Interventions of Interest:

  • betibeglogene autotemcel (Zynteglo, bluebird bio)

The independent appraisal committee unanimously determined that the evidence is adequate to demonstrate that beti-cel provides a net health benefit compared to standard clinical management.

Given the high costs of standard care, cost-effectiveness modeling finds beti-cel meets commonly accepted value thresholds at an anticipated price of $2.1 million — if that price is subject to an 80% payback for treatment failure.

Consideration should be given to assigning a lower share of cost-offsets to determinations of fair pricing, but if pricing and payment agreements match those assumed in this report, payers should use the FDA label as the guide to coverage policy without seeking to narrow coverage using clinical trial eligibility criteria.

Final Documents

According to ICER’s Chief Medical Officer, David Rind, MD:

“Beta-thalassemia is a serious blood disorder, and while treatment has improved, patients with transfusion-dependent-thalassemia (TDT) still have decreased life expectancy and burdensome care that impacts all aspects of their lives. Previously, a minority of patients had access to curative allogeneic bone marrow transplant; beti-cel provides an additional potentially curative option for many patients with TDT. New potentially curative therapies for beta-thalassemia bring the promise of considerable lifetime benefit, but there also remains substantial uncertainty regarding longer-term safety and the durability of benefits. Beti-cel is cost-effective at a high price in part because it offsets current very high costs of care; a somewhat lower price would be needed if half of those offsets were returned to the medical system.”

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