January 2015 update
Equal and easy access to MDS drugs is crucial for all MDS patients.
Here is a quick update on the MDS specific treatments available currently :

  • azacitidine (Vidaza) – NICE and SMC recommended for routine NHS use – available nationwide in the UK
    Licensed for IPSS risk groups Intermediate 2 and high-risk MDS, AML and CMML.
    Must be given for at least 6 cycles – followed by a biopsy to check response to the drug.

  • lenalidomide (Revlimid) – NICE and SMC recommended for routine use in NHS:
    Licensed for isolated del 5q MDS patients who are transfusion dependent. 

    For patients with del 5q MDS plus 1 other chromosomal abnormality – lenalidomide is available through the Cancer Drug Fund. 

    Lenalidomide is sometimes also used for MDS patients without del 5q – either as part of a trial, or via IFRs (Individual Funding Requests), and has shown good results for many patients.
    Please speak to your haematologist to find out if you may benefit from trying this drug out.

 

  • deferasirox (Exjade) – this oral iron chelation should be available anywhere in the UK, but patients may need to try the traditional pump treatment deferoxamine first – to evaluate its ease of use.
    Let us know if you need/wish to be on this oral chelation drug but experience funding problems
    For MDS patients on regular blood transfusion and with a high ferritin level.

Always contact us should you have problems accessing any drugs recommended by your haematologist.  Local commissioning can vary unfortunately.
Always also involve your MP if you have any difficulties accessing medication to help with your MDS.

March 2013:
Azacitidine now seems to be easily available throughout the country – which is wonderful news.
Should anyone encounter any issues however – please contact us.
As a reminder – azacitidine must be given and funded for at least 4 cycles to establish whether the patient is responding to the drug (unless of course clinicians decide to stop the treatment for clinical reasons).
Some patients may not respond until 6 cycles – so discuss this with your physician.

Other issues our patients encounter:
Oral iron chelation drug: Some patients have reported access/funding problems for the drug Exjade (deferasirox).
Generally speaking, patients who need iron chelation – due to numerous blood transfusions – are first asked to use the Desferal (deferoxamine) pump or injections to reduce their ferritin levels.
If this method proves unsuitable for patients – or when patients do not tolerated the desferal pump, the oral chelator Exjade (deferasirox) can be prescribed and must be funded by the PCT (Primary Care Trust).
Each PCT will have issues their own funding guidelines about this – so it is worth checking those and discussing this with your haematologist or specialist nurse should you encounter problems.

Also – always inform your MP about any treatment access issues you encounter.  This is of special importance with the new commissioning methods in place.

And of course – send us details of the problems – so we can help – and highlight the issues.

August 2012:
Access to MDS treatment has improved immensely since the NICE approval of MDS drug azacitidine in February 2011.

See all details in article below.  Azacitidine is now available on the NHS in all hospitals – and funding is now no longer an issue.

Should you encounter any difficulties – please contact us.

We will shortly also include more news about treatment access.

 

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 (CML) 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, who are still waiting for a decision from The Scottish Medicines Consortium (SMC). 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 re-submit 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 recommendation for use of azacitidine was obtained after the manufacturer of the drug agreed a revised patient access scheme with the Department of Health in which azacitidine for the treatment of myelodysplastic syndromes, chronic myelomonocytic leukaemia and acute myeloid leukaemia would be available with a discount. This discount is commercial-in-confidence.

Implementation of this new NICE guideline:

When a NICE technology appraisal recommends use of a drug or treatment, or other technology, the NHS must usually provide funding and resources for it within 3 months of the guidance being published. Please do let us know if you experience any problems accessing azacitidine during this time, as the funding for the drug moves from the Cancer Drug Fund to a standard NHS drug recommended by NICE.

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