Beti-cel was rated a B+ for lingering questions about durability and unknown risks.
The Institute for Clinical and Economic Review (ICER) has concluded that bluebird bio’s lentiviral gene therapy betibeglogeneautotemcel is superior to standard of care for patients with β-thalassemia, however, the magnitude of this superiority is still uncertain.1
“Beta-thalassemia is a serious blood disorder, and while treatment has improved, patients with transfusion-dependent thalassemia (TDT) still have decreased life expectancy and large burdens of care that impact all aspects of their lives,” David Rind, MD, chief medical officer, ICER, said in a statement.2 “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.”
ICER analyzed efficacy and safety data from 5 studies of beti-cel: 2 phase I/2 trials (HGB-204 and HGB-205), 2 phase 3 trials (NorthStar 2 and NorthStar 3), and 1 long-term follow-up (LTFU) cohort study (LTF-303), with a greater emphasis on the phase 3 trials compared to earlier trials due to manufacturing changes in between.1
Overall, 89% of patients (n = 56) treated with bet-cel achieved transfusion independence in the phase 3 trials. The LTFU revealed that transfusion independence was sustained for a median of 42 months (range, 23-87). Few serious adverse events occurred, but uncertainty remains due to known risks with myeloablative conditioning and unknown risks and durability. With these factors in mind, the ICER gace the gene therapy a rating of B+.
ICER also found that many stakeholders, including patients with TDT and their families, were likely to remain on standard of care treatments including transfusion and chelation even if beti-cel proves to be a durable cure with an excellent safety profile.
ICER also assessed cost-effectiveness of beti-cel treatment and determined that it meets commonly accepted value thresholds given the high annual costs of standard care.The anticipated price is $2.1 million with an 80% payback option for patients who do not achieve and maintain transfusion independence over a 5-year period.
“The manufacturer publicly suggested pricing based on the value of beti-cel to patients rather than based on offsetting other costs, and so we are highlighting 2 different pricing analyses, one in which half the cost-offsets provided by elimination of the need for expensive ongoing therapy are ’shared‘ with society and not included in calculating a fair price,” Rind added.2
ICER determined that discounts between 15% and 38% off the anticipated price are required to meet commonly accepted thresholds if half of the lifetime cost savings from the therapy are returned to society.1