MDS patients achieve access to vital new treatment option

January 2015 update
Equal and easy access to MDS drugs is crucial for all MDS patients. Here is an 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.

ULTRA Challenge series in 2015

Walk it, Jog it or Run it – choose your distance!

 It’s Your Challenge – Your Way!

If you’re looking for a challenge in 2015 you might be tempted to join any of the following 2015 events and help raise funds for our charity.

Several people have already signed up – but we need more brave participants to help us maintain our services in future.

Click here to read more about our first 2015 Ultra fundraising Team.

These challenges are organised by Action Challenge. Further information from the organisers about the ULTRA Challenge 2015 series below:

Whether you set out from London towards the coast, along the river or canal towpath into the countryside, or make a weekend of it on the Isle of Wight – push yourself further in 2015. Walk, Jog, or Run a full 100 km distance – with some training, our support & famous hospitality – it’s achievable! With 50 km & 25 km options also – there’s a Challenge here for everyone! Join as a Team or as an Individual, and fundraise for a Charity of your choice. It’s Your Challenge, Your Way, for Your Charity!

These series of events are all fully supported, with rest stops, medics, free food & drinks, marshals along the route, and massages at various rest stops. At the finish line, you’ll be welcomed back with a glass of bubbly, commemorative t-shirt, and a finisher’s medal to cheers and celebrations from family and friends! And if that wasn’t good enough, you’ll go home proud of your achievement, safe in the knowledge that you’ve made a real difference in helping your charity!

To sign up any of the Ultra Challenges, please see the website links below and follow the instructions to register. By you taking part, it would be helping to make a real difference to your chosen charity and we know it’s an amazing experience that you’ll never forget.

• 100km – £49 to register with a minimum fundraising target of £475 for your chosen charity
• 50km – £39 to register with a minimum fundraising target of £295 for for your chosen charity
• 25km – £29 to register with a minimum fundraising target of £175 for for your chosen charity

London 2 Brighton Challenge, 23rd – 24th May 2015

Hundreds of registrations have already been flooding in for one of our most popular and iconic events! New for 2015 is the 44km second half route addition, which means participants can now choose between this; the first 56km or the full 100km. This is now one of the UK’s greatest endurance walking challenges, and the UK’s largest Ultra-Marathon run.

For further details about the London 2 Brighton Challenge or to register, click here.

Grand Union Challenge, 27th – 28th June 2015

London’s Great Escape saw a fantastic first year launch and is now ready for 2015 too! A scenic challenge that follows the Grand Union canal out of London towards the Chilterns, this has all kinds of routes available including 25km (a choice of all 4 legs!), 50km and 100km. Push yourself further for 2015 and explore this fantastic hidden green corridor out of the capital – it’s one of London’s best kept secrets!

For further details about the Grand Union Challenge or to register click here.

Isle of Wight Challenge, 2nd – 3rd May, 2015

With a brand new date for 2015 – the Isle of Wight Challenge has partnered with the Isle of Wight walking festival – now part of Europe’s biggest walking festival! A 106 km coastal loop route, or challengers can tackle a 30km route to the needles, or a ‘half-way’ 56km challenge – all centring around our ‘Base Camp’. Arguably the toughest challenge of the series – but will be one of the most rewarding!

For further details about the Isle of Wight Challenge event or to register here.

The London 2 Cambridge Challenge, 29th – 30th August 2015

New for 2015 – The London 2 Cambridge Challenge is a 100km or 50km walk or run following the quiet and pretty River Lea out of the capital. The route continues across wild flower meadows and through farmland, occasionally passing through quaint rural villages, before joining a direct route into Cambridge. An iconic and historic city to mark the end of an epic challenge.

For further details about the London 2 Cambridge Challenge or to register click here.

Thames Path Challenge, 12th – 13th September 2015

Due to be 2015’s biggest challenge event of the Series, this also has the multi route options available with the 25km, 50km and 100km routes. With most of the year to fundraise and train, this is the biggest and most memorable journey from start to finish. With it’s beautiful and varied scenery, this certainly makes for one of the best events of the series, as well as being the perfect event for teams!

For further details about the Thames Path Challenge or to register click here.

Please get in touch to tell us more about your fundraising activities – we would love to feature your fundraising story on our website and support your efforts!

We have a supply of materials including: T-shirts, badges, posters and information leaflets – ready for you to order. Do let us know if there is anything else we can do to help.

Call our main office on 02077337558 or email us on mds-uk@mds-foundation.org

MDS – Call to Action Report

MDS World Awareness Day is taking place on Saturday 25th October, where patients and patient groups come together to raise awareness of MDS and help to improve the standard of treatment for these patients.

