Bridging the gap: Global caregiver perspectives on awareness, motivators, and barriers to clinical trial participation in sickle cell disease

Abstract

Abstract ”≥Background: The generation of robust and widely applicable data from clinical trials for sickle cell disease (SCD) depends on recruiting a large, demographically diverse participant cohort. For many people with SCD (PwSCD), especially children and adolescents, the decision to participate in a clinical trial is taken collaboratively with their caregivers and healthcare professionals (HCPs). The global Learnings and Insights into Sickle Cell Trial Experiences (LISTEN) Survey investigated attitudes toward clinical trial participation among PwSCD, caregivers, and HCPs, and identified key motivators and barriers affecting decisions to participate (James J et al. Blood 2023;142[Suppl 1]:2498). Here we present global data from the caregiver respondents to the LISTEN Survey. Methods: Between October 6, 2022 and August 22, 2023, caregivers from 17 countries completed a structured quantitative survey. Eligible participants were ≥18 years old, supported PwSCD aged ≥12 years, held advisory or decision-making roles regarding treatment, and were not formally compensated caregivers. The survey assessed caregivers' awareness of clinical trials, prior trial participation by the person they care for, caregiver willingness for the person they care for to participate, and key decision-making drivers. Caregivers rated the importance of potential motivators and barriers on a 7-point scale (from not at all to extremely important) and ranked preferred types of trial information. Results: A total of 199 caregivers, with a median age of 39 (interquartile range [IQR]: 33–45) years, completed the LISTEN Survey. Most caregivers were female (78%), parents/‌guardians (75%), and cared for one person with SCD (80%). The median age of the person they cared for was 16 (IQR: 12–21) years. Most caregivers supported the person they care for “every time” or “most of the time” when meeting HCPs (72%), going to medical appointments (71%), taking their treatment (68%), and deciding what treatment to follow (78%). Overall, 43% of caregivers lacked awareness of clinical trials (had not heard of them or did not know what they were), and awareness varied by geographic region. Lack of awareness was prevalent in India (n=12/12; 100%), Middle East/North Africa (MENA; n=20/29; 69%), and Sub-Saharan Africa (SSA; n=34/56; 61%) in contrast to South America (n=7/23; 30%), Europe (n=9/43; 21%), and North America (n=4/36; 11%). For caregivers who had heard of clinical trials (n=147), awareness came from a variety of information sources, including HCPs (16%), medical centers (15%), and browsing the internet (14%). More than half (54%) of all caregivers were likely or very likely to want the person they care for to participate in a clinical trial for SCD. Despite this, only 19% reported the person with SCD had participated, which comprised 30/113 (27%) caregivers who knew a little or a lot about clinical trials and 7/86 (8%) who lacked awareness. A key motivator that caregivers deemed extremely or very important when helping the person with SCD to decide whether to take part in a clinical trial was the potential to better manage symptoms (54%), and a key barrier was the trial treatment having different side effects than currently experienced (54%). Caregivers most frequently ranked the safety measures in place (45%) and how the treatment would improve patients' lives (42%) as the most important factors (first or second of eight) to know about the trial, and most frequently ranked speaking to experts who are running the trial (53%) as the most important action (first or second of five) once they received trial information. Conclusions: Caregivers, primarily of adolescents and young adults with SCD, expressed great interest in clinical trial participation but reported low enrollment. Limited caregiver awareness of clinical trials, particularly in India, MENA, and SSA, may be a barrier. Enhancing caregiver engagement through targeted, multichannel educational strategies that highlight symptom management potential and safety profiles could improve uptake of clinical trial opportunities by PwSCD.