Don’t rain on our parade!

The story of The Reluctant Advocate continues


14 June 2012 London – AGNSS Meeting to Evaluate Eculizumab for aHUS.

This was it, this is what we had been preparing for. The aHUS Trustee for Patients Voice was the only representative for patients allowed to attend. The only one allowed to speak and was allotted 5 minutes to present  to the Committee Members.
Alexion were there , the bulk of the evidence submission was theirs. The case for clinical effectiveness and safety , the cost effectiveness and explanation of how eculizumab was priced was for them to make. The clinical explanation and support for treatment  was provided by Tim Goodship.
As participants aHUSUK had been given rights to look at the written evidence presented to the Committee Members. There was over 700 pages of evidence including around 30 pages for the patient voice research paper that aHUSUK had submitted. Reading all this would change my understanding of aHUS  immeasurably and few patient advocates would get this education  anywhere.
There were reports on the eculizumab trials , there were estimates of patient numbers projected forward five years, there were the costs of eculizumab , there were the costs of dialysis and plasma exchange but no mention of costs related to damage done by dialysis. There were life expectancy  estimates with or without eculizumab, there was research on the quality of life of dialysis patients. There was even a “cost per QALY”.
The Cost per Quality Adjusted Life Year was an health service indicator of the cost effectiveness of new medicines and technologies compared with existing treatments. It involved estimates of costs of each ,  life expectancy in years depending on treatment used , and the quality of those year assessed on a scale between 0 and 1, where 1 was excellent health and 0 was no life. The difference in the quality of life for those on each treatment , say 0.9 for one and 0.2 for another to give  0.7 was multiplied by the difference in the number of life years to give the quality adjusted life years which when divided into the difference in the costs of each treatment to give the “cost per QALY” . There is a little bit more jiggery pokery using accounting techniques  to get to the figure. It was complicated but flawed.
Normally for medicines looked at elsewhere in the health service the QALY result would have to be lower  than £30,000 per QALY, but AGNSS was not bound by that as it was designed for technologies for rare diseases. Just as well as based on the evidence given to Committee,  the Cost of QALY was many times that figure. In a way it merely demonstrated  that Eculizimab was an ultra orphan drug. It produced excellent clinical outcomes but was it reasonably priced?
aHUSUK’s job was to show how debilitating and life threatening aHUS was and that Eculizumab offered benefits “beyond price”. We had our Patient Voice Report but we also had five minutes to get the point across too. It was import that every second of the 5 minutes was used and no more. Every word had to count. Three trustees and their families met and spent 8 hours designing and developing the talk and its supporting visuals.In run through after run through words were changed and times were cut until the optimum was reached. A five-minute talk emerged which said all that needed to be said and said it plainly enough.
“One of the best presentation we have ever had” said the Chair of AGNSS after the Trustee for Patient Voice  sat down after delivering the talk. A few questions followed and aHUSUK’s job was done and from around the room there was a sense that a good case had been made. So much so that when the next speaker got up to speak even he had to apologise for “raining on our parade”. . aHUS patients had felt the deluge of their illness so one drop more made little effect.His talk was about critiquing the evidence , he read his presentation out and sat down
We could do no more. The stakeholders including Alexion representatives and Prof Goodship , left the room. AGNSS went into a closed discussion during which they could call on stakeholders to return for further questions. We were not called. We did not know what had been said nor decided; we would not know because whatever they recommended would need to be given to the  Minister of Health , who at that time was The Earl Howe , to decide on whether to accept their recommendation. We were told it was in a metaphorical “black box” until the Minster opened it and made his decision to accept it or not. Only then we would know what the fate of aHUS patients would be.
The meeting ended. We waited.
Len Woodward was a founding Trustee of aHUSUK and a founding affiliate  of the aHUS alliance. He is also a Director Trustee of aHUS alliance Global Action. By his own admission he is a reluctant patient advocate , it is something that he had not been prepared for, and which he has no natural inclination to do. But for nearly 8 years now that is what he has been doing.
In this series of blogs Len Woodward explains and describes how he became an aHUS advocate and where the journey has been taking him to this day.

The Reluctant Advocate:  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

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