Starting from December, Belgium will reimburse the world’s most expensive drug, Zolgensma, which is used for treatment against the rare disease spinal muscular atrophy (SMA).
The treatment became more widely known in Belgium two years ago when the family of Pia, a toddler who suffered from the disorder, successfully crowdfunded enough money to afford the medicine, which can cost up to €2 million.
“Access to such an important medicine should no longer depend on collection campaigns: the solidarity of the health insurance is needed here,” Federal Health Minister Frank Vandenbroucke said on Friday in a press release.
SMA is a rare, incurable and hereditary neuromuscular disease that results in the loss of motor neurons and progressive muscle wasting, and that affects around ten babies every year in Belgium.
Before the medicine could be reimbursed, it had to receive approval to be marketed in the European Union. By May 2020, it had been given the green light by both the European Medicines Agency (EMA), which issued a favourable opinion on the drug’s usefulness and effectiveness, and by the European Comission.
Now, following negotiations in collaboration with Ireland and the Netherlands, Belgium reached an agreement with Swedish producer Novartis to have Zolgensma repaid from the end of this year.
The contract between Novartis and Belgium, signed on Thursday evening, states that Zolgensma will be reimbursed for children up to two years old (or weighing less than 13.5 kg) with symptomatic SMA type one or patients with presymptomatic SMA with up to three copies of the SMN2 gene.
Without treatment, a baby diagnosed with type one of the disease would have a life expectancy of up to two years. Although the treatment had a successful outcome of baby Pia so far, it is yet unclear what the long-term effect of the medicine will be.
Meanwhile, Flanders is working on organising a system for regular screening of all newborns to detect and better help those with pre-symptomatic SMA, as is already the case in Wallonia, where there is organised screening from the first months of a baby’s life.
Alongside SMA, six other diseases will be added to the list of rare disorders that will be added to the checklist for newborn screening in Flanders.