A decision by the drug maker Novartis (NVS) to put a $2.1 million price tag on its latest product, a one-time treatment for a rare and fatal childhood disease, has sparked a national debate about just how much society should pay for the medicines it needs.

But for Tina Anderson, whose son will soon celebrate his fourth birthday thanks to the Novartis treatment, there is no debate. Her son, Malachi, was born with the most severe form of spinal muscular atrophy. Doctors said he’d be lucky to survive 12 months. But in late 2015, she and her family got him into a clinical trial in which he received Novartis’ gene therapy free of cost. More than three years later, he defies his diagnosis every day.

“To me, you cannot put a price on your child’s life,” said Anderson, who lives in Mansfield, Ohio. “If tomorrow we were told to pay back everything, we would. We would figure it out. Because our son is now alive and well.”

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Novartis has argued that its therapy, approved last month as Zolgensma, is cost-effective even at $2.1 million. SMA is a progressive disease that gradually erodes muscular function. Patients often need wheelchairs and at-home care, and many suffer from lung infections that require hospitalization, all of which can add up to far more than the cost of Zolgensma.

Khrystal Davis’ son, Hunter, lived exactly that experience. Before he started getting a different treatment for SMA, a Biogen (BIIB) drug called Spinraza, he underwent a pair of lengthy hospitalizations that ran up a bill exceeding $1.5 million, a cost that comes on top of the thousands spent on medical equipment he needs to survive. But since getting on Spinraza in 2016, Hunter hasn’t needed a single hospitalization, said Davis, who lives in Austin, Texas. Spinraza, dosed every four months, has a list price of $375,000 a year, and she said its benefits are more than worth the cost.

“It has actually saved us money,” said Davis, who advocates for rare disease families and has testified before the Food and Drug Administration. “And it’s also really improved our quality of life.”

But some experts on drug pricing believe that Zolgensma’s price tag sets a worrying precedent.

A one-time payment of $2.1 million may look like a bargain compared with a lifetime of Spinraza, but if drug companies continue to price each new therapy at a premium to the last, the system might buckle beneath the cost. SMA affects only a few thousand patients in the U.S., but gene therapies for hemophilia and other genetic diseases are on the horizon. If each is priced like Zolgensma, the aggregate cost, passed down through insurers and across the health care system, could become untenable.

The question now is how soon insurers will add Zolgensma to their policies. Novartis has offered to allow payers to spread the cost of Zolgensma out over five years. Cigna (CI), one of the nation’s largest pharmacy benefit managers, agreed to offer that deal to its employer and insurer clients.

Zolgensma’s FDA approval, announced May 24, was an emotional wallop to SMA parents. Their experiences — the grim prognoses from doctors, the frantic internet research, the devastating statistics — might now become relics of medical history. Future parents will learn about Zolgensma in the same breath as the diagnosis.

“I ugly-cried for like three hours,” Anderson said. “I was so excited.”

The ultimate goal, SMA parents said, is to expand the number of states that require newborns to be screened for the disease and ensure that both Zolgensma and Spinraza are available within days of diagnosis. The disease can progress rapidly, causing untreated children to miss milestones and develop disabilities that can be irreversible. If parents can get treatment immediately, they can give their children a better chance of leading a healthy life.

Matteo
Matteo Almeida Courtesy Almeida family

That’s what happened with Nicole Almeida. She was five months pregnant when she learned her first child would be born with SMA.

“You can take him home, love him, take lots of pictures,” she remembers doctors saying, “but he won’t make it to his first birthday.”

Then she got in touch with Cure SMA, a nonprofit that supports families and funds research, and learned about the Zolgensma trial. She called the study coordinators once a week to make sure they didn’t forget about her son. When Matteo was born, in 2015, the Almeidas confirmed his diagnosis as quickly as they could and made the three-day drive from their Miami home to Nationwide Children’s Hospital in Ohio. There, on Aug. 6, at 27 days old, Matteo got his infusion of Zolgensma.

Almost four years later, Matteo runs, jumps, and “talks constantly,” Nicole Almeida said. He’s going to start school in August, all thanks to what the family calls “the miracle medicine that makes him run fast.”

Like Malachi Anderson, Matteo got his dose of Zolgensma for free. But his mother said she’d gladly pay for a medicine so effective. Her son’s life has been free of hospitalizations, wheelchairs, and ventilators. And, contrary to what doctors once predicted, he’s alive.

“If you look at it that way, so you paid $2.1 million up front, but then you’re not going to spend for the next God knows how many years,” she said. “To me it makes sense.”

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  • With the US health care system, as citizens, we are always one disease or accident away from total financial devastation, even when properly insured. It is really sad.

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