When scientists are able to develop a new treatment for a rare disease that severely impacts the lives of very young children, it’s generally considered good news.
And in the case of the newest announcement from Novartis, it is. But it’s not that simple.
The drugmaker, who is parent to the company AveXis, recently revealed a new treatment for a debilitating genetic disease called spinal muscular atrophy. The condition affects up to 25,000 people and is currently the top genetic cause of death for infants, and a new gene therapy has recently gained FDA approval to treat it.
But it seems there’s a catch: the one-time treatment, called Zolgensma, comes with the largest price tag for any drug treatment in the world: $2.1 million.
So, even in a country known for its skyrocketing prescription costs, is this defensible? Novartis CEO Vas Narasimhan believes the price tag is fair when you consider the tradeoff, stating "Zolgensma could create a lifetime of possibilities for the children and families impacted by this devastating condition.”
Initially, the company publicly discussed pricing possibilities that could reach up to $5 million, which is why Dr. Steve Pearson, the president of the Institute for Clinical and Economic Review, is declaring this actual price “a positive outcome.” But while many experts acknowledge that treatment for this condition is already quite expensive, neither of these defenses have been enough to insulate the drug maker – and industry at large – from backlash.
David Mitchell, founder of the advocacy group Patients For Affordable Drugs, released a statement Friday calling the situation “emblematic of our broken system.” And while Novartis suggests that an initial balloon payment for “a lifetime of benefits” is an improvement on the pay-as-you-go model for chronic illnesses, Mitchell is critical, saying: "We didn't pay for the polio vaccine based on the future cost savings for kids who didn't need to live in iron lungs."