David Hall, Chairman of the MDS UK Patient Support Group and MDS patient said, “We welcome this decision by NICE to re-appraise azacitidine and believe it has been brought about by the weight of patient, expert and clinical opinion behind the arguments put to the Appeal Panel. We only wish that the Appraisal Committee had considered these points in its initial appraisal and made this life-extending therapy available to patients from early this year. Instead patients are still waiting to see if they will be thrown a lifeline.”

In March 2010, NICE recommended that azacitidine should not be provided through the NHS on cost-effectiveness grounds while recognising that it was clinically effective and that it should be seen as a life-extending end-of-life treatment. In June this decision was appealed by eight organisations, including three patient groups and four healthcare professional bodies.
Approximately four in 100,000 people in the UK have MDS which can lead to complications such as recurrent or life-threatening infections or bleeding and which can progress to an aggressive form of leukaemia with an average survival period of a few months only. Azacitidine is the only licensed medicine proven to prolong survival for people with higher risk MDS and could be suitable for 700 patients per year in England and Wales. It has been shown to improve patients’ quality of life substantially, reduce dependence on blood transfusions and is less toxic than chemotherapy.
The MDS UK Patient Support Group awaits more details about the £200 million Cancer Drug Fund promised by the Government with interest. However it will come too late for the many MDS patients who have anxiously been awaiting the outcome of this very long NICE appraisal.

David Hall said, “We are pleased to see this government commitment to making all clinically proven cancer treatments available to patients through the NHS. We hope that the Cancer Drug Fund will be set up in such a way as to ensure that funding follows clinicians’ decisions as, in our experience, clinicians are best placed to advise on the most appropriate treatment for MDS patients.”
The NICE announcement can be found at the NICE website.
Organisations which appealed the NICE decision: MDS UK Patient Support Group, the Rarer Cancer Forum, the Leukaemia Society, the National Cancer Research Institute Haematological Oncology Clinical Studies Group, the Royal College of Physicians’ Medical Oncology Joint Special Committee, the Royal College of Pathologists, the British Society for Haematology and Celgene Ltd.

The doctors have called on the Government’s drugs rationing body to give terminally ill cancer patients the drug azacitidine.

The medication treats rare cancers of the blood and bone marrow.

But the drug, also known as Vidaza, has been turned down by the National Institute for Health and Clinical Excellence (Nice).

The organisation says at a cost of around £45,000 a year the drug is not cost effective.
On average it offers terminally ill patients around an extra nine months of life.

Read the full article at Telegraph.co.uk

MDS UK Patient Support Group is one of the groups appealing the NICE decision on 1 June 2010.

Please sign a petition asking the Prime Minister to ensure treatment for MDS patients in the UK matches the rest of Europe.

Check our campaigns page for more ways you can help out before the appeal on 1st June 2010.

Please sign a petition asking the Prime Minister to ensure treatment for MDS patients in the UK matches the rest of Europe.

Check our campaigns page for more ways you can help out before the appeal on 1st June 2010.

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, “We are extremely disappointed that the Scottish Medicines Consortium has rejected the only drug proven to give MDS patients more time and a better quality of life. Azacitidine is the only licensed drug currently available to treat MDS specifically and is available to patients throughout most of the European Union. So while a patient in Romania can have access to the most innovative and effective treatment for MDS, this decision means a patient in Edinburgh or Glasgow will be denied that access.”

In supporting the MDS UK Patient Support Group, Tony Gavin the Director of Campaigning and Advocacy for Leukaemia CARE said, “Denying this innovative treatment to MDS patients and those progressing to acute myeloid leukaemia, will sentence them to a poorer quality of life and an earlier than necessary death. There is no effective existing therapy available currently, and to deny patients this innovative new treatment for MDS is unacceptable. Further to a review of the economic and cost-effectiveness criteria, the SMC must give increased consideration to quality of life issues in appraising treatments for rarer diseases.”

The MDS UK Patient Group announced last month that it is appealing jointly with the Rarer Cancer Forum and the Leukaemia Society against the negative recommendation on azacitidine issued in early March by the National Institute for Health and Clinical Excellence (NICE). Celgene Ltd, the manufacturer of Vidaza (azacitidine), the Royal College of Pathologists and the British Society for Haematology are also appealing and the National Cancer Research Institute Haematological Oncology Clinical Studies Group and the Royal College of Physicians’ Medical Oncology Joint Special Committee have lodged a joint appeal against the NICE guidance.

