Motivators and barriers affecting decisions to participate in sickle cell disease clinical trials in the global Learning and Insights into Sickle Cell Trial Experiences (LISTEN) Survey: global and regional findings

Background Limited clinical trial awareness, inadequate funding and training of health-care professionals, and inequitable clinical trial participation opportunities hinder improvements in treatment outcomes for people with sickle cell disease. The aim of this study was to inform the design and delivery of future clinical trials to maximise patient recruitment and ensure that future clinical research delivers the best outcomes for the sickle cell disease community. Methods The LISTEN Survey comprised an online questionnaire available in 11 languages and adapted for digital, telephone, or paper completion depending on the region. People with sickle cell disease aged 18 years or older were recruited through patient organisations, health-care professionals, and agencies across 17 countries. Respondents rated and ranked the importance of factors influencing their decision to participate in a clinical trial. Responses were analysed descriptively, with missing data handled using predefined non-response options. Findings Between Oct 6, 2022 and Aug 22, 2023, 1145 people responded to the survey. Of these, 662 (58%) were female and 482 (42%) were male, and 307 (27%) were located in sub-Saharan African. 373 (33%) had been invited to participate in a clinical trial for sickle cell disease, with more limited participation in sub-Saharan Africa (48 [16%] of 307), the Middle East and North Africa (15 [11%] of 142), and India (0 of 78). Motivating factors rated as extremely or very important were the potential to better manage symptoms (573 [50%] of 1145), the opportunity to try a new treatment that might work better (572 [50%] of 1145), and increasing knowledge of sickle cell disease (574 [50%] of 1145). Regional ratings were similar or showed minor differences for questions on potential impact of participation on daily life, but differed for the impact of receiving trial treatment and wider trial impact. The potential for side-effects (587 [51%] of 1145) was an important barrier to clinical trial participation. The most important information people with sickle cell disease wanted to know included planned safety measures (643 [56%] of 1145) and how the treatment works (547 [48%] of 1145). The most important further considerations were the opportunity to speak with experts running the clinical trial (572 [50%] of 1145) and people with sickle cell disease already participating in the clinical trial (562 [49%] of 1145). Interpretation Balanced and tailored communication about clinical trials to the sickle cell disease community should be prioritised to optimise participation opportunities, thereby enhancing representation and generalisability of the results. Funding Novo Nordisk Health Care (Zurich, Switzerland).