Here comes the sun

The Reluctant Advocate journey continues.
aHUSUK formally responded to the Evaluation Consultation Document by 25 March 2014 as requested by NICE. We challenged its decision to not recommend Eculizumab for aHUS , but agreed to do what we could to find evidence on affordability  and asserted that NICE and Alexion find sustainable solutions on price.
There was no NICE meeting in April as planned. instead consultees were asked to comment on the accuracy of a piece of research that had been done for the NICE committee by some financial consultants. The 60 page report was of a study  that had compared 26 ( out of 168) highly specialised technologies( drugs , appliances and surgery)  including eculizumab for PNH ( but not aHUS) in the NHS’ Specialised Service portfolio.
It adopted a range of 10 or more quantitative performance indicators about severity of illness , impact on health , costs , value for money,  innovation cost ( R&D) , and impact on specialised service delivery and budget to provide a holistic quantitative view of  the relative merits of these technologies.
It was realistic about the data barriers that this attempt at comparison faced for all the technologies included in the study. So for each indicator it assessed the data reliability  ( scale of 0 to 3) that could be put on it for each of the technologies.
It was a remarkable bit of research on rare disease technology evaluation that , as far as is known, has never got into the public domain.
The analysis presented to us was very difficult to understand, really difficult.
But what could be observed was that except  for the annual price per patient ( based on adult doses at licensed dose levels) of eculizumab , aHUS would compare favourably with other illnesses such as PNH and Cystic Fibrosis which had already been approved. Given its clinical effectiveness and exceptional Quality Adjusted Life Years scores, the comparative value of eculizumab for aHUS was looking good to us, And we said as much.
We might not be able to run faster than the “ opportunity cost” lion chasing us but we could run faster than others it was chasing.
Rare Disease Day had come and Rare Disease Day had gone. aHUSUK had not sensed that there was much interest or concern by the wider Rare Disease community in the plight of aHUS patients. It would be normal for others to think what was happening would not happen to them ; and in any case they would do a better job of it anyway. Fair enough.
For all the “better togetherness” of rare diseases in the drive to get equity for patients with such diseases, there was an emerging awareness  that as far as health care access to cash strapped providers is concerned there was a sense of competition too.
All I knew was that we had not said it was unfair that PNH patients got Eculizumab and we didn’t . I had also signed a petition supporting Cystic Fibrosis patients getting access to a drug they needed.
It was also making me think though, could “single mindedness” result from being a patient advocate, or was it a quality for doing it in the first place?
Back to NICE , we knew that instead of a meeting on the 24th April, aHUSUK had to meet an extended response period by 6 May to give our views on the Report. Soon after  that we were informed that NICE were planning for a Final Evaluation Decision in September and for that the next meeting of the Committee  would be  on 23rd July in Manchester.
I had booked my summer holiday , my  flight out would be on 23rd July!
Here comes the sun, maybe?
Still time to participate in the alliance’s 2019 Rare Disease Day Video click here for more information

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