health research funding allocations with population health economic goals. Many of the systems required to deploy value-adaptive designs within a publicly funded health system already exist and, with increased application, experience, and refinement they have the potential to deliver improved value for money.
临床试验的价值适应性设计是一组新兴的方法，旨在为临床研究带来更大价值。在公共资助的卫生系统中，人们对使用这些方法的兴趣与日俱增。价值适应性设计允许根据反映其对医疗系统总体价值的标准对试验进行“进行中”的更改，这些标准包括所研究技术的成本效益、试验运行成本以及带给患者的总体健康益处。这些试验设计有可能明确地平衡适应性临床试验的成本和效益与受后续任何卫生技术采用决策影响的人群预期的健康经济效益。它们还可能提高从试验所花费预算中学习的预期价值。
本文介绍了公共资助临床试验的价值适应性设计。它讨论了在卫生技术评估中实现“物有所值”的理念、“价值适应性”的含义以及这些设计的关键特征。描述并通过来自英国的三个回顾性案例研究说明了一种价值适应性设计背后的方法——Chick等人（2017年）提出的双臂临床试验的基于价值的序贯模型。本文最后回顾了利益相关者提供的一系列观点以及我们自己的想法，涉及实施价值适应性方法所需的实际机会和变革。
价值适应性临床试验设计有可能使卫生研究资金分配与人群健康经济目标保持一致。在公共资助的卫生系统中部署价值适应性设计所需的许多系统已经存在，并且随着应用、经验的增加和完善，它们有可能带来更高的性价比。
