Sickle cell gene therapy cost-effective if less than $2 million per person
Two models projected fewer pain crisis events with gene therapy over a lifetime, which can offset the high upfront administration costs, study authors wrote.
Sickle cell gene therapy is beneficial and likely cost-effective if the price stays below $2 million per person, a modeling study found.
Researchers applied two independently developed simulation models to CMS claims data from 2008 to 2016 to evaluate the cost-effectiveness of gene therapy for sickle cell disease versus common care. The models simulated the progression of sickle cell disease under real-world-based care methods to estimate costs and outcomes over a lifetime from both the health care sector and societal perspectives with and without gene therapy. The models assumed that gene therapy would cost $2 million per patient. The findings were published Jan. 23 by Annals of Internal Medicine.
One model estimated an incremental cost-effectiveness ratio (ICER) of $193,000 per quality-adjusted life-year (QALY) and the second estimated an ICER of $427,000 per QALY. Under the societal perspective, the first model estimated an ICER of $126,000 per QALY and the second estimated an ICER of $281,000 per QALY. The differences between models were primarily because they considered slightly different target populations and because the first model incorporated quality-of-life (QOL) effects of splenic sequestration, priapism, and acute chest syndrome. From a societal perspective, acceptable (>90% confidence) value-based prices ranged from $1 million to $2.5 million.
The authors noted that both models projected fewer pain crisis events with gene therapy over a lifetime, which can offset the high upfront administration costs, greatly improve patients' prospects for long-term employment, decrease or possibly eliminate caregiver burden, and substantially improve recipients' life expectancy and recipients' and caregivers' quality of life. Both models found that the effect of gene therapy on mortality was one of the top sensitive parameters. The duration of benefit is among the primary uncertainties surrounding gene therapy that will most affect its cost-effectiveness.
“This issue is particularly important because only 1 gene therapy trial (n=25) has reported outcomes, with a median follow-up of 17.3 months (range, 3.7 to 37.6 months),” the authors wrote. “As longer-term follow-up results are presented for more patients in the early trials, and results from different gene therapies are reported, it will be important to compare results predicted here with those observed and reevaluate value as needed.”