A lottery with lives hanging in the balance

Whose child might live, and whose might not? In the case of children with a rare disease, a lottery devised by Novartis may be a factor.

The lottery will randomly select children with spinal muscular atrophy, or SMA, to receive the company's gene therapy, Zolgensma, for free. The company aims to give away 100 doses of the extraordinarily expensive in countries where it is not yet available.

The ethics of this situation are fraught. On one hand, more kids do get access to the medication, when otherwise they might not. But framing the recipients as “lucky winners” is insensitive to everyone else.

“We applied for it because we desperately want this drug for our daughter, but you’re putting your daughter’s well-being and longevity in the hands of a lottery,” one parent told STAT’s Andrew Joseph. “I guess it’s the fairest way to decide who gets the drug and who doesn’t, but yeah, it’s not a great feeling.”