The UK MDS Forum is thrilled that NICE have approved the use of azacitidine in the NHS in England within its European licence. Reactions to this very important news from members of The Forum and MDS UK Patients Support Group are posted here for you to read. It is hoped that the drug will similarly become available in Wales, Scotland and Northern Ireland through due process very soon.

PRESS RELEASE

MDS patients achieve access to vital new treatment option
National Institute for Health and Clinical Excellence recommend azacitidine is available through the NHS

London, 17 February 2011 – The National Institute for Health and Clinical Excellence
(NICE) announced today that Vidaza (azacitidine), the only licensed drug available
specifically to treat myelodysplastic syndromes (MDS) - a range of life-threatening bone
marrow disorders - will be available through the NHS. The MDS UK Patient Support Group,
which has been in consultation with NICE during the approval process and campaigned on
behalf and in conjunction with MDS patients across the UK, welcomes this announcement.

There are nearly 3,000 new cases of MDS in the UK each year and many people newly
diagnosed with MDS have not heard of this disorder before. A person with MDS will suffer
from chronic tiredness and weakness due to the often extremely low levels of haemoglobin,
owing to a malfunction in the bone marrow in producing the correct quantity and quality of
blood cells. This is debilitating in itself and often requires regular blood transfusions.

Azacitidine is an anticancer drug that is thought to work by re-establishing cells’ natural
mechanisms to control abnormal growth. The final appraisal determination by NICE
recommends the use of azacitidine for the treatment of MDS, chronic myelomonocytic
leukaemia (CMML) and acute myeloid leukaemia (AML) following a revision to the patient
access scheme provided by Celgene (the manufacturers of azacitidine). Rodney Taylor,
Deputy Chairman of MDS UK Patient Support Group said, "I am delighted to hear of NICE's
decision to recommend azacitidine for these patient groups which can benefit from this form
of treatment. Having been on azacitidine myself, I know how effective it can be in promoting
a good quality of life, giving independence from blood transfusions and allowing patients to
lead a normal family life. Azacitidine is the only specific treatment for MDS that improves
quality of life, prolongs survival and delays disease progression. It is great news that many
more patients will now be able to benefit from it."

While the announcement is good news for MDS patients in England and Wales, MDS UK
Patient Support Group is concerned that access to this vital new treatment is still denied to
MDS patients in Scotland, as azacitidine has yet to be re-submitted to The Scottish Medicines
Consortium (SMC) following a rejection in April 2010. We urge all concerned to apply both
maximum effort and the highest priority to bring azacitidine to Scotland, in line with England
and Wales. “Clearly I am delighted that NICE has approved azacitidine for use in England
and Wales in conjunction with the associated patient access scheme,” said Dominic Culligan,
Consultant Haematologist, Aberdeen Royal Infirmary, Scotland. “I hope that Celgene will resubmit
azacitidine to the SMC as a matter of urgency, so that further consideration can be
given to making this important treatment for high risk MDS and some patients with AML
available in Scotland.”

It is a distressing reality that, during the protracted evaluation process attendant to the successful
outcome of this appraisal, some MDS patients have progressed to AML, and some did not survive,
in the absence of azacitidine. MDS UK Patient Support Group are aware of the complex financial
constraints and cost effectiveness criteria attendant to the adoption of new drugs, and ask only that
even more effort is applied in reaching speedier positive conclusions in critical, end of life
situations such as MDS.

This website is still being developed, and we would be delighted to receive suggestions for content and improvement, notification of corrections etc at mds@hartleytaylor.co.uk.

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Please send your comments to dominic.culligan@nhs.net