RARE Daily

ICER Draft Evidence Report on Bluebird Gene Therapy Zynteglo Finds It Provides Value at $2.1 Million

April 14, 2022

The independent, nonprofit drug value watchdog The Institute for Clinical and Economic Review issued a draft evidence report on beta thalassemia that found that Bluebird Bio gene therapy Zynteglo provided value at a price of $2.1 million if paid through an outcomes-based contract for patients with sustained transfusion independence.

Beta-thalassemia is a rare blood disorder with the potential for high morbidity and mortality. Transfusion dependent thalassemia (TDT), the most severe form of this disease, is managed through lifelong regular blood transfusions and iron chelation therapy to avert the consequences of iron overload.

Although life expectancy has increased with improved treatments, the report said patients with TDT still suffer from a decreased quality of life and are required to live near a medical center that can provide thalassemia care. Some patients receive transfusions as often as every two weeks, and nearly all patients plan their lives around transfusions.

Zynteglo is a gene therapy that provides a potentially curative option for patients with TDT. It is manufactured ex vivo using an individual’s own hematopoietic stem cells. A lentiviral vector is then used to add functional copies of the β-globin gene to patients’ hematopoietic stem cells. The modified cells are then infused back into the patient following conditioning chemotherapy.

The Food and Drug Administration is reviewing the company’s marketing application for Zynteglo and is expected to make a decision by August 19, 2022.

ICER said the company’s public statements indicates the pricing will be consistent with an outcome-based payment plan of five equal yearly payments totaling $2.1 million for individuals who achieve and maintain transfusion independence.

The review looked at five clinical studies of the gene therapy, although ICER gave greater weight to the phase 3 studies. It said transfusion independence in the phase 3 trials was achieved in 89 percent of the patients who received the gene therapy. Nevertheless, it said the duration of the studies were not long enough to remove uncertainty regarding the durability of effect over a longer time period.

ICER scored Zynteglo a B+ with evidence that demonstrates that it is superior overall to the current standard of care but said that the magnitude of that overall net health benefit is less certain, ranging between incremental to substantial.

Bluebird has said it will price the therapy based on its clinical value to patients rather than on its ability to offset costs to current therapy. ICER said in a scenario analysis designed to mirror that approach, when half of the cost offsets from treatment are retained by the health system, cost effectiveness estimates exceeded $200,000 per equal value of life years gained, which shows that cost offsets have a substantial impact on the cost effectiveness of this treatment.

On April 26, 2022, as part of ICER’s Early Insights Webinar Series, ICER’s President Steve Pearson, will present the initial findings of this draft report. ICER will review all comments and incorporate any necessary changes in the Evidence Report and Revised Voting Questions that will be posted on or about June 2, 2022. All comments and ICER’s response to comments will be posted publicly along with the Evidence Report.

The Evidence Report will be the subject of a virtual public meeting of the New England Comparative Effectiveness Public Advisory Council on June 17, 2022. During the meeting, the independent council will vote on key questions raised in the report.

Author: Rare Daily Staff

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