Gene therapies for sickle cell disease are likely to be cost-effective in the US when priced below $2M. Based on the University of Washington Medicine and Fred Hutchinson Cancer Center Study estimates suggest that—at least from a societal perspective—the value-based price for sickle cell disease (SCD) gene therapies ranging from $1M to $2.5M would be “acceptable.”
The authors note that these estimates could still be affected by the costs of pre-treatment myeloablative conditioning, caregiver quality of life, and the effect of the gene therapy on long-term survival.
Lead investigator Anirban Basu, in an interview with The American Journal of Managed Care, said that “gene therapies show great promise in bringing a widely applicable curative approach to SCDs,” which typically cuts patients’ life expectancies short and compromises their quality of life.
“Our analyses provide detailed early evidence of such therapies’ potential value and value-based prices, and showcases a comprehensive approach to valuing innovative therapies,” Basu said.