NICE recommends new treatment for CML


London, England (January 13, 2012) – NICE, the healthcare guidance body, has today published final guidance recommending a new treatment for chronic myeloid leukaemia (CML). Final guidance recommends nilotinib (Tasigna, Novartis) for the treatment of the chronic and accelerated phases of CML that is resistant or intolerant to standard-dose imatinib. Dasatinib (Sprycel, Bristol-Myers Squibb) is not recommended for CML that is resistant or intolerant to standard-dose imatinib, and high-dose imatinib (Glivec, Novartis) is not recommended for CML that is resistant to standard-dose imatinib.


Commenting on the guidance, Professor Carole Longson, Health Technology Evaluation Centre Director at NICE said: "We are very pleased to be able to recommend nilotinib as a treatment option for the chronic and accelerated phases in people who are resistant or intolerant to standard dose imatinib. CML is a chronic condition, meaning the drugs will be used for a long period of time and at over £30,000 per patient per year, nilotinib is expensive. However the manufacturer has agreed to provide it to the NHS at a discounted price. This reduction in cost enabled the independent Committee to approve nilotinib for use."


The manufacturer has requested that the size of the discount remains confidential.


The Institute received two appeals on its final draft guidance for the CML appraisal. Both appeals were dismissed on all counts. When an appeal has been held, regardless of the outcome, NICE always publishes guidance as soon as possible. This means that publication doesn’t necessarily take place on the regular NICE publication day, which is the fourth Wednesday of the month.



About the CML guidance


1. The guidance will be available on the NICE website from 13 January 2012 at


2. Chronic myeloid leukaemia is a very rare condition that affects around 560 people in the UK each year. Many are treated with a drug called imatinib. If this treatment does not work, the current options are interferon-alfa, hydroxycarbamide or a bone marrow transplant.


3. The Committee agreed that it was clear that dasatinib, nilotinib and high-dose imatinib provided clinical benefit for people with imatinib-resistant CML. However, the lack of the evidence base meant that the magnitude of the benefit was uncertain.


Most of the clinical-effectiveness evidence came from trials that included a mixed population of people with imatinib-resistant CML and people with imatinib intolerance, and in the trials that reported response rates separately, CML in people with imatinib intolerance generally had a higher response rate to dasatinib and nilotinib than people with imatinib-resistant CML.


4. When considering the manufacturer’s updated analysis submitted during consultation on the first draft, the committee felt that the ICER of £22,800 per QALY gained (for nilotinib compared with hydroxycarbamide) was too optimistic. However, they accepted that with the patient access scheme in place, the use of nilotinib for the treatment of imatinib-resistant CML could be regarded as a cost-effective use of NHS resources.


The committee concluded that the ICER for dasatinib compared with hydroxycarbamide for the imatinib resistant population would be higher than £43,800 per QALY gained and could be considerably more.


The Committee noted that high-dose imatinib was dominated in all cost-effectiveness analyses; that is, it was more expensive and less effective than the other treatments.


When discussing the cost effectiveness of the technologies for the treatment of chronic-phase CML in people who have imatinib intolerance, the committee acknowledged the difficulties of undertaking an assessment without reasonable comparative evidence, relying on surrogate outcomes and uncertain treatment durations. However, it was aware that the effectiveness of dasatinib and nilotinib was likely to be greater in people with imatinib intolerance than in people with imatinib-resistant CML. Noting the uncertainties in these analyses, particularly about treatment duration, the Committee concluded that dasatinib and nilotinib were likely to be at least as cost effective in people with imatinib intolerance as in people with imatinib-resistant CML and, as such, the cost effectiveness of dasatinib and nilotinib for people with imatinib intolerance could be inferred from the cost effectiveness in people with imatinib-resistant CML.


5. In October 2003 NICE published guidance (technology appraisal guidance 70  recommending standard-dose imatinib (400mg) for the first-line treatment of chronic myeloid leukaemia (CML). High-dose imatinib was only recommended in the context of clinical trials.


In July 2010, it was decided that the Final Appraisal Determination (FAD) or final draft recommendations for dasatinib and nilotinib in people with imatinib-intolerant chronic myeloid leukaemia could be influenced by the outcome of the appraisal for people with imatinib-resistant chronic myeloid leukaemia.


The release of the document was therefore delayed until the release of the FAD for dasatinib, nilotinib and high-dose imatinib in people with imatinib-resistant chronic myeloid leukaemia – this topic was discussed at the Appraisal Committee C meeting on Thursday 9 June 2011. Following the discussion, NICE released one FAD combining the recommendations for both people with imatinib-resistant chronic myeloid leukaemia and with imatinib-intolerant chronic myeloid leukaemia.



About NICE


6. The National Institute for Health and Clinical Excellence (NICE) is the independent organisation responsible for providing national guidance and standards on the promotion of good health and the prevention and treatment of ill health


7. NICE produces guidance in three areas of health:


  • public health – guidance on the promotion of good health and the prevention of ill health for those working in the NHS, local authorities and the wider public and voluntary sector
  • health technologies – guidance on the use of new and existing medicines, treatments, medical technologies (including devices and diagnostics) and procedures within the NHS
  • clinical practice – guidance on the appropriate treatment and care of people with specific diseases and conditions within the NHS.


8. NICE produces standards for patient care:


  • quality standards – these reflect the very best in high quality patient care, to help healthcare practitioners and commissioners of care deliver excellent services
  • Quality and Outcomes Framework – NICE develops the clinical and health improvement indicators in the QOF, the Department of Health scheme which rewards GPs for how well they care for patients


9. NICE provides advice and support on putting NICE guidance and standards into practice through its implementation programme, and it collates and accredits high quality health guidance, research and information to help health professionals deliver the best patient care through NHS Evidence.


This page was last updated: 12 January 2012



National Institute for Health and Clinical Excellence (NICE), 13.01.2012 (tB).


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