To coincide with this important date and together with blood cancer charity Leukaemia CARE, MDS UK is launching a report:
‘Addressing the management of MDS in elderly patients’, calling for better care for older MDS patients whose care needs are still unmet.
This document is available for download, here: Call to Action report

There is growing evidence to suggest that cancer is under treated in elderly patients, an example of this is with MDS, which predominately affects patients aged over 60. The report reveals that elderly people with MDS are not consistently being managed according to official treatment guidelines, resulting in substandard care.

What are we calling for?

We are calling for all of those working within the field of MDS and its treatment to take action and implement the recommendations outlined in the report to improve patient care and experiences.

If you have any questions about the report, or would like to help us in disseminating this information, please get in touch.

Please do take a copy of this important document to your MP as well.

The report is the outcome of a round table with geriatricians, MDS experts, haematology nurses and patient groups.
We would like to thank all involved in the production of this report for their input and expertise.

Further information for clinical staff:

• British Society of Haematology MDS Treatment Guidelines – please download here MDS Guidelines 2014-BSH (for clinical staff).
• MDS GP FACT SHEET August 2013 – for GP’s

For everyone:

If you would like to find out more and support MDS World Awareness Day – please go to:

• News post on MDS World Awareness Day
• MDS UK Patient Support Group Facebook
• hashtag #MDSBloodCancer on Twitter
• MDS Alliance website – the new international umbrella group for MDS Patient groups

Thank you
MDS UK & Leukaemia CARE

MDS UK Contacts:
Tel 0207 733 7558 – Helpline 9am to 6pm – Mon-Fri
Email mds-uk@mds-foundation.org
Facebook MDS UK Patient Support Group
Twitter @MDS_UK

Leukaemia CARE Contacts:
communications@leukaemiacare.org.uk
Tel: 01905 755977 – 24-hour CARE Line: 08088 010 444 (free from landlines and all major mobile networks) www.leukaemiacare.org.uk
Like us on Facebook: LeukaemiaCARE
Follow us on Twitter: @LeukaemiaCAREuk
Join us on LinkedIn: Leukaemia-care
Pin it on Pinterest: LeukaemiaCARE

                                    MDS World Awareness Day – Preparations for 2015

We are preparing for MDS Day 2015.  Do contact us if you’d like to participate in any events around the country to mark this international awareness day for Myelodysplastic Syndromes.
See below for MDS day 2014 – in the UK and in other countries.

MDS World Awareness Day – 25th October 2014

MDS Patient Support Groups worldwide are preparing throughout this week to mark the day on Saturday, 25th October 2014.

In the UK, patients and relatives are making use of our busy Facebook page to post pictures, messages and facts about MDS.

In Ireland, our Irish group is holding a patient meeting on Saturday

In Germany, our colleagues are having a patient information day in Munich.

Spain is hosting a radio talk, and football game.

In the USA, the MDS Foundation will be hosting a twitter chat on Saturday, on behalf of the MDS Alliance, the newly created umbrella group for all MDS support groups worldwide.
Read more on this topic and see all our colleagues worldwide here: Worldwide MDS Groups

And more about previous World Awareness Days.

We will be posting pictures and messages over the next few days to illustrate events and patient contributions.
Please also check our Patient Stories page – being updated regularly: MDS Patient Stories.

Here are the first photos of our preparation day at King’s, attended by staff and patients.  We also displayed information from Delete Blood Cancer UK, Anthony Nolan, and Leukaemia and Lymphoma Research, and Leukaemia CARE.

Thank you to all who dropped by today and picked up their badges for the 25th October – MDS World Awareness Day.

On Friday 24th – we ask you to pay particular attention to a document we will be releasing together with Leukaemia CARE, about care and treatment of older MDS patients – a Call to Action – adressed to all who look after MDS patients.

NHS England launches consultation on proposed changes to Cancer Drug Fund Standard Operating Procedures

Patients and individuals are encouraged to comment and respond to this important consultation.
The deadline is the midnight on Friday 31st October.

MDS patients have benefited enormously from the CDF to date, without which many patients would not have had access to drugs such as azacitidine and lenalidomide prior to the NICE approval.
The changes are indicative of a need to again monitor costs of new drugs – in order to sustain a system that will benefit all cancer patients.

MDS UK has attended a briefing event organised by CDF for patients groups on 20^th October – and will submit comments to NHS England on behalf of MDS patients.

