Co-production of randomized clinical trials with patients: a case study in autologous hematopoietic stem cell...

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.