If you want Patient Involvement there are no compromises

There is a new healthcare industry. Patient involvement became inclusive as ‘patient and public involvement’ (PPI) and is now rapidly becoming ‘public involvement’. In research it is acquiring an energy, a workforce, an academic community all of its own. We have advice from regulatory bodies, published standards, endless guidance and retrospective analyses of experience in small very specific case studies. We have started to see systematic reviews, with inconclusive conclusions. We have a journal of our own. We even have privatisation as an issue. It is however a uniquely British development and I would argue that we have lost sight of what it is really all about.

Let me put my cards on the table. I attended my first research meeting as a cancer patient in 2002. I am a journalist by background with a long management experience. I am curious, analytical, occasionally creative, and sometimes abrasive. In 2005 I co-wrote the patient involvement plans of UKCRN, the first iteration of what has become NIHR CRN. I attended the DoH interview from which the contract was awarded and took the part-time role of Associate Director for Patient Involvement. I had to relinquish that in 2007 when I required further treatment for my cancer which left me disabled.

I have spent a lot of time in the last year with the largest European cancer research organisation, EORTC. The European Organisation for the Research & Treatment of Cancer is not an EU institution although it does get some project funding from EU sources. EORTC is a Belgian charity, founded over 60 years ago, is currently running or in follow-up with about 200 clinical trials, and employs about 200 staff – methodologists, statisticians, doctors and research scientists. It has partnerships across the world, engages hundreds of European clinician/researchers with specific tumour specialisms and has an influential policy voice. EORTC is considering how patient involvement can be brought into its core activities, there is already some involvement in the tumour study groups. Note that this is patient involvement, there is no role for the public.

In 8 of the last 13 years I have been a patient member of faculty for the MCCR Workshop run in Europe. There is a similar workshop in the USA. This Workshop is an intensive six day course for young oncologists from all around the world. They all have an ambition for research in their clinical career. About a quarter of them are working in the NHS. Many of these young doctors have never talked with a patient about their research before – the exceptions being those from the NHS. None of them however wants a non-patient to be involved with the study they develop on the course. They can accept that I may not have experience of the specific tumour type they are proposing to research but they recognise that I bring a viewpoint from an experience they have not had and that I understand issues in a different way although it is one which is relevant to their plans.

This experience is making me focus quite hard on what I think is essential. I want to get away from the plans that large-scale UK organisations seem to have to simplify terminology and create ill-defined systems which drive relevance out of the original intent.

Our intent back in 2005 was patient involvement. There was a lot of discussion about the use of the word ‘patient’. Cancer was using ‘consumer’ (as NCRI still does) because it allowed carers to be included, in mental health the term ‘service user’ allowed inclusion of family/carers to address some of the communications challenges which exist. Whatever the term actually used the binding principle was that of the ‘lived experience’ – although we had not defined that term then.  The objective was to replicate in the wider healthcare research community some of the benefits which the introduction of a structure for patient involvement had achieved in cancer research through NCRN.  That in turn had learned from HIV/AIDS research in the 1990s where the Medical Research Council had opened its doors to patient involvement.

Patient involvement in research has a clear objective, to enable better research, relevant to patients, which can be explained easily to patients, implemented more readily and can realise benefits for patients more quickly. When I joined my first cancer research meeting in 2002 that was the aim and it remains the aim.

So how did the primary need for ‘patient involvement’ become subsumed, its purposes obscured, into a woolly agenda driven out of organisation speak and something ill-defined called ‘inclusion’? I will let others answer that question. Then there is that appalling trio of PPI. Get rid of it.

Lets get back to core principles. You can have as much or as little ‘public involvement’ as you like, just get on and do it and don’t bother me with it. 

We should focus on ‘patient involvement’ and resolving the challenges it faces. The issue of identifying impact should be rejected. No research group assesses its staff on their ‘impact’, the view is more rounded than that.  Involving patients is a cultural issue, it represents change, and such change can only be assessed by looking at the organisational culture in a structured analytical manner.  As far as I am aware only the NCRI has ever done this, and then perhaps not consciously.  Considering the performance of individuals is a different matter and is closely related to the challenges of recruiting, training and supporting patients to be involved. We find it difficult to identify and turn away poor performers. Many organisations, including NCRI, have solved all of this but the indications are that each resolution is specific to each situation. We can do this. It is not easy but it can be done. The will is there among researchers and among patients, if only you can get to them, given all the obstructions that are put in the way.

