Ask the right questions, get the right evidence

This is the second of my two thoughts about pressures being faced by patient advocacy.

There have been a number of recent articles in journals lamenting the failure of patient advocates to respect the principles of evidence-based medicine (EBM). It is a consistent low grumble from the science community, to which a small group are deeply wedded, while a majority probably have some sympathy with the point but also have some sympathy with the stance being taken by patient advocates.

There has been a change in the attitudes of regulators. Not that long ago they too were wedded to the evidence-base and they would maintain that this principle still underpins their decision-making. But in that decision-making, and therefore in the processes and procedures they employ, they are being more pragmatic. That has been partly driven by patient advocacy.

Some voices in the regulatory community are also identifying the issue which I believe needs to be addressed and which EBM protagonists seem to be studiously ignoring.  It is quite a big question.

Are we gathering the right evidence in clinical trials and in the way we measure and report real-world clinical care?

No-one in medical research seems to be addressing this effectively. A few are tinkering around the edge of the issue and for some, the idea that we are not gathering the right evidence, is like voicing heresy.

Cancer clinical trials are focussed on some measure of survival or tumour response to treatment. The so-called ‘gold standard’ measure of Overall Survival becomes difficult when a treatment is one of a succession of different therapies. A wide range of surrogate measures are used as a substitute. These are often dependent on some degree of investigator interpretation so are inherently unreliable, a factor which it is convenient to forget.  Side effects of treatment are an important issue and these are reported too, usually using an investigator applied judgement of severity against the CTCAE standards.

All this is a focus on objective measurement of the disease and the effect the treatment has on the disease. No-one is denying that this is important but where are the measures focussed on the patients themselves and the effect on them, their subjective reporting.

This is what patient advocates have been pursuing with their regulatory campaigns which the EBM stalwarts claim ignore the evidence-base. The advocates have an evidence-base in patient stories and anecdotes, but it is not one which they are in a position to measure, the professionals are not measuring it and have been ignoring it for more than twenty years.  So the EBM complainants, writing stridently about patient advocates not playing their game, would do well to look at themselves and their colleagues.

Are they asking all the right questions: are they gathering all the right evidence?

There is a side issue here about patient involvement. It is an issue which patient involvement cannot easily address and could be used as an excuse for inaction. Untrained involved patients do not necessarily see this ‘right evidence’ issue, they are often overwhelmed with understanding the planned outcomes and endpoints in a study. This assumes, of course, that they are consulted at a timepoint when they can actually input such ideas.

The second side issue is that we have much better ‘big data’ now than we have ever had. The ability to look at clinical practice across years, aggregating the experience of thousands of patients, means that retrospective analysis can provide valuable information and new insights. The question is, are we asking all the right questions in these analyses if the right questions were not asked when the data was being first gathered?

So the challenge for the data analysts is not to turn EBM into a self-justifying paralysis of ideas which ignores the patient?

NHS Cancer Performance – a helter-skelter of political incompetence

Heigh ho, its off to work we go. Those dwarves in Snow White had an enduring philosophy of hard work, tempered by compassion. I think their approach is mirrored by today’s cancer advocates, whether from professional or patient ranks. We have to speak truth to power, whether that is a wicked witch or a Tory government.

This government’s attitude to cancer stinks. It is now getting better after eight years of disinterest and decline. No doubt when challenged the government will point to new money arriving, they will not record the fact that performance standards are now lower than in 2012 when those standards were last revised and accepted, by them. Most of those standards repeated what had been in place for many years already and had been measuring the success of the policies and practices put in place by Professor Sir Mike Richards, the first national cancer director. The Coalition government had been in power for two years by 2012 and the disastrous policies of Conservative Secretary of State for Health Andrew Lansley (now ennobled) were beginning to bite. He was sacked in 2012 but the policies did not change.

This decline will continue, possibly for several more years. It takes time to turn the tanker round (another misplaced metaphor). It took time for the tanker to go off course in the early years of Tory rule because there was great momentum. The loss of the Cancer Networks in 2012 destroyed the NHS’s ability to learn and adapt in small ways, the dispersal of experienced staff with extensive knowledge into other roles wiped out a major resource. The recent invention of Cancer Alliances will hopefully correct that mistake but it will take time for the expertise to rebuild.

