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NICE CDF Rapid Reconsideration Committee meeting for bosutinib

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)       

  

Excellent post and thank you for that.

Whilst I understand the NICE process and the necessity of ensuring that drugs are objectively reviewed, it does seriously concern me that NICE impedes clinical advancement and just is not appropriate for new generation cancern drugs. 

For many years I was on imatanib in clinical trial and funded by Leukaemia and Cancer research charities.   It took NICE way too long to get it on the "approved" list so that as we stand today it's the effective treatment of choice. 
 

Let's hope there's positive outcome and opportunity is taken to broaden viable treatment options.

You make an interesting point and what's called NICE 's 'new approach', and broader policy, is all about reducing the timelines between clinical trial data becoming available and patients being able to access the drugs they need.

Superficially the proposed reforms to the process have the capability to deliver that although there remain doubts about the capacity to do so (that is, are there the physical resources in place to undertake the evaluation work involved ).

My concern is more with whether there has been a full digestion by evaluation bodies like NICE of the science driven reality which is of patient populations, like that for CML which is already very small given its a very rare cancer, being increasingly fragmented into ever smaller clinically distinct sub groups.

One obvious example of which is that sub group of patients exhibiting the unambiguously identified T315i mutation where ponatinib is the only TKI capable of activity against the mutation.

The anticipated total annual UK T315i patient population is about 18 patients.

In particular, my concern is whether it is realistic to expect that the at scale and depth of clinical evidence required to meet the demands for clinical effectiveness that apply to drugs for patient populations many times larger in size is realistic (ie are there enough patients available to recruited into large scale clinical trials) and feasible (ie economically sane) in such cases.

Currently there is no sign that NICE is willing to concede on this increasingly important issue.

No amount of acceleration of process will change the likely end result for drugs that fall in this category.

The problem is that this is increasingly the direction of travel for research effort.

Its a problem that regulators (ie the bodies that grant licenses to drugs) now appear willing to address but evaluators like NICE (whose concern is with whether a drug is both clinically and cost effective compared to treatments already in routine use in the NHS in England) seem unwilling to tackle head on.

I'm not unsympathetic to their dilemma but the current process produces, at least in terms of the availability of TKIs for CML, a kind of madhouse of a sequencing system in the route to a TKI most likely to be effective where patients are compelled to switch to an inappropriate TKI in order to attempt to access one that is more appropriate or, much worse, end up in a cul de sac where further progress is impossible even though clinical practice indicates this to be nonsensical. 

One final procedural point, we heard from NICE today that the committee's recommendation for bosutinib will be published at the end of this month. 

 

         

Thread continued from: 
NICE Rapid Reconsideration of dasatinib and bosutinib