News & Commentary

Here’s Zolgensma! (That’ll Be $2 Million)


Although as we went to press Novartis hadn’t set a price for Zolgensma (onasemnogene abeparvovec), a gene therapy that treats spinal muscular atrophy (SMA), financial analysts see a $2 million price tag coming down the pike. Remember when a million-dollar price tag seemed high?

SMA makes infants’ muscles waste away. “Without treatment, victims of the most severe form typically die before their second birthday, making SMA the most common genetic cause of infant death,” the Wall Street Journal reported. About 500 U.S. babies are born with SMA every year, and around 300 have the most severe version.

Zolgensma will rocket into the health care system trailing clouds of hope but also of dread. “A therapy is useless if no one can afford it,” Cathryn Donaldson, a spokeswoman for America’s Health Insurance Plans, said to the newspaper.

The FDA appeared ready to approve Zolgensma in May but that’s only the beginning. The agency’s on track to approve 10 to 25 gene therapies a year until 2025. The prices are likely to be high by any standard.

Take Luxturna (voretigene neparvovec-rzyl), for instance. This gene therapy for inherited sight loss is the only one the FDA has approved so far. Price: $850,000.

Ron Philip, chief commercial officer of Spark Therapeutics, which makes Luxturna, tells the WSJ that success comes at a price. “The whole system was built for chronic care, and never intended for this curative model. We’ve never had to tackle these issues.”

But help may be on the way for payers.

Spark and other pharma companies are working on devising installment payments for insurers, or having payments based on value.

That addresses the concern insurers have about paying the entire bill upfront for treatments that have not been monitored over time. Zolgensma, for instance, was first given to a patient five years ago in a clinical trial.

Thomas Morrow, MD, a member of the Managed Care Editorial Advisory Board, wrote the Tomorrow’s Medicine column for Managed Care for more than 15 years until June 2018. Morrow predicts there will be a lot of trial and error before the health system settles on a payment mechanism all stakeholders can live with. He even quotes Winston Churchill on this: “You can always count on the Americans to do the right thing after they have tried everything else.”

Morrow contends that insurers have little ability to say no to covering an effective treatment for a genetic disease in the absence of alternatives.

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