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Gene Therapy for Sickle Cell Disease Likely Cost-Effective at

Medically reviewed by Carmen Pope, BPharm. Last updated on Jan 23, 2024.

By Elana Gotkine HealthDay Reporter

MONDAY, Jan. 22, 2024 -- Gene therapy for sickle cell disease (SCD) below $2 million is likely to be cost-effective, according to a study published online Jan. 23 in the Annals of Internal Medicine.

Anirban Basu, Ph.D., from The Comparative Health Outcomes, Policy & Economics Institute at the University of Washington in Seattle, and colleagues examined the cost-effectiveness of gene therapy for SCD and its value-based prices in a comparative modeling analysis conducted across two independently developed simulation models (University of Washington Model for Economic Analysis of Sickle Cell Cure [UW-MEASURE] and Fred Hutchinson Institute Sickle Cell Disease Outcomes Research and Economics Model [FH-HISCORE]) among persons eligible for gene therapy.

The researchers estimated incremental cost-effectiveness ratios of $193,000 and $427,000 per quality-adjusted life year (QALY) with UW-MEASURE and FH-HISCORE, respectively, under the health care sector perspective, and at an assumed $2 million price for gene therapy. From the societal perspective, the corresponding estimates were $126,000 and $281,000 per QALY. Acceptable value-based prices ranged from $1 million to $2.5 million from a societal perspective, depending on the use of alternative effective metrics or equity-informed threshold values. The results were sensitive to costs of myeloablative conditioning before gene therapy, the effect on caregiver quality of life, and the impact of gene therapy on long-term survival.

"Our results suggest that gene therapy for SCD can bring substantial benefits to this population and provide evidence for the proper reimbursement level for these therapies by Centers for Medicare & Medicaid Services," the authors write.

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Disclaimer: Statistical data in medical articles provide general trends and do not pertain to individuals. Individual factors can vary greatly. Always seek personalized medical advice for individual healthcare decisions.

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Posted January 2024

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