Trial: Discontinuation of dasatinib

The results of this trial represent an incredible leap forward for CML patients who, like me, are on dasatinib (sprycel). For the patients in this trial, nearly 50% who stopped dasatinib maintained a deep molecular response. The other 50% started taking tablets again and all regained a deep molecular response.

This represents a huge benefit for the patient who could, effectively, remain drug-free but it also represent an economic benefit. What was once considered an expensive drug could soon be considered a drug-for-life for only half of the patients who take it. This could be enough to present a new case to NHS England over funding.

Thanks, Kris

Taken from The Lancet haematology

Summary
Background
First-line imatinib treatment can be successfully discontinued in patients with chronic myeloid leukaemia after deep molecular response has been sustained for at least 2 years. We investigated the safety and efficacy of discontinuing second-line or subsequent dasatinib after at least 1 year of deep molecular response.

Methods
The Dasatinib Discontinuation trial was a prospective multicentre trial done in Japan. Eligible patients taking dasatinib and with confirmed stable deep molecular response were enrolled between April 1, 2011, and March 31, 2012. All patients received dasatinib consolidation therapy for at least 1 year. In those with sustained deep molecular response, dasatinib was discontinued. Patients were followed up every month in year 1 (clinical cutoff), every 3 months in year 2, and every 6 months in year 3 for deep molecular response and immunological profiles. The primary endpoint was the proportion of patients with treatment-free remission at 6 months after discontinuation. Molecular relapse was defined as loss of deep molecular response at any assessment. This study is registered, number UMIN000005130.

Findings
88 patients were enrolled in the consolidation phase, 24 were excluded from the discontinuation phase due to fluctuations in BCR-ABL1 transcript levels. One patient was excluded because of positive expression of major and minor BCR-ABL1 transcripts in chronic myeloid leukaemia cells and the detection of minor BCR-ABL1 transcripts during consolidation. Thus, 63 patients discontinued dasatinib treatment. The 25 patients who were excluded from discontinuation continued to receive dasatinib and none showed disease progression. Median follow-up was 20·0 months (IQR 16·5–24·0). Of the 63 patients who discontinued and were not excluded, 30 patients maintained deep molecular response while 33 patients had molecular relapses, all within the first 7 months after discontinuation. The estimated overall treatment-free remission was 49% (95% CI 36–61) at 6 months. No severe treatment-related toxic effects were seen. Treatment was restarted in the 33 patients with relapse; rapid molecular responses were seen in all 33 patients, of whom 29 (88%) regained deep molecular response within 3 months, as did the remaining four by 6 months.

Interpretation
Dasatinib discontinuation after sustained deep molecular response for more than 1 year is feasible.

Funding
Epidemiological and Clinical Research Information Network (ECRIN).

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

A Ode To The Man From Derby

I’ve spent an inordinate amount of time pontificating whether I should post this. It’s whimsy and self-indulgent, it isn’t going to become a new category; I don’t see a huge demand for blood cancer poetry, but this is part of my story and I am grateful for my monthly tablet delivery.

Hopefully you’ll find this funny or amusing and that you’ll forgive the interruption from more serious matters. Thanks, Kris

An Ode To The Man from Derby
Once a month, on a Friday; a visit from a man from Derby.
He brings me tablets that keep me alive; we have a chat, on my drive.
How’s the weather, traffic, van? Small-talk with this superb man.
We never talk about health or me; he delivers the cancer medicine you see.
He fears the answer, it’s in his eyes. If only he’d ask, big surprise.
I’m feeling good and doing well; Docs are pleased with my blood cells.
And then he’s gone, my knight from BUPA to guarantee someone else’s future.
What he’s not aware of though; he’s part of the team that keeps me on-the-go.
So thank you Derby-man, I love you very much; perhaps one day I’ll tell you as much.
But until then let’s keep our love secret and keep your visits about my treatment.
Joking aside, thank you my friend; my life comes in a box; on which you I depend.
KG

Kris Griffin from Access CML Drugs

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

Ponatinib Access: Denied

I recently sent in a Freedom of Information request to NHS England to find out how many patients in England had requested ponatinib for chronic myeloid leukaemia and who would not already be entitled to it on the NHS. Currently only patients with the T315l mutation are able to have the drug prescribed by their doctor, with other patients who want the drug having to get a clinician to make a special request (an Individual Funding Request, or IFR) to the Cancer Drugs Fund, which NHS England runs.

I was shocked by the response to my query, that of the 14 patients who requested ponatinib (from April 2013 to March 2015), just 2 of them were granted access to the drug and the other 12 were denied. It seems short-sighted of NHS England not to allow patients access to a drug which could benefit them when others have stopped working, and when the only other option is often a stem cell transplant.

With such small patient numbers NICE won’t even consider appraising ponatinib, the CDF is supposed to act as a support system for patients to access drugs for rarer cancers, but the system clearly currently isn’t working.

Patients in England are again missing out compared to their counterparts in Wales, where the drug is fully approved for all CML patients.

