Abstract
INTRODUCTION: Sickle cell disease (SCD) is the most common inherited blood disorder in the United States. The Cure Sickle Cell Initiative (CureSCi) was created by the National Heart, Lung and Blood Institute (NHLBI) to improve the lives of people with SCD by accelerating the advancement of safe and promising genetic therapies, engaging the SCD community and healthcare providers, and encouraging collaboration among stakeholders. CureSCi is a collaborative, patient-focused research effort that includes patients at every level of the Initiative. Patient engagement is a key component, particularly during the development of clinical trials. OBJECTIVE: To describe the processes and strategies utilized by the CureSCi to engage individuals living with SCD about genetic therapy clinical trials. METHODS: The CureSCi Patient Engagement team conducted nine Community Outreach visits in 2019, each of which was structured to include separate group interviews with patients and medical providers. A set of guidance questions was developed for each audience and the interviews lasted approximately 60 minutes. RESULTS: Patients were asked about their participation in a clinical trial, and approximately 50% had participated in a trial. SCD individuals reported trusting their medical provider to provide them with information relevant to trials. Medical providers emphasized the individual, family, and health aspects of SCD that warrant additional investigation. Providers reported barriers encountered by patients as transportation, childcare support, infertility, and trust in the healthcare system. CONCLUSION: Lessons learned from the community have helped to influence recommendations for clinical trial design and key messaging development for the Cure Sickle Cell Initiative.