I spent an exhausting day in Westminster meeting parliamentarians this week. Henry Smith MP, Mark Durkan MP and Nic Dakin MP all gave up their valuable time to sit with me in Portcullis House to discuss Khalid Younis, Chronic Myeloid Leukaemia (CML) and the problem we currently face with the lack of an appraisal system for new drugs.
All three MPs were incredibly kind with their time and very supportive. They all agreed to support a letter from Roger Godsiff MP (Khalid’s member of parliament) to the Prime Minister asking him to intervene in Khalid’s case as well as agreeing to submit written parliamentary questions where appropriate. All three were very concerned with the issues I raised and offered lots of advice and opinion on how we can move forward.
It’s great to have days like this. We’ve made friends with more, influential, people who now know about the struggle CML patients face as well as issues facing the wider cancer community. Although to their credit all three men were very aware of the restrictions faced getting the right drugs to the right people in a timely and cost-effective way. I certainly appreciated the balance in conversation; we all know that the funding pot is finite but to have a productive discussion on how to address this in a fair and progressive way, ensures we, as patients, become part of the solution, rather than part of the problem.
I’ll progress this by suggesting some written questions we can submit and go on record by thanking all three gentlemen very much for caring about CML patients.
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.
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.
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.
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.
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.
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.
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
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
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
It was a conversation with my good friend and fellow Chronic Myeloid Leukaemia (CML) patient Nigel Deekes that made me decide to skip the CML Horizons conference this year and instead explore the wider world of health.
Our conversation centred around whether we were CML advocates or cancer advocates, my attendance at WIRED Health 2015 in London this year suggests an even wider remit. CML is an orphan disease, just 650 of us diagnosed each year, it’s hard to get noticed. It’s even harder to position and relate our condition to the general health marketplace. When we position ourselves as having a rare disease it doesn’t make us commercially attractive to people looking to solve problems. That sounded less harsh in my head.
WIRED is a monthly American magazine that reports on how emerging technologies affect culture, the economy and politics. It is headquartered in San Francisco, California and has been in publication since January 1993. Several spin-offs have been launched including: WIRED UK, WIRED Italia, WIRED Japan and WIRED Germany.
Now in its second year, WIRED Health is designed to introduce, explain and predict trends in the medical and personal healthcare industries. It is a showcase for innovators using technology to re-imagine the health sector.
From senior NHS representatives to disruptive entrepreneurs, investors to international suppliers, the WIRED Health delegate list is a shortlist of some of the most exciting and knowledgeable figures in the international health sector. And me!
This is an exclusive event, the registration fee of over £1,000 exemplifies that, but upon entering it certainly feels like you’ve achieved the next level of a decent video game. I’m not ashamed to admit that I had to work hard to stay with the agenda – the day was long and packed to the rafters with innovation and challenge.
That started from the moment I entered the networking room; the Carbon Black wheelchair was being showcased on the conference floor. The chair has been featured in a recent copy of WIRED. It’s an incredible piece of engineering: stylish, supportive and minimal but above all light. So light I could pick it up, with ease, with one hand. After a productive conversation with the Carbon Black team about its use in education I tweeted a photo of it. From my meagre network, a deluge of retweets and favourites from across the planet. I wasn’t in Kansas anymore. Kids, this was cutting edge stuff.
Editor of WIRED magazine, David Rowan with Kris Griffin
The affable and assured editor of WIRED UK, David Rowan, kicked things off by setting the scene, that we would be told stories, that we would cover all the bases and that we would be challenged. It was, in fact, David’s exquisite questioning of the speakers that provided the most balance to the events of the day, as host he pitched it perfectly.
Clive G Brown, CTO of Nanopore Technologies, spoke about how his company’s USB stick-sized DNA sequencer will enable an “internet of living things”. He spoke of how he could see consumers using this medical device and having results go to the cloud for diagnosis. Old DNA sequencing machines cost hundreds of thousands of pounds and are huge, the MinION costs just £650. Clive has a vision for immediate results on a device that can be run anywhere, he used an analogy of building it into a toothbrush so your biology can be sequenced every morning and monitored for changes.
