Cost-effectiveness of exagamglogene autotemcel gene-edited therapy in patients with sickle cell disease with recurrent vaso-occlusive crises in the United States - PubMed
3 hours ago
- #cost-effectiveness
- #gene-edited therapy
- #sickle cell disease
- Exagamglogene autotemcel (exa-cel) is a one-time gene-edited therapy approved in the US for sickle cell disease (SCD) patients aged ≥12 with recurrent vaso-occlusive crises (VOCs).
- Standard of care (SOC) for SCD includes symptomatic care, hydroxyurea, and/or red blood cell transfusions.
- A Markov model compared exa-cel to SOC, showing exa-cel improves survival by 30.8 years (mean age of death: 74.5 vs. 43.6).
- Exa-cel reduces VOC events by 77 (7 vs. 84) and lowers undiscounted disease-related costs by $3.34M ($0.55M vs. $3.89M).
- Exa-cel patients experience fewer acute and chronic complications compared to SOC.
- The incremental cost-effectiveness ratio (ICER) for exa-cel vs. SOC was $16,800 per QALY from the payer perspective and dominant (cost-saving and more effective) from the societal perspective.
- Exa-cel is projected to be a cost-effective treatment, improving survival, reducing VOCs, and lowering long-term healthcare costs.