ZURICH: Just weeks after Novartis floated the idea that $4-5 million was fair value for its new gene therapy against a deadly neuromuscular disease, a major benefits manager is pushing back.
The Swiss drugmaker’s assessment of AVXS-101’s value for treating spinal muscular atrophy (SMA) has put the company front-and-centre in the debate over what “super drugs”, for rare diseases afflicting relatively few patients, are really worth.
Among the first to react was pharmacy benefits manager Express Scripts, which helps U.S. employers manage workers’ prescription costs.
Its chief medical officer, Steve Miller, told Reuters he “loves the science” behind Novartis’s therapy, a potential cure for newborns who are diagnosed early.
But $4 million or more per patient?
“You just can’t keep pushing these price points up,” Miller said. “I just don’t think we can allow it. It is not sustainable over time.”
Novartis, which bought U.S.-based AveXis for $8.7 billion in April to add the SMA therapy to its portfolio, is still mulling its asking price as it awaits U.S. Food and Drug Administration approval, likely in early 2019.
But the company has begun its campaign to convince insurance groups and governments to cover AVXS-101, contending the one-and-done infusion will save society money over the long haul, even with a cost near the highest ever for a one-time therapy.
There’s now only one approved drug for SMA, Biogen’s two-year-old Spinraza, and it is listed at $750,000 for the first year and $350,000 thereafter. Spinraza is not a cure and must be taken indefinitely.
“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, said.
“Four million dollars is a significant amount of money, but we believe this is a cost-effective point.”
Though newborns may stand to benefit the most from AVXS-101, depending on the durability of its effect, the therapy is also being tested in older SMA patients with more advanced disease in hopes it will improve their symptoms, too.
A diagnosis of SMA, which affects one in 10,000 live births, is devastating. Forty per cent of victims have the severest form and historically die within months.
Children with less severe SMA can live to adulthood, although with profound physical disabilities. Though cognitively normal, many cannot feed themselves and require 24-hour care, wheelchairs and machines to help them breathe and cough.
Janice Kress, a Pennsylvania woman, lost her grandson to the disease at 5 months.
Today she volunteers for SMA charity events and knows families who have fought their U.S. insurers for access to Biogen’s Spinraza, as payers seek to rein in costs using eligibility criteria like age or when symptoms began.
“A child’s life — how can you say no?” Kress said.
As Novartis prepares to launch AVXS-101, it also hopes for tacit endorsement of its pricing strategy from the non-profit Institute for Clinical and Economic Review (ICER), which is currently reviewing the cost-effectiveness of SMA therapies.
The Boston-based non-profit, established in 2006, carries out cost-benefit analyses on drugs that it calls independent of “Big Pharma”, insurers and government.
Unlike European price regulators, ICER cannot dictate costs.
But it has steadily gained influence in the U.S. pricing debate, as companies like Express Scripts and CVS Caremark and governments rely on its analyses.
ICER has conducted 11 assessments in 2018, some covering multiple drugs.