Cancer patient support groups to appeal negative NICE decision

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

For more media coverage please download our file  Press coverage  (Adobe PDF)

Only 200 patients helped by £50m fund for extra cancer drugs

Only a tiny proportion of the Government’s interim fund for cancer drugs has been claimed, almost a third of the way through its six-month lifespan, The Times has discovered.

The £50 million emergency fund for drugs not approved by the NHS watchdog has been used for only 200 patients, with doctors finding the application process bureaucratic and confusing.

Wide regional variations also emerge from figures obtained by The Times from regional strategic health authorities, with experts warning of a return to a “postcode lottery”. For example, while the NHS in the West Midlands has paid out £500,000 from the fund on 22 patients, in the South Central region only £38,000 has been spent on ten patients.

The fund allows doctors to pay for expensive cancer drugs that have not been deemed cost-effective by the National Institute for Health and Clinical Excellence.

Before the general election David Cameron promised a £200-million-a-year cancer drugs fund to allow these drugs to be prescribed on the NHS. The interim £50 million, which opened in October, will be available until the main fund begins in the spring. However, figures compiled from England’s ten NHS regions show that only about 200 people have been treated under the scheme nationwide, a tenth of independent projections, while in Yorkshire the number of applications is in single figures.

About £2 million, 4 per cent of the total, has been spent in the five regions which revealed spending figures. The remaining regions have had similar numbers of applications, suggesting that well under 10 per cent of the £50 million has been claimed.

Experts said that many doctors did not understand how to access the scheme, while others did not want to use it. A survey of hundreds of oncologists carried out by Professor Lesley Fallowfield of the Univeristy of Sussex said: “Many of the doctors had no idea what the policy of their trust was. Many doctors are still uncertain because it’s somewhat ambiguous as to what the process is.”

Different areas have the power to allow different drugs, with Avastin, a cancer drug rejected by Nice, automatically available under the fund in some parts of the country, but not in others.

Professor Fallowfield, a psychologist who studies how cancer is treated, said: “The process to access this fund is still unclear and my anxiety generally is that panels in different SHAs are making their own idiosyncratic decisions.”

She said there was a danger that those who “shout the loudest” could get treatment, not those who need it most.

Doctors also felt that they should put their responsibility to the wider NHS ahead of individual patients, the survey found, meaning that they often did not want to apply for unapproved drugs.

“Many doctors were thinking ‘I’m not sure that NICE hasn’t made the right call’,” Professor Fallowfield said.

A spokesman for the Department of Health said it was monitoring applications and would assess the fund in April.

COST AND EFFECT

Avastin (Bevacizumab) In advanced bowel cancer, can prolong life by six weeks on average, but NICE ruled £21,000 per patient was not cost effective.

Azacitidine In rare cancers of the blood and bone marrow, shown to extend life by nine months on average, but NICE provisionally ruled that £45,000 a head was too much.

Imatinib Used on chronic myeloid leukaemia and intestinal tumours, but NICE said there was not enough evidence to justify £19,500 per patient to give it to people whose tumours had been removed.

Temsirolimus (Torisel) Not approved for patients with kidney cancer.

Source: The Times – 22/11/2010

Latest update on how the Interim Cancer Drug Fund will be implemented at your local Strategic Health Authority level.
Check your SHA guidelines availability of azacitidine.
Information is published by the Rarer Cancers Foundation website.

Please take part in the current Macmillan campaign, as their priorities are very closely linked to MDS issues:

“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

In preliminary draft guidance issued for public consultation NICE has not recommended azacitidine (Vidaza, Celgene) as a treatment option for people who have the following conditions, and are not eligible for haematopoietic stem cell transplantation: intermediate-2 and high-risk myelodysplastic syndromes, chronic myelomonocytic leukaemia and acute myeloid leukaemia.

The committee decided that although azacitadine was shown to be clinically effective, the benefits to patients could not justify the high cost of the drug.

This draft guidance has been issued for consultation. NICE has not yet issued final guidance to the NHS.

Publication of this second appraisal consultation document follows the return of the appraisal to the independent Appraisal Committee after appeals against the Institute’s final appraisal determination were partially upheld. The appeals were received from the manufacturer of azacitidine (Celgene), a joint appeal from the Royal College of Pathologists and British Society of Haematology, a joint appeal from the Leukaemia Society, Rarer Cancer Forum and MDS UK Patient Support Group and one from the Royal College of Physicians Medical Oncology Joint Special Committee.

The Appeal Panel requested that the Appraisal Committee reconsider the guidance, taking account of a wider range of comparators than originally considered. The Appeal Panel also suggested that the Appraisal Committee examine data on quality of life submitted by MDS UK.

NICE Chief Executive, Sir Andrew Dillon said: “The independent Appraisal Committee reconsidered the evidence on the effectiveness of azacitidine, when compared with best supportive care and low-dose chemotherapy. They also considered the additional data submitted by MDS UK on quality of life. However, the Committee again concluded that the drug could not be recommended as a cost effective use of NHS resources. This is a very expensive drug, even with the small discount offered by the manufacturer.”

“Although we are very disappointed not to be able to recommend this treatment, we have assessed it fairly and on precisely the same basis as other drugs used for rarer conditions.”

Myelodysplastic syndromes (MDS) are a group of bone marrow disorders, where the marrow doesn’t produce enough of one or more types of blood cells. The majority of patients with MDS receive best supportive care in current clinical practice and some patients receive low dose chemotherapy. There are approximately 700 patients with MDS in England and Wales.

Notes:

1. The guidance is available here.
2. According to the manufacturer’s estimates, azacitidine costs approximately £45,000 per patient.
3. The manufacturer of azacitidine has agreed a patient access scheme with the Department of Health, in which azacitidine for the treatment of myelodysplastic syndromes, chronic myelomonocytic leukaemia and acute myeloid leukaemia will be available with a 7% reduction in the acquisition cost.
4. The committee agreed that azacitidine did fit the criteria to be considered under the supplementary advice for end of life medicines; however, the magnitude of additional weight that would need to be assigned to the original QALY for the cost effectiveness of the drug to fall within the current threshold range would be too great, even when the patient access scheme was incorporated.
5. The committee agreed that the most plausible ICER for azacitidine in the overall patient population was approximately £59,000 per QALY gained.

4th March 2010 – NICE has just issued their FINAL decision on the cost-effectiveness HTA for azacitidine (Vidaza). They DO NOT recommend the use of the drug for NHS patients.

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Prime minister’s office responds to the AML (Acute Myeloid Leukaemia) petition for azacitidine:

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The government’s drug rationing body, NICE, is to be stripped of its power to turn down new medicines for use in the NHS.

Nice to lose powers to decide on new drugs  Guardian article who first reported the news on Saturday 30th October 2010

Drug victory for patients: ‘Penny-pinching’ NICE stripped of power to ban life-saving drugs  Daily Mail article 1st November 2010

Awareness of rarer cancers Further article by John Leech MP – who tabled the adjournment debate on Rarer Cancers and MDS on the 26th October in Parliament. He also tabled an EDM (Early Day Motion).

Disappointment as Vidaza appeal fails to sway NICE rejection  PharmaTimes article on latest NICE decision 4th November 2010 –

“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.

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