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.

中文翻译：

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与患者共同开展随机临床试验：硬皮病患者自体造血干细胞移植的案例研究

越来越多的人认为，临床试验很难招募参与者，因为它们没有回答患者愿意或能够使用的关键问题或研究治疗方法。本研究探讨了患者偏好的获取如何帮助随机对照试验 (RCT) 的设计者了解改变治疗或试验设计的可修改方面对招募的影响。采用焦点小组和离散选择实验（DCE）调查来了解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 Logit 模型估计对治疗七个属性（有效性、近期和长期风险、护理团队组成和经验、成本、旅行距离）的偏好，并用于预测随机对照试验的参与情况。278 名硬皮病患者回答了这项调查。所有 AHSCT 治疗属性都会显着影响偏好。治疗效果和晚期并发症的风险对参与者的选择影响最大，但与治疗中心的距离和费用等可改变的因素也会影响偏好。根据最近一项试验的参与情况（33%）调整了预测的招募率，并建议以较低的患者成本提供离家较近的治疗，并采用全面的多学科护理，可以将参与率提高到 51%。通过患者参与的方法来诱导对 AHSCT 治疗方案不同特征的偏好，我们能够预测硬皮病患者参与随机对照试验的决定因素。了解人们关心的问题和愿意做出的权衡可以为临床研究设计提供信息，提高招募率和对感兴趣的治疗的潜在接受率。