People with the ‘lived experience’, not the public, will change research for the better. The easy term is ‘patient involvement’ but we have to treat that as a generic, accepting that it needs refining in different situations. Properly implemented involvement of people with the lived experience is what we must be talking about and getting on with. 

Lets not lose sight of this.

Patient Involvement is NOT a sub-set of Public Involvement

I happily acknowledge the experience and thought about this issue which others have expressed but I do not want us to lose sight of the uniqueness of patient involvement. I care little about public involvement, I want to see patient involvement where it matters in areas where research can improve the quality of treatment and care. The differences with public involvement are more than semantics, it is about ‘lived experience’.

I also acknowledge that not everyone likes the term patient and that differing disease contexts use slightly different language. That is why the phrase ‘lived experience’ is so important. It includes carers, especially in those diseases and contexts where patient communication is problematic, where children are concerned, and in diseases where survival is poor.

In much of clinical research unconditional patient involvement is necessary. The lived experience, viewpoint and thinking of patients and carers cannot be provided by anyone else, can influence study design and provides important insights throughout the life of a clinical trial.

Whether any single person can be representative of ‘lived experience’ is a matter for discussion within each context. It is important and should be thought through by patients and researchers together prior to starting work on the study. It is of little use recruiting me to comment on a bone sarcoma study which impacts on teenagers, I am a soft tissue sarcoma patient and my teenage years are distant past. 

It is worth recalling that when the UKCRN was formed back in 2005 (it is the precursor to NIHR CRN CC) it had an Associate Director for Patient Involvement. Public involvement was a sideline issue.

So let me be vocal and unequivocal, patient involvement represented by the ‘lived experience’ is not a sub-set of public involvement and should never be gratuitously lumped into it for academic or organisational convenience. 

Your diagnosis madam – collateral damage

No cancer patient should be seen as collateral damage after a clinical procedure which diagnoses  otherwise unexpected cancer.

That is what happens with most uterine leiomyosarcoma and some other gynaecological sarcoma diagnoses. These patients are among the worst treated cancer patients because not only are they usually diagnosed after radical surgery, many are subjected to inappropriate adjuvant radiotherapy with consequential long term side effects, and some are given adjuvant chemotherapy which has no evidence of benefit – except perhaps to assuage the guilt of their gynaecologist.

The gynaecology community has been challenged over these issues yet takes few steps to try to answer the problems. In the UK we are moving forward by requiring all these patients to be referred for joint care at diagnosis by a specialist sarcoma team. Hopefully this will reduce the burden of adjuvant therapy, improve follow-up and open up access to appropriate new sarcoma treatments. This has met consistent resistance but the NHS is enforcing it. Elsewhere in the world it does not of course apply.

Gynae sarcomas account for less than 1% of all gynae cancers. Many are hidden by uterine fibroids and are only discovered after surgery. Sometimes that surgery breaks up fibroids and distributes sarcoma cells around. As far as gynaecologists are concerned that is ‘tough luck’, nothing they could have done better, disease spreads, patient dies (eventually), collateral damage.

Where there are problems in healthcare there is usually research. Around gynae sarcomas the only research is with drugs for post-operative adjuvant therapy or advanced disease, valuable but not enough and nothing yet found to make a significant difference. Gynae and sarcoma specialists collaborate in these studies. There is no research into trying to identify a biomarker from blood or imaging to try and achieve a diagnosis before surgery where fibroids are concerned. There must be other ideas. No-one is ‘thinking outside the box’. Sarcoma clinicians cannot do that research, they don’t see the patients until they are already collateral damage.

I first met gynae sarcoma patients online in email support lists. I have known several personally and I know some survivors. Of course for some the journey has been supported by good and caring clinicians and its not all bad news. But it is a hidden issue, no-one talks about it. There are so few of these patients to start with that creating a campaign has been impossible. We need to energise this in different ways, its not enough just to be shouting from the sidelines.

The first step is for healthcare systems to openly recognise this problem. In the UK we are slowly moving forward. The NHS is taking steps because of pressure from the sarcoma community. NICE needs to recognise that it has failed these patients. NIHR and other research funders should identify and fund a priority. NCRI should be encouraging researchers. Women’s cancer advocates need to wake up to this challenge. But that is just the UK.

Lets care about collateral damage, research could solve this.

Journal publishing – get rid of the pay wall

There is a situation which we, as patients, have been slow to challenge.

