Parents React to Novartis’ International “Baby Lottery” for Zolgensma with Hope But Also With Outrage

According to a recent article in Euronews, this year one hundred children who were diagnosed with spinal muscular atrophy (SMA) will receive Zolgensma, the world’s highest-priced drug, at no cost.

Novartis, a global healthcare company, is calling this unique give-a-way a lottery or random draw. Doctors have been encouraged to submit the names of their eligible patients as of January 2, 2020.

Mixed Emotions From the Spinal Muscular Atrophy Community

Although this is a generous move by the drug company, Novartis, there are still thousands of families throughout the world who are unable to afford the treatment for their babies. This is one reason for the mixed reception to the Novartis initiative.

Patient representatives have voiced concern about the emotional effect of the lottery on the majority of parents who want to do everything possible to save their babies.

About Zolgensma

Zolgensma received USFDA approval in 2019. It is a one-time therapy that is priced at $2.1 million per treatment. The drug is administered via a sixty-minute intravenous infusion. Only one dose is required.

The drug sends out a healthy copy of the survival motor neuron1 gene (SMN1) to cells in the body. When the cells reach the spinal cord the SMN1 gene begins to produce normal SMN protein. The protein encourages motor neurons to function normally and improves the child’s chance of survival.

Although Zolengsma cannot reverse any damage already caused by SMA, it is able to stop the deterioration of nerve cells and give the children a chance at normal development.

About Spinal Muscular Atrophy (SMA)

SMA may cause permanent paralysis or even death within a year or two of birth. Spinal muscular atrophy (SMA) is a group of hereditary diseases that progressively destroys motor neurons. These are the nerve cells in the brain stem and spinal cord that control essential skeletal muscle activity such as speaking, walking, breathing, and swallowing, leading to muscle weakness and atrophy. Motor neurons control movement in the arms, legs, chest, face, throat, and tongue.

Concern Among the European Medical Community

SMA Europe has come forward with its concerns about the lottery. It issued a statement saying that thousands of babies with SMA will be forced into a competition for the free treatment.

The statement further objected to any drug company being in a position to decide whether or not someone gets a lifesaving treatment. It believes that medicine should be prescribed according to the priorities determined by the clinic.

The European Union is currently considering marketing approval of Zolgensma. Separately, France and some other countries have a system that allows access to therapies prior to their formal approval or negotiated price.

About the Developer

AveXis, the company that developed Zolgensma, was acquired by Novartis. An AveXis spokesman said that the company met with ethics experts in an effort to ensure that the allocation of Zolgensma was fair. The company said that the lottery was the best system at this time to make the drug available on an equitable basis.

The president of AveXis claimed that at this juncture it is unable to produce enough medication to treat all the SMA babies requiring the drug. On a more positive note, he said that they expect to begin to triple production by the start of 2021.

A Universal Dilemma

The worldwide health systems have an ongoing problem deciding how the pharmaceutical companies can be rewarded for their continued innovation while still considering that patients have access to precious life-saving drugs. Zolgensma was originally funded by many charities that had an interest in the discovery of drugs to treat SMA.

Looking at the price from Novartis’ point of view, the company claims that Zolgensma is extremely expensive to produce. Novartis has poured billions of dollars into its purchase of AveXis which it says far surpasses the investments set out by either private or public partnerships.

Crowdfunding

Several parents have set up crowdfunding operations in an effort to pay for Zolgensma. Ellen de Meyer of Belgium successfully raised 1.9 million euros to pay for the treatment of Zolgensma for her infant daughter Pia.

Their doctor coordinated the arrangements and Pia, who is now thirteen months old, received her treatment in an Antwerp hospital.

Pia’s mother is amazed at her daughter’s progress. She explains that there is an improvement in hand coordination, an end to her respiratory problems and that she is able to roll over.

Upon hearing about the lottery, Ellen admitted that she feels sorry for the parents involved. She cannot imagine the emotions and stress of waiting every two weeks to see if your child’s name will be drawn.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

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