A device like this could be revolutionary in the field of testing epidemics, environmental monitoring and infectious disease control. It’s predictive and preemptive. It could allow general self quantification in the same way diabetes patients track their blood sugar. For any leukaemia patient, like me, who has to have regular blood tests or bone marrow biopsies, several narratives converged for me at this particular moment in time.
Imagine a device that meant we wouldn’t have to visit our consultant regularly but still allowed us to do regular blood tests. It would pick issues up sooner, share results, save the NHS lots of money and do away with bone marrow biopsies. Imagine a device that could track chromosome abnormalities and check immediately for the philadelphia chromosome. The device you see me holding in my hand could be the key to unlocking all of that. This could also be the key to mass screening and therefore the saving of many lives. No pressure there then.
CTO of Oxford Nanopore Technologies, Clive Brown with Kris griffin holding the MinION device.
Clive is clearly a man who wants to keep expectations in check but I can’t help but get excited. I spoke to the team during one of the breaks and we are going to talk. This is one to watch!
I managed to get my breath back whilst listening to Matteo Lai from Empatica talking about his wearable sensor, Embrace. It looks for small changes in the body that could signify a seizure and would call a carer automatically. Wearable technology was one of the key themes of the day and this device offered excellent monitoring and safeguarding for just £200.
We learnt from Brad Perkins at Human Longevity Inc that human genome sequencing can give us a better understanding of our biology and with our increased computational power we can identify the root cause of ageing. This was a tough one to grasp but ultimately with shared ‘big’ data and deep analysis we are able to learn more about the human body and how we can adapt to environments that potentially harm us.
The WIRED Health team deserve great credit for including a patient advocate in their section on augmenting the human. Nigel Ackland lost his arm in 2006 in an industrial injury and struggled with NHS solutions and psychological scars. He is now a pioneer of the bebionic3 prosthetic hand and presented to us, as only a patient can, the true impact it had on his life. He inspired without a PowerPoint, from the heart and with passion about how the revolution technology can have an impact on a person’s life. It’s easy to forget during a conference that at the very core level we are talking about people and not products. This was an incredible demonstration.
Sophie de Oliveira Barata, Director of the Alternative Limb Project.
At this point I took a break from the main room and ventured into the BUPA Startup Stage for an hour. This stage ran alongside the main stage and gave some of the most exciting new companies in the health sector an opportunity to present to an audience.
In a very short period of time I learnt about the following:
Buddy Enterprise: a digital life-planning tool for people with depression and anxiety. The user keeps a text-message diary of how they are feeling, helping to reinforce positive behaviours.
Chiaro: a wearable fitness product for women called Elvie that tracks, guides, corrects and visualises the user’s pelvic floor exercises.
Cupris Health: developed smartphone-connected medical devices. Their otoscope and ophthalmoscope can capture, store and send data securely for diagnosis.
Galvanic: their PIP is a device that allows people to measure and manage stress levels, they used the analogy that the device uncloaks stress and help people manage it better. The biosensor and app rely on electrodermal activity to determine its user’s emotional stress.
GoodSAM App: a tool which alerts those with medical training to nearby emergencies, so potentially life-saving interventions can be given before the arrival of emergency services.
Peak: a mobile brain-training app that’s used by millions worldwide (including me now) to track and improve their cognitive skills with fun and challenging games. By providing better insight to our cognitive ability it can detect decline and help us improve and produce optimum performance through environmental factors.
The companies varied in their development journey but all of them had identified a key area of the health market and were looking to improve and save lives. The BUPA Startup Stage really felt like we were getting a glimpse into the future.
The core content was on the main stage, I had to return. I’m sad that I may have missed some more valuable health technology presentations.
