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)

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

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

Front page of the Independent: Cost of CML Drugs

Well, look here. Front page of The Independent yesterday with “the news” that CML drug prices are way too high. It’s something we’ve known for ages and it’s fantastic that the damaging price of these drugs is being brought to public attention.
I think it is important that we consider the other side of the argument for a moment though, and this is not an attempt to justify the prices and defend the pharmas. These drugs are saving lives and, in some cases, curing a form of cancer. What price on that? These drugs have revolutionised CML treatment and have certainly kept me alive. Do I care about the price? No. I care that they’ve saved my life and it helps that I live in the UK and don’t have to find that money. Do I care that there are people on this planet dying from CML. YES – and this is the issue.   Morally these pharmaceutical companies have an obligation to service each territory to ensure these drugs are available at an affordable price. There are plenty of opportunities to cover research costs but there is bigger picture stuff here…even bigger than the shareholders of these companies. Let’s not use these prices as a stick to beat the industry with, after-all they’ve come up with something truly amazing that is going to change the face of healthcare forever. Instead let’s appeal to their common decency, their moral compass and put the pressure on them to deliver a fair solution. This applies to you too BMS. Your failure to provide a Patient Access Scheme in the England has meant the NHS has not been able to deliver dasatinib as a standard treatment.
What a fantastic opportunity for people to do the right thing. Will it happen? Probably not but I must have faith in the human condition. We can put pressure on the pharmaceuticals,  we can write, ask the awkward questions and make sure they know every time a CML patient dies because they can’t afford the treatment they need. Let’s be brave about this, we thank the industry with all our heart, but the time has come to be bigger than the bottom line.  Kris

The wounded surgeon plies the steel
That questions the distempered part;
Beneath the bleeding hands we feel
The sharp compassion of the healer’s art
Resolving the enigma of the fever chart.
T.S. Eliot (1888-1965) – Four Quartets ‘East Coker’ (1940) pt. 4

The real cancer killer: rip-off prices for drugs set by ‘profiteering’ Big Pharma giants

An influential group of cancer experts has warned that the high prices charged by pharmaceutical companies for cancer drugs are effectively condemning patients to death.

The group of more than 100 leading cancer physicians from around the world, including nine from the UK, accuse the drug industry of “profiteering” – making a profit by unethical methods such as by raising the cost of grain after a natural disaster.

Of the 12 drugs approved by the Food and Drug Administration in the US in 2012, 11 were priced above $100,000 (£65,000) per patient per year. In addition the price of existing drugs of proven effectiveness has been increased by up to threefold.

The specialists say: “What determines a morally justifiable ‘just price’ for a cancer drug? A reasonable drug price  should maintain healthy pharmaceutical industry profits without being viewed as ‘profiteering’. This term [profiteering] may apply to the trend of high drug prices where a life threatening medical condition is the disaster.”

The high prices mean the drugs may not be approved by the National Institute for Clinical Excellence in the UK forcing doctors to fill in a 14 page application apply to the Cancer Drugs fund for British patients who could benefit from them.

In addition, the rising cost of existing drugs in a cash limited health service such as the NHS means treatment is denied to other patients with other conditions.

The authors of the article, published in the journal Blood, are all specialists in blood cancers such as leukaemia, where cancer drugs have proved most effective.

One of the best known – imatinib, whose brand name is Glivec – has proved so successful in chronic myeloid leukaemia that patients who a decade ago survived for a few years can now look forward to a near-normal life expectancy.

But the cost of Glivec has risen from £18,000 per patient per year to around £21,000 in the UK, and from $30,000 to $92,000 in the US. This is despite the fact that all research costs were covered by the original price, and the number of patients treated and the length of time they are on the drug have both vastly increased because of the drug’s success.

Daniel Vasella, former chairman and chief executive of Novartis, the manufacturer, said the original price charged for Glivec in 2001 was considered “high but worthwhile” and was estimated to yield annual revenues of $900 million, enough to cover its development cost in two years. A decade later Its annual revenues in 2012 were $4.7 billion (£3 billion).

The cancer specialists say the revenue earned by Glivec over the last ten years “represent generous profits to the company”. But this has put heavy pressure on those who have to foot the bill. “Grateful patients may have become the financial victims of the treatment success, having to pay the high price annually to stay alive”.

In the US even those with health insurance may pay an average of 20 per cent of drug prices out of pocket. Drug prices are the single most frequent cause of personal bankruptcies in the US.

Three new drugs have been approved for chronic myeloid leukaemia in the last year by the FDA but the prices are “astronomical” the authors say at up to $138,000 a year per patient.

Worldwide only about a quarter of the patients with chronic myeloid leukaemia who could benefit have access to drugs because of the cost. “A small fraction are rich enough to pay individually, and most are treated intermittently or not at all. The effects of these financial pressures on long term survival… are yet unknown.”