Further information about the consultation and proposed changes below:

The consultation is focused on a number of proposals to change the current Standard Operating Procedure, which sets out how the fund is managed.
There are three proposed changes:
• Implementation of a re-evaluation process to assess the drugs on the current national list, and remove those which represent the lowest levels of clinical benefit
• Inclusion into this re-evaluation system of a confidential element which assesses the average cost of a drug per patient in relation to the clinical benefit delivered
• Inclusion of an option which enables pharmaceutical companies to make adjustments to drug prices, in order to allow their drug/indication to remain on the CDF list

The proposals represent a significant change to the way the CDF is currently managed, and therefore NHS England is keen to test out the proposals with as many people as possible who have an interest in the fund.

Details of the consultation, and how you can submit responses, can be found on the CONSULTATION PAGE, where you will find two documents and an online consultation survey:
• A CONSULTATION GUIDE – this document introduces the Standard Operating Procedure; explains why we are consulting on it, and sets out the proposals for change and associated questions;
• THE PROPOSED STANDARD OPERATING PROCEDURE FOR THE CDF – this document sets out of the proposed changes to the current procedures, and are clearly highlighted for ease of reference.

If you have any questions about the materials listed above please contact: england.cdfconsultation@nhs.net 

28/08/14 – NHS England have announced the allocation of further funds for the Cancer Drug Fund (CDF)

This is good news for cancer patients, whilst we are waiting to see how NICE will finally alter their appraisal system, following the recent consultation on Value Based Assessments (VBA).

However – the CDF is only a temporary solution to a long-term problem.
Until NICE resolves the issues it has with appraising drugs for rare diseases, and working with scarce data, we will continue to need a CDF to temporarily plug the gaps.
HTA stakeholders do assist NICE in every way they can – in their search for a better understanding or more precise data, or to correct misunderstandings – but this takes time, efforts and money.  And in rare or life-threatening diseases – time is something most patients don’t have much of.

Here is the full article, from the CDF webpage:
“NHS England sets out plan for a sustainable Cancer Drugs Fund – 28 August 2014 – 00:01

NHS England has today pledged a £160m boost over two years for the Cancer Drugs Fund, giving more patients access to innovative drugs not routinely available on the NHS.

Two new drugs will now also be added to the Fund; enzalutamide for prostate cancer and lenalidomide for a group of patients with myelodysplastic syndrome, a rare blood condition.  (* see note below for lenalidomide clarification)

With an annual budget of £200m, now rising by 40 per cent to £280m, the Fund has delivered important benefits for more than 55,000 patients since it began in 2010.

Professor Peter Clark, Chair of the Cancer Drugs Fund (CDF), has written to NHS England Chief Executive Simon Stevens recommending CDF’s panel of experts re-evaluate a number of drugs currently on the list, making evidence-based decisions about what will deliver greatest benefit to patients. NHS England has accepted and agreed these proposals. Copies of the letters are on the NHS England website.

Professor Clark, who is an oncologist, said: “The Cancer Drugs Fund has had a positive impact on cancer treatment for patients, with 2,000 people gaining access to the Fund every month. To ensure patients continue to have access to the best innovative treatments now and in the future, we must re-evaluate some of the drugs on the list. This is absolutely the right thing to do for patients, and I am pleased Simon Stevens has accepted my recommendations.”

Responding to Professor Clark’s proposal, Simon Stevens, NHS England’s Chief Executive, said: “Since its inception, The Cancer Drugs Fund has successfully enabled the funding of new cancer medicines for tens of thousands of patients in England. We want to build on that success by expanding funding again this year to enable cancer patients to access innovative effective new treatments.

“Within this extra funding we want to ensure the drugs provide maximum benefits to patients and that the CDF incentivises responsible pricing by drug companies. We want to create a sustainable Cancer Drugs Fund that will ensure patients don’t miss out on innovative treatments that are on the horizon.”

Mr Stevens has also asked Professor Clark to work with NICE, patient groups and the Association of the British Pharmaceutical Industry (ABPI) to develop options for ensuring greater alignment between CDF and NICE assessment processes. The CDF panel will also develop options for a new Evaluation through Commissioning scheme. This would allow the evaluation of the clinical effectiveness of promising new drugs, which are not currently supported by enough evidence to allow NICE to recommend they are made routinely available.

The CDF panel, comprising doctors, pharmacists and patient representatives, will review all available evidence for a number of drugs currently on the list. The panel’s decisions will be published in due course.”
Cancer Drug Fund article

“Sir Andrew Dillon, Nice chief executive, suggested in an interview with the Financial Times last week that, once the CDF expired, a new way should be found to evaluate drugs. This would potentially allow drugs to be launched for an interim period so that information could be gleaned about their effectiveness before a final price was agreed.”   Financial Times – full article.

