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A medical concern that produces the most expensive drug in the world – Zolgensma – for children suffering from spinal muscular atrophy came up with an unusual idea that was criticized by parents of sick children. The company wants to draw a randomly selected child who will receive free therapy. There are as many as 100 doses of the drug to be drawn.
Zolgensma randomly selected among sick children
Novartis is a concern that produces the most expensive medicine in the world. Zolgensma is a drug approved in the United States for the treatment of spinal muscular atrophy in children under two years of age. The one-time cost of the therapy is $ 2,1 million. So far, the drug has not been approved for use in Europe.
The company decided to sponsor 100 doses of the drug for small patients in an unusual way. Every few weeks, he intends to randomly select one sick child who will receive free therapy. The lottery is aimed mainly at young patients from the United States, but also children from Germany can take part in it. The Paul Ehrlich Institute in Lagen, which studies vaccines and biomedical medicines in Germany, has stated that “there is no reason to oppose Novartis’ agenda”.
Therapy with Zolgensma is a single infusion. During treatment, the damaged SMN1 gene is replaced with a functional copy. The cost of the therapy, converted into PLN, is about PLN 8,2 million.
Parents outraged by the competition for Zolgensma
Novratis explains that the lottery is a response to the criticism that continues to fall on the company over the horrendously high price of the drug. Unfortunately, the idea of drawing turned out to be completely wrong. First, the lottery is criticized by the parents of sick children in Europe. On the one hand, they appreciate the fact that the company will offer access to therapy to children around the world, and on the other, they believe that the lottery system is unfair because it excludes non-US patients.
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SMA Europe also found it disturbing that the “draw” of children for free therapy would take place without the involvement of clinical specialists. Patients with SMA are to be selected blind. All this makes life-saving therapy for a child with SMA a welcome reward and forces children to compete for treatment.
Therapy with Zolgensma
Zolgensma is a drug that has been approved by the United States Food and Drug Administration (FDA) for use in children under two years of age with spinal muscular atrophy (SMA). It is available in the US from May 2019. The drug is produced by the Swiss medical concern Novartis. The cost of the therapy is USD 2,1 million, i.e. over PLN 8 million.
Treatment consists of replacing the missing or defective SMN1 gene that is responsible for the occurrence of spinal muscle atrophy with a working copy of it. In the case of Zolgensma therapy, the age of the sick patient is important. The drug is only given to children under the age of two.
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