Faculty of Pharmaceutical Sciences, University of British Columbia, 4625-2405 Wesbrook Mall, Vancouver, BC, V6T 1Z3, Canada.
Centre for Health Economics Research and Evaluation, University Technology Sydney, Sydney, NSW, Australia.
Trials. 2021 Sep 9;22(1):611. doi: 10.1186/s13063-021-05575-0.
Increasingly, it is argued that clinical trials struggle to recruit participants because they do not respond to key questions or study treatments that patients will be willing or able to use. This study explores how elicitation of patient-preferences can help designers of randomized controlled trials (RCTs) understand the impact of changing modifiable aspects of treatments or trial design on recruitment.
Focus groups and a discrete choice experiment (DCE) survey were used to elicit preferences of people with scleroderma for autologous hematopoietic stem cell transplant (AHSCT) treatment interventions. Preferences for seven attributes of treatment (effectiveness, immediate and long-term risk, care team composition and experience, cost, travel distance) were estimated using a mixed-logit model and used to predict participation in RCTs.
Two hundred seventy-eight people with scleroderma answered the survey. All AHSCT treatment attributes significantly influenced preferences. Treatment effectiveness and risk of late complications contributed the most to participants' choices, but modifiable factors of distance to treatment center and cost also affected preferences. Predicted recruitment rates calibrated with participation in a recent trial (33%) and suggest offering a treatment closer to home, at lower patient cost, and with holistic, multidisciplinary care could increase participation to 51%.
Through a patient engaged approach to preference elicitation for different features of AHSCT treatment options, we were able to predict what drives the decisions of people with scleroderma to participate in RCTs. Knowledge regarding concerns and the trade-offs people are willing to make can inform clinical study design, improving recruitment rates and potential uptake of the treatment of interest.
越来越多的人认为，临床试验难以招募参与者，因为它们无法回答关键问题或研究治疗方法，而这些问题或治疗方法是患者愿意或能够使用的。本研究探讨了如何通过引出患者偏好，帮助随机对照试验（RCT）的设计者了解改变治疗方法或试验设计中可修改的方面对招募的影响。
使用焦点小组和离散选择实验（DCE）调查来引出硬皮病患者对自体造血干细胞移植（AHSCT）治疗干预的偏好。使用混合对数模型估计治疗的七个属性（有效性、即刻和长期风险、护理团队组成和经验、成本、旅行距离）的偏好，并用于预测 RCT 的参与度。
278 名硬皮病患者回答了调查。AHSCT 治疗的所有属性都显著影响了偏好。治疗效果和晚期并发症的风险对参与者的选择贡献最大，但治疗中心的距离和成本等可修改因素也影响了偏好。预测的招募率与最近一项试验的参与率（33%）相匹配，并表明提供离家更近、患者成本更低、采用整体多学科护理的治疗方法可能会将参与率提高到 51%。
通过对 AHSCT 治疗选择的不同特征进行患者参与式偏好引出，我们能够预测是什么驱动硬皮病患者决定参与 RCT。了解人们的关注问题和愿意做出的权衡取舍，可以为临床研究设计提供信息，提高招募率和潜在的治疗接受率。