“The next Government has the power to invest in the future of cancer care. We need every parliamentary candidate to make a firm commitment to cancer support, and you can make this happen.

It only takes two minutes to send the template email to your local candidates, asking them to support our three calls on cancer.”

Click here to access the webpage: Macmillan campaign for parliamentary candidates

This petition was set up on the Number 10 Petition website by patient Donald Facey to ask the Prime Minister to “overturn the decision by the National Institute for Health and Clinical Excellence (NICE) to approve a life-saving new cancer drug called Azacitidine which will help all people who suffer from acute myeloid leukaemia to have the right to live.” This petition collected 525 signatures.
The Prime Minister’s Office has responded to that petition and you can view it here.

The petition set up by the MDS UK Forum and Prof Mufti is still open and we urge everyone who has not yet done so to sign it – and forward it to as many people as possible – family, friends, colleagues, neighbours: MDS Petition – please sign it now

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: http://guidance.nice.org.uk/TA/Wave18/19
2. Information on Vidaza (azacitidine), which is manufactured by Celgene Ltd, can be found at http://emc.medicines.org.uk/medicine/21645/PIL/Vidaza+25+mg+ml+powder+for+suspension+for+injection/
   
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. http://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(09)70003-8/fulltext
4. The Rarer Cancers Forum report can be found at: http://www.rarercancers.org.uk/news/current/exceptionalprog.final.pdf

Media Coverage

For more media coverage please download our free PDF

The Final Appraisal Determination issued by NICE states that azacitidine is not recommended as a treatment option for people with intermediate-2 or high risk myelodysplastic syndromes (MDS), nor is it recommended for patients with forms of chronic myelomonocytic leukaemia or acute myeloid leukaemia. While NICE recognised that azacitidine was clinically effective and that it should be seen as a life-extending end-of-life treatment, it decided that it should not be provided through the NHS on cost-effectiveness grounds.

An appeal is currently being considered. We may require your urgent assistance should an appeal process be possible.

Please check our website regularly in the week of the 8th March.

Please also check our campaigns page for more details on our work to gain access to azacitidine for NHS patients

Documents

• Final Press Release (Adobe PDF)
• Coverage (Adobe PDF))
• Professor Rodney Taylor, an MDS Patient, was interviewed on BBC Radio’s Today Programme (4 March 2010) where he spoke about the need for the NICE HTA process to be more flexible in the evaluation of medicines for rarer cancers that affect only small patient groups. Listen to the clip.

“Scientists at King’s have developed a vaccine treatment for Leukaemia that can be used to stop the disease returning after chemotherapy or bone marrow transplant. The vaccine is due to be tested on patients for the first time. Eventually it is hoped the drug, which activates the body’s own immune system against the leukaemia, could be used to treat other types of cancers. ” KCL website.

Please find more information in the links below:
Official King’s College website:

http://www.kcl.ac.uk/news/news_details.php?news_id=1257&year=2010

Daily Telegraph – 4 Jan 2010

http://www.telegraph.co.uk/health/healthnews/6872451/Leukaemia-vaccine-being-developed.html

NHS Choices – Health news – 4 Jan 2010

http://www.nhs.uk/news/2010/01January/Pages/Leukaemia-vaccine-to-be-tested.aspx

http://www.thisislondon.co.uk/standard/article-23791598-nhs-refusal-to-give-me-drugs-is-death-sentence.do

http://www.cmlsupport.org.uk/?q=node/1302

http://www.yourlocalguardian.co.uk/news/local/richmondnews/4839519.Cancer_sufferer_fights_for_lifesaving_drugs/

For MDS and azacitidine – we are currently awaiting NICE’s decision whether to recommend the drug or not for NHS patients.
The 3rd hearing has taken place yesterday, 7th Jan 2010.
We should have the result of this decision in about 2 weeks time.

If you have been denied access to azacitidine recently, please contact us

Please also sign our petition, requesting azacitidine to be made available on the NHS.
We need as many signatures as possible!
Please ask your friends, families, colleagues to sign as well:

http://www.mdspatientsupport.org.uk/Vidaza_Petition.html

Thank you