Hurry up and wait

  • 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.

It did not take long to realise the meaning of the saying “Hurry up and wait” as far as the NHS was concerned. “To be done by” dates for third parties, which aHUSUK the name of the charity formed in the Wimpole St, had become, were fixed in stone ,whilst dates for the NHS were flexible.
So aHUSUK’s first meeting with the Advisory Group for National Specialised Services (AGNSS) was held on 31 October 2011 and not early in October as was expected. Two Trustees attended the meeting, the Chair of the charity and our daughter. The three-hour meeting was being held to discuss just the scope of Eculizumab for aHUS, not to evaluate it. The “evaluation meeting” was now predicted to be held in June 2012. So much for it being over by Christmas 2011, and, amazingly, eight months would be needed just to get ready for the Advisory Group to evaluate the drug.
The meeting was also the first opportunity to meet some of the members of the Group. Also the people from the NHS who managed the whole process and whom we became reliant on as we learned what was needed, as none of us had done anything like this before.
It was also the first encounter with employees of Alexion and their consultant advisors.
Professor Tim Goodship also attended the meeting.
My daughter’s recollection of the meeting was how welcoming and hospitable everyone was with refreshments laid on for an early morning starting meeting due to finish at lunchtime. As she was the only one attending the meeting who was living with aHUS ( and on dialysis ) she was asked to give a brief introduction of her experience. Perhaps the most telling illustration she could give was that in front of her was a small glass tumbler of water which was still full. Although everyone had been kindly offering her drinks before the meeting ,and in the breaks , she said that that cup represented her total fluid intake allowance for the day. The food laid on had also contained too much salt and potassium  related items and was harmful for someone reliant on dialysis. These are the kind of day to day challenges that those not familiar with dialysis struggle to understand. In such simple ways the patients voice was already resonating.
Later, the Chair of AGNSS spoke to her and commented on how well she looked despite fourteen years of dialysis and asked whether she would wish to have an eculizumab supported transplant. “In a heartbeat..” was her immediate response “…as it would mean Freedom”.
The meeting also introduced the principles on which the decision-making framework had been designed with the patient’s needs seen as out front and central. It was a decision-making process that had been developed specifically for health technologies for those with rare and complex diseases diseases. It was for  treatments for less than 500 patients affected in England. Although no one really knew the exact number,there were fewer than 200 aHUS patients in England.
The underpinning principles were:
Societal value
Best practice
Sustainable Cost
Health Gain
The framework developed from these principles required evidence to show:
Does eculizumab work?
Is it the best way of delivering the service?
Is it a reasonable cost to the public?
Does it add value to society?
For each of these elements criteria were set and evidence would be gathered to determine if they were met. But the Group would take a holistic view across all criteria in their final recommendation.
aHUSUK believed most of the criteria would be met and could provide evidence to support that. However  the “reasonable cost “ test, when eculizumab was reputedly the most expensive drug in the world, was going to be a challenge even though aHUS patients would think it was reasonable for their needs! aHUS patients lived, if they survived, with the impact of the alternative and could say what that was like.
Trustees left the meeting with the task of providing a “Patient Submission” by 30th January 2012. No firm format was given for it as the Group were still consulting organisations who had previously gone through the process in the past , and to come up with a novel way of doing it. Given the lack of resources and experience that aHUSUK had, it was likely that we would be given access to consultants to help with the submission.
This was just the start. It was what the charity  had really been created to do and it needed a lot of work.
The time it was going take depended on the NHS, so the completion date was going to slip!
There would also be a lot of hurrying  up and waiting to come.
* For earlier parts click here.

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