This excellent graphic clearly shows that in the ponatinib PACE trial, patients benefited from ponatinib after they had failed other TKIs at various stages of disease progression.

University of York – not helpful

I’ve been highly critical of NICE (National Institute for Health and Care Excellence) in the past but I’ll give credit where it’s due. In this BBC report University of York researchers suggest the Cancer Drugs Fund (CDF) is particularly poor value, diverting money from other patient services. They argue the drugs advice body, NICE, has set its price threshold too high.

Researchers at York say the funding level should be closer to £13,000 to provide the most benefit across the NHS instead of a £20,000 to £30,000 limit that NICE currently work with. Thankfully Sir Andrew Dillon, chief executive of NICE, said: “Unless you think that drug companies will be prepared to lower their prices in an unprecedented way, using a threshold of £13,000 per QALY would mean the NHS closing the door on most new treatments.” I applaud his position on this occasion.

What the researchers at the University of York have failed to take into account is the cost to develop a drug can run to hundreds of millions of pounds and someone has to pay for this. I’m not a defender of pharma and I still believe pharma should do more to make drugs more affordable but this type of scaremongering is unhelpful. We need to work with pharma and not impose dangerous limits on treatment. Readers of this blog will know that the new CML treatments which cost much more than £13,000 per year are, essentially, curing people with Chronic Myeloid Leukaemia. Who knows where these advances will take us, impose limits and we halt progress.

I need to read the report in its entirety but from this overview it appears the conclusions from York aren’t helpful, aren’t clever and don’t take into account the future of cancer treatment. Their recommendations would mean people would die. Perhaps if someone from York is reading this they would get in touch, perhaps we could meet up and perhaps they could tell me just how much my life is worth. I’d also like to ask them what price they put on a cure for cancer and if their report took this into account?

Kris Griffin
Access CML Drugs

BBC Health: NICE ‘sets price too high for NHS medicines’

On World CML Day Leukaemia Patients Worldwide Unite

Taken from www.cmladvocates.net – an important hub of CML advocate activity:

On World CML Day 9/22, leukaemia patients worldwide unite to address the courage and hope required to live with chronic myeloid leukaemia, and call for access to best available treatment and care for all patients 

 

Group by group, advocate by advocate, and survivor by survivor: on World CML Day on 22 September, the global patient community is raising awareness about the needs of people living with chronic myeloid leukaemia (CML). Simultaneous events, publications and meetings on all continents put a spotlight on the real needs of patients and their relatives, and demonstrate the power and unity of a unique worldwide community that knows no borders. Their brand-new book “Faces of Courage and Hope” further provides an insight into the journeys of 16 CML patients in 15 countries.  

 

Members of the CML Advocates Network, which is today comprised of 90 patient advocacy groups in 68 countries, are honouring World CML Day on September 22nd 2014 through actionable events taking place in their communities. All events worldwide aim to underscore the importance of the needs of people living with CML. The needs of patients in different regions vary largely – from basic challenges of access to treatment and care, over uncertainty on the quality of treatments, to living with cancer as a chronic disease.
 
Although CML is today often referred to as a model disease how blocking essential mechanisms of a tumour can bring cancer to a halt, not all that glitters is gold. Even though there has been a medical revolution in treating CML over the past decade, not every patient has access to best available treatment and care, and many patients struggle to live with cancer as a chronic, life-long disease.

 

There are obvious disparities in terms of access to treatment and diagnostics. We cannot turn our eyes from the fact that socio-economic barriers are present in many countries, where patients are lost in debates around high drug prices and drug quality of copy drugs, combined with the lack of access to quality diagnostics. Just in May 2014, CML patients worldwide called for quality and consistency when new drugs are introduced to treat their cancer. Since none of the new drugs provide for a cure of CML yet, even in countries where access to expensive cancer treatments is ensured, patients are facing challenges with the impact of a life-long treatment on their quality of life. The community’s goal is clear: All CML patients shall have access to optimal and affordable treatment and care, giving them a quality of life as close as possible to normality. There is still a long way to go.

 

Key initiatives on World CML Day 2014

Again in 2014, plenty of events are being carried out by patient organizations throughout the world in order to raise awareness of the needs of people living with CML worldwide. The initiatives are listed on the CML Advocates Network website at  http://www.cmladvocates.net/worldcmlday2014/worldcmlday2014/initiatives
 
On World CML Day 2014, the CML Advocates Network publishes an English-language book Faces of Courage and Hope that illustrates the faces and cancer journeys of 16 CML patients from 15 countries. Men and women of different ages, ethnicities and backgrounds, each share their unique experience in coping with life-altering circumstances and how they refocused their lives. The book has been shipped to CML patient organizations in 56 countries, and will be available on Amazon.co.uk for worldwide shipping from 22 September 2014. For more information see: http://www.cmladvocates.net/book-faces-of-courage-and-hope

On 9/22, the CML Advocates Network also publishes a patient-friendly summary of the European LeukemiaNET’s CML Treatment Recommendations in English, Italian, French, German, Hebrew, Dutch, Russian, Polish, Spanish, Portuguese and Arabic. The summary will allow patients to understand the consensus recommendations provided by a global group of CML experts, coordinated by the European LeukemiaNET.

