$2.8m gene therapy treatment is America's most expensive drug ever

Sure, it's pricey, but not using it would be more expensive, says drug maker

A one-time gene therapy treatment recently approved by America's FDA is gaining attention not just for its clinical value, but for its price: at $2.8 million, Bluebird Bio's Zynteglo is the most expensive drug in US history.

Zynteglo is a treatment designed to tackle the rare condition known as transfusion-dependent beta-thalassemia (TDBT), a blood disorder that reduces hemoglobin and red blood cell counts in the bloodstream, leading to reduced oxygen delivery. Each dose is tailored to an individual's genetic profile for maximum effectiveness.

Transfusion-dependent patients have to undergo red blood cell transfusions as frequently as every two weeks. According to Bluebird, clinical trials of Zynteglo resulted in 89 percent of patients achieving "transfusion independence," defined as not needing a transfusion for at least 12 months. 

Of Zynteglo's price, Bluebird said $2.8 million is actually a deal, as "the lifetime cost of medical care for a patient with transfusion-dependent beta-thalassemia can reach up to $6.4 million."

According to the FDA's statement on Zynteglo's approval, the medication regulator fast-tracked the drug through a pediatric disease voucher. TDBT is an inherited disease that significantly lowers life expectancy, with the average US patient suffering from the condition dying at age 37. Bluebird estimates between 1,300 and 1,500 people in the US suffer from the disease.

TDBT is caused by genetic mutations, which gene therapies are designed to target, repair or supercede, and return cells to normal working order. Zynteglo works by adding functional, modified copies of the gene that causes TDBT to a patient's blood, a process that Bluebird calls "one time," but said can still take months to administer.

Bluebird said the drug will be available beginning in the fourth quarter of 2022.

The most expensive drug ever

At $2.8 million for a treatment, Zynteglo dethroned the previously most-expensive drug, another gene therapy product called Zolgensma priced at $2.1 million. 

According to Reuters, gene therapies are often curative and priced steeply, making them a difficult sell for insurance companies. In the case of Zolgensma, produced by Texas-based Novartis Gene Therapies, the drug was too pricey for many insurers, leaving patients without access to the medication. 

Bluebird, which said it set the price of Zynteglo so high "in recognition of its robust and sustained clinical benefit," appears to at least be aware of possible blowback from putting a multi-million dollar price tag on a potentially life-saving drug, coming out early with plans to help finance the treatment.

According to Bluebird, the company is working with commercial and government payers to establish plans for an upfront payment with an outcomes-based agreement that will see companies refunded 80 percent of the cost of therapy if the treatment doesn't work. So that's all right, then.

Bluebird said it's in "late-stage negotiations with leading commercial payers" that could make it available on "dozens of plans." The company said it's also working with state Medicaid agencies, which represent 80 percent of publicly-insured TDBT patients, to ensure they have access as well. 

Arguing that such an expensive drug is actually a bargain, which Bluebird has asserted, may not be the best approach. Despite the life-saving nature of Novartis' gene therapy drug, insurers simply wouldn't pay out, forcing the company to negotiate similar discounts to the ones Bluebird is proposing out of the gate, presumably in a bid to avoid such a conversation. ®

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