Are $3M gene therapies the new normal? Experts say bluebird will be a test case for a market ready for prime time

Drug pricing experts generally agree that bluebird bio’s two recently approved gene therapies and their multimillion-dollar price tags aren’t going to be one-offs as a wave of new cell and gene therapies makes its way to the market.

The FDA’s recent approvals for bluebird’s $2.8 million Zynteglo — with ICER supporting the price and an 80% rebate if patients don’t achieve transfusion independence — and the $3 million Skysona, approved under accelerated approval, are likely to be the norm for gene therapy prices moving forward, particularly if they can reduce costs elsewhere in the health care ecosystem, experts said.

Daniel Ollendorf

Daniel Ollendorf, director of value measurement & global health initiatives at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center, told Endpoints News in a phone interview that the trend behind multimillion-plus gene therapies is an extension of what began with Novartis’ $2.1 million spinal muscular atrophy gene therapy Zolgensma, which is still priced at about half of the 10-year current cost of chronic SMA therapy, and became a blockbuster for Novartis last year with more than $1.35 billion in annual sales.

But the expectation is that these high list prices will come with risk-sharing agreements and refunds if the products don’t work so payers don’t have to bear the full brunt of the financial risk, Ollendorf said. And he noted that some gene therapies don’t lend themselves as well to tracking milestones, but that isn’t the case for observing transfusion independence in those receiving Zynteglo.

Still, when Zynteglo first won European approval in 2019, the nearly $2 million price tag seemed like a surmountable hurdle, but the therapy has since been withdrawn as the company struggled with reimbursement, and with its finances, eventually laying off almost a third of its staff in April. Analysts don’t expect either of the two recently approved gene therapies to bring in peak sales of more than $100 million annually.

Mark Trusheim, strategic director of NEWDIGS at the Center for Biomedical System Design at Tufts Medical Center, told Endpoints that he expects about 64 product-indication approvals for cell and gene therapies through 2030, with about half of those in this durable gene therapy space that can command these multimillion-dollar list prices.

“Moving forward, each new product will be judged on the specific benefits its evidence demonstrates,” Trusheim noted. “That said, more such list prices before discounts and outcomes contracts results would seem likely in the future.”

But the question of whether such high list prices are sustainable has been on the minds of even the regulators who aren’t allowed to factor prices into their regulatory decisions.

“Reimbursement is the 800-pound gorilla in the room,” CBER director Peter Marks noted at an event in May, as he mused that if there was better manufacturing of smaller batches of AAV vectors for gene therapies, for example, he said, there might be a way to put together a portfolio of these smaller-population products that would then be viable.

Moving forward, companies may look to take their gene therapies to the US first as a test for wider acceptance, and as the price points are typically higher here than in Europe, Ollendorf said.

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