ICER report: Evidence adequate of beti-cel benefit for patients with beta-thalassemia
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Adequate evidence exists to show betibeglogene autotemcel confers a net health benefit vs. standard clinical management for patients with beta-thalassemia, according to a final evidence report by Institute for Clinical and Economic Review.
The gene therapy, also known as beti-cel (bluebird bio), also meets commonly accepted value thresholds at a $2.1 million estimated annual price with an 80% payback option for patients who do not remain transfusion-independent over a 5-year period, cost-effectiveness modeling showed.
“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,” David Rind, MD, MSc, chief medical officer of Institute for Clinical and Economic Review (ICER), said in a press release. “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.”
Beti-cel is a one-time ex vivo gene therapy that adds functional copies of a modified form of the beta-globin gene into a patient’s hematopoietic stem cells. The therapy received FDA priority review in November, with a revised Prescription Drug User Fee Act date of Aug. 19.
Members of New England CEPAC, one of ICER’s independent evidence appraisal panels, reviewed ICER’s report on beti-cel last month. The systematic review yielded five beti-cel studies, including two phase 3 trials that showed 90% of patients who received beti-cel achieved transfusion independence and sustained it over median follow-up of 42 months (range, 23-88), according to an ICER summary of the report.
“However, this duration is not long enough to remove uncertainty regarding the durability of effect over a longer time period,” the summary stated. “Because of the uncertainty about these risks and the durability of the clinical benefit, we judge that the evidence demonstrates that beti-cel is superior overall to the current standard of care, but the magnitude of that overall net health benefit is less certain, ranging from incremental to substantial (‘B+’).”
The panel voted 12-0 in favor of the B+ evidence rating and 9-3 that beti-cel represents a “high” long-term value for the price.
If the pricing and payment structure is the same as the report assumes, the FDA label should serve as the guide to coverage policy without seeking to narrow coverage using criteria for clinical trial eligibility, according to the press release.
References:
- ICER publishes final evidence report and policy recommendations on beti-cel gene therapy for beta thalassemia (press release). Available at: icer.org/news-insights/press-releases/icer-publishes-final-evidence-report-and-policy-recommendations-on-beti-cel-gene-therapy-for-beta-thalassemia/. Published July 19, 2022. Accessed July 20, 2022.
- Report at a glance: Beta thalassemia. Available at: icer.org/wp-content/uploads/2022/07/Beta-Thalssemia-RAAG_July-2022.pdf. Accessed July 20, 2022.
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