Patient Motivators Key for Participation in SCD Clinical Trials Free

During a poster session at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition, Biree Andemariam, MD, a hematologist and founding director of the New England Sickle Cell Institute at the University of Connecticut Health Center, presented findings on different motivators that influence whether people with sickle cell disease (SCD) choose to participate in clinical trials.^1,2

Patient and physician perceptions of the potential value of clinical trials can vary substantially. Individuals with SCD can be motivated or deterred from joining trials due to many factors, which can negatively affect patient recruitment and retention. Previous studies have identified barriers such as overly demanding study requirements, general lack of knowledge about clinical trials, worries about potential health effects, and an overall lack of trust in health care systems.^3,4

To gain more insight into these perceptions, experts designed the Learning and Insights in Sickle Cell Trial Experiences (LISTEN) survey. This included 1,145 adults with SCD from 17 countries, as well as 361 SCD health care providers, who gave their perceptions of patients’ motivations. The survey used a 7-point scale to assess the importance of motivators to people with SCD, for example, better management of symptoms, or barriers like side effects or missing work. Interim results were presented at the 2023 ASH annual meeting, but Dr. Andemariam expanded on this with more complete analyses.^1,2,5

Of the global SCD survey patient participants, 23% reported that they were either unlikely (10%) or very unlikely (13%) to participate in a clinical trial. In contrast, 54% were either likely (36%) or very likely (18%) to participate if invited, and 23% were undecided.¹

Using regression analyses, the team showed that survey participants’ ratings of the importance of various motivators on the 7-point scale had an association with their likelihood of wanting to participate in a clinical trial (standardized regression coefficient [β] = 0.38; p<0.01). They also identified additional positively associated contextual factors, such as frequent interactions 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; p<0.01 for all factors).¹

“Motivators to participating in clinical trials and SCD may have a greater impact on individuals’ willingness to participate than potential barriers,” Dr. Andemariam said. “Because these positive aspects of clinical trials appear to resonate with most people with SCD, health care providers, industry, and patient organizations should consider this when implementing initiatives to increase awareness and understanding of clinical trials.”

During a second poster presentation, Dr. Ande­mariam reported specifically on results from the U.S. subanalysis, which included 193 people with SCD, 40% of whom had taken part in a clinical trial for SCD, as well as 76 U.S. SCD health care providers.²

Although patient and provider perceptions of barriers were aligned in many categories, such as the potential effects of travel requirements or missed school or work, some differed. For example, when it came to being able to see a SCD provider more often, patients were more likely to rate this as very or extremely important (41% of patients vs. 29% of providers; p=0.067). Providers also underestimated the importance of the patients’ perception of different side effects from their trial treatment (ranked as very or extremely important by 54% of patients vs. 32% of providers; p=0.001).²

The survey identified “other people with SCD” as the channel that people with SCD most trusted to receive information about clinical trials (52% fully trust). “Yet health care providers reported referring patients mainly to the pharmaceutical companies sponsoring the trial [54%] or to hospitals or medical centers [47%] for SCD trial information, as opposed to referring patients to people with SCD,” said Dr. Andemariam. “This disconnect is a key insight into the recruitment of patients for participation in clinical trials.”

Any conflicts of interest declared by the authors can be found in the original abstract.

References

1. Andemariam B, Mahlangu J, Colombatti R, et al. 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. Abstract 1137. Presented at the 66th American Society of Hematology Annual Meeting and Exposition; December 7, 2024; San Diego, California.
2. Andemariam B, Wufus A, Hecht E, et al. Motivators and barriers for people with sickle cell disease participating in clinical trials: United States findings from the LISTEN survey. Abstract 1139. Presented at the 66th American Society of Hematology Annual Meeting and Exposition; December 7, 2024; San Diego, California.
3. Patterson CA, Chavez V, Mondestin V, et al. Clinical trial decision making in pediatric sickle cell disease: a qualitative study of perceived benefits and barriers to participation. J Pediatr Hematol Oncol. 2015;37(6):415-422.
4. Lee LH, Whisenton LH, Benger J, et al. A community-centered approach to sickle cell disease and clinical trial participation: an evaluation of perceptions, facilitators, and barriers. Blood Adv. 2021;5(23):5323-5331.
5. James J, Andemariam B, Mahlangu J, et al. Motivators and barriers for people with sickle cell disease participating in clinical trials: global findings from the LISTEN survey. Blood. 2023;142(Suppl 1):2498–2500.