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UMC asks for approval for very expensive medicine against muscle disease

Utrecht University Medical Center has submitted a request to the health inspectorate to have children with the rare fatal muscle disease SMA treated with a drug that has not yet been approved. As a result, sick Dutch children can be registered for a raffle for the expensive medicine.

For the time being, with 1.9 million euros, the most expensive medicine in the world, Zolgensma is only approved in the US. But at the end of last year, Swiss pharmaceutical company Novartis announced that it would raffle the drug to one hundred children with SMA.

That decision led to criticism from European health ministers. “The great uncertainty and lack of transparency are totally unacceptable,” the Dutch minister Bruins stated in a joint statement, among others. “It only causes more suffering to the families involved.”

Last straw

Parents see the medicine as a last straw; the drug would work better and longer than any other, approved drug for SMA: Spinraza, which costs around 83,000 euros per injection and has been included in the basic health insurance package since this month.

“I agree with the minister that a lottery is not the right way to get a life-saving drug. But it is this or nothing for us,” said Arwen van Pelt, mother of a baby with SMA, yesterday.

Urgent need

The UMC today emphasizes in a press release that the Zolgensma draw “is at odds with equal access to care for all patients”, but that “due to the urgent need of some parents” a request for approval of the drug has been submitted to the inspection . This means that parents can still participate in the draw.

To Omroep West, Arwen van Pelt responds with joy and at the same time angrily to the news, because she states that doctors have always told her that registration for the draw was not possible. “So they lied to us as parents,” says. “We now have to wait and see what the inspection will say. But the confidence in what we are hearing is a bit gone.”

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