What do we need? When do we need it?

… The more you refuse to hear my voice (ooh-weh ooh-weh ooh-weh ooh-weh).

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

Back to the U.K. ( see previous) and OK putting the rhetoric aside for a while , we were facing another hurdle but just what could we do about it?
We were by then a rare disease organisation with  the families of about 15 or so aHUS patients as members . We knew of another 15 or so  aHUS patients  at least; but they were not prepared to join with us. There could have  been the families of another 150 aHUS patients but they probably did not know what was happening for them and about them.
We had to decide what we wanted,  and then have plans to act which would not overwhelm a small ,and it has to be remembered , still ill group of people, with little or no resources.
We could not be political,  aHUS people are from all sides of the political spectrum . It would too easy to go to a newspaper which supports an opposition political party to have a go at the political party in power. That be would wrong.
On the day of the Government’s announcement about the AGNSS outcome, the aHUSUK Secretary was in Parliament attending and talking to a meeting called by the Opposition Health Minister about Rare Disease treatment access. This politician had been banging on  for months in debates about how the implementation of Government reforms of the NHS would present high risks to patients. On that day the aHUSUK Secretary was able to give him a newsworthy example of how Government changes had put a small group of patients at grave risk. He did nothing.
Neither did the Health Minister who made the unjust decision and who was not even prepared to meet and discuss his decision with us.
A media campaign was out of the question . We sought advice and were told it would cost us over £100,000 and we would have to do a lot of the work. We could not afford that.
We would have to find a way that reporters and journalists would come to us for free, but remembering that the Government and its agencies had public relations budgets of £ millions. It would be an  unfair competition. Our strength was we were the victims in more ways than could be imagined.
But what would be our message about what we wanted.
We wanted AGNSS recommendation implemented
We wanted it done quickly
We wanted aHUS dialysis patients in scope
We wanted to influence NICE from the outset .
With the latter we were conceding then that we would be the “guinea pigs” for NICE’s new process but in return for that, we wanted aHUS patients, there and then, to be treated equitably while the review took place.
We also wanted equity built into what NICE did.
So our aim was “to get eculizumab right then for aHUS patients who needed it for as long as they needed it”
So then

“What do we need……..?”


  “When do we need it? “



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  (This article)

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