11th December 2013 Part 1

It was a cold, crisp and frosty morning in Manchester, England on 11th of December 2013 . Above the sky was vivid blue when it could be glimpsed through the fog that shrouded much of the UK that day. Road conditions were poor as I set off to the city.

Once inside the NICE office in the centre of Manchester , we were taken to the 20th floor of the Tower and the view confirmed that, above the fog, the day was bright and clear into the distance ;but all the well known Manchester landmarks were invisible.

The Evaluation Committee meeting began with the Chair explaining  how the meeting would run. After introductions and declarations of conflict of interests the witnesses were addressed in turn. Giving  evidence, apart from ourselves, was Prof. Tim Goodship and a Dr Rodney Gilbert from Southampton Hospital, the NHS Specialised Services leaders ( including Dr Edmund Jessop  2019 Winner of EURORDIS’ “Policy Maker  Award”) , and a team from Alexion. The academic guys from Sheffield University, who were expert in health economic evidence critique, were also there. The Evaluating Committee was made up of clinical experts from a range of medical roles, some lay members, and were supported by officials from NICE itself , some of whom we had already met.

No patient presentation had to be made, instead one of the Evaluation Committee’s lay members  summarised the case from our patient submission. His conclusion was the evidence was “rich and robust” and that “it made a compelling case for a call on NHS resources”.

WOW ! The only issue now was that I and the other patient representative said nothing that would detract from that view! The only criticism of our report was that there not enough stories about patients who had received eculizumab. That was true but few had accessed it so far and only some of those who were trialists were known to us at the time.

It had been  difficult to find aHUS patients, as we had told NICE at the outset, and eculizumab had not been approved so by definition such patients were few and far between.  However my aHUSUK  patient expert colleague was a recipient of eculizumab but was not included in the report as it had been completed before she joined us. She was able to tell the Committee how ill she had been with loss of kidney function and needing dialysis after plasma exchange became less and less effective. She had been included in the eculizumab trial and was given doses of the drug. Soon her aHUS came under control  and she recovered some kidney function. She came off dialysis and began to feel better. With better health she returned to work and got married. She was also starting a family. That is what eculizumab can do.

When asked for any concluding comments I remembered what my daughter had said at the first AGNSS meeting. Eculizumab for those on dialysis meant FREEDOM.  Freedom from living a life around a dialysis machine, Freedom to eat and drink as we all do, Freedom to work and have a full life again. It goes without saying that those newly onseting would no longer need to know what that aHUS life would be like.

Unexpectedly the expert from Sheffield University in his critique mentioned an article on the aHUSUK website about a visit to Milan to find out more about the pioneering work of Dr Gianluigi Ardissino in adjusting and tapering the doses of eculizumab in his patients ,and with some withdrawing from treatment once stable. This of course meant, unlike the “licensed full dose for life” on which the Committee had to consider the drug , that the average cost per patient would be much less. We could not have brought that up, it was not part of our evidence, but now  it was part of the discussion and some doubt about the true treatment cost had crept in. Eculizumab when needed for as long as needed . It should not be wasted at that price.

The morning session went very quickly and there was a break for lunch. The meeting was going very well. In part of the afternoon session  Alexion was  asked to talk to the Committee without anyone else present; such was the commercially sensitive nature of the talk about its price for eculizumab.

It was a relief when the meeting ended and “witnesses were released”. I do not know if it had been the coldness of the day or the dryness of the air-conditioning, but by the beginning of the afternoon my voice was disappearing and became croaky! Maybe it was nature’s way of saying “ shut up and say no more, the patients’ voice had already done its job”.

The Committee would spend the rest of  meeting making its decision in private. Our main concern was about what Alexion had said during its closed session, it could be very detrimental.

We had been told that the next step would be the release of the Evaluation Decision document ( the “product” of this stage) . It was likely to be published sometime in late January 2014 ( after our third Christmas as a charity).

Personally none of that seemed to matter to me for now because during the lunch session a message had got to me (I was not one for mobile phones!) . It was from my wife. The hospital had called. There could be a kidney for my daughter.

There was no rush!

 Link to 11th December 2013 (Part 2) here.

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