Every year around 50 babies are born in Germany who never learn to sit or walk because of a genetic defect. Chances are they will have to die before their second birthday. A new therapy against spinal muscular atrophy (SMA) has been available since May last year: Zolgensma. An injection of this drug is enough to replace the defective gene. So far it is only sold in the USA. It costs $ 2.1 million. Four German families have obtained treatment from a health insurance company using a lawyer. In other countries, Facebook collects.
Now the manufacturer Novartis is giving away the treatment. On 100 patients each. Worldwide. The lot decides on the award. There has already been a raffle, the next drawing date is expected to be February 17th.
Is this a good solution?
Intended for the families who have been drawn, because they receive the help they long for without having to beg or go to court. Health insurance companies also save a lot of money. And incidentally, the media reports on the unusual lottery procedure attract a lot of attention from the manufacturer, precisely in the phase in which European drug authorities are consulting about the approval and reimbursement of the most expensive drug in the world.
So it happens that the drug donation is accompanied by suspicion. Jana Brandt from Sebnitz in Saxony is one of the parents who, with the help of a lawyer, managed to get her child injected. That was in November. Now John is ten months old, and now he can stretch his arms out for his mother. And he begins to turn: "He'll make it to the side, not to the stomach," says the mother. If she had been unsuccessful, she would have taken part in the lottery, she says. And yet the 31-year-old has concerns. The raffle is a "game of chance that is played for life".
Novartis seeks understanding
Inge Schwersenz of the patient association Initiative SMA sees it similarly. "Imagine you have a child with the diagnosis and you have to put it in a pot. That's scary!" She wonders why Novartis has not applied for a hardship program, as the law provides, when therapies are lacking for a life-threatening illness in a country. Then the manufacturer can deliver a product without approval. In a clearly regulated and monitored framework instead of by lot.
If you inquire at Novartis in Basel, you will receive a prepared letter that "for all criticism of this procedure" solicits understanding. "We only have very limited cans. Only one plant is currently licensed for production," the message continues. But two other plants are under construction. They would probably go into operation in 2021.
What is almost forgotten in the discussion is the fact that there is already a remedy for muscle loss. It comes from the laboratories of the US company Biogen and has been used in Germany for three years. However, treatment with Spinraza is more difficult for the patient. Instead of a single infusion, the drug must be injected again and again into the spinal fluid. And in the long run, this remedy may be even more expensive than its competitor. Although there are costs of between € 300,000 and € 550,000 a year, the treatment can take several years.