For the desperate parents of terminally ill babies, a new gene therapy is a ray of hope in the darkest of times. But what if the agent is not yet approved, if it is two million euros per dose, the most expensive drug in the world?
The Swiss pharmaceutical manufacturer Novartis is giving away starting on Monday (3. February), treatments for 100 infants and young children up to 2 years. Could apply to families from all over the world, whose child suffers from spinal muscular atrophy (SMA), and for which no other therapy helps. This is a survival lottery, as critics say?
Marina mantle’s son, Michael, is born in 2018 with SMA. “Six weeks ago we thought he would no longer be healthy to the core, then he moved suddenly,” she told the German press Agency. In the hospital, then the devastating diagnosis: SMA. The family is devastated.
Michael has SMA. For the parents is a devastating diagnosis
Then Michael gets the first 2017 approved SMA-drug Spinraza the US company Biogen. It helps many, but not Michael. “He got the lung problems, could mucus expectoration hardly,” says mantle. Nearly 50 children are born in Germany as Michael with the worst Form of SMA in the year.
Last hope on the lottery
Then a coat of Zolgensma from Novartis, approved in may 2019 in the United States hear. She struggles, wants to be rejected is a hardship exception, start a fundraiser, then your health insurance company, a distracting. Michael gets the drug in September 2019, exceptionally, although it is not yet approved. “It’s good for him, he can turn around now self-employed, he can sit,” she says today. Such a development had previously been impossible.
"It is very difficult to tell parents that their child is there. It is the worst thing I have to do."
“Other parents are also desperate and struggling,” says mantle. They criticized that the admission of Zolgensma in Europe, have to wait for. “Of course, a lottery is not right, but at least Novartis does anything,” she says.
No more doses available
Around 200 patients were treated in the United States so far, with Zolgensma. On the lottery, Novartis came to their own information, with an ethics Council, because it wanted to provide the therapy in the world as quickly as possible, says a spokesperson for Novartis.
Only questions Concerned: Why didn’t the Novartis instead of 100 treatments for the nearly two million euros per dose 1000 treatments at a low price? The production capacities were limited, says the spokeswoman. More than 100 cans could not deliver the only plant in Illinois in the United States this year, in addition to the expected orders.
For the price per dose, the costs for the other drug for SMA children was crucial, says Dave Lennon, head of the manufacturer AveXis, which is part of Novartis. Zolgensma, which is administered only once, was grossed over ten years, half as expensive as Spinraza that needs to be injected every four months.
In order to give all patients a fair Chance, just came randomly out of the question, the Novartis spokeswoman. “It is a Dilemma,” she acknowledges. “We just didn’t have as many cans as we like. For all the criticism of this method, there is a lack for now of alternative proposals.”
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Fair trial – says Novartis
Medical ethicists Norbert W. Paul contradicts. Ethical it would have been a clear set of criteria as a prerequisite for the administration of the drug to define, says the Professor of the Mainz University medical center. For example, whether there is for the children, alternative therapies, whether a clinic was in the vicinity that can deal with gene therapy, whether a follow-up treatment and in case of emergency is also a crisis in supply was possible.
The lottery, he refuses to accept it. “Novartis, subject to the approval, in order to have a foothold in the market and to make so much pressure that the admission does not seem to be necessary in runs with this charge out of compassion,” he says. It was like a covert marketing campaign. It is the impression, as if it were the drug to be a magic bullet, and as the standard therapy with Spinraza was bad or a cheap variant to emergent. “The is not so,” stresses Paul. “But, of course, desperate parents, grasping at any straw. Even more disturbing, a raffle is.”
Monday decides the lot
Rejection also comes from the Federal managing Director of the German society for Muscle disorders (DGM), Joachim shoot. “This is a Dilemma Situation at the expense of the parents,” he says. “If someone has a medical indication, he must have access to the drug”, he demands. The Ministry of health, registration authorities, Doctors and parents would have to finally come to the table to find the best way forward. “Of course we are pleased in principle that there is therapies. Because no one would have five years ago is still expected.”
On Monday one of Novartis contract research organization is now for the first time, the name of one of the children to get the treatment for free. Every few weeks, another draw follows. How many applications were received, says Novartis. If the home country of the child allows for the treatment with the unapproved drug, and a treatment center there is, can Zolgensma within weeks administered.
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