Not even the sixth time was a charm for the pharmaceutical company Biogen in its efforts to get muscle wasting disease treatment Spinraza approved for use in a wider group of patients than the small number who currently have access to the treatment.
The Danish Medicines Council recently rejected a new price proposal from Biogen for the drug, which in practice is only currently approved in Denmark for newborns and children aged under 8 with type I or type II SMA. No other patient group has access to the treatment.
"The council has received information that Biogen has proposed a new price for nusinersen (Spinraza) as the offer does not have any accompanying new data and concerns a group of patients who are not included in the current recommendation, the chair has concluded that the discount does not give sufficient reason to reconsider the council's recommendation for nusinersen," the minutes from the latest council meeting state.
Biogen executives cannot understand the decision over the drug, which was approved for treatment of rare muscle wasting disease spinal muscular atrophy (SMA) in 2016 by the FDA and later by the EMA.
We have tried so many times because Denmark is a sore spot for Biogen, as it is the only country in Europe that does not use the treatment for all children and young people with SMA. We have therefore bent over backwards for this treatment
Biogen's Danish branch has now tried to get the medicines council to expand the approved patient group a total of six times.
"We have tried so many times because Denmark is a sore spot for Biogen, as it is the only country in Europe that does not use the treatment for all children and young people with SMA. We have therefore bent over backwards for this treatment," says Tashia Lentz, CEO of Biogen Denmark, to MedWatch.
In Norway, Sweden and Finland, Spinraza is approved for standard usage in children and young people aged under 18, explains the CEO, who took over from former boss Janne Harder in the spring. In Denmark, the patient group is limited to pre-symptomatic newborns, and children who were diagnosed before they turned 2 and have had symptoms for a maximum of four years before starting treatment.
Since she stepped into the CEO role, the expansion of the use of Spinraza has been one of Lentz's top priorities.
"We will not give up – I will not give up. I will continue the dialog with the Medicines Council, and Biogen and I will do everything we can to ensure more Danish patients have access to Spinraza," she told MedWatch when she joined the company in May.
Impossible to provide the data the council wants
In Denmark, the pharmacy's official purchase price for Spinraza is DKK 572,233 (USD 90,290) per vial, totaling a treament price of DKK 3.4m (USD 536,600) in the first year and DKK 1.7m (USD 268,300) for subsequent years – however, hospitals buy the medicine at a confidential discount, meaning the actual price is estimated to be lower. Nevertheless, the drug has been the subject of discussion and debate because of its price tag.
This was relevant last summer, when the Danish Medicines Council declined to expand the use of Spinraza to include a broader segment of patients.
Like today, the reason given for the rejection then was the drug's price, which the council thought was too high. In addition, according to the council, the drug lacks documentation for its efficacy. Nontheless, the decision provoked furor among patient organizations and politicians, and the Danish Association of the Pharmaceutical Industry (Lif), also voiced its stance that the drug should be reevaluated.
The Danish Medicines Council's expert committee also wanted to widen the usage to more patients.
"Back then, just as now, Biogen wanted Denmark to expand the treatment so that more children and young people with SMA could receive treatment in Denmark. Today, only children aged 8 or under receive the treatment in Denmark, and newborns. It's around 4-6 children a year," says Lentz.
"We've gone very far – how far I can't say of course. The price offers to Amgros (the procurement center for the Danish regions) are confidential," she explains.
In its rejection statement, the Danish Medicines Council mentions that your new offer might come with a price reduction but lacks new data supporting the wider usage of Spinraza. Why can't you offer new data, if it could potentially change the council's stance?
"The data the council wants is impossible for us to deliver. The treatment is already well-regarded and offered in other countries for children and young people, and in several places also for adults. So, it's unethical to create studies in which some people get the product and others don't," says Lentz.
Biogen has also made Denmark a new offer: an outcome-based deal, which is also called a pay-per-performance program, which takes into account the treatment's effect on patients.
"However, we haven't succeeded in getting through with that," Lentz states.
Hopes competitor has more success
Biogen's Spinraza isn't the only spinal muscular atrophy medicine that the Danish Medicines Council is unwilling to make widely available. Roche, too, was met with closed doors for its competing treatment Evrysdi (risdiplam).
In a council meeting on Sep. 1, the minutes of which have only just been publicized, the council submitted its opinion that Evrysdi be recommended for the same patient group as Biogen's Spinraza.
This decision comes in spite of Roche's application that includes a data set with a wider age distribution than Spinraza's.
I have to admit that I hope our competitor has more success than we've had. For the sake of the children.
What's Biogen's next move? Are you giving up?
"I've taken a moment to think. I'm the new CEO at Biogen in Denmark, and I had great expectations that I was finally going to succeed in getting more Danish children and young people into SMA treatment. There's still no treatment for them, and I have to admit that I hope our competitor has more success than we've had. For the sake of the children," says Lentz.
The Danish Medicines Council will take its final stance on Roche's Evrysdi during its next council meeting on Sep. 29.