CURRENT CLINICAL TRIALS FOR NEW THERAPIES IN SICKLE CELL DISEASE DO NOT ADEQUATELY REPRESENT THE PATIENT POPULATION

Background:
Sickle cell disease (SCD) is a progressive disorder characterized by acute pain crisis (APC), organ damage, and early mortality. Despite the (few) disease-modifying therapies available, many individuals experience inadequate symptom control or treatment intolerance. Treatment effectiveness varies by genotypes, age, and social contexts, highlighting SCD heterogeneity. The gold standard for clinical trials (CT) (randomized-placebo control with APC as an endpoint) may be suboptimal and has contributed to unsuccessful trials. Further, two recently approved SCD therapies were withdrawn from Europe after real-world or later-stage data raised concerns about safety and efficacy.