An ethical allocation scheme for scarce gene therapies in sickle cell disease and transfusion-dependent β-thalassemia

Abstract

Novel gene therapies (GTs) for sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) are severely limited by manufacturing, intensive care delivery, and high costs, requiring an ethically justified framework for allocating treatment within GT programs. Using an Accountability for Reasonableness process, we worked with a multidisciplinary committee to develop our program's initial GT allocation framework. This defines the population eligible for GT among patients with SCD and TDT, balancing inclusivity and safety. Among those eligible, prioritization is based on the following: (1) disease prevalence-based proportionality; (2) those who may not be eligible for GT in the future ("sickest first" due to impending organ failure); (3) those without an allogeneic donor; and (4) lottery. Transparent, adaptable frameworks are crucial for just GT allocation. Collaboration across programs is essential for equitable access and mitigating gamesmanship. Although this single-center framework cannot address systemic inequities, we hope our transparent process serves as a model for other programs.