According to a story from Xconomy, a recent report from the Institute for Clinical and Economic Review (ICER), based on Boston, is calling for reductions to the price tags of critical therapies for spinal muscular atrophy, a rare, progressive neuromuscular disorder. The report finds that nusinersen (marketed as Spinraza), which is currently the only approved therapy for spinal muscular atrophy, is way overpriced. Nusinersen is manufactured by Biogen. The report also says that the projected price tag for a new and currently experimental therapy from Novartis is also too high.
About Spinal Muscular Atrophy (SMA)
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. There are a variety of management strategies for spinal muscular atrophy, but it is still usually fatal in its most severe forms. To learn more about spinal muscular atrophy, click here.
Effectiveness vs. Cost Effectiveness
Nusinersen was a groundbreaking moment in spinal muscular atrophy treatment and was first cleared for use in 2016. The experimental therapy from Novartis, called Zolgensma, could be another major advancement that would require only a single dose of treatment for patients to see long term (and potentially lifelong) benefit. While there is no question that these drugs do and can offer major improvements for spinal muscular atrophy patients, ICER was still unable to deem them cost effective.
For spinal muscular atrophy patients that have yet to see symptoms, ICER projected that the annual price for nusinersen would need to drop to $65,000 per year or less, which is far under the current price tag of $750,000 for the first year and $375,000 for every year afterwards. Similary, ICER valued Novartis’ new drug at $900,000 per treatment, far under the potential price tag of $4 or $5 million.
To the surprise of no one, both Biogen and Novartis have taken issue with the contents of ICER’s report.
