According to a story from The Washington Post, two families affected by the rare disease spinal muscular atrophy have cause for celebration after UnitedHealthcare agreed to cover their treatment with Zolgensma, a recently approved gene therapy that has the potential to provide lifelong benefits to patients, effectively curing the disease. The single use therapy costs over $2 million per dose. This follows prior denials of coverage for the therapy from the company. However, UnitedHealthcare now says that it has approved all requests for coverage for Zolgensma.
About Spinal Muscular Atrophy
Spinal muscular atrophy is a type of neuromuscular disorder in which the motor neurons are destroyed, leading to muscle wasting. In many cases, the disease is lethal. This disorder is linked to genetic defects of the SMN1 gene. This gene encodes a protein called SMN, and when not present in certain amounts, neurons are unable to function. There are different kinds of spinal muscular atrophy that are categorized by when symptoms first appear. These symptoms may include loss of reflexes, muscle weakness and poor muscle tone, problems with feeding and swallowing, developmental delays, respiratory muscle weakness, tongue twitching, and a bell shaped torso. The most effective treatment currently available for the disease is called Zolgensma. To learn more about spinal muscular atrophy, click here.
Insurance Companies: Doing Their Job, Sometimes, But With Reluctance
The story highlights the potential for future standoffs between patients, doctors, and insurance companies over covering treatment with gene therapies, which, while having great potential to treat and even cure many genetic diseases, will almost certainly have high price tags. 21 month old Daryn Sullivan was one of the patients that will receive coverage for the drug; Zolgensma is approved for patients under two years so her mother Lauren was understandably relieved when she received a call from the company.
Because of the great cost of the drug insurance companies are not very keen on helping their customers that need it. Daryn was initially denied coverage because of supposed lack of symptoms, and competitor insurance company Anthem won’t cover the drug for patients greater than six months old despite the fact that some patient families may not have been diagnosed by then. The company claims that this severe and extreme restriction is backed by clinical data. UnitedHealthcare would also deny coverage to patients who they deem have too extensive nerve loss despite the lack of evidence demonstrating that the drug isn’t effective in patients with more advanced disease.