In a new study published in the Journal of Market Access & Health Policy, researchers conducted an economic evaluation of the use of betibeglogene autotemcel (beti-cel) gene therapy in the treatment of transfusion-dependent b-thalassemia.

The study involved development of a model to estimate incremental cost-effective ratios (ICERs) based on quality-adjusted life-years (QALYs) for the use of beti-cel compared with standard of care treatment for patients with b-thalassemia using lifelong blood transfusions and chelation therapy. The model was designed to consider a US commercial payer perspective.

The patient population included in this analysis consisted of patients with transfusion-dependent b-thalassemia who were between the ages of 2 and 50 years, and the model involved evaluations over patients’ lifetimes. Some outcomes included in the model were death, disease relapse, engraftment failure, liver complication, cardiac complication, other complications, cardiac death. QALYs were calculated with a discount of 3% per year.

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Clinical trial data from were used to define clinical parameters with beti-cel, and real-world information using US MarketScan data were used to estimate iron levels with standard of care therapy. Data were also obtained from published literature to estimate levels of iron overload-associated complications and mortality.


Costs of treatment over a lifetime, which included a mean age at entry of 22.5 years, were $572,107 with beti-cel and $2,038,384 with the standard of care; the cost associated with beti-cel treatment did not include the cost of beti-cel itself. The difference in cost between these treatment strategies was attributed to costs of blood transfusions, iron chelation therapy, and iron monitoring that are required with standard-of-care treatment and not with beti-cel therapy.

The researchers assumed a net cost of $238,709 associated with beti-cel therapy. This resulted in an ICER of $34,833 per QALY gained with the use of beti-cel in this analysis. A probabilistic sensitivity analysis suggested that costs were greater with beti-cel than with standard-of-care in 500 simulations, but that beti-cel demonstrated greater effectiveness. With a willingness-to-pay threshold greater than $89,000 per QALY gained, beti-cel showed cost-effectiveness against the standard of care across tests.

The research team concluded that in a US-payer model, the use of beti-cel was cost effective compared with standard of care transfusion- and chelation-based therapy for transfusion-dependent b-thalassemia.

Disclosures: Some authors have declared affiliations with or received grant support from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.


Kansal AR, Reifsnider OS, Brand SB, et al. Economic evaluation of betibeglogene autotemcel (beti-cel) gene addition therapy in transfusion-dependent β-thalassemia. J Mark Access Health Policy. 2021;9(1):1922028. doi:10.1080/20016689.2021.1922028