The plan to hold a lottery to give away 100 free doses of the gene therapy Zolgensma has been met with consternation, the Guardian reports.
The US Food and Drug Administration approved Novartis' Zolgensma in May for children with spinal muscular atrophy who are under the age of two. The disease causes paralysis, breathing difficulties, and death, and Zolgensma is a one-time treatment that delivers a working copy of the SMN gene to prevent its progression. But Zolgensma is also expensive, with a list price of $2.1 million.
According to the Guardian, parents of children with SMA are put off by both the drug's high cost, but also the lottery. "SMA is still the biggest killer of children under two," Lucy Frost, whose son has the disease and who is a trustee of TreatSMA tells it. "Imagine parents putting a child in a draw every two weeks to see if their life can be saved. It will have a huge emotional impact."
A Novartis spokesperson says the drug is fairly priced, as it is a one-time treatment, and that it costs less than the standard of care treatment, the Guardian adds, noting that AveXis, which developed the drug and was bought by Novartis, says it is working to "design sustainable and ethical solutions" to address access concerns.