In the UK, patients are shielded from the “direct economic anxieties of illness”, the article says.  But Professor Jane Apperley, chair of the Department of Haematology at Imperial College, London, and one of the authors, said high drug prices were still a cause of harm in Britain .

“The price of a drug heavily influences the decision of NICE whether we can prescribe it on the NHS. I am chief of service at Imperial College and we are constantly being asked to reduce our spending. We have to look very carefully at the cost of the drugs we use.”

“Of course we need the pharmaceutical industry to go on developing new drugs. It is very exciting that a number of cancers are now becoming susceptible to these new drugs. But the rising cost is unsustainable. “

“The drugs are very effective at keeping people alive. But if they are priced out of what you can afford you know that you can keep people alive but you can’t afford to do so. It is completely unsustainable for the NHS because the costs are going up every year. We need a serious dialogue about whether we can sustain these costs.”

The authors of the article in Blood conclude: “We believe the unsustainable drug prices may be causing harm to patients. Advocating for lower drug prices is a necessity to save the lives of patients who cannot afford them. We believe drug prices should reflect objective measures of benefit, but should not exceed values that harm our patients and societies.”

The group say they intend to organise regular meetings and campaign for lower cancer drug prices.

A spokesperson for the UK charity Beating Blood Cancers said: “As a charity we want to see an ethical approach to drug pricing . There is no point in us investing in research if the pricing policy means drugs won’t be available to patients.”

In a statement to The Independent, Novartis said: “We recognize that sustainability of health care systems is a complex topic and we welcome the opportunity to be part of the dialogue.  Our critical role, as one of many parties working towards improving cancer care, is to discover and develop innovative treatments.”

“ Novartis innovation in chronic myeloid leukemia (CML) has changed the course of the disease. Before Glivec(imatinib)* and Tasigna (nilotinib), the five-year survival in CML was only 30 percent. Today, nine out of ten patients with CML have a normal lifespan and are leading productive lives.”

“Over the years, our programs have evolved to improve patient access to our medicines. We work together with government health care systems, charities and other payers to build successful cost-sharing models.”

Expert view: ‘Price of drugs is harming patients’

The following is an extract from an article, contributed to by more than 100 leading cancer physicians from around the world, including nine from the UK, published in the journal, Blood.

This perspective reflects the views of a large group of CML experts, who believe the current [high] prices of drugs may compromise access of needy patients to highly effective therapy, and are harmful to the sustainability of our national healthcare systems…

If drug price reflects value, then it should be proportional to the benefit to patients in objective measures, such as survival prolongation, degree of tumour shrinkage, or improved quality of life. For many tumours, drug prices do not reflect these endpoints, since most anti-cancer drugs provide minor survival benefits, if at all.

As physicians, we… believe the unsustainable drug prices in CML and cancer may be causing harm to patients. Advocating for lower drug prices is a necessity to save the lives of patients who cannot afford them … For CML, and for other cancers, we believe drug prices should reflect objective measures of benefit, but should also not exceed values that harm our patients and societies.”

An ethical price tag? Cancer drugs

Brands used for the treatment of chronic myeloid leukaemia

Imatinib (Glivec) £21,000 per patient per year – Novartis
Designed from first principles, it proved hugely effective and unexpectedly turned into a blockbuster, earning billions of pounds for its makers.

Nilotinib (Tasigna) £21,000 – Novartis
Designed for patients who fail to respond to Glivec, Novartis reduced the cost to get it past Nice, whilst increasing the cost of Glivec.

Dasatinib (Sprycel) £31,000 – Bristol Myers Squibb
Also designed for patients who cannot take Glivec. But it has not been approved by Nice for use on the NHS because of its high cost.

Bosutinib (Bosulif) £76,000 – Pfizer
For patients who suffer side-effects from the other drugs. It won approval in the US in 2012 but is awaiting a licence in the UK.

Omacetaxine (Synribo) £100,000 – Teva
For patients who cannot tolerate other drugs. Approved in US in 2012 but awaiting licence in the UK.

Ponatinib (Iclusig) £90,000 – Ariad
A third-generation drug which works in a different way. Approved in the US in 2012 but awaiting a licence in the UK.

JEREMY LAURANCE    MONDAY 29 APRIL 2013

http://www.independent.co.uk/news/uk/home-news/the-real-cancer-killer-ripoff-prices-for-drugs-set-by-profiteering-big-pharma-giants-8591825.html

Suzie’s story

I received inspirational story from a blog reader – I hope Suzie keeps us up-to-date with her progress and that we all join together to wish her health and the greatest happiness. Kris  

I would like to share my story…..