We have also seen major steps forward on such issues as early diagnosis. Sir Harpal Kumar’s Task Force produced an excellent report which the NHS accepted. What is not in the report is a recommendation that hospital operating capacity needs to expand to cope with more people being diagnosed earlier. The austerity-led reduction in capacity, seen by waiting lists in almost every surgical discipline, is impacting on cancer treatment targets and will continue to do so, possibly for years.

Maybe politicians did not see the linkage. Cutting nursing numbers impacts on areas such as surgical recovery, intensive care and theatre staff. Without the proper safe staffing levels surgery cannot take place. Closing one operating theatre for half the week so you can use what staff you have left more efficiently extends waiting times. What chance do performance standards have then?  At the same time improve diagnostic procedures in primary care thus increasing referrals into secondary care, and you extend the queue at the other end.

The equation is clear. One good + one bad = all bad. Sorry Harpal.

Increasing cancer resources with more surgeons or oncologists will not solve the cancer waiting time issues. It requires a major overhaul of staffing resources down the whole chain of treatment increasing the capacity of the NHS. It takes three years to train a nurse to staff level, and then further training for specialist care. At the very moment when we are about to exit the EU and possibly lose access to a major staffing resource, what chance rapid improvement?

Talk about screwing it all up. How to kill people invisibly. We made huge steps forward in the early 2000s. The now largely forgotten report by Professors Sir Ken Calman and Dame Deirdre Hine in 1995 records what cancer care was like then. We may not sink back to those standards but 2004, here we come.

The raw Performance Data is here

Excellent commentary on the data in the Guardian

PS: a real delight to see and have a chat with Sir Ken Calman at the NCRI Conference last week. Enjoying his role as Chancellor of Glasgow University where he first became a professor in the early 1980s.

PROs at the centre of cancer care

Chemotherapy or supportive and palliative care? Its a question which oncologists are increasingly addressing with their patients. People with advanced solid tumours, that is cancers which have spread to secondary sites, are very rarely curable, although maintaining stable disease for a significant time is increasingly possible. Symptomatic treatment with the aim of delivering a high quality of life may be preferable to a so-called curative approach.

One of the communication challenges is the widespread belief that chemotherapy in these circumstances will deliver a cure. Oncologists can do their best to help patients to understand the situation but they cannot educate family and friends. Patients often lack the necessary mastery of the information or the skills to convince their family of the reality. Family pressures to undergo chemotherapy can be very real for those who are already vulnerable.

The new targeted therapies do change the picture for some patients. If your tumour carries a genetic or biological target which is treatable extra time can be bought, sometimes with few side effects, although there can be a financial cost and cure remains rare. These drug costs are becoming a significant part of the NHS cancer drugs budget and the struggle to keep costs under control is highlighting the challenge which society faces, to understand the holistic value of these treatments.

Value lies in the underlying reality that secondary solid tumours kill their host. Patients without secondary tumours usually survive their cancer. Treatment with high cost drugs to prevent secondaries is a ‘holy grail’ for pharmaceutical companies, the demand would be very profitable. Their urge for acceptance of their costly new treatments excites the media, which rarely reports the realities.

Today’s realities offer a different picture. Early diagnosis and skilled primary treatment are the surest ways of delivering better survival. The renewed NHS focus on this is welcome. Cure represents value. However, there will always be people who, for whatever reasons, present late and with secondary tumours. Proper supportive care is needed for them and the NHS is not as good at responding to this need as it could be either in primary or secondary care. Attention to value is required.

The value issue principally focuses on those with secondary tumours which develop while in follow-up or care. How does treatment affect a patient’s life, whether that is chemo, new targeted drugs, interventions such as surgery or radiotherapy, or symptomatic care. What is the balance with cost? Health economics looks at ‘cost effectiveness’ but this is more about how a patient clinically responds, and for how long, to any treatment rather than how it affects their life.

Looking to meet the modern demand for an acronym Value can be defined this way (from a patient viewpoint):

Variations in my Actual Life and Usual Experience

Attempts to look more holistically at value to capture actual life and usual experience through the lens of patient reported outcomes (PROs), are still at an early stage. The tools which have been developed so far are clumsy and the challenges of implementing them effectively are still being worked out. There are good people working on this (not enough of them are actual patients) but many research organisations give it a low priority so funding is small and progress slow.

