NICE process begins it will soon be Christmas

In April 2013 we heard that NICE would not get to the evaluating and decision step until December 2013 , which would be over two years since aHUSUK had been created , none of the trustee advocates had expected that.

aHUS Advocacy in the UK – The Reluctant Advocate:  A series on the story of the battle of how eculizumab treatment became available to those people diagnosed with atypical HUS in the UK

NICE manages each evaluation as a project . Each evaluation is assigned a Project Manager. A Project Management Methodology is used to plan and control the operation in a logical manner.
The evaluation is broken down into stages and each stage results in a “product” , such as a document which defines the outcome and decisions made.
Each stage has a gathering evidence step, evaluating and decision step and documenting and communicating the decision step and then a consultation or quality assurance review step ( or steps as the latter can be reperated) before the stage is complete and the project moves  on a  stage.
In April 2013 we heard that NICE would not get to the evaluating and decision step until December 2013 , which would be over two years since aHUSUK had been created , none of the trustee advocates had expected that.
Although we were also preoccupied with the Clinical Priorities Advisory Group we began the NICE process by responding to a request to comment on the scope of the “eculizumab” project . We answered the questions posed and then attended a meeting on 13 July at NICE’s office. We were shocked by the numbers attending the meeting ; NICE tended to make its consultations public affairs. Anyway it seemed that aHUSUK had responded with the most detail and the conversation mostly addressed our views.
The initial stage of  the NICE evaluation  began with this scoping stage -where it establishes and defines what they are going to examine and how they will do it .
They decided :-

  • they were reviewing eculizumab;
  • to be used for treating atypical Haemolytic Uraemic Syndrome;
  • and compare it with other treatments including plasma exchange and dialysis and combined liver/kidney transplants;
  • they will gather clinical and economic evidence from the manufacturer;
  • they will gather patient impact data from the patient organisations;
  • the evidence will be gathered in a format they have devised for the Evaluation Group led by Prof . Jackson;
  • evidence will be gathered over the next eight to eleven weeks;
  • NICE will only meet with evidence suppliers if they need to  test understanding or seek more data;
  • Otherwise the next meeting will be on 11 December 2013 when the NICE HST Committee meets to make a decision.
So aHUSUK  now had to complete its Patient Voice Submission in less than two months ,which in effect meant improving the original AGNSS submission by adding the Welsh Patients testimonies, the data gathered from a Survey Monkey and for  other meetings, including CPAG . We hoped members ,including those from Scotland ,would continue to feed into the process and make it a very compelling document.
The meeting was a very positive experience because the  NICE staff  were keen to develop their an ultra orphan evaluation methodology,and  so many clinical experts attended who were very supportive of eculizumab for their patients.
Looking back despite how much we had developed as a patient group , our understanding of aHUS within the context of all thrombotic microangiopathies was just beginning. That knowledge would have benefited us more in understanding potential patient numbers for inclusion in the scope for treatment. We would know now  that it was only those TMAs that were “Complement mediated” which should be in scope, not other secondary causes of aHUS, thereby reducing the costs to be afforded. . Although there are overlaps between categories  that could apply.

With the scope defined, the next step would be supplying evidence of what it is like for those affected by aHUS, That evidence was called for in late July  and had to completed by 9th September. Less than 2 months.  Fortunately aHUSUK already had its evidence . Indeed we had improved it with interviews  of 4 Welsh patients and a carer. Doing it ourselves it revealed how hard a job it was to interview and record participants. Then transcribe the interview word for word before analysing the thread of the discussion to determine unique or confirming comments to use , and specific “sound bites” to make the point in a memorable way.
Except NICE had decided to use an on line questionnaire for patient organisations, but when we tried to copy and paste the content of our evidence research into the  relevant questions , the system crashed. We were providing “too much” evidence but  to precis it would ruin and diminish the work we had done. We were going through  an unprecedented third evaluation process not of our making . We complained to NICE and it was agreed that we would map and cross reference  relevant questions in their form to the sections of our research document , and we would append our full report to read. That plus  some administrative details and closing comments was our submission and it was provided on time.
The next step in the evaluation process would be the holding of a meeting of the Evaluation Committee. We  would be expected to attend. Indeed two representatives were allowed and it was decided that I and another trustee, who had been an eculizumab trialist, would provide any additional answers for the Committee at this  meeting.
NICE unlike AGNSS did not want a presentation, so we had no control over what we would expect to say. So we made a list of possible points we wanted to get across when a more or less  relevant question was raised. We would answer the question prepared for but not necessarily the question asked! All the contact with politicians was now rubbing off on us.
Eventually we were advised that the Committee would hold its meeting in public, unlike the AGNSS meeting at which only stakeholders attended along with the Committee.
It was scheduled  for 11th December 2013 and the result would not be known until 2014 but with an intention to complete the project by July 2014. A year on from the scoping meeting and nearly three years since aHUSUK was created.
In that time  an additional 75  English patients were likely to have onset with aHUS most of whom would not have known about the illness they had. At least now they had the safety net of the interim service.
Another Christmas was to pass before we would see the outcome of what we had come together to achieve.
It was not be a foregone conclusion and our anxiety about having to do this again was evident in  this extract from a press release by the charity shows.


