One step at a time

For the  Reluctant Advocate story so far click here
NICE was not ready to begin its work on eculizumab when it took over responsibility for the job as part of the NHS reorganisation. It had done no preliminary preparation because the organisation itself was going through change and the outgoing Chair of NICE , who had known about taking on this responsibility for at least 7 months, decided to leave the management of its implementation to his replacement. The replacement would take over from 1 April 2013. No joking.
However there were a number of people who had been given the job of communicating the change decision . A meeting had been called with potential stakeholders to explain the implications. aHUSUK had not been invited to attend. This did not auger well as a start.
However having complained about NICE’s snub to aHUS patients we were invited to meet them in their London office.
Understandably  we told them that we did not believe we should be going through this again having gone through it with AGNSS , we were not happy to do so. We said that we did not think they would come to a different conclusion. We said to remove any doubt it needed to build equity into its process and properly address the affordability question. We insisted that getting the NHS to let it be known how many aHUS patients there were and who needed treatment and for how long. We could not believe there was as many patients  as estimated which had raised doubts about affordability.
By then we had found out the work on treatment adjustment taking place in Milan clinic , having heard about it from the alliance affiliate from Italy but this was not what we meant . Just the mix of patients on different doses levels for weight would have a bearing  on the actual average cost per patient. Similarly we did not believe the projected number of patients within five years needing eculizumab for life was right. Neither was the estimate of existing numbers of patients . If that basic budget forecast was flawed how could affordability be assessed!
We were told NICE would try to devise a methodology for comparing resources on an opportunity cost basis as part of its decision making. However there was a feeling that this would not be robust. We did not believe that the relative societal costs would be adequately reflected. aHUSUK had made contact with a Professor Jennifer Roberts of the London School of Hygiene and Tropical Diseases. Prof Roberts along with Professor Jennifer Busby of the USA were eminent authorities on the true cost to society of e.coli outbreaks , of which  typical HUS and its implications had been researched.The costs off the alternative to eculizumab they found were higher that those used in the AGNSS process,  including the impact on society. The morbidity and outcome for aHUS patient not transplantable  would be higher still.  We thought that NICE should look at that too.
Finally we asserted that this whole  process would be improved if the NICE committee had a qualified accountant on board to give a professional opinion on the profitability of the price of the drug because that was a key determinant in the decision. Health economics was not enough.
So before we got into the process we had made clear that unless changes were made a similar non conclusive outcome would be likely due to incomplete evidence.
Another example of aHUSUK’s advocacy going beyond just giving  the patient voice about the disease. However for the process we would initially be giving evidence about the illness again. We would have to bide our time on the fiinance and economics.
We will get there just one step at a time.

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