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What?! Expensive yet lifesaving drug for newborns being offered through random lottery

baby toe treatmentThe world’s most expensive drug, Zolgensma, has a price tag of $2.1 million and is approved only in the U.S. It’s a one-shot cure for newborns with spinal muscular atrophy, a deadly inherited disease. Now its maker, Novartis, has launched a lottery-style program to provide the drug free of charge to a limited number of patients outside the U.S.

Patient advocates question the moral and emotional consequences of such a plan.

“It’s really too crude,” Kacper Rucinski, co-founder of U.K.-based patient-advocacy group TreatSMA, told The Wall Street Journal. “They are making patients compete. Which will be the lucky one? That’s not helpful.”

The Guardian in London reports that Zolgensma is designed to add a functional copy of a gene that is missing in babies born with spinal muscular atrophy. It is not a cure.

There is limited data concerning the drug’s safety and it was approved by the U.S. Food and Drug Administration following trials involving just 68 children.

Companies are allowed to give out unapproved drugs for free in some countries under “compassionate use” programs. This provides seriously ill patients with experimental treatments when they have exhausted other options. Under Novartis’s lottery-style approach, doctors can submit requests for the treatment with eligible patients entered into a drawing every two weeks. Once regulators in a country or region approves the drug, the free lottery program would end there.

Parents would have preferred a program that prioritized the patients or countries according to need, Rucinski told the Wall Street Journal. Zolgensma is one of the first in a new wave of gene therapies that promise to cure inherited diseases with a single treatment. Despite the high price tag, Novartis claims the drug has been selling well since its launch.

Novartis is one of the world’s wealthiest drug companies. It defends the $2.1 million cost by pointing out that it costs less than a competing drug, Spinraza, in the long term. This lifelong rival treatment costs $750,000 for the first year and the $375,000 for each year thereafter.

Do you think lifesaving drugs should be given out in a random lottery? Share your thoughts with Voices for Affordable Health.