Department of Quantitative Health Sciences, Mayo Clinic, Rochester, MN.
Department of Quantitative Health Sciences, Mayo Clinic, Rochester, MN.
Mayo Clin Proc. 2023 Aug;98(8):1241-1253. doi: 10.1016/j.mayocp.2023.04.013.
Clinical trials have been the bedrock of research to evaluate the safety and efficacy of new medical, surgical, or other interventions. Traditional "explanatory" clinical trials have aimed to explain a biological cause (new treatment) and effect (patient outcome) while controlling for many factors that might impact the evaluation, such as restricted eligibility criteria, frequent follow-up visits, and multiple clinical and laboratory measures. Despite the benefits of a well-controlled clinical trial, compromises have been made that can limit who might benefit from a new intervention, can increase complexity of the conduct of a trial, or that lead to excessively long durations of trials. An alternative approach to evaluate the effectiveness of an intervention is based on "pragmatic" clinical trials, which consider how an intervention affects a patient's condition in the real world, accounting for how to optimize an intervention within the operations of busy and diverse clinical practices. Although we describe explanatory and pragmatic trial designs as separate approaches, there is a continuum of approaches that intersect. Some key points are the need to maintain scientific rigor, increase efficiency of clinical trials operations, ensure that trial results can be generalized to a broad spectrum of patients, and balance the needs of real-world clinical care. Pragmatic trials can leverage technology and telecommunication strategies of decentralized trials to further reach underrepresented and underserved patients to close the health disparity gaps.
临床试验一直是评估新的医疗、手术或其他干预措施的安全性和疗效的基础。传统的“解释性”临床试验旨在解释生物学原因（新的治疗方法）和效果（患者结果），同时控制许多可能影响评估的因素，如限制资格标准、频繁的随访和多项临床和实验室措施。尽管有精心控制的临床试验的好处，但也做出了一些妥协，这些妥协可能会限制新干预措施的受益人群，增加试验实施的复杂性，或者导致试验持续时间过长。另一种评估干预措施有效性的方法是基于“实用”临床试验，它考虑了干预措施如何在现实世界中影响患者的病情，同时考虑了如何在繁忙和多样化的临床实践中优化干预措施。虽然我们将解释性和实用性试验设计描述为两种独立的方法，但实际上它们是一个连续的过程。一些关键点是需要保持科学严谨性、提高临床试验操作的效率、确保试验结果可以推广到广泛的患者群体，并平衡现实世界临床护理的需求。实用性试验可以利用分散试验的技术和电信策略，进一步接触代表性不足和服务不足的患者，缩小健康差距。
