The U.S. Food and Drug Administration recently approved a drug that costs $2.1 million — a potential cure for a childhood disorder that afflicts children under 2 years of age.
According to Bloomberg, Novartis AG’s Zolgensma is considered to be a gene therapy targeting children who have an illness called spinal muscular atrophy.
The drugmaker based in Basel, Switzerland is willing to wheel and deal, offering payment options and refunds for those who have a failed treatment and also offering discounts to insurance companies who provide swift coverage.
Roger Stark, a health care analyst for the Washington Policy Center, said the high cost would be worth it for families.
“I don't have an opinion on the price, mainly because I don't know the cost of the R&D,” Stark told the FDA Reporter. “It is cheaper than the other alternative and is a one-time treatment. From a purely financial perspective, you could weigh the cost of the therapy against the infant's lifetime productivity which is likely to be higher. From an ethical standpoint, one could say that any potentially curative treatment is worth the cost — especially from the family's standpoint.”
Stark believes gene therapy very well could be the future.
Stark then was asked what kind of precedent this has set.
“This question touches on the whole issue of orphan drugs and therapies. Is it worthwhile for a company to spend the resources to find cures for a very select few patients? I'm not sure anyone can provide a cut-off number for potential patients below which resources should not be spent. In general, I would say the precedent is good,” Stark said.
Stark added there is another issue involved when it comes to gene therapies.
“Many, if not most, gene therapies will be directed toward childhood conditions. Half of all children in Washington state and about 45 percent of kids nationally are in the Medicaid or S-CHIP entitlements. In other words, taxpayers, not insurance companies or the families, will be funding the cost of these expensive therapies for a high percentage of children. Will the government seek price controls. Will the government limit who gets the therapies? There is a lot to be worked out here,” Stark said.
In a press release, a gene therapy investigator and the Novartis chief executive officer talked about the rare disease and the possible cure.
“A diagnosis of SMA is devastating, leaving untreated babies who have the most severe form with painfully short, highly medicalized lives, during which they are unable to lift their heads, sit or roll, have difficulty swallowing and breathing and need 24-hour care,” said Jerry Mendell, principal investigator at the Center for Gene Therapy at The Abigail Wexner Research Institute of Nationwide Children's Hospital in Columbus, Ohio in a press release. “In the START clinical trial we conducted with Zolgensma, all children were alive at the conclusion of the study and many were able to sit, roll, crawl, play and some could walk. This level of efficacy, delivered as a single, one-time therapy, is truly remarkable and provides a level of unprecedented hope for families battling SMA Type 1. We now have data four years out from the trial, and we see the durability of this gene therapy.”
“The approval of Zolgensma is a testament to the transformational impact gene therapies can have in reimagining the treatment of life-threatening genetic diseases like spinal muscular atrophy,” said Vas Narasimhan, CEO of Novartis. “We believe Zolgensma could create a lifetime of possibilities for the children and families impacted by this devastating condition.”
