NICE have today published their Appraisal Consultation Document (ACD) on the evaluation of ponatinib. ACD publication is a significant milestone event in the evaluation process. The provisional recommendation contains both good and bad news. The good news is for that often neglected and very numerically small constituency, patients in accelerated or blast phases, where the recommendation is a positive (a ‘yes’).
The bad news is that the recommendation is negative (a ‘no’) for patients in chronic phase. In technical terms, and assuming this situation remains unchanged at the end of the process (ie the publication of statutorily supported Guidance), this means that ponatinib is recommended for routine use in the NHS in England for adult CML patients in accelerated or blast phases who are resistant to, or intolerant of, dasatinib or nilotinib and for whom subsequent treatment with imatinib is not appropriate. In addition, ponatinib is recommended for any patient with the T315i mutation in any of the three phases of CML. Ponatinib is currently accessible on a case by case basis via applications to the Cancer Drugs Fund (CDF) but only to those patients, in any CML phase, with the T315i mutation so the Committee’s recommendation amounts to an endorsement of that situation.
We are obviously very disappointed with this outcome. The more so given the vast majority (over 90%) of patients are diagnosed in chronic phase. We will of course take advantage of the opportunity to comment on the ACD, as I am sure will the clinicians who have participated in the process as ‘clinical experts’, and hope that NICE will revise their positive recommendation to include chronic phase patients. However Its important to stress that past experience indicates this is likely to prove an uphill struggle and that the discussion at the second NICE Committee meeting on March 16th will reflect that.
Below is the link for those who would like to read the ACD (see the last document dated 13th February 2017 in the Appraisal Consultation column) . In addition to the detailed description of the Committee’s deliberations, there is also a shorter, easy read summary document available if you scroll down the attachment.
NB: The appraisal also covered the use of ponatinib to treat acute lymphoblastic leukaemia (ALL) but we will not comment on this topic and its evaluation.