Children with cancer are often prescribed non-refundable adult medicine. “Some parents have to take out a loan.”
Cancer in children is rare. Nevertheless, the disease affects around 320 Belgian children under the age of 15 and 180 15-19-year-olds each year.
The good news: Survival rates have improved dramatically, with 80 percent of all children with cancer still living five years after diagnosis.
The bad news: There are almost no added drugs for childhood cancer. Between 1995 and 2021, the European Medicines Watchdog (EMA) approved more than 169 adult cancer drugs. For children, there were 16, according to a recent study in the European Journal of Cancer.
- There is very little specific cancer medicine for children on the market.
- Pediatric oncologists prescribe drugs developed for adults, which they know from their own research that they are also clinically useful in children.
- That medication is not always reimbursed, so parents struggle with high bills.
‘Pharmacy companies do too little for pediatric cancers, although it must be said that it is very difficult’, says Delphine Heenen, who in 2017 founded the Belgian patient organization KickCancer. Her 17-year-old son Raphaël was diagnosed with a tumor in his foot in 2013 at the age of nine.
We ask for automatic subsidy of off-label medication prescribed to children as part of standard treatment.
Because there is little cancer medicine for children, pediatric oncologists prescribe off-label drugs: drugs designed and approved for adults that they have found to be clinically useful for children.
“This is about decades old, but also new cancer medicine for adults,” says pediatric oncologist Bram De Wilde (UZ Gent). “In the United States and in Europe, pharmaceutical companies have for years been required to add a pediatric research plan to their approval file for a new drug. For example, there must be treatment options for children.”
“But these studies often do not extend beyond the safety of the medicine in children. A final phase study was not always performed on children and presented to the European medical watchdog,” says De Wilde. Such a study looks at the effectiveness of the treatment. ‘Therefore, this medicine does not receive a grant, even though we know from our academic research that it works and that we administer it effectively.’
As a result, one in four parents of children with cancer has difficulty paying for non-refundable cancer medication each year, estimates KickCancer, which is hosting a conference on Saturday with all the actors involved.
‘I never calculated it exactly myself,’ says Heenen. Her son Raphaël has already had a relapse three times. ‘In 2016 alone, we paid 30,000 euros for one type of off-label cancer drug, a chemotherapy that had not yet been reimbursed at the time. We had to draw on our savings’.
‘I also know parents who had to take out a loan to pay an invoice of 125,000 euros. How high the cost of non-supplemental medicine will depend on factors such as age, medicine and type of cancer. ‘
I know parents who had to take out a loan to pay an invoice of 125,000 euros.
According to an estimate by KickCancer, together with UZ Gent and consultant Bain, parents in our country spend a total of 1 million euros each year on non-refundable medicines for pediatric cancers. ‘That number dates from 2019 and is probably higher in the meantime,’ says Heenen.
UZ Ghent is trying to help parents cover the costs, says pediatric oncologist Bram De Wilde. ‘We submit cases to RIZIV / INAMI’s special solidarity fund to receive compensation. This usually only happens in one out of three cases. ‘
‘We also encourage charities and sometimes pharmaceutical companies to make a medicine available for free. But all those steps require a lot of administration and create uncertainty for the patients. ‘
KickCancer therefore argues for a structural solution. ‘We ask that off-label medications prescribed as part of the standard treatment for children be automatically reimbursed,’ says Heenen. ‘We also suggest that people from RIZIV / INMI and pediatric oncologists meet every year to see what medication is allocated to each type of pediatric cancer.’