$4 million a dose for upcoming Novartis drug 'not sustainable,' Express Scripts says

Express Scripts, a pharmacy benefits manager, is pushing back against Novartis, which floated the idea that its new gene therapy against a deadly neuromuscular disease was worth more than $4 million per dose, according to Reuters.

The drug in question is AVXS-101, which was proven effective in a 15-person trial for patients with spinal muscular atrophy, a genetic disease affecting voluntary muscle movement. In its most severe form, the disease is fatal for patients before age 2. In the trial, infants with SMA who received AVXS-101 treatment had a 100 percent survival rate after 24 months. The trial data prompted Novartis to purchase the gene therapy maker AveXis for $8.7 billion in April.

Among the first to react to Novartis' idea was Express Scripts, one of the largest PBMs in the U.S.

Express Scripts CMO Steve Miller, MD, told Reuters he "loves the science" behind the Novartis therapy, but charging $4 million a dose is "not sustainable."

"You just can’t keep pushing these price points up," Dr. Miller said. "I just don’t think we can allow it. It is not sustainable over time."

While Novartis awaits FDA approval for the drug, expected to come early next year, the company has begun its campaign to persuade  insurers to cover it. The president of AveXis has argued that the one-time cure will save money in the long run, even at a cost of $4 million.

"When we look at 10-year costs, you see somewhere between $2.5 million to $5 million being spent by societies to care for these types of patients," Dave Lennon, AveXis’s president, told Reuters. "Four million dollars is a significant amount of money, but we believe this is a cost-effective point."

It is unclear whether Novartis will be able to persuade payers to spend millions of dollars up front with no guarantee of benefit — especially as drug prices remain a top concern in the U.S. Novartis executives stressed that the actual drug price would be disclosed after it receives regulatory clearance.

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