Khalid Younis – A Letter From Number 10

In October I wrote this letter to David Cameron, our Prime Minister.

Dear Prime Minister,

The photo below is of me with my new friend, his name is Khalid Younis. We both have a rare form of leukaemia called Chronic Myeloid Leukaemia (CML). Treatment for CML was revolutionised in 2001 when Time magazine hailed a drug called imatinib a magic bullet against cancer. Now, 14 years later, my friend cannot access a new, potentially life-saving drug called ponatinib because it hasn’t been appraised by NICE.

Khalid Younis and Kris Griffin

I don’t understand why the cancer drug fund process was closed alongside the appraisal process, we find ourselves falling through an administrative gap. Khalid’s situation has attracted nearly 8,000 signatures on a petition and the story has been featured in the Daily Mail, Daily Mirror and the local Birmingham newspapers. We’ve contacted Khalid’s MP, Roger Godsiff and my MP, Mark Garnier. Mark knows my work and has always been really supportive.

Whilst I realise you have many responsibilities and must receive many letters like this; if you have time to meet or discuss this issue and help us I would be extremely grateful.

Ponatinib offers the last real hope Khalid has, not being able to access it in England is devastating.

Yours sincerely,

Kris Griffin (Mr)

Last week I received this reply:

Of course I’m disappointed, I’m not sure if I ever thought he’d agree to a meeting and fix the problem. My issue with this letter is that the Department of Health is part of the problem. They’ve allowed this issue to manifest and within the space of two days (last week) I’ve managed to extract three different dates when they expect the appraisal process to be ready. Not ready for use…oh no..ready for it to be reviewed. In the meantime, we don’t have an appraisal process and by that point we won’t have a cancer drugs fund. I don’t want to write a ‘told you so’ letter to our PM.

So, I’ll be writing to Mr Cameron again, perhaps I’ll also include the 8,000 signatures on this petition and ask him what he thinks the Department of Health will do; because from where I’m sitting they are making the situation worse.

Kris Griffin – Access CML Drugs

Letters from NHS England and 10 Downing Street

Some very pertinent housekeeping. Earlier in the year David Ryner from CML Support asked me, Bloodwise, Leukaemia Care and CML-UK (Facebook) to co-sign a letter he penned to Simon Stevens (Chief Executive of NHS England) and Prime Minister, David Cameron. The letters and responses are all posted below.

The letter from Professor Sean Duffy confirms that the new model for the evaluation of drugs, including cancer drugs, following the Accelerated Access Review’ s report will be launched on April 1st 2016. Not good enough. I want to make this clear. We currently don’t have a method of evaluating new drugs and the old method was flawed (see the issues ponatinib had with small population numbers). This gap in service and the delisting of life-saving drugs is going to kill people.

I’m making this extra clear because a politician I’ve spoken to recently questioned me, quite ferociously, on the launch date of the new model. I know that this politician subscribes to my blog updates and I hope that they now have all of the facts they need to do something about the issue and register their protest.

A huge thanks to David Ryner from CML Support for coordinating this activity.

Kris Griffin

LETTER TO SIMON STEVENS

Dear Mr Stevens,

We are writing to you regarding the recent announcements relating to the Cancer Drugs Fund (CDF) and the specific decisions that have been taken on treatments for chronic myeloid leukaemia (CML).

As patient groups representing the concerns of patients with CML, we are particularly alarmed by both the inclusion of CML treatments in the next review of the CDF for delisting at the end of this month, and by the suggestion that there may not be any further meetings of the CDF panel to consider new treatments or indications for the remainder of the 2015/16 financial year.

In particular, I would like to draw your attention to the situation currently facing two medicines which treat patients with more advanced CML, who therefore face severely limited treatment options. Bosutinib is scheduled for review at the next meeting of the CDF panel at the end of this month. Ponatinib, a drug which has never been appraised by NICE due to its small patient population, is only available through the CDF for patients with the T315i mutation, rather than in its full licensed indication. Ponatinib was due to be assessed by the CDF panel for its full licence in June, before the cancellation of the last scheduled meeting.