Regulators and legislators need to step into this area. Their work is increasingly relying on good data drawn directly from patients. The oncologist and patient who today are having that difficult conversation about chemo or not need the data too. Patient Reported Outcomes are the future shape of healthcare regulation and practice. They are not a nice add-on which appeals to ethics committees, they are an essential element in the science of treating people with disease. PROs can deliver an understanding of real Value to society. They must be taken seriously.

Good start Sally, have another go!

I am a great admirer of Professor Sally Davies, our Chief Medical officer. She is an innovative and determined leader, who listens and sets direction after due consideration of the issues involved. Her enthusiasm for genetics and its potential is to be admired but the recent Generation Genome publication, which gained wide press coverage earlier in July because of its implications for cancer, is a flawed document. It is a great shame, its an opportunity missed

The 256 page report also has some sloppiness which betokens haste. Chapter 3 is followed by Chapter 9 which has been transposed into Chapter 4. Chapter 9 really is Chapter 9 and not to be mistaken for the earlier Chapter 9 which is now Chapter 4. Confused – so was I until I worked it out. However this is a trivial slip.

The report is a fascinating overview of genetic medicine covering a lot more than cancer. I learned a lot from it – indeed I have not finished reading it yet. I am only competent to comment on the cancer chapter and not about the science.

It presents a one-eyed view of cancer treatment. The assumption that genetic science will be the only treatment that is needed for cancer comes across strongly. The word ‘surgery’ is only mentioned once yet surgery (plus adjuvant therapy) currently cures more than 50% of cancer patients and if we diagnosed them earlier it could be many more. No mention of these important self-evident truths. There is a real-life context for genetic cancer medicine.

A lot is made of ‘big data’ and its potential to impact on clinical decision-making. This is true regardless of genetic information, although such information will undoubtedly add immensely to the potential. The paper alludes to being able to select patients for treatment but does not consider the moral and ethical issues of empowering computers to make individualised assumptions which are used to deny treatment.

If the intention was to create the idea that surgery and radiotherapy (plus all the other modes of treatment) will be redundant in the future and that cancer treatment will solely rely on costly designer drugs, the chapter succeeds. It talks about personalised medicine as if that is the exclusive characteristic of new generation drugs. In my experience surgery is the most personalised medicine there is. Someone needs to cut out the hype and wake-up to the real life context – Dame Sally please take note. Case Study 1 describes a colon cancer patient with inoperable liver metastases and how cetuximab reduced them to become operable. There is no mention that the patient would have had surgery for his primary tumour and that without that intervention treating the metastases was inappropriate. Surgery to remove the residual metastases is mentioned – the only use of the word surgery in the whole chapter. Even Case Study 1 seems to have been distorted to support the idea! And there isn’t a Case Study 2.

This over enthusiasm and lack of context does a dis-service to what would otherwise be a useful paper. It has some relevance to what is happening today and clearly points to a lot of tremendous scientific and clinical advances. It will have an impact for surgery and radiotherapy, no mention of that. The tremendous advances in imaging and the relationship with that work is ignored. It focuses on drugs alone and then makes no mention of the fact that today there are more known mutations than there are drugs to treat them.

Genetics will undoubtedly create change. There will be new and valued diagnostic information although the ability to act on it will develop over time. Curative surgery plus radiotherapy, curing most patients, will change slowly as evidence to support change appears. There will be new adjuvant therapies and maintenance therapies. The precision medicine impact will be largely to the way advanced disease is treated, when successive metastatic events introduce new mutations which can be identified though techniques such as liquid biopsy. This will deliver patient benefit. Patients currently on a path to end-of-life care will have that pathway extended, although at largely unknown cost.

We need to have a balanced and inclusive view rooted in our multidisciplinary world. Patients need a range of skills and knowledge to support their survival, or their pathway to dying, that must be reflected.

Good start Sally, have another go.