In addition, the CML Advocates Network provided buttons with the community’s World CML Day logo – a total of 35,000 ‘World CML Day’ buttons were distributed to organisations in 34 countries on all continents to raise awareness about CML.

22 September – the global date for awareness about CML

The date chosen for World CML Day (9/22) adds symbolic significance to the campaigns of the patient community: World CML Day is honoured on September 22nd because 9/22 represents the genetic change of Chromosomes 9 and 22 that is the cause of Chronic Myeloid Leukemia (CML), a rare cancer that is characterised by an unregulated growth of white blood cells. Just about 15 years ago, CML was a lethal disease for the majority of patients. Nowadays, CML is no longer a death sentence. If patients are treated effectively with recommended treatments, they can have a life expectancy similar to that of the general population.

 

Since 2008, patient organisations around the world have initiated events and projects on World CML Day to raise awareness of the needs of patients living with CML and to sensitize not only the public, but also politicians and medical professionals regarding CML. “All united, all unique!” is the theme that brings the groups together. It is a reminder of how each person and each group experiencing a rare cancer will have their own journey. As Jan Geissler, CML patient and co-founder of the CML Advocates Network shares, “Since the first meeting of a handful of CML groups back in 2003, it is impressive to see the growth in intensity of activity in the CML patient community. Today 90 groups are members of the worldwide network. We are not only meeting once in a year at our CML Horizons conference – we are supporting each other across borders, and are jointly collaborating with all stakeholders, throughout the year. Working together is the key to improving support and care for CML patients in all regions.”

 

Thanks to all the groups around the world participating in World CML Day, together we are, “All united, all unique!”

 

About the CML Advocates Network

The CML Advocates Network is a patient-run network and platform for patient organisations supporting patients and relatives affected by Chronic Myeloid Leukemia (CML). Launched in 2007 by four founding patient organizations, it has now grown to a network of 90 leukaemia patient groups in 68 countries, offering support to patient leaders by sharing of best practice, information and advice for CML patient leaders. Individual patient support as well as country-specific policy work is being covered by its member organisations on the country level. The CML Advocates Network is hosted by the patient-driven, non-profit Leukaemia Patient Advocates Foundation in Bern, Switzerland.

 

Members of the CML Advocates Network 

See the list of all 90 member organizations here: http://www.cmladvocates.net/cml-groups-list

World CML Day

US health chief becomes new NHS boss…

The appointment of the “US health chief” as the new NHS boss worries me too. The following article appeared on the BBC last week:

A senior executive at a private US health firm has been appointed to lead NHS England – the most powerful body in the health service. Simon Stevens, who has worked as an NHS manager and acted as a health adviser to Labour, will take over from Sir David Nicholson in April.
http://www.bbc.co.uk/news/health-24635890

I’d echo the comments from the Unite representative – was there really nobody in the current NHS structure or the Third Sector capable of doing this job? I believe that there are many people in government who would like to see the privatisation of the NHS, in some areas we have already seen this happen. These American medical values are worrying and represent a polar opposite to a service that should be free at the point of delivery. if we need reminding of those guiding principles again:

Aneurin Bevan, on July 5 1948, the NHS was based on three core principles:

• that it meet the needs of everyone
• that it be free at the point of delivery
• that it be based on clinical need, not ability to pay

These three principles have guided the development of the NHS over more than 60 years and remain at its core.
http://www.nhs.uk/NHSEngland/thenhs/about/Pages/nhscoreprinciples.aspx 

I hope that I’m being cynical again, after-all what type of barbaric society would allow medical care to hinge on a person’s wealth?

Cartoon by http://www.christopherkeelty.com

What is MY PCR survey…

A survey worth spreading…Kris

Dear PCR Campaign Supporters,

As many of you know, 9/22 was World CML Day, the date that symbolically represents the genetic change of Chromosomes 9 and 22 that causes CML. This also marks the one year anniversary of the “What is MY PCR” campaign. In honor of these two events, we are excited to announce the official launch of the PCR Campaign Impact Survey!

Our goal is to gather feedback from you as campaign participants to help us measure our impact and identify areas in which we can strengthen the campaign in 2014. Your voice is critical to shaping this campaign, and we want to hear your feedback! The survey is posted on the campaign website http://www.whatismypcr.org and we encourage you to share this opportunity within your networks as well.

The survey will be open from September 2013 to December 2013. At the conclusion of the survey, we will compile the results into a report in early 2014 to be shared with you and your networks. Currently available in English, Spanish and Thai, we will continue to add translations over the next two months as we receive them from the PCR campaign partners.

We look forward to receiving your feedback. Complete the online survey now!

http://www.surveymonkey.com/s/WhatIsMyPCRImpact

Best wishes for World CML Day,
From PCR Campaign Headquarters