One man’s unique vision and dream of ending unnecessary iatrogenic infection worldwide via dirty needles has led to the World Health Organisation (WHO) launching their third ever global policy. 1.3 million people die each year through medical syringe misuse, that’s twice the number of people killed by malaria. Marc Koska invented a new syringe that can’t be reused and therefore can’t infect someone else. It’s the same cost, uses the same machinery to manufacture and is used in the same way, yet he found resistance. It was perceived that this disruptive technology meant the medical supply industry would have to sell more syringes, which they were reluctant to do as they were sold as a loss leaders. It was only through WHO policy change and funding that the breakthrough happened. Manufacturers are now on board, ministries are on board and they are doing a healthcare worker and public awareness programme under the Lifesaver banner.
Every $1 spent on new syringe saves $14 treating disease in each country they are used due to transference being less of an issue. Manufacturers don’t have to make more syringes after all, there is less disease and people are heather. Marc suggested that we should, “make progress profitable, things will go faster.” It’s a logical step but feels uncomfortable. It always does when we bring the word profit into the sphere of health. We don’t live in an ideological world and to this end we need to start being realistic about pharma, about treatment and about talking economics in the health sector. I agree with Marc, economic viability opens doors.
Rory Sutherland, Vice Chairman of Ogilvy & Mather UK.
Rory Sutherland is an incredible speaker and no amount of words from me can do him justice. He’s a serial TED speaker as well as one of the brightest minds on the planet in behavioural economics. His logical, disruptive analysis of many different areas provides us with a breath of fresh air on problems we have long given up on. Rather than present a solution to issues in healthcare, he spoke about a new way of thinking to solve problems. He spoke about getting lucky, testing stupid things and the appearance of choice; that we don’t notice when we get a poor choice, we simply make a poor decision. Red or white wine anyone? It was incredibly refreshing to have a session that focuses on why, rather than how.
Professor Tony Young is now the national clinical director for innovation for NHS England and the co-founder of the Anglia Ruskin MedTech Campus. Previously as a health professional and innovative maverick he wouldn’t be told it couldn’t be done. This attitude allowed him to fight through a stagnant system and improve patient care through innovation and bold funding. His incredible vision will brighten our health services and benefit the system. He’s realises that we are able to innovate at scale and get better at prevention, which will create a better, faster, smarter NHS.
A slide from Tony Young, NHS England on creating a system for delivery.
Sonia Trigueros delivered a science-heavy session on a nanoscale approach to cancer. It was perhaps unfair to expect her to drop this information on our heads in just 20 mins, she gave it a valiant try. It’s clear that this is an incredibly important area of research and the work at nano level and with nano-hybrids can unlock huge advancement in cancer therapy. It was worrying to hear that the biggest thing holding her work back is finance, she spends 80% of her time looking for funding!
Neuroscientists from the world’s most respected universities shared their discoveries about the brain. Sarah-Jayne Blakemore from University College London debunked myths about Brain Gym, left and right brain people and how we are seduced by neuroscience. Her field of interest was education and adolescence in particular, looking at how an environment can shape the development of the brain. Can we adapt education to fit the best way a child develops?
Eleanor A Maguire from University College London followed up with memory and the function of the hippocampus. She looked at how damage disrupts memory and why some people are better at remembering than others. She explained the change of brain structure through intense environmental factors and used London taxi drivers as an example; when examined their brains show a level of plasticity after taking on ‘the knowledge’. It normalised when they retire.
John F Cryan from University College Cork rounded this area off with a session on the relationship between the gut and the brain. We speak of a gut feeling but discovering the medical relationship was fascinating; brain health can be linked to healthy microbiota in the gut. On a more practical level, treatment for Clostridium difficile infection has a 90% success rate when a faecal transplant is administered…that’s a poo transplant.
Optimising Performance was always going to submit to an element of masculine chest beating, especially when two Formula One brands followed each other. Thankfully Adam Gazzaley from the University of California and his study of the processes of the neural mechanisms of memory and how they affect childhood development, dementia and ageing served as an excellent opt in to the content that followed. His study explored how cognitive abilities can be enhanced via engagement with custom-designed video games. The results were astonishing and once again showed an incredible level of plasticity to the brain and showed through optimisation there was great potential for brain function at any age.
