The manufacturer of the drug eculizumab has recommended a lifelong treatment, but the drug costs around 450,000 euros per patient per year. There were rumors calling for the compensation to be lifted. A national study on shortened administration of the medicine was therefore conducted.
relapse
The drug is given to patients with atypical hemolytic uremic syndrome (AHUS) with kidney disease. While three months of treatment is sufficient for most patients, there are also cases where patients relapse. For this group it works and then resumes hiring again.
For those who need the drug longer, the time between drug administration can be extended, the researchers found. It remains important that patients remain under the supervision of the hospital.
What does the disease involve?
In atypical hemolytic uremic syndrome (aHUS) kidney disease, blood clots form in the small blood vessels of the kidneys. Patients develop anemia, platelet deficiency, and kidney damage. This leads to kidney failure.
It is a rare condition, with five to ten new patients in the Netherlands each year, both among children and adults. Patients can be treated with the drug eculizumab. With this treatment, the small affected blood vessels in the kidney repair themselves, eliminating blood clots in these blood vessels.
Ruud Coolen van Brakel, director of the Institute for the Responsible Use of Medicine, is very satisfied with the practice-oriented research. According to him, especially with medicines for rare diseases, the effect in practice should be considered due to the costs of these medicines. It is not always easy. “The reality of paper is sometimes different from how something works for a patient.”
According to Coolen van Brakel, the government needs to look into how expensive a particular medicine is when it comes to a small group of patients. “Expensive drugs sometimes work for a certain group, while they don’t work for other patients with the same disease. Often the reason is discovered only after extensive research, almost per patient. But it is very expensive.”
No intent
He does not think that the manufacturer of the drug intentionally provided incorrect information. “Manufacturers also don’t expect many patients to benefit from their drugs or take too many. So no one will take them anymore.”
