Novartis to give Longmont-made Zolgensma in lottery, sparking ethical questions

LONGMONT — AveXis Inc. and parent company Novartis AG (NYSE: NVS) is planning to give away doses of the world’s most expensive drug outside of the United States next year in a lottery, raising concerns about equity in drug access and the larger drug-pricing system.

In a statement, AveXis said it would offer 50 free doses per year in 2019 and 2020 to patients in countries where the drug has not been approved for sale, including Japan and members of the European Union.

The company said it plans to continue the lottery program past 2020, and it consulted a bioethics advisory committee while developing the program.

“AveXis designed a program anchored in principles of fairness, clinical need and global accessibility to best determine the equitable global distribution of a finite number of doses that doesn’t favor one child or country over another,” it said.

AveXis is in the final stages of retrofitting a 692,000-square-foot former AstraZeneca plant in Longmont to produce Zolgensma alongside a sister plant in Raleigh, North Carolina.

The drug treats spinal muscular atrophy (SMA), a rare genetic disorder that is estimated to affect one in every 6,000 to 10,000 newborns. Children born with SMA do not have functioning motor neurons in their spines and progressively lose muscle strength and cause movement, eating and respiratory problems. The majority of patients die before their second birthday.

Zolgensma is the most expensive drug in the world at $2.1 million for a one-time infusion.

AveXis and Novartis were chided by the U.S. Food and Drug Administration earlier this year after they learned some of the drug’s animal-testing data was tampered with and not reported to regulators until after it approved Zolgensma for sale in May. Both the FDA and Novartis say the drug is still safe and effective enough to remain on the market.

Implementing a lottery for a life-saving disease aimed specifically at infants instead of giving the doses away by need has raised concerns from patient-access and medical-ethics advocates.

Dr. Daniel Goldberg, an associate professor at the University of Colorado Anschutz Medical Campus’ Center for Bioethics and Humanities, told BizWest there will be varying opinions on the lottery, and one could view the lottery as fair in an economic sense because FDA-approved treatments for SMA are a scarce resource.

However, he believes the lottery and Zolgensma’s price illustrates the larger way of how drugs are priced in the U.S., which he describes as “profoundly, structurally unjust.”

“This drug has a huge (price) on it and it affects a very small number of very vulnerable people, and that’s why people are eyeing this one,” he said. “But to me, this is just a symptom of the larger structural injustice that we have, which is that we have an unbelievable conflict of interest. We’ve chosen in this country not to have any way of actually controlling prices.”

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