I attended, with a patient being treated with ponatinib, the Committee meeting in Manchester for the NICE appraisal of ponatinib on the 18th of January.
Ponatinib is currently only accessible to CML patients, in any phase, with the T315i mutation and only after an application has been made to the Cancer Drugs Fund for reimbursement of the cost of treatment.
NICE are, however, evaluating (appraising) the cost and clinical effectiveness of the full licensed indication which is:
chronic-phase, accelerated-phase or blast-phase chronic myeloid leukaemia (CML) who are resistant to dasatinib or nilotinib, who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate, or who have the T315I mutation
Prior to the meeting both CML Support and the patient had submitted responses to the evaluation by completing the standard NICE template for 'patient experts'.
Two clinicians also attended the meeting, as 'clinical experts'.
Although its notoriously difficult to predict accurately the outcome of the process, my personal impression of the meeting was that it went well.
That's to say there was nothing in the paperwork we had received previously or that was raised during the course of the meeting that seemed to indicate there were some very fundamental problems with the clinical performance of ponatinib or, as importantly, with the way in which cost effectiveness had been established.
These are encouraging signs although it should also be said that the degree of 'uncertainty' present due to the status accorded to the clinical trial and the data it generated together with the range of values (for ponatinib relative to currently available NHS treatments) derived for cost effectiveness makes for this to be a very cautious assessment.
The patient we nominated gave a brilliant description of the benefit she had obtained from ponatinib treatment in the very short time allocated to her and we are very grateful to her for the contribution she has made. Professor Jane Apperley was also outstanding giving an authoritative, decisive and fluent response to both the NICE presentations and subsequent discussion.
The next step will be our being sent either an Appraisal Consultation Document (ACD) on the 6th February or a Final Appraisal Determination (FAD) on the 20th February.
The ACD is an interim document that we, and others, will be permitted to comment on whereas a FAD is a final recommendation (unless there is an appeal and in particular an appeal that is successful).
My guess is that an ACD will be issued but I would be very happy to be proved wrong if a FAD with a positive recommendation is issued instead. Assuming there is no appeal, ponatinib would be well on the way to becoming available for all patients who meet the criteria for its use.
I'll update next month.
