London, 23 March 2010 – The MDS UK Patient Support Group has announced that it has lodged an appeal against the negative recommendation issued earlier this month by the National Institute for Health and Clinical Excellence (NICE) for the use of Vidaza (azacitidine) in patients with higher-risk myelodysplastic syndrome (MDS). The Leukaemia Society and the Rarer Cancers Forum are also appealing the NICE guidance.

Approximately four in 100,000 people in the UK have MDS, a group of debilitating bone marrow diseases that lead to complications such as recurrent or life-threatening infections or bleeding. Most MDS patients have to rely on frequent blood transfusions to manage anaemia and extreme fatigue. While the average survival of patients with MDS is about twenty months, nearly a third (30%) progress to acute myeloid leukaemia (AML), a very aggressive and resistant form of leukaemia with an average survival period of a few months only.

A study published in The Lancet Oncology demonstrated that the median overall survival for higher-risk MDS patients receiving azacitidine was 24.5 months compared with 15 months for patients receiving conventional care such as supportive care or chemotherapy – a difference of 9.5 months. The study also showed that at two years, the survival rate for patients receiving azacitidine was just over 50%, nearly double that of patients receiving conventional care (26%).

David Hall, Chairman of the MDS UK Patient Support Group and MDS patient said, “The negative recommendation is a return to hopelessness for UK MDS patients who have glimpsed hope in the EU-approval of the first therapy for malignant bone marrow disease that is a significant advance in medical treatment for this condition. The recommendations also create an ethical dilemma for doctors treating this condition. How can it be in the patient’s best interest to deny them an average of an extra nine months of life? Some patients have their lives extended by much longer than the average nine months.

“We believe strongly that the underlying appraisal methodology used by NICE is unfair and lacks transparency. We also believe that the recommendations are perverse as the appraisal committee completely ignored evidence presented to it on quality of life and does not understand the nature of this very rare disease.”

MDS UK considers that a negative recommendation for azacitidine is unreasonable given the 9.5 month average extension to life – more than three times the figure required by NICE’s end-of-life criteria and longer than the extensions to life accepted by NICE in the past – and the very small patient population. There are approximately 700 people with higher risk MDS in the UK and any financial burden on the NHS is unlikely to be significant.

MDS UK believes that NICE ignored available quality of life evidence, in particular by not considering information gathered from more than 1,000 patients worldwide by the international patient body, the MDS Foundation. This patient information provides strong evidence that patients treated with azacitidine have a significantly improved quality of life.

Finally, MDS UK believes NICE has made recommendations that are incompatible with the Human Rights Act 1998- particularly in terms of preventing foreseeable loss of life and discrimination against the elderly and newly-diagnosed patients – as well as the General Medical Council’s code of ethics. Not being able to offer patients azacitidine and the chance of an extra extension to life flies in the face of doctors’ ethical obligations and may lead to negligence claims against the NHS.

The MDS UK Patient Support Group endorses the report released last week (Monday 15 March) by the Rarer Cancers Forum (RCF) which stated that NICE has rejected cancer treatments which could have benefited up to 16,000 patients in spite of its new guidelines for the appraisal of life-extending, end-of-life treatments. The RCF said that the way NICE is interpreting these guidelines is confusing and runs counter to the spirit of the 2008 report from Professor Mike Richards, the Government’s national clinical director for cancer: ‘Improving access to medicines for NHS patients’.

  1. The NICE press release announcing the organisation’s Final Appraisal Determination on azacitidine can be found at the NICE website: here
  2. Information on Vidaza (azacitidine), which is manufactured by Celgene Ltd, can be found at EMC website: here
  3. Published survival data includes: Fenaux P; Mufti GJ; Hellstrom-Lindberg E et al. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009 Mar;10(3):223-32. Epub 2009 Feb 18.: here
  4. The Rarer Cancers Forum report can be found here http://www.rarercancers.org.uk/news/current/exceptionalprog.final.pdf

Media Coverage

Outlet URL / Headline Date
BBC News Online Cancer groups challenge NICE ruling 23 March
Community Newswire Read the article… 23 March
Daily Mail Online Read the article… 23 March
Mail on Sunday Read the article… 28 March
The Pharma Letter Read the article… 23 March
Scrip Read the article… 23 March
The Pink Sheet Read the article… 23 March
Pharmacy Europe Read the article… 23 March
Pharmajobs Read the article…
Nursing in Practice Read the article… 23 March
Nursing Times Read the article… 23 March
Birmingham Wired Read the article… 23 March
Pushpi News Read the article… 23 March
Mizoram Express Read the article… 23 March
University of Information Read the article… 23 March
Paper News Read the article… 23 March
World News Read the article… 23 March
Sain Sunsite Read the article… 23 March
HC2D Read the article… 23 March
Free Republic Read the article… 23 March
Life Science Dynamics Read the article… 23 March
Michelmores Read the article… 23 March
Cancer Support Groups Read the article… 25 March

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