Zolgensma, a one-shot gene therapy, is the world’s most expensive medicine. What if you’re not covered?
Gene therapy is bringing out the best—and worst—in America’s health-care system. Zolgensma, the first systemic gene therapy of its kind that the U.S. Food and Drug Administration has approved, appears to cure in one shot a rare muscle-destroying disease that can kill infants and toddlers. (The kids still carry the gene mutation; they just don’t exhibit the eventually fatal symptoms.) It’s also the world’s priciest medication. Novartis AG set the cost at $2.1 million after the FDA approval came down on May 24, and some families have been left scrambling for ways to get the drug in its first several months on the market.
The medical system always faces a learning curve with a new treatment, particularly one this revolutionary, but the stakes for patients who could benefit from Zolgensma have made things that much tougher. Only 400 American babies a year are born with spinal muscular atrophy (SMA), but left untreated, it kills them before they turn 2 years old.
“It’s a challenge anytime you introduce a new therapy into the marketplace to gain understanding from the insurers,” says Dave Lennon, president of the Novartis division that developed Zolgensma. All major insurance companies cover the treatment, he says, but one-third of them have criteria more restrictive than the FDA’s, which says any patient under 2 can have it. “It’s still very much an evolving marketplace,” he says. “We want to make sure the insurance companies and the community understand the gaps in coverage and help us address that.”
Bu hikaye Bloomberg Businessweek dergisinin August 26, 2019 sayısından alınmıştır.
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Bu hikaye Bloomberg Businessweek dergisinin August 26, 2019 sayısından alınmıştır.
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