This situation for those patients needing access to ponatinib is particularly acute; with NHS England’s Commissioning Intentions for 2015/16 committing to producing algorithms for all chemotherapy within the year, ponatinib now faces the prospect of being effectively excluded from the CML algorithm entirely, with the exception of the T35i mutation.

When the threat to bosutinib is factored in and with the exception of the minuscule number exhibiting the T315i mutation, patients in England now face a lack of access to two of the five drugs that are currently available to them. The clinical effectiveness of these drugs in being able to secure optimal responses at speed and scale relative to the current entry level CML inhibitor, imatinib, now over a decade old, is well established.

The Government’s Accelerated Access Review (AAR) demonstrates a welcome recognition that current evaluation processes require revision to ensure they are fit for purpose in assessing the new generation of innovative products, including targeted therapies for CML. We recognise, too, that the CDF needs to adapt its processes to remain in step with the wider Government agenda.

It is therefore bewildering, contradictory and illogical for NHS England’s real time activity to be moving in the opposite direction of travel in reversing, rather than accelerating, access to targeted therapies for CML. This is made even more remarkable given the fact that, relative to other CDF list treatments, the performance of this class of drugs has been considered outstanding when judged against standard measures of survival. As a result, the overwhelming majority of patients are now able to secure decades of benefit from these home-based oral therapies, with their lives returning to near normal (and patients enjoying near-normal life expectancy) following treatment.

Such marked improvements in CML patient outcomes have been achieved by the steady increase in targeted therapies. We believe that to withdraw the opportunity from patients who would benefit from targeted CML therapies such as ponatinib and bosutinib is both discriminatory and perverse and we would strongly urge you to reconsider this decision by NHS England.

Yours sincerely,

xxxx

cc. Rt Hon Jeremy Hunt MP

RESPONSES

from the Department of Health (Malcolm Jones)

from NHS England (Professor Sean Duffy)

LETTER TO RT HON DAVID CAMERON

Dear Prime Minister,

We are writing to you following the intervention you recently made to NHS England regarding its consideration of the funding of medicines for a number of rarer diseases, to make you aware of the situation patients with chronic myeloid leukaemia (CML), a rare form of blood cancer, currently face.

As patient groups representing patients with CML, we were concerned with recent announcements relating to the Cancer Drugs Fund (CDF) and the specific decisions that have been taken regarding treatments for CML. We have great concerns about the fact that the CDF panel will not now consider any new treatments or indications for the remainder of the 2015/16 financial year, meaning new and innovative treatments for CML will remain unavailable to patients, and that CML treatments currently available on the Fund are at risk of being delisted.

CML is treated with targeted therapies which have ensured marked improvements in patient outcomes but mean patient sub populations are small. Patients need to have a wide range of treatment options available to them because of the problem of resistance to medicines, as well as contraindications and co-morbidities which mean some patients are unable to tolerate certain drugs currently within the treatment pathway.

Patients with more advanced CML face severely limited treatment options, with two of the five CML drugs either at-risk or unavailable to all patients who would benefit. Bosutinib, a second-line treatment for CML, is at risk of being delisted from the CDF following its inclusion in the review of current treatments conducted by the CDF panel on the 29th and 30th July. Ponatinib, a drug which has never been appraised by NICE due to its small patient population, is currently only available through the CDF for patients with the T315i mutation, rather than in its full licensed indication. Ponatinib was due to be assessed by the CDF panel for its full licence in June, before the cancellation of the last scheduled meeting, and now has no opportunity to be appraised for clinical and cost effectiveness, meaning the wider CML patient population are unable to access the drug other than through Individual Funding Requests (IFRs).

The clinical effectiveness of both drugs in being able to secure optimal responses at speed and scale relative to the current entry level CML inhibitor, imatinib, now over a decade old, is well established. The following comment from a patient on ponatinib, which was used in the CML Support Group submission to the SMC in Scotland – who approved the drug for its full licence – confirms its clinical effectiveness; “Ponatinib for me represents a quantum leap forward in the treatment of my CML and the impact of this condition on my family and work life. For me, even though I am likely to have to take this for life, ponatinib represents the optimum treatment that I could have expected and hoped for beyond the major trauma and loss of employment that the only other “ total “ cure , a bone marrow transplant, represents.”

