December 20, 2019

Yesterday, the pharma company Novartis rolled out a lottery program that will provide the world’s most expensive medicine, Zolgensma, for free in countries where it hasn't been approved.

Zolgensma is a gene therapy that treats spinal muscular atrophy, an inherited disease that causes loss of muscle control (usually in babies) and is often fatal. The one-time treatment costs $2.1 million.

How it works: Doctors will be able to submit requests for the treatment, and patients will be entered into a drawing every two weeks. AveXis, the Novartis unit handling the program, said it plans to award up to 100 free doses.

Patient groups objected to the move, arguing the ethics of Novartis's lottery are questionable. “They are making patients compete,” said Kacper Rucinski, cofounder of TreatSMA.

Critics think Novartis should have a system that prioritizes the neediest patients or countries instead of a lottery.

Looking ahead...doctors can submit requests starting Jan. 2, and drawings will begin Feb. 3.