More from the reluctant advocate.
The July meeting of NICE left us worried.
The meeting had been held on 23rd July as planned. I attended with my fellow aHUSUK Trustee expecting to just view proceedings but we were seated in front of the Committee.
Most of the early part of the meeting was taken up by the Chair reprising what the evidence had been and explaining how the committee had arrived at its previous decision not to recommend eculizumab for aHUS.
Although the Chair did acknowledge that the Committee had received several letters from patients /families who had benefited from eculizumab to address the comment by a committee member of not having enough testimony from eculizumab patients at an earlier meeting.
aHUSUK had also submitted some estimates of the likely cost of treating aHUS patients for the next five years with revised figures for numbers and average dose cost These were acknowledged. It was our aim to create uncertainty about any unaffordability.
Little was expected from us , the focus was on Alexion and its evidence about price, or value as its representatives preferred to talk about. Nothing apparently had changed. There had been no patient access offer.
Several committee members asked the Alexion representative in a variety of ways to explain the price of eculizumab and how it broke down into production , R&D, profit etc but they were not willing , or unable to do so. “That was accountancy ” explained the health economics advisor. I just looked at the NICE officials we had met nearly 18 months ago.
I had to leave the meeting early because I had a flight to catch I was going on my summer holidays. As I left the room I was interviewed by the Channel 4 film team . They had been filming the proceedings. I was quite downbeat about it. Alexion’s performance had been disconcerting.
We had been told that an evaluation document would be released in September. It turned out to be October and a further meeting would be needed. Prior to the meeting we received a copy of the decision.
“Eculizumab, within its marketing authorisation, is recommended for funding for treating atypical haemolytic uraemic syndrome, only if all the following arrangements are in place:
- coordination of eculizumab use through an expert centre
- monitoring systems to record the number of people with a diagnosis of atypical haemolytic uraemic syndrome and the number who have eculizumab, and the dose and duration of treatment
- a national protocol for starting and stopping eculizumab for clinical reasons
- a research programme with robust methods to evaluate when stopping treatment or dose adjustment might occur.
The long‑term budget impact of eculizumab for treating atypical haemolytic uraemic syndrome is uncertain but will be considerable. NHS England and the company (Alexion Pharma UK) should consider what opportunities might exist to reduce the cost of eculizumab to the NHS”
The decision was qualified. We did not care. We agreed with them.
It was October 2014 and ,subject to someone appealing against the decision and against aHUS patients, every aHUS patient in England and subsequently Wales and Northern Ireland, ) could access eculizumab free at the point of treatment delivery when they needed it for as long as they needed it. Scotland would be different to England and would not approve eculizumab for Scottish patients , nor PNH patients , but that is another story!
We jumped for Joy.
The final NICE meeting in November was positive and short.
Two memorable moments were when for some reason the actual base figure for those receiving eculizumab emerged. It was less than the base figure on which we had estimated the five year cost of treating aHUS . We had given NICE costings based on a range of base patient numbers from low to high. Our mid point patient figure had used to show how NICE’s budget estimate had been vastly overstated by well over £100m.
The Chair of the Committee looked at me I was busily recalculating the budget effect. I said if the revealed base patient numbers figure was correct, it would take another £30million off our estimated five year budget. Had we known at the time another £40millon could have been knocked off the budget as nearly two thirds of the aHUS dialysis patients ,who we expected to have eculizumab supported transplants, have not yet been transplanted. No one knows why.
We had also raised another affordability argument. aHUSUK had found out in a news item in the Summer about the way in which Pharma agree drug prices with UK’s Department of Health. Agreed and operated by the Association British Pharmaceutical Industries, it set out the framework for price listing and discounting processes.
The new five year agreement had been radical. It set a no growth in the NHS drug budget for drugs for two years funded by rebates taken from the sales of all Companies listed in the agreement , of which Alexion was one. In subsequent years some growth of budget was allowed, but new higher rebates would be expected.
aHUSUK had said that this was in effect in reduction in price and would apply to eculizumab sales for PNH too.
At the meeting NICE were confused and questioned Alexion’s representative about it, who seemed perplexed. Reasonably so as they had failed to raise this in the first place. As confusion rained around I said it was aHUSUK who raised it and made the point that it would further reduce the cost of treatment as far as the NHS was concerned. Though no one knew where the money was.
NICE argued it was not relevant to their decision as they were comparing eculizumab with other drugs and their prices too would be reduced. I was not too bothered about that as we had already seen evidence that aHUS could run faster than PNH and Cystic Fibrosis from the “Opportunity Cost Lion!
NICE had told us the the day it would release the decision in the public domain. The Channel 4 film crew came to our house to record our reaction and I got to read out the recommendation. My only speaking appearance in the whole documentary. I did not care as we had got it.
Over three years had passed since aHUSUK and my volunteering began , and now we had finally got it for all aHUS patients when they needed it for as long as they needed it.
It would take a bit more time for it to be turned into NHS England commissioning policy , it would be given three months to do that. Before that there was time given for any appeals against the decision, and aHUS patients, to be made and reviewed.
There were none and on 28 January 2015 ( yep another Christmas had passed!) NICE made its final announcement on its guidance for eculizumab to treat aHUS. ( click here to see NICE’s full guidance).
The Channel 4 documentary was broadcast in February.
In the first week of April 2015 NHS England revised its temporary policy for treatment to became its final policy going forward.
Since then around 100 patients have received eculizumab when they became ill and went to their NHS for help.
No lobbying MPs , no media interviews, no gathering and presenting evidence to decision makers for them.
Their NHS , once a diagnosis for aHUS was made , just gave them a treatment.
Is that not all they could want?
Isn’t that world class?