MAY 23, 2019 11:29 PM PDT

Miracle Drug for Rare Genetic Disease Causes Price Concerns

WRITTEN BY: Nouran Amin

A newly approved “miracle drug” called Zolgensma will be used to treat children with spinal muscular atrophy, or SMA. Unfortunately, the therapeutic comes with a price target of $2.1 million for just a single dose to treat a child. However, Vas Narasimhan, the CEO of Novartis, the maker of Zolgensma, believes such a price is justifiable and thinks insurance companies should cover the cost.

“Critics are not actually thinking about how our healthcare systems work,” Vas Narasimhan said. “For transplants, we spend $3 million to $5 million [per patient] with far less efficacy than we see with medicine like this. We’re trying to price this at 50 percent of what is the current cost of care over ten years for these children.”

“I'm sure the company's going to try and push through laws to require insurers to pay for these drugs. The advocacy community that are often funded by the drug companies are going to push legislators to require insurers to pay for these drugs,” says Dr. Peter Bach, an epidemiologist and the director of the Center for Health Policy and Outcomes at Memorial Sloan Kettering Cancer Center in New York City.

According to the National Institute of Neurological Disorders and Stroke, babies diagnosed with SMA will later have weakened muscles as the disease progresses and eventually patients lose the ability to walk, talk, eat, and even breathe. SMA is a fatal genetic disease and affects approximately 1 in 11,000 babies with about 1 in every 50 Americans is a genetic carrier.

Learn more about Spinal Muscular Atrophy (SMA):

“In our early studies we’ve had 15 children who’ve been followed out to approximately four years and all the children are alive and doing well,” says Narasimhan. “The thing about SMA you have to remember is, children are developing mentally normally. So these are very bright children. They’ve lost the ability to move. So any regaining of function allows these children to start to experience life again. And I think that’s what we’re all here to do.”

Source: BioSpace

About the Author
Doctorate (PhD)
Nouran is a scientist, educator, and life-long learner with a passion for making science more communicable. When not busy in the lab isolating blood macrophages, she enjoys writing on various STEM topics.
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