No one said it would be easy

The reluctant advocate story continues:

aHUSUK had received the Evaluation Consideration Document the week before Rare Disease Day 2014 and we participated in Rare Disease Day events knowing what we knew but not able to say anything in public. So patients and others would not be aware.

NICE was minded NOT to recommend eculizumab for aHUS.

The prime reason was Alexion’s inability to explain why the price of eculizumab  was what it was.

We could not imagine that a company like Alexion had no clue about why its price was what it was but we were at a loss to understand why it was unwilling to explain itself. Despite assurances that  it would do all it could on price for use of eculizumab for aHUS. , it had failed.

The clinical and patient case had been made. Alexion did little  different to what it had done at AGNSS . It had not made an offer for a patient access scheme nor was it able to explain its price. An accountant could easily explain it. It is what we said to NICE at the outset.

aHUSUK was as baffled by this outcome as it is convinced that as a patient organisation,  it could not have done more to make its case. NICE had  told us, as did AGNSS before it, the patient case was made.

aHUSUK was dissatisfied with the way aHUS patients have been treated by our heath authorities over the past two years. aHUS patients must have felt devastated at hearing what NICE is now telling them.

We were back to where we were and having to keep that  secret for a week built up our frustration and annoyance. We were unhappy with both Alexion and NICE .

I was invited to talk through the decision with the NICE patient engagement team in early March.  I said before I left for the meeting that if NICE wanted to say “no” we would be facing a Final Evaluation Document at this point so maybe it was a case of “needing to read between the lines” . At the meeting I think I was forthright and angry as I felt aHUS patients would be about this latest hold up. In the  explanation  I got from NICE I was told to “read between the lines”. I left assuring them we would do all we could do to lobby Alexion to concede what we had implored them to do on price( which we subsequently did in a meeting with Alexion). I also said that we would challenge the figures being used by NICE to question the affordability of aHUS patient treatment.

So reading between the lines NICE would wish to say “yes” but needed more reasons  to justify doing so. Those reasons relied mostly on Alexion.

NICE said there would be another meeting in April after the formal responses to the ECD had been received from all.

So this was now set to go on and into the Summer.

aHUSUK view on it wasDrawn out over almost two years ,three separate evaluations have been carried out of a drug that everybody admits is clinically wonderful and life transforming. Has any other group of patients ever been put through such an ordeal and had its hopes raised, then dashed, then raised again now dashed again? And all the patients have done is to be unfortunate enough to have a rare  genetic condition.

Fortunately we had CPAG’s Interim Policy because ,without it, what had happened to aHUS patients would have been  unbearable . Although it is yet to be confirmed by NHS England, whatever NICE’s final recommendation is to be the drug will not be withdrawn from those already benefiting from it to save their lives and preserve their kidney function.

The sticking point has existed for 21 months now and aHUSUK had , on a number of occasions in that time ,exhorted both the NHS and Alexion to get round the table to talk, clarify and collaborate on a sustainable  solution  because simply that is all that is needed.But our exhortations so far had been in vain.

It looks now as though  NICE sending aHUS patients back to square one was inevitable, even if unforgivable ;but we can only hope that at last,  a suitable and sustainable solution can be found.  So it is now up to those, whose job it is to do so ,to use the next few weeks to simply get it done.”

Enduring all this makes a mockery of those who would claim later that eculizumab for aHUS Patients was got by going through a “loophole” denied to them. Some bloody loophole!

It is not easy being an advocate. It is not irrational to not want  to be one. For us it was not as simple as just occasionally putting some sentimental quotes on Facebook. it was now like being in a business environment but not paid. No one said it would be easy.

More about the Reluctant Advocate’s experiences so far read here.

Get something done to raise awareness about aHUS and Rare Diseases join in with the Rare Disease Day Video Project more Information here.