Hiding in plain sight were neighbours Republic of Ireland and Iceland.
The south of Ireland is well known as the place where eculizumab is manufactured. It is in a factory just outside of Dublin.
There are aHUS patients there. Probably a couple of dozen or more.
There is no aHUS patient organisation nor has any patient reached out to us to our knowledge.
The situation in the Republic is:
Eculizumab is available but provision is complicated . Eculizumab is approved for reimbursement by the Health Service Executive (HSE) for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) through a Managed Access Process. This funding application process is in place solely to address long-term chronic funding issues for these indications and is not designed to assist hospitals in making acute financial decisions. Therefore, short-term in-patient management remains within the in-patient hospital governance.
Getting access to the drug was opposed by politicians who had things like below to say about Alexion
“Leo Varadkar criticises US drug firm’s ‘aggressive’ policy was a headline of a news paper featurong a story of the then Health Minister criticising how much profit Alexion made and how much its CEO was paid.!
“The health policy makers also considered suing US firm over high price of live-saving drug. It was astronomical they said.”
aHUS research in the Republic of Ireland is scarce. One published article (behind a paywall) is
Eculizumab reimbursed in Ireland despite high costs. PharmacoEcon Outcomes News 755, 23 (2016). https://doi.org/10.1007/s40274-016-3130-1
Another article by Rizman and Madeuman describes an unusual case of Idiopathic Atypical Haemolytic Uraemic Syndrome presenting with acute dystonia.
Oddly since the manufacturer pays its corporation tax there ( ROI has a notoriously low corporation tax rate.) there is a societal reluctance in the country against paying such a high price for the drug. But campaigning by PNH patients has helped get a decision to fund it.
Ravulizumab is not available for ROI aHUS patients.
Change the “r” to “c” and you get Iceland.
A country which Global Action is aware that there are aHUS patients despite a very small population. We wrote about it a couple of years ago READ IT HERE. but overlooked it in the original listing .
It affects only one family. Over five generations they have seen incomplete penetrance of the disease even among one set of twins!
We don’t know who they are but the lead of the research Prof. Diana Karpman from Sweden is well known to us.
Any leads on aHUS patients in any country not included in the table below are welcome. They ill be followed up and added to the Town list.
Now 63 neighbours!
Article No 753
aHUS Awareness Day is on 24th of September.
