Tag: personalised medicine

Twenty years on from Calman Hine …

four covers
The new Cancer Strategy is a remarkable piece of work for its breadth and depth of detailing. This could, and deserves to be, as far reaching and valuable in terms of patient benefit as the Cancer Plan of 2000. If you look back to Calman Hine in 1995, through the Cancer Plan, then the 2007 Cancer Reform Strategy you get documents from very different times in the evolution of cancer care all of which help provide the foundations for the new Strategy. The 2011 Cancer Outcomes Strategy is probably best forgotten as rooted in post-election political nonsense.

The important thing about this plan is that it is grounded in well researched and understood realities. It is do-able and it can shift the quality of care upwards as well as handle the growing numbers of patients. The 2000 Cancer Plan had some lofty ambitions; the 2007 CRS trod into unfamiliar territory; this is going to put real buildings on the secure foundations these two pieces of work created.

The six headline priorities map out clearly some key targets and the Government cannot ignore the value that these offer.
A radical upgrade in prevention and public health. Smoking and obesity are the high prioritiy targets.
A national ambition to achieve earlier diagnosis. The ambition set out is that, by 2020, 95 per cent of patients referred for testing by a GP are definitively diagnosed with cancer, or
cancer is ruled out, and that patients get this result within four weeks.
Patient experience on a par with clinical effectiveness and safety. Improving the use of IT, making CNS more accessible, and making the National Cancer Patient Experience Survey an annual event.
Transformation in support for people living with and beyond cancer. By 2020 every person
with cancer should have a comprehensive plan that outlines treatment as well as post-treatment support and care. CCGs must commission appropriate End of Life care, in accordance with NICE guidelines and the Choice Review.
Investment to deliver a modern high-quality service. A plan for radiotherapy machines (linacs) covering upgrading and replacement. A permanent and sustainable model for the Cancer Drugs Fund. A national molecular diagnostics service to guide more personalised prevention, screening and treatment. Plans to address critical deficits in the cancer workforce.
Overhauled processes for commissioning, accountability and provision. Most treatment to be commissioned at population sizes above CCG level. By 2016, a network of Cancer Alliances should be established across the country to address service variation and support redesign.

I really like the way that the three strands of what was unfamiliar territory in 2007 now come together as key elements in the Strategy – early diagnosis, data and information, and survivorship. In 2007 we felt we were being bold tackling three completely new strands of of activity. NAEDI, NCIN and the National Cancer Survivorship Initiative all broke new ground and have achieved so much in seven years that their work is being embedded and taken further in the new Strategy.

The treatment chapters are probably the areas where most interest will be shown. Here the big hidden issues about workforce come to the fore strongly – not enough oncologists, radiologists, radiographers, CNS etc and a complete absence (until now) of any meaningful national workforce planning. The importance of new investment in linacs is something we have seen before and will, if there is continuing absence of planning, see again. These two areas are perhaps a shattering condemnation of the last five years – the absence of planning, the slow but obvious decay of service standards and the long term potential for patient harm. By contrast the big headline issue, high cost drugs, can be glossed over. The call for the Cancer Drugs Fund policy review to come up with a sustainable answer to the problem will resonate with NHS England.

At a personal level I am delighted at the new emphasis on palliative care and end of life care. I have long believed that palliative care and oncology practice were not separate disciplines and that this should be recognised clinically. It is in the strategy. Similarly those of us that work with local support groups will be aware that end of life care is far from integrated. It is usually community based, with lack of clear leadership, multiple provider agencies, leaving patients feeling as though they have been dumped. The message is about getting acts together.

For patients perhaps most striking priority is the call to be heard, to be supported through the NHS jungle, and to be given results as of rightn rather than when it is convenient. There is a strong emphases on research, other patient reported measures, and patient involvement. With CQC getting stronger in the regulatory mix (and a friend of cancer patients influential in this) plus the restoration of Peer Review, albeit with a new name, patients will have watchful allies as well. Stratified medicine is another patient friendly development. The full implications are not yet understood so the need for focus on the opportunity and being flexible to explore it is the message.