Download Generation Genome (Government website)

Precision medicine – promotion is an ethical challenge

Precision medicine is the carefully chosen term for what has been called ‘personalised medicine’. It is more appropriate but it still hides a range of issues affecting cancer patients which the promoters are not keen to discuss. When my eye was caught by the headline Cancer Care is Rapidly Changing — Here’s How You Can Keep Up it was no surprise to find precision medicine over promoted as usual.

The story describes the launch this week of Precision Medicine for Me which claims to take a new slant on the subject. It is a collaboration of more than a dozen patient organizations, patient advocates, startups, and industry leaders with the admirable objective to inspire and help patients understand and navigate the potential of new tumor testing, and raise awareness of the latest treatments and trials.

They discuss the challenge of getting tumours tested for genetic mutations, the fact that few centres which treat cancer have the capability yet, and the fact that it is costly. They skate around the fact that drugs may not be available to treat mutations which have been identified, drugs which might work may not be approved or funded for certain cancers, and that clinical trials of unproven treatments may be the only route open to a patient. It is a US initiative and the collaborators have a cancer background. One patient is named who has benefited from the approach.

Precision medicine is an important development, however it has a long term view even though it may benefit a select (and growing) group of patients in the meantime. Precision Medicine for Me is focussed on lung cancer, five of the collaborators are lung cancer specific. It is a matter of great regret that their story failed to focus on that specific point and made a promise to all cancer patients which cannot be met.

To illustrate the issues it may be useful to recall that one specific sarcoma benefited from one of the very first ‘precision’ drugs. Imatinib for GIST was first studied back in 2000 and was the fastest drug to achieve a licence when it was approved in the US and Europe. The rise of secondary mutations resistant to imatinib, commonly after about two years, has been followed by further drugs to treat them, or some of them. We now have strategies designed to delay resistance to imatinib. Mutation analysis has identified more than 40 secondary mutations. We have many patients alive after well over a decade of treatment, and all that is truly welcome. We also have patients who have died because the secondary mutations they faced are untreatable. Hopes we might have had for rapid development of similar treatments for other sarcomas have been dashed long ago.

This is a tough reality.

The truth is that at present more cancer mutations are being found each year than there are drugs to treat them. The FDA in 2016 licensed treatments for 11 cancer indications – 2 were extensions of an existing licensed drug, 2 were for side-effects – a total of 7 new cancer therapies (by the way one was for sarcoma). EMA’s activity was similar. This is a recipe for frustration – science can find the mutations but drug development cannot deliver the treatments anywhere near fast enough.

So beware promoting precision medicine. The danger of raising expectations in people desperate for treatment makes it unjustifiable. The ethics of unbalanced promotion are questionable, the science should be discussed but even the vaguest promises should be avoided. Ideally only specific clinical situations should be reported. The GIST story should be remembered and if it is replicated in other tumours it should be openly talked about.

Oncology – a changing environment

In a few weeks time I shall have my 18th cancer birthday – a coming of age. I have had cancer for over 25% of my life (just). Although some experiences which cancer patients face have passed me by there are many which remain strong in memory. Some are confined to sarcoma, so I am something of a specialist.

Last week I had my latest check-up – all clear once again. It is hard to explain the sense of relief this brings. Those who do not know what it is to live under seemingly constant threat cannot imagine the tension and anxiety that dominates the days before a check-up. My last treatment is now three and a half years ago (thoracic metastectomy). It took time to recover and get going again, and then I was hit by back problems. Now I have diabetes and other niggling issues to address, so its not an easy downhill run.

I talked about this check-up anxiety (often known as scanxiety) with Dr Mike Leahy, my consultant. Mike has looked after me for twelve years, has managed recurrent diagnoses, helped and advised on many issues, but has never had to treat me. He is a medical oncologist and his training and scientific knowledge primarily concern drugs to treat cancer. Sarcoma throws up some of the most challenging situations, disease like Ewings sarcoma, rare tumours with few (if any) effective treatments, cancers in young people, so the challenges are many. He has been working with members of the team at the Christie Hospital on the anxiety issue as part of a pilot project (Christie PlanBe) looking at the holistic needs of patients with advanced disease. The aim is to develop and teach strategies for self-management and it was heartening to find that some of the strategies I have developed for myself are included and are working for others too.

