Sickle Cell Disease: A Cure Within Reach, Justice Still Distant

By Abdirazak Mohamed and Athanasia Agapiou

Sickle cell disease (SCD) remains one of the most underrepresented conditions in clinical research despite the urgent need for new therapies. Affecting primarily individuals of African, Middle Eastern, and South Asian descent, SCD presents unique clinical, social, and cultural challenges that make trial recruitment and retention especially difficult. This white paper explores the critical gaps in traditional clinical trial approaches for SCD and outlines strategies to create more equitable, patient-centered studies.

Conventional site selection, recruitment practices, and trial logistics can often fail to meet the needs of the SCD community. Mistrust in healthcare systems, compounded by decades of systemic inequities, further hinders participation. To address these barriers, partnering with trusted community organizations, prioritizing diverse site networks, and leveraging decentralized clinical trial (DCT) models is recommended to reduce patient burden. Integrating digital health technologies (DHTs) and flexible visit schedules can also enhance trial accessibility and engagement.

Real-world examples illustrate how thoughtful protocol design, cultural competency training, and proactive patient engagement can drive meaningful improvements in SCD trial performance. By transforming how trials are designed and conducted, sponsors and researchers can accelerate the development of life-changing therapies for individuals living with sickle cell disease.