Gene Therapy Not Cost-Effective for Sickle Cell by Conventional Measures

MONDAY, June 5, 2023 (HealthDay News) -- For persons with sickle cell disease (SCD), gene therapy seems not to be cost-effective per conventional cost-effectiveness analysis (CEA) standards and can be equitable per distributional CEA (DCEA) standards, which incorporate the effects of treatments on disparities, according to a study published online May 30 in the Annals of Internal Medicine.

George Goshua, M.D., from Yale University School of Medicine in New Haven, Connecticut, and colleagues compared gene therapy to standard of care (SOC) using conventional CEA and DCEA in a birth cohort of SCD patients. The intervention involved gene therapy at age 12 years or SOC.

The researchers found that for females, gene therapy versus SOC yielded 25.5 versus 15.7 discounted lifetime quality-adjusted life years (QALY), and for males, it yielded 24.4 versus 15.5 discounted lifetime QALY, with costs of $2.8 and $1.0 million for females, respectively, and $2.8 and $1.2 million for males. The incremental cost-effectiveness ratio was $176,000 per QALY for the full SCD population. For gene therapy to be preferred per DCEA standards, the inequality aversion parameter would need to be 0.90 for the full SCD population. In a sensitivity analysis, at a willingness-to-pay threshold of $100,000 per QALY, SOC was favored in 100 and 87.1 percent of females and males, respectively, of 10,000 probabilistic iterations. To meet conventional CEA standards, gene therapy would need to cost less than $1.79 million.

"Goshua and colleagues estimate a cost of $176,000 per healthy year gained, well above the conventional U.S. cost-effectiveness threshold of $100,000," writes the author of an accompanying editorial. "Gene therapy is thus a 'lose-win' proposition -- a lose on cost-effectiveness but a win on equity."

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