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The sound of Sognsvannbekken can be heard right up to five-year-old Adele Elvira’s room at Rikshospitalet in Oslo.
She can and will do a lot, but her body stops to do exactly the same as children her age.
RIKSHOSPITALET: There will be many visits to Oslo for the Høydal Sande family who live in Fosnavågen in Sunnmøret. Photo: Aage Aune / TV 2
When Adele Elvira was just over four months old, she was diagnosed with spinal muscular atrophy, often abbreviated SMA. It is a rare and serious muscle disease caused by the disappearance of motor nerve cells and, therefore, also by the disappearance of muscles.
I fight for life
After a long and hard battle, the very expensive drug Spinraza was finally approved for use in Norwegian hospitals. It should be taken four times a year and should stop the development of the disease. The price ended at a staggering million crowns per dose.
RESPIRATORY ASSISTANCE: Adele Elvira has needed respiratory assistance most of her life. Here with parents Karl Erik and Vibeke Høydal Sande. Photo: Aage Aune / TV 2
Doctors said he would be up to three years old, if they were lucky. But Adele Elvira became the first in all of Norway to receive Spinraza. This year she has turned five and she is scheduled to start school next year.
– The life situation has become completely different. Before we started with this, there was a battle over what we could do to make her survive. She is medically stable now, and then it is more about quality of life for her, says Father Karl Erik Høydal Sande.
Considering the most expensive drug in the world
In May, a revolutionary new drug, called Zolgensma, was approved in Europe. It is a gene therapy with completely new technology, which is involved in changing the genes of patients with SMA who have a defective gene.
But that medicine costs. The selling price of a dose of Zolgensma is a staggering 27 million crowns. In return, each patient only needs this dose, unlike Spinraza, which must be dosed four times a year.
THE WORLD’S MOST EXPENSIVE: A dose of Zolgensma has a retail price of NOK 27 million. Photo: Novartis / AP
– The price is probably the highest we have ever seen, says the unit manager of the Norwegian Medicines Agency, Einar Andreassen to TV 2.
The drug is known as the most expensive drug in the world. The Norwegian Medicines Agency is now considering whether Zolgensma should be used in Norway.
– We have now received this case for evaluation. And then we will consider how good this drug is and how much it costs. It then goes to the hospitals, which will decide whether it is an established treatment or not, Andreassen explains.
Back in Rikshospitalet, Adele Elvira receives her 14th dose of Spinraza. The drug has had a good effect on the five-year-old from Sunnmøret, but the parents are happy with the development now taking place in the field.
– Without Spinraza, we wouldn’t have been here today. It has been absolutely crucial that new drugs have arrived and we are completely dependent on ongoing research, says Karl Erik Høydal Sande.
FIVE YEARS: Adele Elvira had a life expectancy of two to three years when she was diagnosed with SMA in 2015. This year she turned five and is seeing a good effect from the drug Spinraza. Photo: Aage Aune / TV 2
There is no doubt that Adele Elvira’s medicine is also expensive. Four times a year, she and her family travel by car or plane to Oslo to receive the dose of Spinraza for about a million crowns.
And it is the price of these drugs and hospital budgets that greatly complicates the discussion.
The difficult discussion
– If a drug is prioritized for a new group of patients, then they are prioritized. Then there are others in the health service who are given lower priority. And those who are given priority lose their offer. This is why it is so important that we also have an assessment of how good these drugs are before they are used, Andreassen says.
The Høydal Sande family greatly appreciates the treatment and support that their youngest daughter has received. Now they hope other parents won’t have to face the same battle they did three years ago.
– The alternative was not good. So we had to have Spinraza and we had to fight to get it. So I wish those who become parents of a child with SMA today did not have to fight as hard as we do, says the father of the five-year-old.
Will have SMA included in newborn screening
The large Swiss pharmaceutical company Novartis markets the drug. AveXis is the biotech company behind the technology and development.
In an email to TV 2, AveXis writes that they are in dialogue with the authorities to collaborate on innovative payment models and solutions.
– For children with SMA to have the best possible effect from the medicine, it is important to treat it as soon as possible. Therefore, SMA should be included in neonatal screening, writes Sissel Rodahl.
She is the leader of Europe’s midsize countries on AveXis.
AveXis or Novartis have not yet answered the question of why the price of Zolgensma is so high.