Talking of restoration. A National Cancer Team! Cancer Alliances! What a damning statement about the NHS reconstruction started by Andrew Lansley and continued by Jeremy Hunt. The National Cancer Action Team and Cancer Networks were only disbanded in 2013. The fact that they need reinventing barely two years later confirms it as the mistake many said it was at the time.

The Independent Taskforce, chaired by Harpal Kumar CEO of Cancer Research UK, should be congratulated on a remarkable piece of work. It was all done in six months, there was wide consultation,evidence was sought and analysed. The work is detailed and considered. If only every policy had this kind of report behind it.

Cancer Drugs Fund – inevitable signal for wider change

If I remember correctly the Cancer Drugs Fund was set up at the same time as the Government announced it was to establish a value-added base to NICE drug appraisal which could support and reward innovation. NICE was left to consult on it, which it diligently did. However it became apparent that without an agreed definition of ‘value’ and without agreed means and methods of measuring and quantifying ‘value’ this was a fruitless task. The current Secretary of State has made no comment on this issue and his dis-interest in value/pricing has left the Cancer Drugs Fund exposed in an unsustainable way that means that the Government just has to keep putting more money in.

The re-working of the Cancer Drugs Fund, now underway, is long overdue. Professor Peter Clark, the well respected oncologist who chairs it, has been left with the difficult task. One can argue that he has been left with the problem when the politicians who created the fund, and NHS England, which now owns it, don’t want that profile. Sadly for him, I don’t know of anyone better equipped for the task. He is, however, going to field a lot of criticism and it is going to echo into the l;eval system and the political arena.

The Fund was set up to ensure that people with a rarer cancer got an equal chance at new technology drugs as those with more common cancers. It was also aimed at treatment which had been refused NICE approval. Among rare cancers are drugs which have applicability to 50 or 60 patients a year and which are never going to be appraised by NICE because the cost of doing so is unreasonable. These should get funded. At the other end of the scale it is questionable whether a drug which NICE has rejected on the grounds of cost effectiveness should receive funding. However such drugs quite often already have an applicability outside the strict indication for which they were appraised by NICE. It is a factor of timing as much as anything else and these new indications can carry a badge of rarity because they are off the standard track of treatment.

The CDF budget has inevitably burgeoned and the focus on drugs is also threatening to distort the evolving balance of treatment opportunity as new technologies in radiotherapy and new therapy methods employing techniques such as sound and light, appear.

The review was inevitable, the squeaks of complaint from pharmaceutical companies are inevitable and the protest of patient groups is inevitable.

What is the answer?

There is no easy answer but if we look at the longer term history, which provides the context for the CDF, we can perhaps see a way through. The answer is in NICE which has consistently failed to recognise cancer as anything different from other disease/treatments areas. It uses a generic means of assessing the quality of benefit achieved by a treatment in isolation from the whole patient context in which that treatment takes place. Its former chairman, Professor Sir Michael Rawlins, instructed committee chairs to be ready to use their judgement. However the whole NICE appraisal process is quite rigorously focussed on the new drug and its immediate treatment purpose, not the value a drug can offer in the widest patient sense. The cost per QALY* benchmark has not altered in 18 years, not even to reflect inflation, and bandages put over the process such as “end of life” criteria are just that, bandages.

Peter Clark and his team are not trying to be another NICE but they are trying to identify criteria which allow them, openly, to refuse funding for a drug which offers little or no value. Let us hope they don’t try to evaluate drugs for rare cancers with the same sriteria as those for common cancers.

Rather than wingeing the pharmaceutical industry should be taking steps to illustrate indisputable value in their new agents. The current clinical trials model which industry (and to be fair regulators too) are wedded to does not do that. It’s a broken system. The patient community is not short of ideas on how to change things. The medical oncology community is also beginning to question the endless ‘me too’ drugs, treating yet another tumour pathway and offering another month of life with yet more side effects but only in common cancers of course**. Schemes like Cancer Research UK’s Stratified Medicine programme are welcome developments which will help change the shape of treatment and of drug regulation. Bring ‘em on.

 

*Quality Adjusted Life Year – a statistically derived measure of patient benefit built on factors which are so opaque you need a degree in Health Economics to argue them.

**Because that’s where you get the quickest payback.

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.

Refs:

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.