I am a 36 year old female pharmacist from Melbourne, Australia. I was diagnosed with CML on 8th May 2008 at the age of 31. My husband and I had been married around a year and we were more than ready to start a family. I went to the GP for a check-up and had some blood tests. I requested a copy of the results of these tests to be posted to me as I hate waiting to see the doctor to find out if I am low in vitamin D or iron or some other fairly insignificant thing. What a shock I got when I opened the envelope one night after work and it said “likely to be chronic myeloid leukaemia” at the bottom of my blood test, most of which was highlighted in red.

I was reluctant to get on the internet and read horror stories so instead I turned to my Merck Manual dated 1999. As this was written before the brilliant discover of imatinib, the prognosis for CML read palliative 3-5 years. I vaguely remember crouching down in the corner of the shower crying and hiding from the world. I’m not sure how I got through the night.

The next day I saw the GP and I don’t think she knew much about CML as she just gave me sleeping tablets, referred me to haematologist and told me to try and not go to “that dark place”. Easier said than done. That was a Friday and my appointment with haem was on the Monday so, believing I was going to die, my husband and I kicked our heels up for the weekend and tried to have as much fun as possible. We drank a lot at the pub and went out for breakfast, lunch and dinner but there was definitely a big cloud hanging over our heads.

After seeing the haematologist, finding out about the brilliant imatinib and being given the opportunity to live, I was thrilled and couldn’t wait to start. I asked about freezing my eggs as we really wanted a baby at some stage but I was told I would be on the treatment for the rest of my life so not to bother basically. I started on a high dose of imatinib (600mg) and continued to work full-time as a pharmacist. I struggled with the side effects, especially fluid retention – I gained 6kg in 48 hours. My face was extremely puffy and my eyes really swollen. Diuretics helped a bit but eventually, after reaching undetectable levels, I had a dose reduction then swapped to nilotinib a year after diagnosis as the imatinib was not working that well. I can’t remember how great my adherence was…

I found nilotinib much, much better but still had fluid retention issues and skin problems (dry, rashes and rosacea). I reached undetectable levels quickly and in September last year I stopped treatment to undergo an IVF cycle in October which was successful first time around (so lucky!). All was going well until the evil leukaemia reared it’s ugly head. In November my BCR-ABL went up from 0.000 to 0.15 then in December it increased to 5%. I was not expecting that at all. I knew it would come back, but I thought it would be quite slow.

I am 14 and a half weeks pregnant and I must now start treatment with interferon for the rest of the pregnancy. I am absolutely dreading it although the thought of my husband and I becoming parents is helping keep me sane. I am starting tomorrow night – YIKES and I am ready to feel like I have the flu permanently which terrifies me to be honest. They say interferon causes depression but I think the thought of going on it is already making me depressed.

On a positive note, before the invention of Gleevec in 2001, interferon would have been the only option to all of us with CML and it usually only prolonged survival so when I am feeling really unwell, I will keep reminding myself how extremely lucky we all are to have these life-saving drugs available. If everything goes well with the pregnancy, I will go straight back on nilotinib when the baby is born although I am going to ask my haematologist whether I can possibly try bosutinib as it sounds a lot better, especially for skin and puffy face issues.

In summary, I hate CML but I feel lucky to have a cancer that has a normal life-expectancy. I hope there will be a cure for CML soon because since I stopped my medication, I have realised how fantastic it is to feel normal and full of energy. I know there are a lot of people working on it and for this I am extremely grateful. That is my story for now and I will keep you posted how the interferon/ pregnancy thing goes…

FDA approves Synribo for chronic myelogenous leukaemia

More fantastic news regarding another drug to treat CML. More hope for those who fail on other TKIs. We must ensure our UK health system funds drugs like this as they become available. Kris

The U.S. Food and Drug Administration today approved Synribo (omacetaxine mepesuccinate) to treat adults with chronic myelogenous leukemia (CML), a blood and bone marrow disease.

An estimated 5,430 people will be diagnosed with CML in 2012, according to the National Institutes of Health. Synribo is intended to be used in patients whose cancer progressed after treatment with at least two drugs from a class called tyrosine kinase inhibitors (TKIs), also used to treat CML.

Synribo blocks certain proteins that promote the development of cancerous cells. It is injected just under the skin (subcutaneously) twice daily for 14 consecutive days over a 28-day cycle until white blood cell counts normalize (hematologic response). Synribo is then administered twice daily for seven consecutive days over a 28-day cycle as long as patients continue to clinically benefit from therapy.

“Today’s approval provides a new treatment option for patients who are resistant to or cannot tolerate other FDA-approved drugs for chronic or accelerated phases of CML,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research. “Synribo is the second drug approved to treat CML in the past two months.”

On Sept. 4, 2012, the FDA approved Bosulif (bosutinib) to treat patients with chronic, accelerated or blast phase Philadelphia chromosome positive CML who are resistant to or who cannot tolerate other therapies.

