viernes, 20 de diciembre de 2019

How do you get gene therapy to children in need? Novartis is running a lottery

The Readout
Damian Garde

How do you get gene therapy to children in need? Novartis is running a lottery

In the U.S., Novartis’s gene therapy for the rare and deadly spinal muscular atrophy can be had for $2.1 million for a one-time dose. But in Europe, where it’s not yet approved, there’s no commercial access. And, because there’s already an approved SMA treatment on the market there, Novartis can’t give it away under compassionate use.

So, as STAT’s Ed Silverman reports, the company has set up a lottery. Starting in 2020, Novartis will set aside as many as 100 doses of its gene therapy, called Zolgensma. Doctors can then nominate babies who would benefit from the treatment. Every two weeks, names will be drawn, and the winners will get the potentially life-saving therapy.

If that rings a little dystopian to you, you’re not alone.

“The fact that the manufacturer is lotterying it strikes me as capturing every injustice about health care globally,” wrote Dr. Steven Joffe, who heads the division of medical ethics at the University of Pennsylvania. “It just fesses up to the reality of access to care in the most honest way I’ve ever seen.”

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