NICE have today published their final recommendations for ponatinib for adult patients being treated in the NHS in England.
We are very pleased to report that they have modified their initial recommendation to limit ponatinib treatment to patients in accelerated or blast phases or to patients in any phase with the T315i mutation.
The NICE Committee have now extended their recommendation to include patients in chronic phase.
With the exception of those with the T315i mutation, patients will still have to be either resistant to, or intolerant of, nilotinib or dasatinib to be eligible for treatment. Imatinib as a subsequent treatment will also have to be considered inappropriate although this is in essence a formality since most patients will already have failed imatinib.
We would like to extend our thanks to Sara Mulvanny who, as our nominated patient expert, provided detailed, concise statements for the NICE Committee and who spoke movingly and eloquently at the NICE Committee meeting some months ago.
All current, and future, patients for whom ponatinib is, or would be, considered an appropriate treatment owe her so much for volunteering to come forward and represent their interests.
Technically, and this should not be a worry to patients, ponatinib will not be available for routine use in the NHS for three months from today's date. Until that time a doctor will still have to make an application to the Cancer Drugs Fund to access ponatinib.
In theory, there is also an option available to appeal against the recommendation but the possibility of this occurring is remote with the possibility of the appeal being successful being even more so.
This publication marks the end of a series of NICE evaluations of TKIs for CML that began over a year ago. For patients in chronic phase the outcome is that all 5 TKIs for CML are now available for their doctors to use. The situation for those few patients in the other two disease phases is also very different now.
I'll post a more lengthy piece on this transformed environment sometime over the next couple of days.
For those with an interest here is the link to the NICE publication:
https://www.nice.org.uk/guidance/gid-ta10060/documents/final-appraisal-d...