We were reassured to read your comments in a letter to the Specialised Healthcare Alliance dated 28th April 2015, in which you stated “I am absolutely committed to ensuring that patients with rare diseases have access to the latest and most effective treatments that represent value to the NHS and deliver benefits to patients.” Any assistance you could offer in ensuring CML patients have access to the full range of effective treatments would be greatly appreciated. In addition, we would be grateful of any clarity you are able to secure on our behalf from NHS England regarding the new system of appraisal – particularly in terms of when the CDF will consider new medicine appraisals, and how medicines for rarer cancers and those with small patient populations will fit into the new system of evaluation – which will replace the current CDF when it ends in March 2016.

Yours sincerely,

RESPONSE

from 10 Downing Street (Ed Whiting)

Update: MPs debate availability of cancer medicines

Following my previous blog post about Tuesday’s Westminster Hall Debate on the availability of cancer medicines, here’s an update on what happened.

A number of MPs from different political parties participated in the debate, sought by Jim Shannon, an MP committed to improving patient access to cancer drugs. This included two MPs I am scheduled to meet with on my next Westminster day in November, Mark Durkan and Nic Dakin, so it’s great to see they already have a good grasp of the issues we face.

Other MPs contributing to the debate included:

• Philip Hollobone, Conservative MP for Kettering
• Margaret Ritchie, SDLP MP for South Down
• Lady Hermon, Independent MP for North Down
• David Simpson, DUP MP for Upper Bann
• Gavin Robinson, DUP MP for Belfast East
• Sir Oliver Heald QC MP, Conservative MP for North East Hertfordshire
• Nick Thomas-Symonds, Labour MP for Torfaen
• Andrew Smith, Labour MP for Oxford East
• Andrew Gwynne, Labour MP for Denton and Reddish
• Marion Fellows, SNP MP for Motherwell and Wishaw
• Mark Tami, Labour MP for Alyn and Deeside

The debate focussed on the general inequity of access to cancer medicines which patients face, and the recent delisting of drugs from the Cancer Drugs Fund (which included a number of drugs for CML). Jim Shannon highlighted the need to develop a long-term, sustainable solution to the issue of the evaluation and funding of cancer medicines, in order to benefit patients.

Life Sciences Minister, George Freeman MP, responded to the debate on behalf on the Government and cited the commitment NHS England has made to implementing the Cancer Taskforce recommendations, which is encouraging. His view was very much that if NICE has recommended a medicine then NHS England has a duty to fund it, which brings into question drugs which NICE has chosen not to even consider appraising due to small patient numbers – such as ponatinib for CML and ALL. He also mentioned the Government’s Accelerated Access Review (AAR), which was due to report this September, but has now been postponed until 2016. Time will tell if the recommendations of the AAR live up to the Minister’s promises but an accelerated scheme that has consistently been delayed doesn’t fill me with great confidence.

Many readers of this blog sent me messages when we announced this Westminster Hall debate, they had written to their MP telling them about it and asking them to attend. If you did this and the name of your MP isn’t on the attendee list above please write to them again and ask why they didn’t attend; urge them to look into the issues surrounding the delisting of drugs from the Cancer Drugs Fund. I’d be happy to speak to them on your behalf or perhaps we can meet them together. It’s vitally important that as a small community we have a voice and are listened to.

Thanks, Kris

28/10/15 – ADDITION – you can watch a recording of the debate on Parliament Live TV here: http://parliamentlive.tv/event/index/6c473be6-564a-43d2-92e8-65aa6cec067d?in=09:30:00

MPs to debate the availability of cancer drugs in Parliament

I am pleased to announce that next Tuesday, 20th October, MPs will take part in a Westminster Hall Debate on the availability of cancer drugs. This has been sought by DUP MP Jim Shannon, a big advocate of cancer patients, following his attendance at a dinner to discuss access to medicines for blood and rarer cancers earlier in the year. The debate will give MPs the opportunity to discuss the significant problems many cancer patients in England are currently facing in terms of gaining access to the medicines they need.

