Some very pertinent housekeeping. Earlier in the year David Ryner from CML Support asked me, Bloodwise, Leukaemia Care and CML-UK (Facebook) to co-sign a letter he penned to Simon Stevens (Chief Executive of NHS England) and Prime Minister, David Cameron. The letters and responses are all posted below.

The letter from Professor Sean Duffy confirms that the new model for the evaluation of drugs, including cancer drugs, following the Accelerated Access Review’ s report will be launched on April 1st 2016. Not good enough. I want to make this clear. We currently don’t have a method of evaluating new drugs and the old method was flawed (see the issues ponatinib had with small population numbers). This gap in service and the delisting of life-saving drugs is going to kill people.

I’m making this extra clear because a politician I’ve spoken to recently questioned me, quite ferociously, on the launch date of the new model. I know that this politician subscribes to my blog updates and I hope that they now have all of the facts they need to do something about the issue and register their protest.

A huge thanks to David Ryner from CML Support for coordinating this activity.

Kris Griffin

LETTER TO SIMON STEVENS

Dear Mr Stevens,

We are writing to you regarding the recent announcements relating to the Cancer Drugs Fund (CDF) and the specific decisions that have been taken on treatments for chronic myeloid leukaemia (CML).

As patient groups representing the concerns of patients with CML, we are particularly alarmed by both the inclusion of CML treatments in the next review of the CDF for delisting at the end of this month, and by the suggestion that there may not be any further meetings of the CDF panel to consider new treatments or indications for the remainder of the 2015/16 financial year.

In particular, I would like to draw your attention to the situation currently facing two medicines which treat patients with more advanced CML, who therefore face severely limited treatment options. Bosutinib is scheduled for review at the next meeting of the CDF panel at the end of this month. Ponatinib, a drug which has never been appraised by NICE due to its small patient population, is only available through the CDF for patients with the T315i mutation, rather than in its full licensed indication. Ponatinib was due to be assessed by the CDF panel for its full licence in June, before the cancellation of the last scheduled meeting.

This situation for those patients needing access to ponatinib is particularly acute; with NHS England’s Commissioning Intentions for 2015/16 committing to producing algorithms for all chemotherapy within the year, ponatinib now faces the prospect of being effectively excluded from the CML algorithm entirely, with the exception of the T35i mutation.

When the threat to bosutinib is factored in and with the exception of the minuscule number exhibiting the T315i mutation, patients in England now face a lack of access to two of the five drugs that are currently available to them. The clinical effectiveness of these drugs in being able to secure optimal responses at speed and scale relative to the current entry level CML inhibitor, imatinib, now over a decade old, is well established.

The Government’s Accelerated Access Review (AAR) demonstrates a welcome recognition that current evaluation processes require revision to ensure they are fit for purpose in assessing the new generation of innovative products, including targeted therapies for CML. We recognise, too, that the CDF needs to adapt its processes to remain in step with the wider Government agenda.

It is therefore bewildering, contradictory and illogical for NHS England’s real time activity to be moving in the opposite direction of travel in reversing, rather than accelerating, access to targeted therapies for CML. This is made even more remarkable given the fact that, relative to other CDF list treatments, the performance of this class of drugs has been considered outstanding when judged against standard measures of survival. As a result, the overwhelming majority of patients are now able to secure decades of benefit from these home-based oral therapies, with their lives returning to near normal (and patients enjoying near-normal life expectancy) following treatment.

Such marked improvements in CML patient outcomes have been achieved by the steady increase in targeted therapies. We believe that to withdraw the opportunity from patients who would benefit from targeted CML therapies such as ponatinib and bosutinib is both discriminatory and perverse and we would strongly urge you to reconsider this decision by NHS England.

Yours sincerely,

xxxx

cc. Rt Hon Jeremy Hunt MP

RESPONSES

from the Department of Health (Malcolm Jones)

from NHS England (Professor Sean Duffy)


LETTER TO RT HON DAVID CAMERON

Dear Prime Minister,

We are writing to you following the intervention you recently made to NHS England regarding its consideration of the funding of medicines for a number of rarer diseases, to make you aware of the situation patients with chronic myeloid leukaemia (CML), a rare form of blood cancer, currently face.

As patient groups representing patients with CML, we were concerned with recent announcements relating to the Cancer Drugs Fund (CDF) and the specific decisions that have been taken regarding treatments for CML. We have great concerns about the fact that the CDF panel will not now consider any new treatments or indications for the remainder of the 2015/16 financial year, meaning new and innovative treatments for CML will remain unavailable to patients, and that CML treatments currently available on the Fund are at risk of being delisted.

CML is treated with targeted therapies which have ensured marked improvements in patient outcomes but mean patient sub populations are small. Patients need to have a wide range of treatment options available to them because of the problem of resistance to medicines, as well as contraindications and co-morbidities which mean some patients are unable to tolerate certain drugs currently within the treatment pathway.

Patients with more advanced CML face severely limited treatment options, with two of the five CML drugs either at-risk or unavailable to all patients who would benefit. Bosutinib, a second-line treatment for CML, is at risk of being delisted from the CDF following its inclusion in the review of current treatments conducted by the CDF panel on the 29th and 30th July. Ponatinib, a drug which has never been appraised by NICE due to its small patient population, is currently only available through the CDF for patients with the T315i mutation, rather than in its full licensed indication. Ponatinib was due to be assessed by the CDF panel for its full licence in June, before the cancellation of the last scheduled meeting, and now has no opportunity to be appraised for clinical and cost effectiveness, meaning the wider CML patient population are unable to access the drug other than through Individual Funding Requests (IFRs).

The clinical effectiveness of both drugs in being able to secure optimal responses at speed and scale relative to the current entry level CML inhibitor, imatinib, now over a decade old, is well established. The following comment from a patient on ponatinib, which was used in the CML Support Group submission to the SMC in Scotland – who approved the drug for its full licence – confirms its clinical effectiveness; “Ponatinib for me represents a quantum leap forward in the treatment of my CML and the impact of this condition on my family and work life. For me, even though I am likely to have to take this for life, ponatinib represents the optimum treatment that I could have expected and hoped for beyond the major trauma and loss of employment that the only other “ total “ cure , a bone marrow transplant, represents.”

We were reassured to read your comments in a letter to the Specialised Healthcare Alliance dated 28th April 2015, in which you stated “I am absolutely committed to ensuring that patients with rare diseases have access to the latest and most effective treatments that represent value to the NHS and deliver benefits to patients.” Any assistance you could offer in ensuring CML patients have access to the full range of effective treatments would be greatly appreciated. In addition, we would be grateful of any clarity you are able to secure on our behalf from NHS England regarding the new system of appraisal – particularly in terms of when the CDF will consider new medicine appraisals, and how medicines for rarer cancers and those with small patient populations will fit into the new system of evaluation – which will replace the current CDF when it ends in March 2016.

Yours sincerely,

RESPONSE

from 10 Downing Street (Ed Whiting)