In the Netherlands, a collaboration of (pediatric) nephrologists nationwide led to a new treatment guideline which in the end enables tailored therapy with the orphan drug eculizumab and a considerable cost reduction. The nationwide study CUREiHUS, initiated by the Radboudumc and led by Dr Nicole van de Kar, pediatric nephrologist, Prof Jack Wetzels, nephrologist and Kioa Wijnsma, PhD candidate, is one of the hallmarks of this new national approach and acquires a lot of (inter)national attention.Eculizumab, nowadays the standard treatment for patients with atypical hemolytic uremic syndrome (aHUS), is considered one of world’s most expensive drugs with costs as high as €500,000 per patient per year. Besides the high price tag of eculizumab, physicians treating aHUS patients have sincere concerns regarding the long term consequences, when eculizumab is used as lifelong maintenance therapy. Especially since there is little to no evidence to support this life long duration. In the Netherlands, this led to the development of a new Dutch guideline, drafted by the National Working Group aHUS, which enables a restrictive eculizumab regimen. The implementation of this guideline led to a whole new political discussion in the Netherlands. Only recently, the National Health Care Institute of the Netherlands declined reimbursement of eculizumab as lifelong therapy. However, with the implementation of this new guideline, which is shown to be (cost) effective and safe, the minister of Health has decided to reimburse eculizumab reimbursed, solely when subscribed according to the new guideline. Instead of lifelong maintenance therapy with the orphan drug eculizumab, patients will receive eculizumab for three months after which therapy will be evaluated and while closely monitored withdrawn if possible. With this nationwide prospective study, abbreviated CUREiHUS (NTR5988), sponsored by ZonMw and Zorgverzekeraars Nederland, this restrictive regimen will be monitored for and evaluated after four years.
Recently the newspaper NRC published a extensive background story regarding this new and unique phenomenon in the Netherlands, interviewing both involved patients as physicians and politicians.
See also: link.
Related news items
Often additional demand for care without decreasing number of admissions Telemonitoring in chronic heart failure not unqualifiedly positive19 October 2021
To contain rising healthcare costs, digitization of healthcare is often seen as a solution. Researchers at the Radboudumc examined the use of telemonitoring in chronic heart failure. Does this reduce hospital admissions and visits to the emergency room?read more
Epigenetics: Immunization is passed on to offspring Mice transmit adaptations to infections to next generations19 October 2021
Does an infection affect the immunization of subsequent generations? It does, according to research now published in Nature Immunology.read more
Why we are curious about 'useless' information A neurocognitive explanation13 October 2021
In our daily lives, we are exposed to an overload of information. We found ourselves to be curious about both 'useful' information such as weather forecasts, and 'useless' information such as the infinite content on social media. A new study provides us a cognitive explanation to such curiosity.read more
Recognition & Rewards: panel discussion 20 October 202111 October 2021
What makes a good researcher? How should we evaluate and reward good academic practices? What metrics, if any, should we use to evaluate university staff and award funding? Could deviating from current international standards be good or bad for Dutch academia?read more
Palliative care for people with Parkinson’s Disease and their family Caregivers Current state of affairs7 October 2021
Advanced stage Parkinson’s disease can cause a variety of symptoms, for which palliative care can be beneficial, though research from the point of view of patients in later stages is still rare. Radboudumc researchers therefore placed their patients perspectives at the center of their recent study.read more
Genetic mutations cause severe magnesium deficiency5 October 2021
Newly discovered errors in DNA change diagnostics and treatment. Jeroen de Baaij tracked down new genetic errors that cause magnesium deficiency and published the findings in the Journal of the American Society of Nephrology.read more