You will have read about the fight I have taken up to help 43 year old Birmingham father of four, Khalid Younis, gain access to ponatinib for his resistant Chronic Myeloid Leukaemia (CML), including the meeting we had last week with his local MP, Roger Godsiff. I have alerted Roger to the fact that this debate is happening and asked if he can raise Khalid’s case with the Minister to see if we can progress the situation further with the Government and NHS England. In addition, an Access CML Drugs briefing has gone out to a number of MPs I’ve met before, or who have an interest in CML, stem cell transplantation or the ending of the Cancer Drugs Fund. The briefing highlights our key issues and the inequity of access English patients currently face compared to those in Scotland and Wales. You can download the briefing below.

Whilst the debate won’t focus solely on CML or blood cancer, I hope Khalid’s story will form part of the discussion and be used to demonstrate the problems which exist with the current evaluation methods used for cancer medicines in England, particularly with the Cancer Drugs Fund (CDF) refusing to appraise any new medicines between now and when the Fund ends in March 2016. I hope the issue of how the National Institute for Health and Care Excellence (NICE) and the CDF work (or don’t!) together will also be discussed; any future system of evaluation for cancer medicines needs to be able to work for cancers with small patient populations and previous drugs have, sadly, fallen down the gap between NICE and the CDF.

I would encourage everyone to contact their local MP and ask them to attend and contribute to the debate. You can find out who your local MP is here. The more MPs that attend, the greater the level of awareness there will be amongst parliamentarians of the issues we face.

I will post again following the debate.

Kris Griffin – founder of Access CML Drugs

ACCESS CML DRUGS – WESTMINSTER HALL DEBATE – BRIEFING

Birmingham Mail story – Leukaemia Man – NOT EXCEPTIONAL!

This is possibly the most important post I’ve carried to date. It compounds all of our fears about our CML drugs and exposes NHS England bureaucracy as a cruel, unsympathetic, misguided fool. This is the story of a father-of-four, who lives in England, and has received the devastating news that he does not qualify for Ponatinib, a treatment freely available in Scotland and Wales. This drug is his last chance and he’s been told that his case is “not exceptional.” Thank you to the Birmingham Mail and to reporter Alison Stacey for covering this and for contacting me and asking for my comment.

This is not an isolated incident and I am in touch with another gentleman who is in a similar position who has been told that he’s not exceptional either, I’ll be writing about him next week.

This, in a city where we have one of the finest blood cancer charities in Europe, Cure Leukaemia, desperately raising money to save lives. Khalid attends the same hospital as me, the QE in Birmingham; I can only imagine their despair at not being able to give him the drugs that he needs.

And the response from NHS England: “NHS England and NICE will shortly be consulting on a proposed new system for commissioning cancer drugs…” SHORTLY!! Written by people who don’t realise the consequence of their actions – cancelled reviews, cancelled meetings and a new process that only exists as a blank sheet of paper. All this whilst people die. If Khalid lived in Scotland or Wales he’d be taking Potaninib right now; afforded another lifeline.

We’re putting a patient pack together right now to help you campaign but in the meantime share the blog post, share the original article and show your support for Khalid. NHS England have to hand the keys to the medicine cabinet over, this is not about extending lives, it’s about saving them – to refuse to do is a death sentence.

Kris Griffin

Click the headline to take you to the original article.

Kings Heath dad denied ‘wonder’ cancer drug and told his case is ‘not exceptional’

Father-of-four Khalid Younis, 43, does not qualify for Ponatinib because of postcode lottery

A dying Birmingham dad has been denied a potentially life-saving cancer drug by the NHS in a postcode lottery scandal – and told his case is ‘not exceptional’.

Father-of-four Khalid Younis, from Kings Heath, has received the devastating news that he does not qualify for Ponatinib, a treatment freely available in Scotland and Wales.

The new ‘wonder’ drug is the only treatment left for the 43-year-old who is battling Chronic Myleoid Leukaemia (CML), as his body has become resistant to all other medicines.

