The US Food and Drug Administration approved a treatment last week for a genetic disease called spinal muscular atrophy that causes infants’ muscles to waste away, potentially killing them before age of two.
The price tag: $2.125 million for a one-time treatment. The list price immediately reignited controversy around the rising costs of drugs and how to pay for them.
The gene therapy, called Zolgensma, will be marketed by AveXis, whose parent company is Novartis. This approval marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases. The potential for gene therapy products to change the lives of those patients who face a terminal condition, or worse, death, provides hope for the future.
The advocacy group Patients for Affordable Drugs, said It is emblematic of our broken system that effectively forces us to pay whatever price drug corporations demand for lifesaving new drugs. Novartis said 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. Cigna Corp. said that the health insurer anticipated “continuing the work we have started with AveXis to find unique solutions like installment payments and outcomes-based agreements for these life changing gene therapies.”
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.
As many as 1 in 8,000 babies is estimated to be affected by one of the genetic mutations that causes it. That includes about 450 to 500 infants in the United States per year. The FDA said the agency plans to help speed up the pace of reviewing products that address unmet medical needs and bringing them to the market. In January, FDA leaders said they expect that the agency will be approving 10 to 20 cell and gene therapy products yearly by 2025.
Novartis called for a new pricing and payment model for one-time treatments like Zolgensma, versus other treatments with costs that accumulate over time. Health-care systems are accustomed to treating chronic diseases with a pay-as-you-go model, spreading costs over months and years. They are currently unprepared to pay for a surge of new, single-treatment therapies with the potential to provide a lifetime of benefit.
I guess the real question is “what are you willing to pay to save your child’s life?’
I filled out an application that said, “In Case of Emergency Notify”. I wrote “Doctor”… What the hell is my mother going to do?