Bluebird Bio Sets $1.8M Ticket For New Gene Therapy Drug

Biotech firm Bluebird Bio has revealed a plan to price their new gene therapy drug at $1.8 million for a single course. The drug is aimed at treating a genetic blood disorder, and Bluebird hopes its promising outcomes will persuade European healthcare officials to pay the premium price for it with evidence of its efficacy.

The new drug therapy is called Zynteglo and it has been approved in the European Union.  Officials say the cost of treatment will be spread out over five years.  In addition, the healthcare system will make the initial installment (315,000 EU); and they will only pay more installments if the treatment is proven effective at helping patients avoid the need for blood transfusions. Should the treatment work, and the patient remain transfusion free, the Boston-based biotech will receive additional, equal payments every year for the next four years. 

Fortunately, the treatment has indeed proven successful in 80 percent of patients, in trials.

Zynteglo is the latest addition to an emerging class of new, transformative therapies that apply to just a small cohort of patients and require only a single administration.  For example, drug regulators in the United States approved Zolgensma last month. This is a gene therapy treatment for spinal muscular atrophy; Novartis listed the drug at a price of $2.1 million (which makes it currently the most expensive drug in the world).

Novartis CEO Vas Narasimhan recently noted the importance of the industry developing these new economic models, and for determining the exact value a “cure” represents when compared against the cost and energy saved over longtime care.  

Additionally, Bluebird Bio chief executive Nick Leschly said these new treatments force the industry to explore creative ways to spread out their costs.  

In a statement, he said, “We have a one-time potentially curative treatment that has a dramatic effect on patients. And the ripple effect of that disruption is leading to what some people view as a challenge.”

He goes on to lament that “traditional model” which, essentially, prescribes a drug to a patient who then must take it—and pay for it—for the rest of their life. 

As such, he connects, the entire pharmaceutical industry continues to explore these creative new payment systems. “Value-based” payment, then, is the latest novel approach to address skyrocketing costs of drugs (in the US; they aren’t quite as expensive in Europe). 

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