According to a story from euronews.com, the drug maker Novartis has announced that it will give away its breakthrough spinal muscular atrophy gene therapy drug Zolgensma to 100 patients each year. However, the method for deciding who will receive it has drawn criticism: a random lottery. The drug was approved last year by the FDA and costs $2.1 million for a treatment, making it the single most expensive therapy in the world.
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. Without prompt treatment, the disease is lethal in many cases. 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.
The Lottery System: Fair or Cruel?
While it is true that the company’s announcement means that 100 more lives will be saved by Zolgensma, the fact remains that there are thousands of babies born with the disease every year around the world—meaning that most patients will lose the lottery and be unable to receive the treatment for free. Patient advocacy groups from around the world, such as SMA Europe, have leveled criticism at the company’s preferred method for distributing the 100 free doses.
“…The programme will make thousands of SMA babies compete with each other for a life-saving treatment, splitting tightly knit communities and turning this experimental drug into a coveted prize.” – SMA Europe
In the US, even patients with expansive insurance coverage are having difficulty getting Zolgensma because of its extreme price. This makes the lottery system feel like little more than a twist of the knife for patient families who are already suffering from the trauma of a potentially fatal diagnosis—a piling on of further unnecessary emotional anguish.
The company claims that the system was decided upon based on the fact that the production of the treatment currently cannot meet the pace necessary to treat every single patient that needs it. Therefore, the lottery system was determined to be the fairest method of distribution.
There is no question the Zolgensma is effective. Ellen de Meyer of Belgium was able to successfully raise the money necessary so that her daughter could get treated, and the results have been impressive.
However, even Ellen, whose daughter’s life was saved by the gene therapy, expressed reservations about the lottery system:
“I cannot imagine to get your hopes up every two weeks, hoping your child’s name will be drawn from the lottery, only to be disappointed again.” – Ellen de Meyer
