Informed Representation of Persons Living with Sickle Cell Disease in the US and Factors That Influence Clinical Trial Participation
Authors: Towne Sara D , Huml Raymond , Barlow Linda , Keuker Christopher , Rajgopal Ujwala , Shah Radhika , Miller Alice D. 
Published: December 2023

Rationale:
Sickle cell disease (SCD) is a group of blood disorders that results in an abnormality in the protein hemoglobin found in red blood cells. This leads to a rigid, sickle-like shape under certain circumstances. Sickle cell disease is typically inherited, and the most common type is ‘SS’ disease or sickle cell anemia. As of 2015, about 4.4m people have SCD, while 43m have the sickle cell trait. There is currently no pharmacological cure for SCD. The disease highlights racial and socioeconomic disparities in healthcare with treatment options, opportunities for clinical trial participation, and challenges associated with reimbursement. To enroll sufficient numbers of patients for SCD trials, drug developers need to identify enough patients with SCD, provide them with a compelling reason to enroll and complete these studies and make it possible for them to participate.
Our proposed study aims to leverage a survey targeting patients, caregivers, patient advocacy groups (PAGs), and healthcare providers (HCPs) to ensure a representative understanding of the patient journey in the context of rare chronic diseases, focusing on: Overcoming trial participation constraints by identifying factors affecting patients' readiness to participate in SCD trials.Bridging the gap between the biopharma industry and patient needs to ensure patient-centered approaches.Evaluating select aspects of the economic impact of SCD to aid in more cost-effective treatment development.Promoting Diversity, Equity, and Inclusion in research by using feedback from diverse stakeholders.Addressing challenges in research such as the lack of a definitive pharmacological cure and instead focusing on disease management and quality of life improvements.
Methods:
A 20-question survey, developed by physicians with experience treating both adults and children with SCD, was distributed via social media and through purposeful distribution to subject matter experts on July 7 th, 2023, targeting patients, caregivers, PAGs, and HCPs in the US to seek a better understanding of the barriers to SCD clinical trial enrollment, retention, and access to current treatments. The range of questions covers: Barriers to clinical trialsBarriers and facilitators to clinical trial participationThe range of trials currently available to patientsBarriers to advocacyAccess to current treatmentsClinical trial impacts on the SCD communityStrategies of PAGs to advocate and provide resources to patients and caregiversLevel of the impact of PAGs on legislation and disease management considerationsEducational material resourcesCost of treatment and access to resourcesDrug safety and privacy concerns
Results:
Access to the survey will close on October 2 nd, 2023. Survey results will be analyzed and presented using descriptive graphics and text statistics. Interim analyses suggest individuals living with SCD hold safety and success of treatment in high importance. Final survey data are anticipated to further suggest the following: a diverse respondent demographic is vital for inclusive and relevant research efforts; acknowledgment of the importance of research and development (R&D) in creating novel treatments for chronic diseases; a need for resources to be directed towards diseases affecting marginalized communities; an understanding that chronic diseases usually lack a complete cure, focusing instead on disease modification and symptom control; and the importance of insights from a wide spectrum of respondents. The results aim to underline the necessity of patient-centric R&D, especially for vulnerable populations and disease-modifying treatments, given the difficulty of curing chronic diseases like SCD. By leveraging this data, stakeholders can better align their efforts toward creating impactful solutions for SCD patients, potentially leading to additional innovative, life-improving treatments.