Former carpet fitter Khalid, a patient at Birmingham’s Queen Elizabeth Hospital, said: “They say I’m not exceptional, but talk to my Mum, talk to my kids, they’ll tell you I’m exceptional.

“It seems crazy. I have even considered moving to Wales so that I can get the treatment, but I worry about putting my family through it.

“We are in a very sad, vulnerable and stressful situation.”

Khalid’s case comes just days after NHS England announced it is cutting 16 drugs from its Cancer Drugs Fund after overspending by £70 million.

For Khalid the postcode cancer lottery seems desperately unfair as living in England means his survival odds have been drastically reduced.

As he is unable to have a stem cell transplant due to a lung condition, the drug would have been his last shot at beating the leukaemia.

“In a way it’s more painful to know that there’s something out there that could treat me, but I just can’t get Ponatinib,’’ said Khalid.

“This is England, the most beautiful country in the world. They can spend millions on Wembley Stadium, but when it comes to a Dad’s cancer treatment there is not enough money.

“I am truly blessed that I have my amazing family and friends around me. I worry that there must be people out there going through the same thing as me, but on their own.”

Patient advocate Kris Griffin explained this in not an uncommon story for CML patients, as NHS England restricts the amount of drugs patients can access.

“We are not talking about extending his life for a few months so he can prepare to say goodbye,” said Kris.

“We talk about finding a cure for cancer and one comes along we say it is too expensive. It’s insane.

“How is a man not ‘exceptionable’, when he has no other option? This could save his life, and to refuse him is inexcusable.”

A spokesman for NHS England said: “We have every sympathy with anyone in this position.

“NHS England and NICE will shortly be consulting on a proposed new system for commissioning cancer drugs. The new system will be designed to provide the NHS with a more systematic approach to getting the best price for cancer drugs, meaning more treatments can be made available, and give a greater focus on evaluation, leading to the best drugs progressing swiftly to routine commissioning.”
21:00, 16 SEPTEMBER 2015
BY ALISON STACEY

Khalid Younis (Birmingham Mail)

Cancer Drugs Fund Cuts CML Drugs (and what we can do about it)

Details from the recent announcement:

The Cancer Drugs Fund in England will no longer pay for 16 medicines, used in 23 separate cancer treatments.
All the drugs on the Cancer Drugs Fund list have been rejected by the NHS as a whole because they do not provide enough benefit for the amount they cost.
At the beginning of 2015, there were 84 funded therapies, but after a series of culls there are now just 41.
The fund was set up by Prime Minister David Cameron to provide access to such medication. However, NHS England announced that the fund was due to go £100m over budget in 2014-15.
The drugs will be formally removed on 4 November and the announcement will not affect patients currently receiving treatment through the fund.
Patients affected: Blood cancer – 1,759 patients.
The Rarer Cancers Foundation said the news was a “hammer blow” and estimated that 5,500 patients across a spectrum of cancers would miss out.

Source: Cancer drugs fund cuts 23 treatments from BBC News.
The official announcement from NHS England can be found here.

Before I start it is imperative to start that the announcement will not affect patients currently receiving treatment through the fund.

I’ve read a lot of things over the last few days. I’ve heard many opinions and chewed a lot of fat. Any way you look at this recent decision, it’s hard to take any positives from it; that’s clearly why emotions are running so high. But, let’s remember what Yoda taught us:

“Fear is the path to the dark side. Fear leads to anger. Anger leads to hate. Hate leads to suffering.“

This is not a situation for finger-pointing or blaming people, countries or administrations we perceive to be at fault or guilty for a variety of suspected sins. One of the silliest suggestions I’ve read is that if that if we weren’t talking so many refugees in to the UK we would be able to afford the CML drugs. Not the case. Health economics doesn’t work like this. I’m not a fan of the Trident programme but I’m not daft enough to think that by scrapping it and saving billions we’d immediately be rewarded with the drugs we need. No, it’s more likely we’d get another station for High Speed 2. Joke. And for the record, I’m in favour of the UK playing our part and taking refugees.