We also talked about the recent paper in BMJ by retired oncologist Dr Peter Wise and some of the responses he has had. My feeling is that this paper gives an accurate view of what is happening worldwide. The debate comes down to the issue of the patient choice between chemotherapy for advanced cancer and ‘something else’. Too often the ‘something else’ is no treatment, so patients naturally choose chemotherapy. Doctors in healthcare systems which pay them for providing treatment don’t get paid if there is no treatment, so they are quite happy about this decision. In the NHS they get their salary regardless of the decision so there is a trend, and I believe it is a growing trend, to make it clear to patients that symptomatic treatment (sometimes called palliative care) is an appropriate alternative to chemotherapy for advanced disease which is incurable. Painting this in a positive way, while retaining the balance with treatment which patients and families often mistakenly regard as curative, is not easy. There is evidence from studies in Holland that patients and their families cling to false hope. Doctors have to recognise this and find ways of communicating prognosis in a sensitive manner. Patients also want to keep contact with consultants they know when they take this decision, so the role of oncologist as palliative care specialist is evolving.

I have met and worked with many oncologists in my 18 years. Mike has a special place as “my” doctor. That experience builds perspective and he gives an important depth to my understanding. I recognise in oncologists clever people with a high motivation and commitment to patient benefit. Ultimately this is uncompromising in the face of evidence. Importantly the evidence is now steadily tilting the balance away from chemotherapy where the prognosis is poor and oncologists are thinking hard about how they act in this changing environment.


Dying your way – support the Assisted Dying (No2) Bill

An important moment is coming and everyone needs to think quite hard about it. In just over two weeks time the Assisted Dying (No2) Bill is to have its Second Reading in the House of Commons.

The Bill aims to make medically assisted suicide legal in the UK.

This Bill was originally introduced in the House of Lords by Lord Falconer but it lapsed when Parliament was dissolved for the General Election. It is being revived by Mr Rob Marris MP, who won the lottery for Private Members Bills in the new House of Commons.

The Bill has been very carefully framed and negotiated. Remember that Lord Falconer is a lawyer and former Lord Chancellor. In the Lords there was quite strongly felt debate. It lasted over 9 hours, there were over 100 speakers and two attempts to amend it to worthlessness were resisted, being out-voted by quite substantial margins. It was passed by a majority of two-thirds of those voting.

The Bill is a simple piece of legislation. It has very clear safeguards to prevent misuse of the new provisions. The fear that some elderly people may be exploited and bullied into assisted suicide has been addressed very carefully. Every written application has to be approved by the High Court. Doctors are not obliged to help a patient even to make an application and at every step the patient has the absolute right of withdrawal. Someone seeking to use this as a route to bump off a relative opens the way to life imprisonment.

The Bill’s first real test comes in the House of Commons on Friday 11th September. There will be a debate and a vote. There will, no doubt, be some opposed to the Bill.

There are many cancer patients who would like to see the Bill enacted and to have the option of dying at a time of their own choice. Dying from cancer rarely has much dignity. Even when at home and properly supported by medical and caring staff, extremes of medication are often necessary to control pain and other symptoms. The new NICE Guidance on end-of-life care is explicit about what symptoms might be experienced. It details treatment for agitation, anxiety, breathlessness, nausea and vomiting, “noisy respiratory secretions” and pain. It is not a menu, a list of options from which you make a choice. You might suffer all of them. You will not be in control.

If you would like to see this Bill take its next step through the House of Commons and, hopefully, make its way through Parliament to reach Royal Assent, write to your MP and ask him to join those who will support Rob Marris on September 11th by voting in favour of the Assisted Dying (No2) Bill.

End-of-life care should be an openly discussed topic

End of Life care is not usually regarded as a front page headline topic by the popular press, nor is it a frequent discussion point in cafes, pubs or bars. It is an important topic nonetheless, if only because it is the one area of healthcare which very nearly all of us will experience. Sudden death, from whatever cause, is not the usual way to die.

This lack of debate might be understandable if everyone who died did so in a quiet, caring, and capable environment at home or in hospice. In the right hands the well-evidenced Liverpool Care Pathway (LCP) helped a lot of people cared for by those trained in its use. NICE actually recommended it. However the fact is that hospitals are still the main place where people die and there have been horrific accounts of poor and insensitive care of those dying in hospital.

