Lifelong use of the drug eculizumab by people with the rare kidney disease aHUS is often not necessary. In most patients, a three-month course is sufficient. This is what doctors at Radboudumc in Nijmegen conclude on the basis of a five-year study, in collaboration with the other UMCs. Eculizumab is one of the most expensive medicines in the Netherlands: it costs about 450,000 euros per patient per year. Because the manufacturer prescribes lifelong use, this study saves 50 million euros.
In patients with aHUS, small blood clots form in the blood vessels of the kidneys. Without treatment, these become seriously damaged, resulting in kidney failure. Every year, about five to ten people in the Netherlands receive this diagnosis. Since 2012, there has been for eculizumab, which prevents clots. It is given through an IV. According to the manufacturer’s advice, this should be done every two weeks for a lifetime.
Because the drug is so expensive – eculizumab is in seventh place in the list of most expensive drugs – the government wanted to stop the reimbursement in 2016. Thanks to Radboudumc, in collaboration with the Kidney Patients Association, provisional reimbursement was made, provided patients participated in the study.
Radboudumc followed 21 patients, among others. The disease returned in 4 of them. They received the drug again within 48 hours. There is no clinical damage in these patients, the researchers said.
Also other medicines
The researchers are calling for more expensive drugs and their guidelines to be scrutinized. The Dutch Healthcare Authority established this summer that expenditure on medicines is rising so fast that other specialist medical care is being supplanted. Nephrologist Jacques Wetzels, who is involved in the study together with pediatric nephrologist Nicole van de Kar and medical researcher Romy Bouwmeester, says: “Manufacturers make their own guidelines for drug use. They prefer not to use less, they want to earn money.”
Wetzels says that the discussion about expensive drugs should be had. Doctors and patients took a risk. Van de Kar: “Internationally, we and the patients were looked at with suspicion. “Don’t you play Russian roulette with your kidneys?” was said to a patient.”
Margreet de Vries is the mother of Rosa (9), who became critically ill in 2014. Thanks to an observant doctor she received the correct diagnosis. Read also: To swallow or to choke? It can also be different
De Vries: “She barely survived.” Rosa has not needed a cure for four years now. Why did the family participate in this approach? “I find it appalling that the pharmaceutical industry makes so much money from our child’s illness.”
But what weighs just as much, she says, is that it is taxing to spend half a day in the hospital every two weeks. There are also side effects, such as an increased risk of meningitis. De Vries says to remain alert, but that it is valuable that they are now “out of the care circuit”. “We now have a child with an illness, but not a sick child.”