Kruizinga Matthijs D, Stuurman Frederik E, Groeneveld Geert J, Cohen Adam F
Centre for Human Drug Research, Leiden, The Netherlands.
Juliana Children's Hospital, HAGA Teaching Hospital, The Hague, The Netherlands.
Handb Exp Pharmacol. 2019;260:371-397. doi: 10.1007/164_2019_302.
Clinical trials have been conducted since 500 BC. Currently, the methodological gold standard is the randomized controlled clinical trial, introduced by Austin Bradford Hill. This standard has produced enormous amounts of high-quality evidence, resulting in evidence-based clinical guidelines for physicians. However, the current trial paradigm needs to evolve because of the ongoing decrease of the incidence of hard endpoints and spiraling trial costs. While new trial designs, such as adaptive clinical trials, may lead to an increase in efficiency and decrease in costs, we propose a shift towards value-based trial design: a paradigm that mirrors value-based thinking in business and health care. Value-based clinical trials will use technology to focus more on symptoms and endpoints that patients care about, will incorporate fewer research centers, and will measure a state or consequence of disease at home or at work. Furthermore, they will measure the subjective experience of subjects in relation to other objective measurements. Ideally, the endpoints are suitable for individual assessment of the effect of an intervention. The value-based clinical trial of the future will have a low burden for participants, allowing for the inclusion of neglected populations such as children and the elderly, will be data-rich due to a high frequency of measurements, and can be conducted with technology that is already available.
自公元前500年起就开始进行临床试验。目前，方法学的金标准是由奥斯汀·布拉德福德·希尔引入的随机对照临床试验。这一标准产生了大量高质量证据，从而形成了针对医生的循证临床指南。然而，由于硬终点事件发生率持续下降以及试验成本不断攀升，当前的试验模式需要改进。虽然新的试验设计，如适应性临床试验，可能会提高效率并降低成本，但我们建议转向基于价值的试验设计：一种反映商业和医疗保健领域基于价值思维的模式。基于价值的临床试验将利用技术，更多地关注患者关心的症状和终点，减少研究中心的数量，并在家庭或工作场所测量疾病的状态或后果。此外，它们将在与其他客观测量相关的情况下测量受试者的主观体验。理想情况下，这些终点适用于对干预效果的个体评估。未来基于价值的临床试验对参与者的负担较小，能够纳入儿童和老年人等被忽视的人群，由于测量频率高而数据丰富，并且可以使用现有的技术进行。
