ICER Draft Report Finds Beti-Cel is Cost-Effective for Blood Disorder

The gene therapy betibeglogene autotemcel (beti-cel) provides net health benefits to patients with transfusion-dependent beta thalassemia and meets thresholds for cost-effectiveness if an outcomes-based pricing mechanism is used, according to a draft evidence report from the Institute for Clinical and Economic Review (ICER).

Beta thalassemia is a blood disorder caused by the mutation of the HBB gene that leads to reduced or missing synthesis of the beta-globin proteins of hemoglobin, which are components of red blood cells that carry oxygen. This can lead to anemia. Patients who are missing beta-globin have a more severe disorder. In infants six to 24 months of age, this can result in severe anemia, failure to thrive, and end organ damage.

Beti-cel is a one-time gene therapy designed to add the functional copies of a modified form of the beta-globin gene into the patient’s own blood stem cells. It is currently under review at the FDA with a Prescription Drug User Fee Act (PDUFA) date of August 19, 2022.

Bluebird Bio, which developed the therapy, has said it plans to use an outcome-based payment plan of five yearly payments totaling $2.1 million for patients who no longer need transfusions. ICER reviewers based their cost-effective analyses on the manufacturer’s plans compared with standard of care, which includes transfusions and chelation that removes iron from the blood.

Bluebird, however, said the company has not set a price for beti-cel in the United States nor confirmed contracting plans, according to a company spokesperson.

ICER results showed that at the proposed price of $2.1 million per treatment course, all eligible patients could be treated over the span of five years without crossing the ICER budget impact threshold of $734 million per year.

Additionally, they found that patients could be treated without reaching the potential budget impact threshold at three prices (about $1.85 million, $2.11 million, and $2.38 million per course of treatment). This analysis was done at several prices to document the percentage of patients who could be treated without crossing a potential budget impact threshold that is aligned with overall growth in the U.S. economy.

ICER reviewers assessed several clinical trials for beti-cel. In phase 3 trials, 89% of patients who received beti-cel achieved transfusion independence. In phase ½ and phase 3 trials, patients remained without the need for transfusions at a follow-up of 42 months.

ICER reviewers, however, said there is uncertainty about the duration of effect over a longer time, as well as about the side effects in a real-world setting. In trials, side effects were mild, few patients had serious side effects, and no deaths were reported.

Bluebird Bio, however, presented data at the American Society of Hematology in December 2021 of a long-term study (LTF-303) that found showed adult and pediatric patients who require regular transfusions can produce normal or near-normal levels of total hemoglobin and continue to remain transfusion-free, and achieve stable iron markers, through up to seven years of follow-up.

ICER is accepting comments on the draft report until May 10, 2022, and will host a public meeting on June 17, 2022.