Unique approach to orphan drugs in the Netherlands

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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patients will receive eculizumab for three months after which therapy will be evaluated and while closely monitored withdrawn if possible.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: https://www.nrc.nl/nieuws/2017/06/22/slikken-of-stikken-het-kan-ook-anders-11220990-a1564075



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