How People with Sickle Cell Disease Rate Motivators is Associated with the Likelihood of Wanting to Participate in a Clinical Trial: Findings from the Global LISTEN Survey
Authors: Andemariam Biree , Mahlangu Johnny , Colombatti Raffaella , Waller John , Al-Behaisi Samar , Morrell Gareth , James John 
Published: December 2024

Background: The success of clinical trials for sickle cell disease (SCD) and global generalizability of the results rely on securing a sufficiently large and diverse participant pool. The global Learnings and Insights into Sickle Cell Trial Experiences (LISTEN) Survey investigated the attitudes towards SCD clinical trial participation among people with SCD (PwSCD). The survey identified several motivators and barriers affecting decisions to participate in clinical trials. The importance of individualized communication to improve clinical trial access and recruitment was a key finding of the LISTEN Survey (James J et al. Blood 2023;142[Suppl 1]:2498).

Aim: In this LISTEN Survey analysis, we investigate how demographics, characteristics, and factors representing the experiences and attitudes of PwSCD may impact the likelihood of wanting to participate in an SCD clinical trial.

Methods: Between October 2022 and August 2023, adults with SCD (≥18 years old) from 17 countries completed a quantitative survey. The survey assessed the importance of motivators (e.g., better manage symptoms or increase knowledge of SCD) and barriers (e.g., different side effects or missing work/school) that affect decisions to participate in a clinical trial by rating them on a 7-point scale. Using regression analysis, we investigated the association between contextual factors and responses to the survey question, “In general, how likely do you think you are to want to take part in a clinical trial for SCD?” (rated on a 5-point scale from very unlikely to very likely). Contextual factors included demographics and characteristics such as gender, living location, having dependents, life satisfaction, financial situation, and treatment preferences. Additional contextual factors were derived from factor analysis, grouping responses to multiple questions into categories including importance of motivators, importance of barriers, impact of symptoms on daily life, frequency of receiving SCD care and information, frequency of pain management, trust and relationship with healthcare professionals (HCPs), frequency of interaction with the SCD community, and usefulness of SCD information. Key contextual factors identified from the regression analysis were analyzed further.

Results: A total of 1,145 PwSCD (58% female) with a median age of 30 years (interquartile range: 24-38) participated in the LISTEN Survey. Of these, 67% (n=772) had never been invited to participate in an SCD clinical trial. When PwSCD were asked how likely they would be to want to participate in a clinical trial, 13% of respondents (n=146) were very unlikely, 10% (n=118) were unlikely, 23% (n=269) were neither likely nor unlikely, 36% (n=408) were likely, and 18% were (n=204) very likely. Regression analysis revealed that how PwSCD rated the importance of motivators had the greatest association with the likelihood of wanting to participate in a clinical trial (standardized regression coefficient [β]=0.38; p&lt;0.01). Other positively associated contextual factors were frequent interaction with the SCD community (β=0.12), living in a rural location (β=0.11), preferring to receive a different treatment (β=0.09), and frequently receiving SCD care and information (β=0.08; all p&lt;0.01). How PwSCD rated the importance of barriers was not associated. Reporting a comfortable financial situation was negatively associated with the likelihood of wanting to participate in a clinical trial (β=−0.08; p&lt;0.05). Further analysis indicated that the importance of certain motivators was higher among those educated to at least university degree level than those not educated to university degree level. Few differences in the importance of motivators were observed based on household income and rural living location.

Conclusions: HCPs, industry and patient organizations should be aware that individuals' perceptions of the positive aspects of clinical trials in SCD may affect their decision to participate. This awareness is crucial, as 23% of PwSCD who responded to the LISTEN Survey were undecided about participating in a clinical trial, highlighting a need to raise awareness and understanding of clinical trials. Ensuring clinical trial opportunities are accessible to all PwSCD and understanding the reasons that motivate individuals may improve participation in SCD clinical trials.