Synribo is approved under the FDA’s accelerated approval program, which allows the agency to approve a drug to treat a serious disease based on clinical data showing that the drug has an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients. This program provides earlier patient access to promising new drugs while the company conducts additional clinical studies to confirm the drug’s clinical benefit and safe use. Synribo also received orphan-product designation by the FDA because it is intended to treat a rare disease or condition.

The effectiveness of Synribo was evaluated using a combined cohort of patients whose cancer progressed after previous treatment with two or more TKIs. All participants were treated with Synribo.

The drug’s effectiveness in chronic phase CML was demonstrated by a reduction in the percentage of cells expressing the Philadelphia chromosome genetic mutation found in most CML patients. Fourteen out of 76 patients (18.4 percent) achieved a reduction in an average time of 3.5 months. The median length of the reduction was 12.5 months.

In accelerated phase CML, Synribo’s effectiveness was determined by the number of patients who experienced a normalization of white blood cell counts or had no evidence of leukemia (major hematologic response, or MaHR). Results showed five out of 35 patients (14.3 percent) achieved MaHR in an average time of 2.3 months. The median duration of MaHR in these patients was 4.7 months.

The most common side effects reported during clinical studies include a low level of platelets in the blood (thrombocytopenia), low red blood cell count (anemia), a decrease in infection-fighting white blood cells (neutropenia) which may lead to infection and fever (febrile neutropenia), diarrhea, nausea, weakness and fatigue, injection site reaction, and a decrease in the number of lymphocytes in the blood (lymphopenia).

Synribo is marketed by Frazer, Pa.-based Teva Pharmaceuticals. Bosulif is marketed by New York City-based Pfizer.

For more information:

• FDA: Office of Hematology and Oncology Products
• FDA: Approved Drugs: Questions and Answers
• FDA: Drug Innovation
• NIH: Chronic Myelogenous Leukemia

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Source: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm325895.htm

FDA approves Pfizer leukeamia drug – Bosutinib / Bosulif

Whilst I desperately want to celebrate a new CML drug coming into the marketplace I find it hard when I think about the dasatinib debacle we currently find ourselves in here in the UK. If Pfizer do not offer a PAH we will find ourselves lagging even further behind in CML treatment, this being said even a PAH will not guarantee availability.

This does prove to us that developments are still being made and that we must do all we can to ensure patients get access to the drugs they need, regardless of where they live.

I’ve started off with an excellent video interview with Dr. Carlo Gambacorti-Passerini, a prominent CML researcher from the University of Milan:

In this video interview from ASH 2011, Dr. Carlo Gambacorti-Passerini, a prominent CML researcher from the University of Milan Bicocca, gives us an update on bosutinib, a newer Tyrosine Kinase Inhibitor (TKI) that he has studied in-depth.  Once approved, bosutinib may provide one more option for patients and their physicians to better treat the disease while managing side effects.

WASHINGTON | Tue Sep 4, 2012 5:33pm EDT – LINK TO ORIGINAL ARTICLE

(Reuters) – Health regulators on Tuesday approved a Pfizer Inc pill for a rare type of leukemia, another step in the company’s effort to expand its oncology business.

The medicine, called Bosulif, treats chronic myelogenous leukemia (CML), a blood and bone marrow disease that usually affects older adults. About 26,000 Americans live with the cancer, and 5,430 people in the United States expected to be diagnosed with it annually, the U.S. Food and Drug Administration said.

Most people with CML have a specific type of genetic mutation called the Philadelphia chromosome. This mutation causes bone marrow to make an enzyme that triggers the abnormal growth of white blood cells. Bosulif, known generically as bosutinib, blocks the enzyme’s signal that causes the white blood cells to grow.

“We are seeing improvements in the treatment of CML based on a better understanding of the molecular basis of the disease,” Dr. Richard Pazdur, head of the FDA’s cancer drugs center, said in a statement.

Pfizer’s drug is meant for people who have CML with the Philadelophia mutation who cannot tolerate other medicines, such as Novartis AG’s Gleevec, or whose cancer has stopped responding to the older treatments.

The FDA gave Bosulif orphan status, which means it treats a condition or disease that affects fewer than 200,000 people in the United States. The designation comes with a seven-year period of marketing exclusivity.

The medicine is expected to reach peak global sales of $341 million in 2016, according to the average forecast of analysts polled by Thomson Reuters.

Bosulif is the second Pfizer cancer drug to get the FDA’s nod this year, after its kidney cancer drug Inlyta. Investors are looking for signs of the company’s research productivity, to help replace lost revenue from its cholesterol fighter Lipitor. The world’s top-selling drug began facing generic competition last year.

Shares of Pfizer were largely flat at $23.85 in after-market trading on Tuesday.

(Reporting by Anna Yukhananov, additional reporting by Bangalore equities newsroom; Editing by Leslie Gevirtz)