We stand alone on this, fight our corner strategically and productively and make sure our voices are heard. Do I believe that campaigning hard will result in a reversal of this decision? No. But if we allow our voices to fall silent, when the day comes to start appraising drugs again, I want CML drugs to be at the front of people’s minds. I want people to understand that this is a poor decision about drugs that SAVE LIVES. I want the people responsible for the decisions to know that we are NOT faceless. I want them to know our names.

So what should we do? I believe there are two fundamental priorities to focus on:

1. To pressure the health administration groups in England to review decisions, open the appraisal process and ensure that we are part of the process moving forward – with respect to the reconfigured way of deciding which drugs to approve and which to reject.

2. To encourage pharmaceutical companies who manufacture our drugs to reduce their prices through Patient Access Schemes (PAS).

By playing this straight down the middle we position ourselves as the result of both health administration AND pharmaceutical company decisions. The decisions are unfair and unjust but that argument won’t win us any battles. A coordinated, strategic approach will. This means responding to requests for help with media enquiries, visiting Parliament to talk to MPs and writing letters to appropriate parties. It also means making yourself a more informed patient, understanding the process and contributing towards any changes. This is the only way we, as patients, will be part of any changes.

We’re doing this for our generation and the generation of patients that follow us. We’re doing this for the person diagnosed tomorrow who currently has fewer drugs available to them than when I was diagnosed 8 years ago. If that isn’t motivation enough to bring about change then I don’t know what is.

Thanks, Kris

Experts call for equal access to CML therapies in the UK

A quick update, excellent article from the Pharma Times. Kris

Medical experts are slamming a decision by NHS cost regulators that restricts use of Ariad’s Iclusig in the treatment of chronic myeloid leukaemia in England while patients in Scotland and Wales get full access to the novel drug.

Iclusig (ponatinib) was cleared in Europe in 2013 as an orphan drug considered to offer a significant benefit to patients. However, it was not appraised by the National Institute for Health and Care Excellence because of its small target population, leaving the decision on access with the Cancer Drugs Fund, which says it can only be given to patients with the rare T315I mutation, representing about 2%-20% of CML patients.

Results of a poll by the National Cancer Research Institute CML Working Group revealed that 83% of respondents support a change in CDF policy so that Iclusig is available to all appropriate patients in England in line with its approved indication, as the current disparity in access and use of treatments is failing patients.

“The CDF uses a different methodology to score therapies; by assessing the Median Total Drug Cost per Patient this effectively penalises therapies that help patients live longer,” said Mark Tanner, General Manager of ARIAD Pharma UK. “Given recent news about the restructuring of the CDF & NICE, we hope that NHS England will address this anomaly and quickly find a mechanism to allow patients in urgent need the same level of access as their neighbours in Scotland and Wales,” he added.

“Treatments should be available on an equal basis to all people with chronic phase CML across the UK; the decision should be medical, not geographical,” added Prof Mhairi Copland, Chair of NCRI CML Working Group and Professor of Translational Haematology, University of Glasgow, while Dragana Milojkovic, Department of Haematology, Imperial College School of Medicine Hammersmith Hospital, London, noted that “restricted use of therapies imposed by the CDF have seriously limited our ability to treat patients effectively, which is a barrier especially when treatments are used according to their full license elsewhere”.

by SELINA MCKEE

Read the original article here: www.pharmatimes.com/Article/15-08-25/Experts_call_for_equal_access_to_CML_therapies_in_the_UK.aspx#ixzz3jqZxTTre

Letter to Simon Stevens – Chief Exec of NHS England

A group of charities and advocates (including me) have come together to put a letter in front of Simon Stevens, the chief executive of NHS England to ask him to consider the situation for two CML drugs: bosutinib and posatinib.

The letter sets out the case for the retention of bosutinib on the list of Cancer Drugs Fund (CDF) drugs for CML patients in England, and also argues for ponatinib to be made available, via the CDF, for any patient in England that falls within its licensed use providing their clinician thinks it appropriate and the patient agrees to treatment. This would allow CML patients resident in England to enjoy the same level of access CML patients in Wales and Scotland currently enjoy.

