Price discount means NICE can recommend a new drug for chronic myeloid leukaemia
In new draft guidance NICE has recommended nilotinib, also known as Tasigna and made by Novartis, for the treatment of the chronic and accelerated phases of CML (chronic myeloid leukaemia)that is resistant or intolerant to standard-dose imatinib. Dasatinib (Sprycel), made by Bristol-Myers Squibb is not recommended for CML that is resistant or intolerant to standard-dose imatinib, and high-dose imatinib (Glivec), made by Novartis, is not recommended for CML that is resistant to standard-dose imatinib .
In response to the draft guidance Professor Carole Longson, Health Technology Evaluation Centre Director at NICE said: "We are very pleased to be able to recommend nilotinib as a treatment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 of this condition. The committee heard from clinical specialists that in practice dasatinib and nilotinib are equally as effective in treating both imatinib intolerant and imatinib-resistant disease and would be preferred over high-dose imatinib for imatinib-resistant CML. Both treatments are expensive and cost over £30,000 per patient per year - CML is a chronic condition, meaning the drugs will be used for a long period of time. However, during consultation on draft recommendations, the manufacturer of nilotinib agreed to provide the drug to the NHS at a discounted price. This reduction in cost enabled the independent Committee to approve nilotinib for use on the NHS."
The manufacturer has requested that the size of the discount remains confidential.
Both dasatinib, and nilotinib cost over £30,000 per patient per year. Novartis has recently increased the price of imatinib which means the cost per patient is now over £40,000 per year for the high dose of 800mg.
The draft guidance is now with consultees, who have the opportunity to appeal against it. Until NICE issues final guidance, NHS bodies should make decisions locally on the funding of specific treatments. This draft guidance does not mean that people currently taking dasatinib or high-dose imatinib will stop receiving them. They have the option to continue treatment until they and their clinicians consider it appropriate to stop.
Notes to Editors
About the guidance
1. The draft guidance will be available on the NICE website from 18 August 2011 at http://guidance.nice.org.uk/TA/WaveR/105.
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, but 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 manufacturers 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 also felt that they were unable to rely on the updated analysis provided during consultation on the first draft.
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 http://guidance.nice.org.uk/TA70) 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 is releasing one FAD combining the recommendations for both people with imatinib-resistant chronic myeloid leukaemia and with imatinib-intolerant chronic myeloid leukaemia.
1. The National Institute for Health and Care 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
2. 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.
3. 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
4. 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.
 Dasatinib was considered for treating the chronic, accelerated and blast phases of CML. Imatinib was considered for accelerated and blast phases.
This page was last updated: 17 August 2011