健康经济学如何用于自适应临床试验的设计和分析？定性分析。
How can health economics be used in the design and analysis of adaptive clinical trials? A qualitative analysis.
机构信息
School of Health And Related Research, University of Sheffield, Sheffield, UK.
Department of Mathematics and Statistics, University of Reading, Reading, UK.
出版信息
Trials. 2020 Mar 6;21(1):252. doi: 10.1186/s13063-020-4137-2.
INTRODUCTION
Adaptive designs offer a flexible approach, allowing changes to a trial based on examinations of the data as it progresses. Adaptive clinical trials are becoming a popular choice, as the prudent use of finite research budgets and accurate decision-making are priorities for healthcare providers around the world. The methods of health economics, which aim to maximise the health gained for money spent, could be incorporated into the design and analysis of adaptive clinical trials to make them more efficient. We aimed to understand the perspectives of stakeholders in health technology assessments to inform recommendations for the use of health economics in adaptive clinical trials.
METHODS
A qualitative study explored the attitudes of key stakeholders-including researchers, decision-makers and members of the public-towards the use of health economics in the design and analysis of adaptive clinical trials. Data were collected using interviews and focus groups (29 participants). A framework analysis was used to identify themes in the transcripts.
RESULTS
It was considered that answering the clinical research question should be the priority in a clinical trial, notwithstanding the importance of cost-effectiveness for decision-making. Concerns raised by participants included handling the volatile nature of cost data at interim analyses; implementing this approach in global trials; resourcing adaptive trials which are designed and adapted based on health economic outcomes; and training stakeholders in these methods so that they can be implemented and appropriately interpreted.
CONCLUSION
The use of health economics in the design and analysis of adaptive clinical trials has the potential to increase the efficiency of health technology assessments worldwide. Recommendations are made concerning the development of methods allowing the use of health economics in adaptive clinical trials, and suggestions are given to facilitate their implementation in practice.
简介
适应性设计提供了一种灵活的方法，允许根据试验进行过程中数据的检查来改变试验。适应性临床试验正成为一种流行的选择，因为谨慎使用有限的研究预算和准确的决策是全球医疗保健提供者的优先事项。旨在使花费的金钱获得最大健康收益的健康经济学方法，可以纳入适应性临床试验的设计和分析中，以提高其效率。我们旨在了解健康技术评估利益相关者的观点，为在适应性临床试验中使用健康经济学提供建议。
方法
一项定性研究探讨了关键利益相关者（包括研究人员、决策者和公众成员）对在适应性临床试验的设计和分析中使用健康经济学的态度。使用访谈和焦点小组（29 名参与者）收集数据。使用框架分析来识别转录本中的主题。
结果
参与者认为，尽管成本效益对决策很重要，但在临床试验中应优先回答临床研究问题。参与者提出的担忧包括在中期分析中处理成本数据的波动性；在全球试验中实施这种方法；为基于健康经济结果设计和调整的适应性试验提供资源；并对这些方法进行培训，以便能够实施并进行适当解释。
结论
在适应性临床试验的设计和分析中使用健康经济学有可能提高全球健康技术评估的效率。就允许在适应性临床试验中使用健康经济学的方法的发展提出了建议，并就其在实践中的实施提出了建议。
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