When the Food and Drug Administration approved Zolgensma, a life-saving drug for the treatment of spinal muscular atrophy, the parents of young children with rare and fatal illness were delighted.
But this relief was quickly mitigated by the price: The most expensive one-time drug dose
"I was quite shocked," says Sara Stunger of Monroe, Ohio. "You know, as a teacher we definitely do not have $ 2.1 million and I do not know anyone who does it."
Stunger's son, Duke, was diagnosed with the condition as a child. When Zolgensma was approved in May, doctors said the drug was the best option for Duke. But the family insurance company refuses to pay for it.
Without drugs, Duke's future is bleak. In babies with SMA, nerve cells in the brainstem and spinal cord that control the muscles needed to talk, walk, breathe and swallow are destroyed because there is no critical protein. As the disease progresses, muscles weaken and atrophy, and patients lose their ability to walk, eat or even breathe, according to the National Institute of Neurological Disorders and Stroke. told NBC News. "Without treatment, most children will be over 2 years of age."
When the drug was approved, Zolgensma's Novartis said it expects insurance companies to cover the cost of the treatment. Novartis also said that the high cost of the drug is justified, and the one-time treatment is half the cost of 10 years of treatment with an existing SMA drug.
But Butler Healthcare, Stangers Health Insurance Provider, says their arguments for refusing to pay for Zolgensma is that this type of therapy has historically been excluded from the scope. Zolgensma is a type of gene therapy
"To date, gene therapy … is excluded from the benefits provided by our health benefits plans," says Stephanie Hearn, executive director of the Butler Health Plan. This is because despite the potential of gene therapy to cure or cure debilitating diseases, therapies are costly, and health care providers still need to understand how to balance the cost of these costly treatments without jeopardizing the coverage of others on the plan
But this math will become more and more difficult to solve over time, according to David Mitchell, founder of the Patient Protection Group for Affordable Medicines.
"The situation we are seeing now with access to Zolgensma is a problem that will only get worse," Mitchell said, "There are at least 400 other gene therapy therapies, and if they all come to market at 2 million dollars, we can not afford it either as a family or as a nation, "he said.
Novartis told NBC News that a" broad circle of patients "had the medicine covered by his insurance approval, but noted that it was not uncommon for
Just a few days ago a big insurer and a company, UnitedHealthcare, overturned its decision to refuse to pay Zolgensma to two children whose cases have been publicized.The company reported to NBC News that the conversions have happened because they received more information about the cases than the media attention  In the end, these cases are another example of how the healthcare system in the United States is failing patients, says Dr. Albert Wu, intern and professor of health policy and management at Bloomberg School of Public Health Johns Hopkins .
"This is a bad reflection of how our healthcare system is currently working – or not working – The only way people can get paid drugs is through the Hail Mary GoFundMe site or by generating a bad enough press that the payer thinks it is not worth fighting, "said W. Linda Carroll is a regular health care associate of NBC News and Reuters Health. & Facebook