Ahead of its appearance at NICE when eculizumab will be evaluated again, aHUSUK has released the following statement.
Anxious Patient Group calls on NICE to approve only drug treatment for ultra -rare disease
Fight for treatment over three years – NICE Committee in the spotlight as it evaluates first ultra-orphan drug 
On 11th December the National Institute for Health and Care Excellence’s (NICE) First Appraisal meeting is taking place to judge whether eculizumab should be recommended in England as a treatment for the ultra-rare disease atypical Haemolytic Uraemic Syndrome (aHUS).  National commissioning of this drug for the treatment of aHUS was recommended in June 2012 by the Advisory Group for National Specialised Services (AGNSS) which stated “It is clear that eculizumab is an effective treatment for aHUS, and gives a much better quality of life than the alternative treatments”.  Patients’ elation turned to horror when, following this recommendation Health Minister, Earl Howe, unilaterally decided that the drug should not be made available to sufferers, but must undergo a further evaluation by NICE, the first time NICE has ever had to approve an ultra- orphan drug for a rare disease.  Eculizumab has already been approved for treatment of aHUS in forty other countries and is even commissioned nationally for another rare disease in England.
Sufferers of aHUS have been living in a state of limbo.  Some have been able to access the drug, through interim funding measures and clinical trials, and are waiting anxiously to hear if they will be able to continue to take the drug and lead a normal life, or whether they will have to revert to previous treatments such as plasma exchange or much worse, be condemned to renal dialysis for the rest of their much- shortened lives.  The fight to get any access to this life changing treatment has been hard – in March and May of this year we delivered petitions to Downing Street and the House of Commons calling for everyone to be able to have access to this miracle drug.
“Eculizumab is the only treatment for aHUS.  Everyone, including ministers, NICE, AGNSS, CPAG and clinicians in the field, agrees that it is clinically effective. Dialysis and plasma exchange are ways to manage the disease but with this management strategy aHUS sufferers cannot work, cannot travel far from hospital and have to endure horrendous treatment side effects.  Eculizumab allows sufferers to live a normal life, to work and contribute to the economy.  We urge NICE to think differently when it comes to payment for and commissioning of treatments for very rare diseases that have devastating effects on a few sufferers. We trust that  its Evaluation Committee will show it can make well-rounded decisions for rare diseases, like its highly regarded predecessor, AGNSS , and will not condemn all aHUS patients to horrific and foreshortened lives,”.  Ian Mackersie, Secretary of  aHUSUK Patient Group.
“Our lack of confidence in the process of approval for this drug, given it has already been approved by two previous evaluations [AGNNS and CPAG], has meant we have had to fight publicly to try to ensure NICE does the right thing.  The treatment is life transforming.  aHUS patients  previously had no light at the end of the tunnel, now they do.  Surely they too deserve the right to get the very best care available today.  NICE can take that bright horizon away from us and we cannot let that happen.  This is a heavy burden for aHUS sufferers and their families to shoulder in addition to coping with living with this terrible disease”.  Emma Woodward, Trustee, aHUSUK Patient Group.


NATIONAL INSTITUTE FOR HEALTH AND CARE EXCELLENCE (NICE) regarding  Highly Specialised Technology Evaluation (HSTC)

Eculizumab for treating atypical haemolytic uraemic syndrome (aHUS):  2nd Evaluation Consultation Document (ECD)

Click HERE to read the aHUSUK Comments on ‘Consideration of Evidence’, courtesy of



NICE: aHUSUK’s Experience – courtesy of Find A Cure UK

Click HERE


See also a Review of the Reluctant Advocate Series  by MHealth Apps



The Reluctant Advocate:  Read the Series 

(Click Title links below)

A change in gear  Part 1
Where the heck did that come from?  Part 2
Now where is my tutu?  Part 3
Hurry up and Wait  Part 4
Much ado about nothing   Part 5
“It’s  BLOODY  Scary!”  Part 6
Don’t Rain on Our Parade!  Part 7
The Higher you build your Barriers   Part 8
Good Will Come Together?  Part 9
What do We Need?  When do We Need It?  Part 10
The Finest Hour of the Few    Part 11
Affordable Means Able to Afford    Part 12
One Step at a Time   Part 13

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