Petition: 'NICE: Be NICE to Cancer Patients'

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The CML Support Group- Press Release: February 2010


NICE:ACD -

Appraisal Consultation Document for Multiple Technology Appraisal of dasatinib and nilotinib for ‘imatinib intolerant’ Chronic Myeloid Leukaemia




CML, the second most common form of Leukaemia, was until recently an inexorably fatal disease with a life expectancy of 3-5 years from diagnosis in almost all patients for whom stem cell transplantation was not available. However, in 2000 the development of imatinib (Glivec), a revolutionary molecular targeted therapy, significantly changed the clinical management and outcome for most patients. Imatinib is an oral therapy and is so effective that chronic phase CML is now considered to be a chronic condition that is relatively easy to manage. Most (but not all) patients now enjoy a good quality of life and can expect to live out their normal life-span.


NICE published their ACD in November 2009 which included both imatinib resistant and intolerant CML. However, the appraisal committee stated at the ACD meeting on January 15th 2010 that they recognised the validity of some of the concerns and criticisms raised by consultees and expert clinicians in response to the ACD. The committee then took the decision to separate the two sub-sets of patients and continue with a new appraisal of dasatinib and nilotinib which would focus solely on imatinib 'intolerance'.
*Imatinib 'resistance' will now be included as part of the re-appraisal of imatinib later this year.


The Chronic Myeloid Leukaemia (CML) Support Group have grave concerns about the recommendation contained in the new ACD for imatinib intolerant CML. In the consultation document, the appraisal committee does not consider comparative data from international clinical trials of dasatinib and nilotinib to be sufficiently robust to allow for a recommendation of NHS cost effectiveness of either therapy in the treatment imatinib intolerant CML.

We question the validity of this decision because we understand that imatinib (Glivec) intolerant and resistant patients are difficult to separate with clinical precision. We also point out that in actual clinical practice, both categories of patients who have failed imatinib already enjoy increased progression free survival and quality of life benefits from therapy with nilotinib and/or dasatinib within the UK and throughout Europe.

In cases where 'imatinib intolerance' includes haematological side effects, dangerously low blood cell counts means that imatinib cannot be safely prescribed without careful management and close monitoring. In cases where therapy needs to be constantly interrupted, patients are exposed to the risk of developing drug resistant mutations and disease progression. In such cases, without timely access to nilotinib (Tasigna) and/or dasatinib (Sprycel) and where stem cell transplantation is inappropriate, patients who are intolerant to imatinib (Glivec) will undoubtedly suffer progression of their disease and their lives will be put at risk. Separating the two sub-sets of patients in order to assess the imatinib intolerant group in isolation is, in our view, not only cynical but absurd and unethical.

Should the appraisal committee's recommendations be upheld, patients with chronic or accelerated phase CML who prove to be intolerant to imatinib, will be offered other treatment options from a list of far less effective therapies used in the pre-imatinib era. Several of these options can in themselves be life threatening, most have well documented seriously debilitating side effects and one is purely palliative. With the exception of stem cell transplantation, which has relatively high morbidity and mortality rates, in almost all cases of CML the suggested alternative options do nothing to halt the progression of the disease and are rarely used in current clinical practice.


Sandy Craine, a CML patient and director of The CML Support Group said:

”Patients are angry, frightened and very frustrated by this inhumane provisional recommendation by the NICE appraisal committee- a recommendation that seems to us to have much more to do with cost cutting than with a rational appraisal of two highly effective and innovative therapies. If the recommendation is upheld, and NHS funding of these therapies is refused in cases of imatinib intolerance, it will undoubtedly cost lives. It is a shocking prospect that UK citizens, who prove to be intolerant to standard dose imatinib (Glivec) will be denied access to such effective and life-saving alternatives. Clinicians will find themselves unable to treat their patients within internationally agreed expert recommendations outlined by the European Leukaemia Network, with the knowledge that clinicians in Germany, France, the Netherlands and other EU countries, are able to offer their patients the very best therapeutic options available.

NICE have clearly acknowledged that both nilotinib (Tasigna) and dasatinib (Sprycel) are clinically effective and well tolerated oral therapies. It is therefore difficult to understand why they have now singled out a small group of patients and given a provisional recommendation that chronic and accelerated phase (but not blast phase) patients should be denied access to effective and life-saving therapy. As a consequence, this group of patients, who currently represent around 30-40 British citizens of all ages, will suffer an appalling injustice that puts them at great risk of dying from acute (blast) phase CML.”

Nilotinib and dasatinib have both shown some efficacy in the accelerated stage of CML, with dasatinib also showing limited usefulness in the acute (blastic) phase. However, we do not believe that expert clinicians would ever suggest that their patients should wait for ‘resistance' to be established in order that they become eligible for NHS treatment with these alternative therapies. Certainly an informed patient with a good knowledge of how CML ‘works’ would never accept such a high risk strategy.


The CML Support Group believe that NICE has failed to recognise how successful dasatinib and nilotinib have been in the treatment of imatinib intolerant patients and consider that the NICE HTA model does not allow for either the patient experience of their therapy, nor the knowledge and experiences that expert clinicians have gathered from actual clinical practice. "Our view is that the economic model that NICE uses to inform all HTA’s is inappropriate in the context of rare cancers and is of little use in the evaluation of the increasing number of innovative molecular targeted therapies coming from the new frontiers of molecular biology research. We suggest that the use of a Bayesian statistical model would allow for evidence from actual clinical practice to be included in appraisals and would be more relevant in the context of rare cancers and rare diseases like CML."




It should be noted that in Wales, patients in both chronic and accelerated phase CML currently have access to dasatinib as second line therapy. In Scotland both nilotinib (Tasigna) and dasatinib (Sprycel) are available as second line therapy for CML in chronic phase only.

It remains to be seen whether a negative FAD (Final Appraisal Determination) from NICE will encourage the Scottish Medicines Consortium (SMC) to review their previous appraisals of both drugs.

It is very likely that the All Wales Medicines Commissioning Group (AWMCG) will accept the NICE FAD and refuse NHS funding of dasatinib (Sprycel) and nilotinib (Tasigna) in new cases of imatinib (Glivec) intolerant CML.



Nice have stated that they have insufficient data available to assess the even smaller group of imatinib intolerant patients in blast crisis and do not feel they can give any recommendation for this group. It is of some comfort that blast phase patients will continue to have access dasatinib, although by not providing a recommendation for blast crisis patients, whilst at the same time giving a negative recommendation for chronic and accelerated phases even though NICE has stated there is a paucity of data sufficient data here too does reveals some inconsistency.



The prospect of real equity of NHS care and treatment for cancer patients throughout all regions of the UK remains out of reach for the foreseeable future.






Notes:

Documents concerning this ACD can be found on the NICE website at:
http://guidance.nice.org.uk/TA/Wave17/18



Comments from individual members of the public should be made by March 1st at:
http://www.nice.org.uk/guidance/index.jsp?action=article&o=47297



NICE ACD meeting: March 9th 2010

Publication of FAD (Final Appraisal Determination) follows within 30 days