By May 2013, after nearly three months of campaigning by the “few” and getting the issue to the attention of the Health Minister and Department of Health, two notable events happened.
In April NICE had taken on responsibility for evaluating eculizumab for aHUS, but was not ready to do so and it expected to begin its work on eculizumab in December.
The e petition response from the Department of Health confirmed this , but also said that in the meantime another NHS group would look at the service to be given in interim period.
So the NHS had shifted its position and was now prepared for an interim policy to be implemented ahead of NICE . Starting with all new onsets. A newly created Clinical Priorities Advisory Group decided at its first meeting that new onset aHUS patients were a priority for treatment. The first sign of a change of mind but we also wanted to bring aHUS dialysis patients in scope for a transplant . NHS now had to do it via this new group which had been set up in the NHS reforms. Whilst yet another hurdle for aHUS patients to get over there would be no more discriminatory individual funding requests in a post code lottery .
By July 2013 CPAG held its second meeting which aHUSUK Trustee for Patient Voice , along with Alexion and Prof. Goodship were invited to attend and present to the Group ( our research document was the basis of the patient’s voice, it had been added to and improved upon since the AGNSS meeting so we were confident it would do the job.)
Immediately after the meeting we were told that an interim aHUS Service had been approved for all aHUS patients , and it would be included in the NHS Specialised Services list for 2013/14. The service would be interim one pending the review of eculizumab by NICE.
The CPAG meeting was the day after the first formal meeting by NICE to define the scope of the evaluation of eculizumab for aHUS. So with that we were back to the stage we reached with AGNSS in October 2011 but with some progress made for existing patients.
We can never be sure what went on behind the scenes but this shift was announced following the aHUSUK campaign and the noise created by tens of thousands of people who felt we had been poorly treated. Even the Health Minister invited aHUSUK to visit him finally ( Sylwia an aHUSUK member had telephoned a radio programme with the Deputy Prime Minister as a guest and who agreed to arrange a meeting with the Health Minister) and welcomed our intention to help NICE develop its new Highly Specialised Technology evaluation process providing existing aHUS patients were treated.
So we would be doing it in the knowledge that aHUS patients known about there and then would have access to eculizumab even those who were trialists and those who needed a transplant.
If NICE turned down eculizumab for aHUS at the end of its review future aHUS patients , including those on dialysis who could not be transplants in that time, would not be treated.
Our job now was to help NICE make the right decision that eculizumab should be given when needed for as long as is needed.
This concludes the second tranche of chapters in this series for now. Previous chapters can be read at the links below.
The Reluctant Advocate: Read the Series (Click Title links below)
A change in gear Part 1
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
Good Will Come Together? Part 9