There was a Written Parliamentary Question answered by a Department of Health Minister on Monday that confirmed rumours that have been circulating for some weeks that there will be no new applications for drugs to enter the national CDF in the future. This would effectively make ponatinib unavailable in England to all clinically qualifying patients who do not have the T315i mutation.

NHS England will of course say a clinician can make an exceptional needs application for ponatinib treatment for an individual patient but the success rate to date of this type of application has been negligible.

Finally, it’s important to stress that all patients in England currently being treated by bosutinib or ponatinib via a successful application to the CDF will continue to be able to be treated with either drug until they or their clinician decides otherwise.

I’ve posted the content of the letter below and have also included a download to the PDF. It has also been sent to the Secretary of State for Health. Many thanks to David Ryner at CML Support for spearheading this action.

I’ll keep you posted with any progress. Thanks, Kris

Dear Mr Stevens,

We are writing to you regarding the recent announcements relating to the Cancer Drugs Fund (CDF) and the specific decisions that have been taken on treatments for chronic myeloid leukaemia (CML).

As patient groups representing the concerns of patients with CML, we are particularly alarmed by both the inclusion of CML treatments in the next review of the CDF for delisting at the end of this month, and by the suggestion that there may not be any further meetings of the GDF panel to consider new treatments or indications for the remainder of the 2015/16 financial year.

In particular, I would like to draw your attention to the situation currently facing two medicines which treat patients with more advanced CML, who therefore face severely limited treatment options. Bosutinib is scheduled for review at the next meeting of the CDF panel at the end of this month. Ponatinib, a drug which has never been appraised by NICE due to its small patient population, is only available through the GDF for patients with the T315i mutation, rather than in its full licensed indi. cation. Ponatinib was due to be assessed by the CDF panel for its full licence in June, before the cancellation of the last scheduled meeting.

This situation for those patients needing access to ponatinib is particularly acute; with NHS England’s Commissioning Intentions for 2015/16 committing to producing algorithms for all chemotherapy within the year, ponatinib now faces the prospect of being effectively excluded from the CML algorithm entirely, with the exception of the T35i mutation.

When the threat to bosutinib is factored in and with the exception of the minuscule number exhibiting the T315i mutation, patients in England now face a lack of access to two of the five drugs that are currently available to them. The clinical effectiveness of these drugs in being able to secure optimal responses at speed and scale relative to the current entry level CML inhibitor, imatinib, now over a decade old, is well established.

The Government’s Accelerated Access Review (AAR) demonstrates a welcome recognition that current evaluation processes require revision to ensure they are fit for purpose in assessing the new generation of innovative products, including targeted therapies for CML. We recognise, too, that the CDF needs to adapt its processes to remain in step with the wider Government agenda.

It is therefore bewildering, contradictory and illogical for NHS England’s real time activity to be moving in the opposite direction of travel in reversing, rather than accelerating, access to targeted therapies for CML. This is made even more remarkable given the fact that, relative to other CDF list treatments, the performance of this class of drugs has been considered outstanding when judged against standard measures of survival. As a result, the overwhelming majority of patients are now able to secure decades of benefit from these home-based oral therapies, with their lives returning to near normal (and patients enjoying near-normal life expectancy) following treatment.

Such marked improvements in CML patient outcomes have been achieved by the steady increase in targeted therapies. We believe that to withdraw the opportunity from patients who would benefit from targeted CML therapies such as ponatinib and bosutinib is both discriminatory and perverse and we would strongly urge you to reconsider this decision by NHS England.

cc. Rt Hon Jeremy Hunt MP

Yours sincerely,

David Ryner, Chair, Chronic Myeloid Leukaemia Support Group
Chris West, Head of Media and Public Affairs, Leukaemia & Lymphoma Research
Tony Gavin, Director of Campaigns & Advocacy, Leukaemia Care
Nigel Deekes, CML UK
Kris Griffin, UK CML blogger

Simon Stevens Letter PDF

Jeremy Hunt Letter PDF

Westminster Meetings: Maggie Throup MP and Kevin Foster MP

I recently spent another day meeting with MPs in Westminster to talk about chronic myeloid leukaemia and seek their support in helping CML patients access effective medicines. The meetings were with Maggie Throup and Kevin Foster, both newly elected Conservative Members of Parliament who have a keen interest in health matters.