If I say that patients agree to enter clinical studies, which take data from patients, and reports on those studies are then published in medical journals after due review processes have taken place, and are then put on sale, I wonder if you see the problem.

If we as patients want to see the study result, review the data etc we have to pay – usually a fee around $30, sometimes more. We don’t buy because we do not have the resources. We cannot get free access because we do not belong to a body which licences access for its members through Athens or Shibboleth. So we are left with the abstract, at best. Fortunately a number of journals are Open Access, allowing anyone full access to published reports. Some also offer Open Access as a paid option but notably the High Impact journals will usually only offer Open Access if the study sponsor pays a substantial fee up front.

Patient advocates and those relying on understanding research are disadvantaged by the publications system. Some authors are generous and subject to keeping quiet about it let us have a copy of their study report. Sometimes we are able to find a print copy, take photos on a mobile phone and thereby have a reference copy, but that is getting silly isn’t it?

I started to wonder what the ethics of all this are. Patients freely enter studies, unpaid. Their data is gathered, analysed and written up under an ethical process. If it is then published and they cannot access it, is that ethical? The actual patients in that study may well get a copy of the report from the study team which might answer the direct question, but patient advocates and representatives who could use these data in their work for the benefit of patients do not have such access. Is that unethical, or just hard b***** luck?

What makes this worse of course is that patients and the public fund a lot of this research, whether through taxation or charitable giving.

We have to recognise that publishers have to earn a living and that there are various business models available to them in practical terms. We can only applaud those who have an Open Access policy.

It has to be better than this. I cannot see an easy route through but I hope someone can grasp this nettle and help patient representatives get better informed and become more effective.

Personalised medicine and accelerated access

Once upon a time a new cancer drug achieved marketing authorisation after randomised controlled trials demonstrated effectiveness treating carefully calculated numbers of patients with a given disease or condition. The critical patient outcome for the trials was a comparison between the experimental and control arms using a measure of patient survival which gave a high degree of statistical significance. It was also important that safety was demonstrated and that side effects were tolerable and manageable.

We are entering an era where a drug with a specific mode of action for a specific genetic alteration can achieve marketing authorisation in an accelerated process after small clinical studies, offering no comparative data, using endpoints where patient benefit is unclear, and without long term measurement of issues such as survival or side effects. 

We made this transition, seemingly without open debate amongst stakeholders. The move is justified by the term “personalised medicine”, which in its fullest sense would include radiotherapy and surgery but is usually focussed exclusively on drugs. It has been carried along by enthusiasm and hype and it requires genetic testing, the demand for which is driving costs down. Technology is proving disruptive.

But patients have lost something in this transition. The science behind repeating studies, using control arms, good statistical practice, endpoints with real meaning etc has been built-up over years. It is robust good practice and provides a very high level of certainty that authorised drugs deliver patient benefit safely. Based on the data at registration these new drugs promise hope for a few but uncertain patient benefit overall.

It is important to acknowledge that when a treatment has a high probability of treating a disease effectively then practice needs to change so that relevant patients can be treated without delay.  We are now recognising that in doing so we lose important evidence which is very relevant to patients and society, even if it is less important to pharmaceutical companies.

What about dose determination, treatment sequencing, duration of treatment, combination therapies, toxicity management, quality of life, patient compliance? They all get forgotten in the rush to get the treatment into routine practice where we are reassured that any problems which arise will be handled by doctors. Perhaps, but their experience is not shared and collectively analysed as it would be in a study. Evidence based medicine it is not. Nor is it truly personalised medicine, it is simply drug-centred medicine.

However, patients want it. They want the tests and if they are eligible they want the treatment.

Some patients will be denied by cost. These drugs are expensive. Larotrectinib, approved by the FDA for a rare mutation in a wide range of cancers based on data from 55 patients, is estimated to cost over $330,000 a year – assuming anyone lives for a year. Compassionate and discounted programmes are available but no major HTA body has yet got into discussion with the drug company. There will soon be tales of patients selling homes and using crowd-funding to raise the money for drugs like this.

We must move fast to apply scientific process and rigour to personalised medicine. It is not about denying treatment to anyone. Drug companies must not determine what happens, much as they might wish to. At present they are succeeding. We must change the balance. Put patient interests at the centre of drug regulation, not pharma interests.

So what are our interests? Safety (the minimum effective dose), side effects (nature, treatment, duration), duration of response to treatment (combination therapy, therapy sequencing), longevity (real life outcomes), quality of life (in all its dimensions), holistic issues of care, cost.