The abandonment of the Liverpool Care Pathway (LCP) a year ago shows us the nature of that problem. The review into the LCP led by Baroness Julia Neuberger was generally damning of hospital end of life care. A tick-box approach to the LCP de-personalised treatment, they were highly critical of the quality of communications around the dying person, and deeply critical of hospitals’ inability to respond to the needs of families and their loved ones, especially those dying at weekends or in the middle of the night.

“In the wrong hands,” they said, “the Liverpool Care Pathway was an excuse for poor quality care.” They went on “Generic protocols, as the LCP has come to be seen, intended to be applicable for all patients in the last hours or days of their lives, in any setting, are the wrong approach.”

The publication of guidance from the Leadership Alliance for the Care of Dying People followed quickly and set new standards. The Alliance was made up of 21 organisations including the Department of Health, NHS England, NICE, CQC, NIHR and key charities including representation from the Hospice movement. Their guidance is a direct response to the Neuberger review, accepts its recommendations and points to the supporting evidence base to guide local implementation. It confines itself to establishing strategic guiding principles, it does not replace the LCP. No-one following these principles and guidance will reproduce what is bad but they have to make up their own minds about the details of practice and focus that on the individual, not a protocol. The guidance emphasizes the need for individuality of care, giving proper respect to each person and their needs.

The guidance is extensively supported on the NHS Improving Quality website, with direct access to training information, so there is no excuse for any healthcare professional concerned with the care of dying patients not to have referred to it at the very least.

Now NICE is consulting on the draft of new Guidance for care at end of life. This will be authoritative. It is open for comments until the 9th September. At 266 pages its no lightweight and the supporting evidence is a much bigger tome. The group developing the Guidance was chaired by Professor Sam Ahmedzai from Sheffield University, an internationally respected palliative care clinician. If nothing else the list of 67 recommendations is worth reading to take in the breadth and depth involved in creating personalised care at end of life. They also point to four areas of clinical research priority which will hopefully be picked up in the near future.

One issue which affects research is that there is no clinical priority for end of life care research in the NIHR Clinical Research Network. It is a topic which is ignored in the way the Network has been structured. The 30 speciality topics mostly have a disease theme. In all probability the four end-of-life research priorities (and others which arise) will get researched through the cancer theme. The NCRI seems to be alone in clinical research in recognising Palliative Care with its own Clinical Studies Group (chaired by Professor Ahmedzai).

In addition to the clinical issues there is a need to better understand why so many people die in hospital when research shows they wish to die in their own homes. There have been numerous small studies, mostly focussed on one institution. There is no reason to doubt one conclusion that the communications problems and mechanics of transferring uninterrupted active care from a hospital to a GP, who relies on the support of multi-agency resources in the community, are dominant but there is a very real need to find ways of making it better.

There is also a need to find out what are the key measures which can evaluate performance. This may sound a bit callous. Maybe it is one of the characteristics of this topic that there are aspects which feel a bit insensitive. But it does affect all of us and I know that, when it comes to my time, I want to know that I am getting the best care there is.


Love, morphine and whisky

Dr Richard Smith, the former editor of BMJ, raised a storm last week with his blog about dying. He drew broad comparisons between different pathways to death and made the statement that death from cancer can be more acceptable than a death from a lingering disease like dementia.

Maybe his wording was a bit careless and he has acknowledged that in his latest addition to the blog. He certainly attracted a large number of reactions to what he said. Most were highly critical, going way beyond the point of understanding what he was actually saying. There were intensely personal and very painful stories of death from cancer, focussing on an individual, usually in the family of the writer.

Richard was addressing an important issue – we all have to die of something. This is not something that is part of general conversation and culturally it is a topic avoided like the plague. Sudden death, or peaceful death in ones sleep, are the preferred pathways when people do talk about it. Sudden death leaves goodbyes unsaid. Dementia leaves goodbyes behind hanging meaningless in the air. Dying of cancer gives us some time, occasionally it is not very much time, to get things into order, to make our peace with everyone who matters to us, and to accept the inevitable. I liked his line about love morphine and whisky. I am still trying to decide which whisky !

