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
  • Nouran earned her BS and MS in Biology at IUPUI and currently shares her love of science by teaching. She enjoys writing on various topics as well including science & medicine, global health, and conservation biology. She hopes through her writing she can make science more engaging and communicable to the general public.
You May Also Like
APR 21, 2020
Neuroscience
APR 21, 2020
Considerations for Lab Managers in Choosing a Microplate Reader
In today's high-tech, digitized laboratory environments, nobody pays very much attention to the humble plastic micro ...
APR 20, 2020
Genetics & Genomics
APR 20, 2020
Advances in Gene Therapy for Neurons
New research may aid in the development of gene therapies for diseases like Alzheimer's and Parkinson's.
APR 21, 2020
Drug Discovery & Development
APR 21, 2020
Classic Chemotherapeutic Repurposed for Treating Leukemia
Researchers were seeking a promising new strategy to challenge drug resistance in leukemia. They found that targeted low ...
APR 27, 2020
Drug Discovery & Development
APR 27, 2020
Making Cardiac Medicine From Foxglove Plants
Foxglove plants are known to be shaped like bells and have long decorated many gardens. They belong to a genus of plants ...
APR 30, 2020
Drug Discovery & Development
APR 30, 2020
FDA to Approve Remdesivir to Fight COVID-19
A clinical trial investigating the ability of antiviral drug remdesivir to treat COVID-19 has found that it can signific ...
MAY 21, 2020
Cancer
MAY 21, 2020
A New Player in the Regulation of Cancer's Microenvironment
Breast cancer is one of the most common cancers in the world.  Triple-negative breast cancer (TNBC) is a sub-type o ...
Loading Comments...