And then came the price tag: $2.125 million for a one-time treatment.
The gene therapy, called Zolgensma, will be marketed by AveXis, whose parent company is Novartis.
The list price, which was anticipated to be in the low millions, immediately reignited controversy around the rising costs of drugs and how to pay for them.
“It is emblematic of our broken system that effectively forces us to pay whatever price drug corporations demand for lifesaving new drugs,” David Mitchell, founder of the advocacy group Patients For Affordable Drugs, said in a statement Friday.
Novartis leaders said Friday that the price met their targets of cost-effectiveness, with the newly approved treatment costing about half of what another treatment for the disorder, Spinraza, might cost over 10 years. They said that the company will have patient support and payment programs in place and that they do not expect out-of-pocket costs to be prohibitive for patients who need the treatment.
“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,” Mitchell said in criticizing the pricing decision.
Spinal muscular atrophy is an inherited disorder stemming from a defective gene that leads to the death of nerve cells responsible for moving muscles that allow us to walk, talk, breathe and swallow, according to the National Institute of Neurological Disorders and Stroke. Zolgensma replaces a working copy of that gene into those nerve cells before they die and symptoms develop.
Sharpless said Friday that the agency plans to help speed up the pace of reviewing products that address unmet medical needs and bringing them to the market.
“Health-care systems, however, are accustomed to treating chronic diseases with a pay-as-you-go model, spreading costs over months and years,” Narasimhan said. “They are currently unprepared to pay for a surge of new, single-treatment therapies with the potential to provide a lifetime of benefit.”
But Mitchell, a cancer patient himself, said the company was abusing a broken system, “forcing families to answer one question: ‘What are you willing to pay to save your child’s life?’ “