As part of the NICE CDF (Cancer Drugs Fund) Rapid Reconsideration process for bosutinib I attended, with a patient who very kindly volunteered to participate, the committee meeting in Manchester last week.
We 'patient experts', as NICE likes to describe us, were joined by two 'clinical experts' (Dr. Jenny Byrne from Nottingham and Dr. Adam Mead from Oxford) with representatives from bosutinib's manufacturer, Pfizer, also attending.
This was the first meeting for this specially assembled committee, whose lifetime is limited to considering drugs in the current CDF, and was unlike any other NICE committee meeting I have attended.
Over fifty percent of the two and a half hour meeting was devoted to a discussion of the evidence supporting the clinical effectiveness of bosutinib and the reliability of that evidence for making a judgement for how effective bosutinib is for the treatment of CML.
What was different was that the clinical experts were given space and time to lead the discussion with us involved in putting forward the 'patient experience' perspective in support. Evidence from the key clinical trial was of course discussed but it was not allowed to dominate to the exclusion of all else.
We should know the committee's recommendation in a couple of months time.
I am optimistic that their recommendation will not be negative but less certain that it will be a fully positive recommendation that would allow bosutinib to join imatinib and nilotinib in being routinely available in England.
I would not be surprised if the recommendation was a provisional positive recommendation which would mean patients could only obtain access to bosutinib via the new version of the CDF which will be launched on July 1st this year. Access on this basis would only continue whilst further data was gathered to establish its clinical effectiveness.
For the more technically minded with an interest in the NICE process:
* As usual a key clinical issue was survival duration with the comparator being hydroxycarbamide (HU) against the intervention bosutinib. In particular discussion revolved around estimated survival times after HU use following none, a single or multi TKI treatments. In essence what were the most plausible values that could be assigned.
* The other key clinical issue was the (post treatment) benefit obtained following TKI use (in this case with bosutinib) even when treatment failure resulted in discontinuation. Discussion focused on whether all, some or no patient would secure a benefit that endured beyond the discontinuation date or, put another way, would a patient's disease load remain unchanged following unsuccessful treatment remaining as it was before treatment started. If not what survival benefit (expressed in months) would be obtained.
* Pfizer, the manufacturer, had returned (following TA 299) with an acceptance of the ERG's (cumulative survival) model over their own surrogate outcomes (using MCyR as a proxy for OS) model. In addition they also conceded on other issues to align more closely with the ERG.
* Taken together and with a further PAS offer. discussion revolved around the revised ICER values and their improvement against threshold values including those for EoL (in AP & BP only)