Establishing this kind of treatment optimisation is a theme supported by European research organisations, professional associations, patient groups and by a considerable body of influential MEPs. The move calls for protocols to guide treatment and for data to be gathered centrally for all treatments which use an accelerated approval process. By gathering data prospectively according to a protocol we can avoid bias and a picture can emerge dynamically as a treatment gathers numbers of patients. Quite separately the FDA has suggested a form of ‘synthetic’ control group based on historical data to empower comparison. 

This might result in treatments which are not effective, and we might see treatments which patients dislike, using patient reported outcomes. These treatments could be withdrawn, that would be an innovation. This kind of treatment optimisation approach importantly provides a framework for testing lower dose levels than were used in the trials for drug registration. This is not a favoured idea of drug companies either.

Support Treatment Optimisation, it is in our interests as patients.

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?

Pressures are building on patient advocacy

Two pressures are building on patient advocacy. We do need to draw a line here between patient advocacyand patient involvement. The former are patients or professionals working towards a specific objective such as getting a new treatment approved, or raising awareness of an issue amongst politicians. That can be correctly called advocacy. Patient involvementis the drawing in of patients with ‘lived experience’ to a structure so that their experience based input, whether considered or ad hoc, can be taken into account alongside other views. There is of course no clear boundary line, partly because the same patients can be doing both. The two terms can be confused and issues which apply to one can also be inappropriately applied to the other.

What are these two pressures, and why are they unique to theadvocacyagenda?

The first is the influence of pharma companies, the funding they provide to patient charities and the influence that the funding opens up. There are regular, sometimes well informed, articles in both professional journals and the wider press raising concerns.

These concerns are real because recent history shows us what can happen. One example was a breast cancer charity that accepted funding for a policy post which actively promoted that pharma company’s drug to politicians and created patient activity supporting it. Quite often funding was sheltered through a local PR company.  Matters became more subtle in the mid 2000s and funding related to ‘projects’ rather than core activities. One not unusual ‘project’ was funding charity staff to attend an educational conference at a foreign and very pleasant venue, all costs covered. Regulations which required openness about funding followed and pharma companies now declare who and what they have funded in great detail. Charities have no such regulatory requirement from the Charity Commission although many do declare what they have received and for what purpose. Some healthcare charities will not accept industry funding at all and declare that policy openly. It is not tidy and while this lack of openness remains there will always be critics eager to point fingers at one or the other party.

Where this becomes an issue is with regulatory affairs. Patient groups and charities are often asked to provide an input to regulatory discussion, usually a paper, sometimes representation at meetings. Questions about financial influence are part of the preparation and a declaration of potential conflicting interests is requested. Some regulators are less searching than others.  Charities all have a different approach to completion of these requests and I do not believe that any regulator has a requirement that the CEO must sign the declaration.  Understandably charities which have little or no involvement with industry find these forms tiresome, not always understanding their purpose. The aim is not to eliminate input but to provide a perspective which reflects the real-life of working with and supporting patients. I have spoken with regulatory committee members who tell me they weigh up what is put forward by patient advocates at a meeting, taking into account the declared potential for conflicts of interest, but not discounting the input provided.

It is time matters were tidied up. The Charity Commission could regulate how charities declare commercial grants in their accounts and charities themselves could carry a declarations page on their website, as some already do. The regulators could work together to have a common declaration approach and individual advocates should make declarations (already required by some regulators) so that their personal situation is clear.

If there was this kind of clarity pharma company lawyers might be able to stop twitching so much and make the matter of financial support where it is proper, appropriate and open happen more easily. In addition researchers or journalists who get a ‘bee in their bonnet’ about inappropriate influences could be quietened, although I doubt they will ever be eliminated and, to be fair, the questions need to be asked.

The second advocacy influence is about understanding evidence-based medicine. It overlaps with the first issue above because some pharma companies could be accused of fostering a preparedness to ignore evidence. More about that in my next contribution to the debate.

 

 

Questions, questions … but can we get them answered?

Academic interest in patient involvement has been dominated recently by evaluation. We at last have a growing recognition that involvement is not an intervention, like sticking a needle in, but an influence over process which acts subtly and sometimes covertly to effect change over a period of time. There is rarely a single identifiable point of ‘impact’, the change may be subtle and only evident through reporting by those involved, researchers, managers, funders and patients themselves.

