FDA Approves Gene Therapy, Manufacturer Prices Drug at Record $2.8 Million

by Tim Casey   After the FDA approved Zynteglo (betibeglogene autotemcel) on Aug. 17 for the treatment of transfusion-dependent beta-thalassemia, the gene therapy’s manufacturer, bluebird bio, Inc., announced the drug would have a $2.8 million wholesale acquisition cost (WAC). That is the highest WAC for a drug hitting the market in the U.S.  bluebird justifies high price tag  The medication is administered via a one-time intravenous infusion of a patient’s own modified stem cells following chemotherapy. An estimated 1,500 people in the U.S. have transfusion-dependent beta-thalassemia, according to the company, of which bluebird projects 850 could potentially be eligible to receive Zynteglo.    Tom Klima, bluebird’s chief commercial and operating officer, said during a conference call on Aug. 18 that the $2.8 million WAC is “a price directly tied to recognize the value for a potentially curative gene therapy for beta-thalassemia.”  “When pricing Zynteglo, we took into consideration the therapy’s benefit to patients and society, including measures of positive clinical outcomes as well as expected quality of life improvements, health systems’ cost savings, and societal impact of patients and families living lives more fully,” Klima said.  The nonprofit Institute for Clinical and Economic Review (ICER), meanwhile, found last month that Zynteglo would achieve a net health benefit at a price of up to $2.73 million based on an incremental cost-effectiveness ratio of $150,000 per quality-adjusted life year gained. The ICER analysis assumed an arrangement where payers would have an upfront free and could get up to 80% of the cost back if patients do not achieve transfusion independence.   Payers will find themselves in a ‘difficult situation’  “The price is high, but ICER finds it defensible given the expensive and life-altering alternative,” Rubinstein writes in an email. “The existing therapy for those patients is chronic, quite intrusive from a quality-of-life point of view, and is also very expensive over many years. The bluebird bio therapy, for patients who enjoy a durable cure and who do not suffer from adverse effects of the therapy, offers a future life free of this disease.”   Klima said that bluebird is currently in “late-stage discussions” with many U.S. payers, including national PBMs and state Medicaid agencies. Payers, though, are in a difficult situation due to balancing the drug’s high cost and the unmet need for the patient population, according to Ge Bai, a professor at Johns Hopkins University’s schools of public health and business.   “Insurers will balk at the high price tag as they always do with expensive therapies because, other things equal, insurers would like to offer sponsors a lower premium to stay in business,” Bai writes in an email to AIS Health.

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