Continuation of patient advocacy in the real world….
Then came the announcement and the “No decision” conversation. ( click here for that!)
To say aHUSUK trustees were incandescent would be an understatement. To keep us waiting for seven months on a decision that AGNSS had actually made and had approved eculizumab; and to say it was all to be done again because AGNSS was disappearing and to be replaced in April by NICE was absolutely deplorable. Appalling.
We were thwarted as there was no right of appeal. A great injustice had been foisted on to aHUS patients in England all because the NHS was to be re-organised and the Health Minister wanted a review on what “affordability” was and would use aHUS patients to find out. Another set of hurdles for an unfortunate cohort of people with a rare disease.
The Minister said that aHUS patients who needed Eculizumab could seek “Individual Funding Requests” (IFR). This was the process that had failed aHUS patients so far as it sought uniqueness within a rare aHUS cohort so it could not be for all. It had created a postcode lottery and much discrimination even within families, and it was why a National Specialised Service was needed and had been applied for and which AGNSS had agreed to be given . Indeed under the “new” NHS rules, if four patients got IFRs approved for a single therapy it would trigger an application for a National Specialised Service to be considered approved . That is precisely what going through AGNSS had been about. A suitable plot for a Gilbert & Sullivan comic opera or “Catch 22” type novel.
A bit of news that we had heard a few days before the announcement made us scratch our heads. The NHS had approved a national service for a specialised treatment for a rare disease. A rare cohort of those suffering from Cystic Fibrosis. We knew that the Cystic Fibrosis Trust was raising awareness for a drug at the same time as us , I had even signed a petition that they had set up for the drug to be made available , such was our support for rare diseases by then. They were not in AGNSS programme at that time and so were behind us in the “queue”
Except once AGNSS had ended, and before they would need to go to NICE Cystic Fibrosis clinicians, the pharmaceutical company , the patient group and the NHS conspired to develop a bespoke evaluation and funding process while aHUS patients were waiting for the outcome of AGNSS . Within 3 months it delivered a Specialised Service to be delivered Nationally, but not a National Specialised Service which, of course, it could not be. ( Good luck to CF patients it is an awful disease, as bad as aHUS , though perhaps not as immediately life threatening. It did seem that their drug did not appear to be as effective as eculizumab). aHUS patients could now die. It had been predicted that over 10 would die in the coming 12 months.
aHUSUK needed to act and would have to campaign, not for the drug to be approved , we did not need to campaign for that our Patient Voice did its job , now it was the injustice of a decision making process for which we had no right of appeal. It was our appeal.
No right of appeal and the Minister making the decision refused to talk to us.
Oddly at around the same time we heard the results of an application we had made to a large kidney patient organisation, BKPA, it had turned down our request for financial support because it considered us to be a campaign group for patients, not a patient support group. We were doing both, we had not been political but advocating for aHUS patients, an extremely small group of people because of its rarity. Something this industrial size charity could not get its head around at that time.
BKPA would continue to keep large sums of money in its bank account for which reputedly it was getting criticism from the Charity Commission about.We needed funds though as publicity for our cause could cost us. One of our members made a donation to the charity to be used for awareness projects. Along with that came excellent advice because this member had also had experience of campaigning for a specialised service for another rare disease Pulmonary Hypertension which affected her family as aHUS had too.
aHUS people were going to die but had no rights to life, and others surviving would be destroyed through injustice.
They had been treated wrong. So wrong
Something inside was getting so strong.
The higher they build their barriers the taller we became….