We need to look at whole contexts. Science is currently driving us down a route to scavenging a few weeks and months of life at a high cost in the ambition, maybe hopeless but we don’t know that, that along the way the magic cure to cancer will be found. One of his points was that the more we spend on cancer research the more likely it is that more people will die of dementia. That is the worst death on his list of pathways to dying.

There are no easy answers, there may be no answers at all. What Richard Smith has done is, hopefully, to open the debate up a bit wider than it has been. The list of doctors doing that is growing, now we need the patients and carers clamouring to add to that debate, not with emotion, but with a considered view recognising realities, however harsh they may be.

Patients need the integration of Palliative Care and Oncology

At a recent CTOS conference one of the elder statesmen of medical oncology, Dr Bob Benjamin (MD Anderson), came out with a very perceptive comment. Summarising a number of presentations, he suggested that European oncologists treated patients with advanced disease with palliative intent.

“My intent is always curative,” he said, “its just that I am not very successful.”

His use of the word ‘success’ highlights an issue. Is success only about cure? I believe it is important to see success in treating a patient, and cure, as distinct and separate.

An underlying agenda is our reluctance to discuss death and dying. I have lived with advanced cancer for 15 years. The measures taken to control each recurrence have been radical and have been undertaken with curative intent. That has not stopped my mind working hard on the issue of dying, and how I would handle a diagnosis of irrevocably terminal disease. That’s a lot of quite hard thinking. You could argue that my long survival means that I am not representative of the ‘average’ patient. I would merely say that you get what you can from each patient’s experience and I hope that my view has the potential to open up new awareness of what clinical ‘success’ might mean.

I believe the position is simple. Every cancer patient wishes to live a long life but they also want to have a high quality of life, free of pain and other symptoms, and also free from any life-style limiting side-effects of treatment.

As far as the patient is concerned treatment for cancer is not just about the outcome, it’s about the journey. Looking at cancer treatment pathways it is clear that the key to the best outcome is two-fold:

  • Early and accurate diagnosis
  • Followed by primary interventions delivered by specialists

If we then look at outcomes we find that about 50% of patients survive 5 years. The majority of these are cured, if we define cure as the fact that they eventually die of something else.

Most cancer treatment is about extending life. Even for those who will in time be cured it starts with follow-up but for about half of us it evolves into a battle to deliver a cure. Treatment is given with curative intent and patients maintain that belief even when the truth is that it is palliative and the longer it goes on the more obvious that is. Looked at this way oncology faces a challenge. It can be accused of over-medicalising dying, with patients seemingly willing but usually ignorant collaborators.

We have to move on. Palliative care has been rapidly developing and we have to find ways to integrate that knowledge clinically and implement it with patients who are on a pathway to end-of-life, even if that term will only apply years in the future.

But the social and scientific world in which we are working is broken. The patient community is encouraged to believe it has a right to the latest treatment, somehow, and of course the pharmaceutical industry is not going to discourage that.

Current research practice focuses on Clinical Trials whose barely concealed objective is to create demand for drugs at the end of life. They deliver marginal benefit to patients, often with substantial side-effects and at a high cost. In the confusing but important politics of pricing interventions like the Cancer Drugs Fund actually encourage higher pricing from pharmaceutical companies. The CDF was due to disappear when value-based pricing was introduced in 2014. But that dream has vaporised. What a shambles.

What we need to do is to pivot this debate on the patient. The debate must be about each individual, decisions for the individual made by the individual. In cancer the tipping point is the one where the oncologist knows that the disease is unlikely to be beaten. This is the point by which a proper program of supportive and palliative care must already be in place. Patients have a right to a rounded understanding of quality of life so they can take a personalised approach to the treatments which will be offered and make decisions which are right for them.

Four years ago a study in lung cancer was published by Dr Jennifer Temel. A group of patients was randomised to receive either early palliative care with standard oncology care, or standard oncology care alone. The outcome was that not only did the patients receiving the palliative programme have a higher quality of life but they used fewer drugs and lived longer too. Currently the study is being replicated. It is of course not surprising to discover that no pharma company has tried to replicate this study and compare the value of its new drugs with a properly constructed palliative care programme but that is what we need.