Having an interim decision – especially when NICE is unable to make a clear decision, due to uncertainties in the cost-effectiveness financial model – is something MDS UK has suggested at previous NICE appraisal meetings.
We therefore definitely welcome such a suggestion from Andrew Dillon – and hope this will be implemented as soon as possible.  This would most certainly help to reduce delays and would help to gather the necessary practical evidence often missing with rare diseases and scarce trial data.
It may also help to rely less heavily on the current QALY system, which again is inadequate for scarce data.

A further NICE improvement would be to allow all stakeholders – clinical experts, patient experts and pharma – to be present at all NICE appraisal meetings – in order to provide additional expert information and reduce misinterpretations.  Currently only the pharma industry is allowed to participate in all appraisal meetings.
Clinical experts – as well as patient experts are only allowed as public observers – after the initial NICE meeting.

* The CDF has now approved the use of lenalidomide for MDS del 5q patients with 1 more chromosomal abnormality.
NICE has now already approved lenalidomide for del 5q as a single chromosomal abnormality – see our previous News item here:  NICE approves lenalidomide – 20/08/14

Further news and media coverage of the CDF announcement:

Dept of Health press release

Guardian article

Reuters article

Telegraph article

The Independent

Daily Mail article

When looking for a meeting location for our new local Patient Support Group in Bournemouth, we came across a very special hotel – the Grove. Not only does it cater exclusively for cancer patients – it is also a charity – and operates on a not for profit basis.
MDS UK will now start holding it Bournemouth local meetings there – from November 2014.
We would like to thank the Grove for their assistance and cooperation in setting up our meetings there – and look forward to meeting the staff and enjoying the location with our MDS UK patient members.

And we’d like to share the information about this unique hotel with our readers – as the Grove is the UK’s only not for profit hotel for use exclusively by sufferers of Cancer and other life-limiting illnesses, specially adapted to the needs of their guests.

Here are their full details – and a description of services:

The Grove Bournemouth Hotel

UK’s exclusive hotel created for sufferers of life-limiting illnesses

The seaside resort of Bournemouth is the idyllic setting to escape from the world for a while. For Cancer sufferers, often that is exactly what is required – taking their mind off their illness and getting away for a short, well-deserved break. With first class care in mind, The Grove Hotel in Bournemouth has recently been launched, serving as the UK’s only hotel of its kind exclusively for use by sufferers of Cancer and other life-limiting illnesses.

Previously, Albany Lodge was launched to serve this market, and had been running for over 25 years. However, with growing demand, the Albany was no longer fit for purpose and a new and even more charming site needed to be found – resulting in the launch of The Grove.

Situated on Bournemouth’s leafy East Cliff, and just minutes away from the town’s famous 7 miles of golden beaches, The Grove Hotel was created as a haven offering a week or two away from the tough business of getting well, and simply focuses on recuperation and relaxation. During their stay, guests of the hotel will enjoy evenings of entertainment arranged by the hotel and exceptional dining laid on by their team of talented chefs, who are entirely mindful and trained to deal with any specific dietary requirements which are products of the guests’ illnesses. 

To the delight of guests, The Grove has a dedicated beach hut in a prime location so that guests can take advantage of the sunshine in the ultimate comfort. To enhance their stay, complimentary coach tours are arranged by the Hotel to allow guests to explore the surrounding areas of the New Forest and the Purbeck and Jurassic coast.

Given its focus on healthcare, The Grove has a duty nurse available 24 hours a day. They are on hand to offer guidance and support and to arrange any services that they may require. The Grove is owned and operated by Macmillan Caring Locally based in Christchurch Dorset, whose aim is to support people suffering from cancer and other life threatening illnesses, as well as members of their family and carers. As the hotel is a registered charity, they welcome support and donations to help them provide outstanding breaks for their guests.

General Manager of The Grove Hotel, Brenden Howard, spoke about the idea behind the property: “Our aim was to create the UK’s leading hotel exclusively for sufferers of life-threatening diseases. Of course we welcome their family or carers to join them at the hotel which will hopefully enhance their stay. We strive to offer a serenely peaceful and enriching holiday, whilst offering the highest level of service and care.”

Hotel guest, Gillian Burr, commented on her stay: “The staff at The Grove are just wonderful – nothing is too much trouble for them! The food was also fantastic – such a lovely variety every day! This is exactly what I needed to take my mind off things for a week, and I cannot wait for my next holiday here.”

For more information, visit www.thegrovebournemouth.co.uk.

Please click here if you’d like to download their leaflet: The Grove – leaflet, do you need a holiday?

We look forward to our first Bournemouth Group meeting  on 12th November  – click here for details about the meeting.