Motivators and Barriers for People with Sickle Cell Disease Participating in Clinical Trials: United States Findings from the Listen Survey

Introduction: Recruitment and retention of a large and broad group of participants is imperative for the success of clinical trials and the development of new therapies for sickle cell disease (SCD). The Learnings and Insights into Sickle Cell Trial Experiences (LISTEN) Survey was developed to deepen understanding of the motivators and barriers in the recruitment of people with SCD (PwS) for clinical trials. This sub-analysis focuses on the results from the US.

Methods: A diverse group of adults (≥18 years) with SCD and health care professionals (HCPs) involved in the treatment and/or clinical research of SCD from 17 countries completed a quantitative survey between Oct 2022 and June 2023. PwS rated and ranked the importance of specified factors to consider when deciding whether to participate in a clinical trial, and HCPs rated and ranked their perceptions of PwS beliefs on those factors. Factors were grouped into 5 categories (impact on daily life, treatment impact, trial outcomes, trial information, and other sources of information and influence), rated on a 7-point scale from “not at all” to “extremely important,” and ranked from most to least important. The perspectives of PwS and HCPs were compared, and the results presented include the total proportion of respondents who rated factors as “very” or “extremely” important and the factors that were ranked within the top two in each category.

Results: Of 1145 PwS who completed the global LISTEN survey, 193 were from the US (USPwS). Of 193 USPwS, 67% were female with a mean age of 33 years. Almost all USPwS expressed being negatively impacted by SCD, and only 5% reported the disease does not limit them at all. Of USPwS, 40% had taken part in clinical trials for SCD, and the majority had a positive experience. Of 361 global HCP respondents, 76 were from the US (USHCP) with an average of 21 years of experience treating PwS. Of USHCPs, 43% had taken part in clinical trials for SCD, and the majority had a positive experience. For factors relating to impacts on daily life, both USPwS and USHCPs ranked highly the “potential to miss school/work or lose income” (44% USPwS, 45% USHCPs) and “additional travel requirements” (40% USPwS, 49% USHCPs); however, a significant difference was noted on the importance of the “potential to see a SCD specialist more often” (41% USPwS v 29% USHCPs, P=0.067) as “very” or “extremely” important. For factors relating to treatment impact, both USPwS and USHCPs rated the “potential to better manage their symptoms” (56% USPwS, 55% USHCPs) and “opportunity to try a therapy that might work better” (53% USPwS, 51% USHCPs) as “very” or “extremely” important. However, USHCPs significantly underestimated “the trial treatment might have different side effects than their current treatment” (54% USPwS v 32% USHCPs, P=0.001), which was the second most important factor for patients in absolute terms. For factors relating to wider clinical trial outcomes, compared to USPwS responses, USHCPs significantly underestimated the importance of all 5 categories: “feeling that they are supporting new treatment developments that may benefit them” (62% v 22%) “or benefit others” (62% v 22%), “to increase self-knowledge about SCD” (63% v 22%), “the opportunity to receive their own data” (51% v 21%), and “the possibility of receiving treatment regularly after the trial” (47% v 33%). For sources of information, USPwS identified that “HCPs” were the most common channel through which they learn about clinical trials; however, “other PwS” (52%) was their most trusted source. USHCPs also reported referring patients mainly to the “pharmaceutical company sponsoring the trial” (54%) or to “hospitals/medical centers” for SCD trial information (47%), as opposed to referring patients to “other PwS” (18%), whom USPwS trust the most.

Conclusions: Findings from the US sub-analysis of the LISTEN Survey highlighted not only areas of absolute and relative importance of different barriers and motivators, but also areas of significant disconnect between the perspectives of USPwS and USHCPs. USPwS responses suggested USHCPs should place greater emphasis on wider clinical trial outcomes and more deeply discuss potential side effects, travel requirements, and time missed from school/work. These insights can be used to improve the delivery of clinical trial information to enhance patient recruitment and ensure that future clinical research outcomes benefit the needs of the SCD community.