Maggie, who has a background in blood testing having previously worked in pathology labs, had just been to her first Health Select Committee meeting as she has been elected to serve on the group for this parliament. Kevin was on his way to an introductory meeting with ministers at the Department of Health when we met, so both meetings were very timely.

I explained what it means to have CML, the different phases of the disease, and the importance of having a range of medicines available for patients who become resistant or cannot tolerate a particular drug. I updated Maggie and Kevin on the campaigning work I have been doing to improve access for patients, and the difficulties CML patients in England are facing in terms of accessing ponatinib, which is available in Wales in Scotland, as well as the threat of delisting to bosutinib which is currently available through the Cancer Drugs Fund.

Both MPs were sympathetic to the situation, and the meetings will only serve to reinforce the need for more to be done to improve the current system and ensure patients have the best range of treatment options available to them. Once the MPs return after their summer recess I hope to have more parliamentary meetings to continue to raise awareness of the key issues for CML. By then we will know the outcome of the CDF delisting meetings which are currently taking place . It’s vital we keep the pressure on so that the decision makers know we need a range of effective drugs to treat CML, and that no patients should be missing out.

I’m always very appreciative to any MP who spends a little time with me to learn more about CML and wish to send thanks to Maggie and Kevin for their time and understanding.

Kris Griffin

Kevin Foster MP and Kris Griffin

Maggie Throup MP and Kris Griffin

Ponatinib: Scotland 1, Wales 1, England 0

In recent news I reported that Iclusig® (ponatinib) had been granted access to patients in Wales by NHS Wales. You can read the whole story here. I’m pleased to be able to write today to say that patients in Scotland have been given access to ponatinib too! Both countries are providing access for ALL phases of chronic myeloid leukaemia (CML).

In England ponatinib is only available on the Cancer Drugs Fund (CDF) IF the patient has the T315i mutation. We recently reported the majority of patients were being turned down in England after making individual funding requests; full story here.

Whilst you can read the full details of the appraisal by NHS Scotland I’ll draw your attention to this:

A non-comparative phase II study of ponatinib was conducted with primary outcomes of major cytogenetic response in patients with baseline chronic phase CML and major haematologic response in patients with baseline accelerated or blast phase CML or Ph+ALL. Ponatinib demonstrated efficacy in heavily pre-treated CML and Ph+ALL patients who had received dasatinib/nilotinib as second line or further line tyrosine kinase inhibitor therapy or who had the T315I mutation.

The studies of ponatinib show it to be very effective and NHS Scotland have recognised this, but NHS England remain steadfast and refuse to appraise ponatinib because the patient numbers are too low. We find ourselves in a situation where patients aren’t able to access a drug that could save their lives. How can the system be fair when, depending on which NHS authority you come under, will depend on the availability of a drug to you?

There is little that can be done at the moment but once Parliament reconvenes we’ll start to apply pressure and ask direct questions of the organisations that are allowing us to fall behind our counterparts in the United Kingdom.

Thanks, Kris

Advice: following a full submission considered under the orphan and end of life process:

ponatinib (Iclusig®) is accepted for use within NHS Scotland.

Indication under review: Adult patients with
• Chronic phase, accelerated phase, or blast phase chronic myeloid leukaemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.
• Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.

A non-comparative phase II study of ponatinib was conducted with primary outcomes of major cytogenetic response in patients with baseline chronic phase CML and major haematologic response in patients with baseline accelerated or blast phase CML or Ph+ALL. Ponatinib demonstrated efficacy in heavily pre-treated CML and Ph+ALL patients who had received dasatinib/nilotinib as second line or further line tyrosine kinase inhibitor therapy or who had the T315I mutation.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.

Taken from: www.scottishmedicines.org.uk/SMC_Advice/Advice/1032_15_ponatinib_Iclusig/ponatinib_Iclusig