Bluebird Bio has nabbed a green light for its gene therapy Zynteglo, the first one-time treatment for a chronic blood disorder to be cleared for U.S. marketing. Despite the monumental approval, the drug’s $2.8 million cost has given rise to questions of fair pricing.
Zynteglo, also known as beti-cel, won clearance as a one-time gene therapy for transfusion dependent beta-thalassemia (TDT), a rare blood disorder, in patients requiring red blood cell transfusions. The Food and Drug Administration’s decision makes Zynteglo just the third gene therapy to cross the regulatory finish line in the U.S.
Shortly after the approval was announced, Bluebird released the drug’s average wholesale price of $2.8 million. The company said that sum recognizes the potentially curative medicine’s clinical robustness – trials show transfusion-independence was achieved in 89% of beti-cel patients – and the possibility of alleviating a lifetime of healthcare costs associated with regular transfusions.
But that price falls on the upper end of a cost watchdog’s suggested range. The Institute for Clinical and Economic Review (ICER) gave a base case price range of $2.1 million to $2.8 million, noting that if half of the cost-offsets are returned to society, the price range would be $1.3 million to $1.8 million. According to ICER’s assessment, at $2.8 million, zero percent of the savings from the therapy are going back to society.
While that trade-off is bound to ruffle some feathers, the company’s commercial scheme could alleviate criticism. Bluebird is launching Zynteglo with an outcomes-based deal that would refund up to 80% of Zynteglo’s cost if a patient fails to achieve and maintain transfusion independence up to two years after infusion. The lifetime cost of caring for a patient with TDT can reach up to $6.4 million in the U.S., the company said.
It’s not the first time a costly gene therapy has opened the door to fair-pricing questions. Nor is it the only instance of an outcomes-based model being used to reassure payers, who generally want to make novel treatments available to patients but require additional assurances given the lofty upfront costs.
In Zynteglo’s case, payers “have responded favorably” to the approach,” said Bluebird chief commercial and operating officer Tom Klima on a call with investors.
Klima estimated there are 1,500 patients with TDT in the U.S., about 50 of whom are already being treated with the drug. Its commercial teams are in the field this week to begin patient identification and consultation, he said, adding that the firm anticipates “high likelihood of coverage.”
But the company is hoping to avoid the issues it encountered with Zynteglo’s European launch. Bluebird had to cease commercial operations in Europe last summer after what it claimed were difficulties convincing EU governments to pay steep upfront prices for its treatment, then sold under the brand name Zynteglo and priced at $1.8 million per treatment.