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

Abstract

Background 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. Methods 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. Results 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%. Conclusions 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.

The financial burden of accessing care for people with scleroderma in Canada: a patient-oriented, cross-sectional survey

“…We collected information on the out-of-pocket costs incurred by people with scleroderma as part of an international online survey (primarily Canada, the United States, France and the United Kingdom), which aimed to understand the preferences of people with scleroderma for autologous stem-cell transplant treatment (results reported elsewhere). 16 Participants were recruited to an open survey sent to the mailing list of the Scleroderma Patient-centered Intervention Network (SPIN) cohort (a group of people with scleroderma from around the world who participate in SPIN’s online studies) 17 and 2 patient organizations, the Scleroderma Association of British Columbia and the Sclérodermie Québec. Participants were eligible if they reported having a diagnosis of scleroderma.…”

“…The patient-oriented approach in this research study has been described elsewhere. 16 , 22 Briefly, this project began with a conversation between patients, clinicians and researchers, with the aim of understanding how the patient perspective could be elicited and integrated into the design of randomized controlled trials. Two patient partners (T.B., J.B.) were members of the research team and contributed at all stages of the research process.…”