Our ambition should be to reach the point where each patient can make considered decisions of their own about treatment, supported by good information, and expert advice which does not, for whatever reasons, seek to talk-up the latest high-technology route in such a way that it is automatically preferred. I would expect many oncologists to say that this is what they try to do already and to be fair, many try hard and they do their best. A few are changing their hospital’s structures and systems, but it is a few. However I believe that a lack of independent clinical specialists focussed on the whole picture of patient benefit, is work against this happening effectively.

We do not study ‘living with cancer’ anywhere near enough and we need to. Our doctors need tools that look at ‘staying alive’ in a rounded manner. Such tools would be based on behavioural, social, and psychological markers identified through observation, questioning and self-reporting.

A philosophical friend of mine uses a simple equation: Objective + Subjective = Oneness

For him it is a description of a spiritual search. In cancer the objective view is disease-centred while the subjective is patient-centred. We should not, however, lose sight of the spiritual context. The emptiness of a biological diagnosis without a holistic assessment of the patient is something which Dr Nathan Cherny illustrates in a recent paper in JNCI (I leave you with the reference).

Small studies are appearing which support this ‘oneness’ approach. An article published earlier this year in CA: A Cancer Journal for Clinicians looked at the palliative care experience at MD Anderson. It concluded that the earlier palliative care was introduced to patients the more they benefited. There was benefit for the hospital too: fewer emergency visits, fewer hospital admissions and fewer deaths in hospital. With patients preferring to die at home or in hospice, an ambition which the NHS is not good at meeting, there must be a lesson here. At the recent NCRI Conference we were also given examples from Italy and Finland where they are making this concept work. Its not rocket science.

A key component in this approach is communication with the patient’s close family, whether that communication is direct with the cancer doctor, a specialist nurse, or some other intermediary. At the moment family communication often only happens through the patient themselves. There is good anecdotal evidence that well-informed patients can make inappropriate decisions about treatment when pressured by a less well-informed family. The truth is that we do not do communication well. It is unfair to look at it as a clinical problem best resolved by training oncologists or nurses better than we currently do. We need intermediaries acting to support the principles of ‘oneness’.

An outstanding contributor to the growing library of material has been the Harvard Professor of Surgery Atul Gawande, this year’s BBC Radio 4 Reith Lecturer. The lectures are available as podcasts. His book ‘Being Mortal’ was also recently published and is a powerful voice for change. What he describes will probably sound similar in principle to the ‘personalised medicine’ approach driven out of biology, genetic knowledge and chemical innovation. As far as patients are concerned individualised care would be a more meaningful use of the term ‘personalised medicine’ than better chemistry. Gawande proposes systematIc approaches, supported by simple ideas like checklists. People have priorities other than longevity for the end of life, he says, yet they are rarely asked about them and the actions needed can be so simple.


N Engl J Med 2010; 363:733-742  Early Palliative Care for Patients with Metastatic Non–Small-Cell Lung Cancer.  Jennifer S. Temel, M.D., Joseph A. Greer, Ph.D., Alona Muzikansky, M.A., Emily R. Gallagher, R.N., Sonal Admane, M.B., B.S., M.P.H., Vicki A. Jackson, M.D., M.P.H., Constance M. Dahlin, A.P.N., Craig D. Blinderman, M.D., Juliet Jacobsen, M.D., William F. Pirl, M.D., M.P.H., J. Andrew Billings, M.D., and Thomas J. Lynch, M.D.

 JNCI DOI:10.1093/jnci/dju321  Words Matter: Distinguishing “Personalized Medicine” and “Biologically Personalized Therapeutics”.  Nathan I. Cherny, Elisabeth G. E. de Vries, Linda Emanuel, Lesley Fallowfield, Prudence A. Francis, Alberto Gabizon, Martine J. Piccart, David Sidransky, Lior Soussan-Gutman, Chariklia Tziraki

 CA: A Cancer Journal for Clinicians, 64: 223–224. 2014.  Early outpatient referral to palliative care services improves end-of-life care.  Barton, M. K.

This is an abbreviated and slightly updated version of a talk given at the National Cancer Research Institute Conference in November 2014.