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A UK website about atypical Haemolytic Uraemic Syndrome (aHUS)


aHUSUK realises that the case for eculizumab is based on avoiding  the severe consequences of aHUS ,which many patients are facing or  have faced.

Elena’s story in a Devon newspaper today illustrates how successful eculizumab can be and the good news it brings. The article  can be read by clicking here.

Elena was very close to kidney failure and was reliant on the burdensome treatment of plasma exchange. The treatment was not fully effective in stopping her  uncontrolled complement activity which was slowly destroying he kidneys.

Once on eculizumab her complement activity was quickly controlled. Significantly ,however, as aHUSUK is hearing  time and time again, Elena’s kidney function increased. What is being learned is that the sooner  eculizumab is administered after aHUS  diagnosis, the more kidney function is likely to be restored. Something that is not claimed for Plasma Exchange.

The Department of Health talk about the rounded advice needed from NICE ;but as  AGNSS’ balanced scorecard “wheel”  ( click wheel and  see page 5)  of decision making criteria illustrates, their decision that eculizumab should be made available to all who need it was indeed rounded.

Eculizumab was AGNSS’ last appraisal, it was the Minister who decided that NICE would take its responsibility because of changes brought about by the Government’s Health and Social Care Act 2012.Who knows then why someone, and we still do not know who  ,selected one issue   from AGNSS’ balanced score card to justify the case for delay, and then took so long to do it!

There has been plenty of time to look at that one issue in the nine months since AGNSS met to make their decision, but, because of the reorganisation  within  the NHS generally,  and  in NICE  specifically ,the transfer of Ultra Orphan Drug evaluation responsibility,  as well as everything else going on ,was one project too many.So instead of hitting the ground running with an enhanced interim process for the next therapy , there is a gap.

This gap has left eculizumab still  in the frame, and, with the kalydeco appraisal having  already body swerved NICE’s attention, NICE now needs eculizumab to test the success of their project.

Indeed aHUSUK believes it can offer much input to help NICE develop a successful evaluation framework , as we already did with AGNSS through the development of the Patient Voice process.  But when aHUS patients are not receiving the best therapy and there are people on dialysis/plasma exchange who could have their burden of treatment removed, the focus should firstly be on people not process.

  So aHUSUK  think it is completely unacceptable that aHUS patients should now take the hit for the Department of Health’s failings to act properly.That can be put right by a clear statement that all aHUS patients who need it now, whether to avoid kidney failure or to get out of it, should receive eculizumab irrespective of where they live.. …. as AGNSS has already recommended. 

That is what our e petition is all about, and why 3500 people have signed it in just thirteen days!

As Elena says ” “We have decided we should fight for this. We feel responsible to help the others that haven’t experienced it yet.”







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