Attitudes toward clinical trials among patients with sickle cell disease

Abstract

Background A substantial number of planned clinical trials for sickle cell disease (SCD) have terminated early due to insufficient patient enrollment. Purpose To describe attitudes towards clinical trials among a sample of adults with SCD and identify patient-level factors associated with these attitudes. Methods Our data came from a sample [N = 291] of primarily adults with SCD participating in the Improving Patient Outcomes with Respect and Trust (IMPORT) study, which is a federally-funded observational study of SCD patient experiences in seeking health care. Attitudes towards clinical trials were assessed using items from the Perceptions of Participation in Clinical Research instrument. Patient factors examined as potential correlates of clinical trial attitudes were demographics, disease severity, engagement in self-care, trust, healthcare experience ratings, and prior history of participation in clinical trials. Multiple regression analyses were used to identify patient-level correlates of clinical trial attitudes. Results Our sample of SCD patients expressed overwhelmingly favorable attitudes about clinical trials, with 77% to 92% of our sample expressing agreement with a series of positive statements about clinical trials in general. Demographics, engagement in self-care, healthcare experience ratings, and prior trial participation each explained significant portions of the variability in clinical trial attitudes. Limitations The generalizability of our results to the entire SCD population may be of concern as the study participants were all receiving care at comprehensive sickle cell centers and already participating in clinical research. Conclusions Our results suggest that, in principle, adults with SCD enrolled in an observational study express very positive general attitudes about clinical trial participation and that specific factors attached to particular clinical trial opportunities may play a greater role in a SCD patient’s decision to participate than a general unwillingness to participate.

Primary caregiver decision‐making in hematopoietic cell transplantation and gene therapy for sickle cell disease

“…[30][31][32] Historically, African Americans have been reported to have a distrust of the medical community in general, and clinical trials, in particular. 33 Haywood et al 34 report changing attitudes among SCD patients about participation in clinical trials. In their study, more education, better-perceived health status, and previous clinical trial participation were all associated with more positive attitudes about clinical trials.…”

Sickle Cell Clinical Research and Intervention Program (SCCRIP): A lifespan cohort study for sickle cell disease progression from the pediatric stage into adulthood

“…In addition, acceptance of the optional banking of DNA for future genetic studies has been remarkably high (97.9%). Research study acceptance and enrollment have been traditionally low for SCD, [60][61][62][63][64][65] and some reports have indicated that African Americans are less likely than other racial and ethnic groups to support genetic studies that require a broad consent for the use of biospecimens in genetics research. 66,67 We attribute our high rate of enrollment to the possible benefit of genetic studies perceived by our patient population.…”

A re-randomisation design for clinical trials

“…The SWIM trial had difficulties in patient recruitment and, as a result, was terminated early. Other trials in patients with sickle cell disease have faced similar issues in recruitment, and many have also terminated early [ 5 ].…”

“…Finally, it can adversely impact the feasibility of conducting trials for conditions with a small patient pool. For example, a number of trials in patients with sickle cell disease have been terminated early due to insufficient recruitment [ 5 ]. As a result, care for patients with less common conditions may be based on suboptimal evidence from underpowered trials or non-randomised studies.…”