Founding Director of Neuroscience Imaging Center, UCSF, Adam Gazzaley.
Dr Andy Walshe, the high performance director at Red Bull and Geoff McGrath, vice president for McLaren Applied Technologies captivated the audience with the secrets behind elite performance. Walshe spoke about the Red Bull Stratos project, when Felix Baumgartner jumped out of a space capsule from an altitude of approximately 71,580 feet. He was part of the team that coached and trained Baumgartner and looked at extreme training and preparing elite athletes. Whilst this session was light on data, the concept that we should treat doctors and scientists like elite athletes and monitor performance and provide optimum environments in which to function, holds much water. Likewise, McGrath spoke about racing cars; constantly monitoring and evaluating performance from race-to-race. Their innovative developments and breakthroughs in performance can be applied to the healthcare sector and it was fascinating to learn about real-time data processing and predictive analytics being applied to remote biotelemetry of patients in clinical trials, mainly through wearable technology.
Finally, we were presented with the winner of the judged BUPA Startup Stage presentations; Ana Maiques from Neuroelectrics presented her diagnostic and treatment telemedicine platform that helps patients recover from health issues. This incredible real-time device is essentially a cap that fits on the head and can be used for home treatment. It could revolutionise the way we look at stroke and neuropathic pain.
THE quote from Rory Sutherland at WIRED Health 2015.
It was interesting to note that through the day there were several common themes: that disruption can yield excellent results, that there is a big question about who owns the data our bodies produce, that gamification of particular areas is big business and can produce results and that we are finally beginning to understand and learn how our environment can optimise our treatment and performance. These wide ranging breakthroughs and challenges have the potential to shape our destiny, I felt privileged to share a room with the people breaking doors down
And that was it. An incredible day. I have to thank Ariad Pharmaceuticals for sponsoring my attendance. Their foresight to invest in an independent advocate will indirectly affect many CML patients and other networks. I can start to bring a wider approach to my work now; I’ve made some outstanding contacts and I’ve already started to develop some ideas that could have a huge impact on our patient group. I’m incredibly glad I took Rory Sutherland’s advice before I’d even heard it, “test counterintuitive things, because no one else will.”
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.
Didn’t crazy eighties hair rockers Europe write a song like that?
Sorry about my absence, no excuses really – simply a case of balancing all of the things I’m involved in and sadly my gorgeous little blog got bumped down the list. It doesn’t mean I don’t love you. I’m good, had a bit of blip with my blood but all OK now. I’ve got some cool things going on so the blog will spark back into life now. Hurrah!
Firstly I’ll be at Impact Day for Leukaemia & Lymphoma Research this Saturday doing a speaky bit about my journey and their online patient services which are fantastic. Find out more here: www.leukaemialymphomaresearch.org.uk/event/special-events/impact-day-2014 If you are going please drop me a note or tweet me – I’d love to catch up with people.
The following weekend I’m off to Serbia, as a delegate, for CML Horizons 2014. I’m a 3 year veteran of this remarkable event which regularly attracts over 140 participants from almost 60 countries. It’s 3 long days with even longer evenings but it provides a great opportunity to network and get up-to-speed on the latest developments in the CML world. I’m being sponsored to go by Leukaemia & Lymphoma Research (LLR) along with their patient engagement manager. I’m delighted LLR are attending, I’ll be sharing my daily blog entries on their website.
Finally, I don’t do things by half, I’m pursuing some issues with the finance industry about life insurance cover for CML patients. Whilst traditional cancer patient are able to get cover after being in remission for a number of years, it is incredibly difficult to get cover if you have had blood cancer. I think this is partly down to the sector not understanding the new treatments available to us, I’m hoping to work this out. I’ll keep you posted.
I trust you are keeping well? Thanks for sticking with the blog. I’ll post at the weekend from Impact Day.
Access CML Drugs 