As many of you know both dasatinib and bosutinib are only available to a CML patient in England if their doctor makes an application to the Cancer Drugs Fund (CDF). However for the application to be successful a patient's clinical profile must match that set out in the CDF.
I described the changes that are to be made to the way the CDF operates in my 17th February Forum post and NICE are now beginning the implementation (or transition) process for what are called 'Group 1 CDF drugs' (drugs that have already been assessed by NICE, are not recommended on cost effectiveness grounds and are currently only available via the CDF).
In the last two weeks we have been sent details by NICE of their review (officially described as a Rapid Reconsideration) of dasatinib and bosutinib. We have now completed the formal paperwork to allow us to enter the process and have also recruited dasatinib and bosutinib patients to join us at CML Support in participating in the review.
In theory NICE have to approve all of us to participate in the process, but we think this a formality and would be astonished if approval was not forthcoming. The same applies to the specialist clinicians who will also be nominated to participate in the reviews.
The timelines are tight with a deadline for evidence submissions required in April and a NICE Committee meeting, which both ourselves and the two patients will attend, scheduled for May. It is expected NICE will publish their recommendations for both drugs during October of this year at the latest.
In my February post I set out the three NICE recommendations that are available under the new arrangements (a 'Yes', provisional Yes' or a 'No' ).
In both reviews we think a 'No' is the least likely outcome. So unlikely that it can be discounted and we are hopeful in both cases the recommendation will be a 'Yes'. Our thinking is based on the willingness of the companies involved (BMS and Pfizer) to enter into productive discussions with the Department of Health and NICE on the price of dasatinib and bosutinib. However we do not dismiss the possibility of a 'provisional Yes' recommendation, with all that involves - see my February post.
Now we get to the slightly complicated bit.
For various reasons, the reviews are of previous NICE appraisals of bosutinib and dasatinib.
For bosutinib the NICE appraisal number is TA 299 (for previously treated CML patients no matter what their Phase) and for dasatinib the appraisal number is TA 251 (for a first treatment of CML, this appraisal also included nilotinib as well as standard dose imatinib, both of which were consequently recommended for routine use via the NHS in England).
However the scope of NICE appraisals and the CDF clinical profiles, as I mentioned in the opening paragraph, are not the same, but they do capture the varied clinical profiles of the relevant patient populations involved (in the NICE scopes) and ensure more patients can potentially secure routine NHS access to these two drugs than would be the case were only the CDF clinical profiles reviewed. This is something we at CML Support have welcomed.
There may be some confusion for those who have read my 3rd March Forum post on NHS England's proposals for a Commissioning Policy Statement for dasatinib as a 1st line treatment for newly diagnosed patients and a 2nd line treatment in cases where there is imatinib resistance.
We were puzzled by why NICE seem to be duplicating the proposal for 1st line use.
However it seems that this is both a general housekeeping exercise, which joins others that NICE conducts regularly, to maintain the integrity of their Guidance collection and is also a particular requirement of the NHS England Policy Statement proposals.
As a postscript, I want to add that we hope this speedy movement on dasatinib and bosutinib will bring forward a date at which ponatinib, which has never been appraised, enters the NICE process.
We would also like a resolution to the four applications to the CDF, made by two specialist CML clinicians in 2014, which broadened the scope of access to CML drugs to cover the following:
(i) Treatment with dasatinib in all three CML phases where certain mutations are identified that are known to indicate resistance to bosutinib or nilotinib
(ii) Treatment with bosutinib in all three CML phases where certain mutations are identified which indicate resistance to nilotinib
(iii) Treatment with ponatinib in all three CML phases if resistance to two other TKIs has been confirmed
(iv) Treatment with bosutinib or dasatinib for patients in Blast Phase CML.
In a decision published in July 2014 NHS England declined to pursue the standard procedural route for applications. Instead it asked for four issues arising from the CDF Panel's consideration of the applications to be investigated as a part of an action plan.
We asked for a progress report on the action plan in September last year and again in February this year. We are still waiting for a response.