So what is the next challenge for our researchers? There certainly seems to be plenty of  content, the challenge is finding the questions to resolve.  Lets try a list of unanswered questions, not worded yet as proper research questions and to be fair, some might not make it that far:

  • How do we draw the lines between patient and public in involvement?
  • What is “lived experience” for the purpose of defining patient involvement?
  • How do we ensure we get the right people for the right role?
  • How do we report involvement as a key element of research?
  • If we sack an involved patient how do we report that?
  • What questions should we ask researchers to help find the value of involvement?
  • How should we ask researchers those questions?
  • How can we remunerate involved patients without creating conflicts of interest?
  • Are there ethical issues we should address to empower more effective involvement?
  • Is effective involvement pragmatic, or driven by philosophical and cultural change?

That’s ten to start with. I could go on.

One of the challenges is getting these questions, and others like them, aired. In this world of evidence based medicine research proposals are usually supported by a literature search from which the prospective funders can identify that the research question is a valid one. Thus research builds up in an organic manner, with a traceable provenance. In patient involvement there is little evidence, as yet, in the literature with the result that provenance is largely impossible to identify. One of the challenges for researchers who wish to raise questions like these, even as a commentary or discussion point for a journal, is that there is no evidence to point to that the question is important. The editor will not publish. It is a chicken and egg situation.

I am suggesting something which steps outside the usual ‘evidence based medicine’ criteria. As usual, remember that this is a patient making the suggestion, I have only one interest – getting questions answered so that patient involvement improves.

My appeal is that those journals which are happy to report or commentate on patient involvement, few but growing in number, encourage such new questions to be aired and debated relying only on the evidence that there is a question which informed people are discussing. The aim is that researcher groups will pick up on these ideas and have a reference base, however small, to work with when they seek funding for a full research project.

In search of creative input for PPI

Patient and public involvement in research has been gaining a lot of attention in the professional journals recently. I am not sure whether this is a turning point or just a matter of coincidences. It seems that either researchers have woken up to the idea that this is an area where research is needed (it is) or that it is where opportunities lie for writing papers which enhance their CVs.

The paper which seems to have been written solely because it could be written, yet offers nothing to our understanding of patient and public involvement, could be called futile.  However, a paper which is has futile conclusions is not necessarily a bad paper, it may just point to a shortage of evidence. The paper which predictably has futile conclusions, even if the methodology employed is well-validated and robust, should be identified by peer review and rejected. The pursuit of futility is not science.

One problem here is that peer review in most professional journals rarely uses people who work in, or genuinely understand, patient and public involvement. The journals just don’t know who they are and most cannot be bothered to find out. So the chances of a reviewer challenging a paper as having little or no value is small. Usually such papers are sent to methodologists to review, a task they will relish, grist to the mill so to speak.

One of the reverse problems in patient involvement is that a good paper seeking to identify new understanding will be criticised by the methodologists because there is a lack of evidence to support its creative thinking. The lack of evidence is because researchers have not sought it. It can be partly attributed to the fact that creative understanding of patient involvement by those who do understand it is rare, and the ideas on which good research can be built are not aired publicly because journals will not publish them.

Quite a challenge!

We have recently seen a paper looking at “synthesising conceptual frameworks” for PPI in research. It is a commentary by methodologists on a systematic review first published in 2013 which put forward such a conceptual framework and identified priorities for research. The commentary is highly critical of the earlier work and goes on to state; “…this critical appraisal has usefullyhighlighted that there is a continuing, even urgent, needfor a more rigorous synthesis of conceptual frameworksfor PPI.”  The commentary offers no ideas about further research, suggesting that its authors actually know nothing about PPI, confining its suggestions to a need for “more robust conceptualisation” of  PPI frameworks.

From the viewpoint of one involved in patient and public involvement this verges on self-justifying nonsense. What PPI in research heeds is more people getting on with it, professionals and lay people working together.  Our ‘frameworks’ need to be created, reviewed, changed, evolved, reported, and all done in a timely way so we can all learn together.  Conclusion noted, this paper adds nothing. There may be other viewpoints but from that of PPI this was a research paper written for the sake of it. I hope it looks good on a CV.

Contrast the approach of another recent paper, also a critical commentary. It looked at the NIHR Standards published earlier this year. The Standards are seen as a step forward but many limitations are identified. Some are “simplistic”, even “optimistic”, and there is a need for guidance which can make implementation relevant to the objectives of PPI in any organisation or situation.  The conclusion states that the Standards: “… fail to address fundamental questions about when, why and with whom involvement should be undertaken in the first place.”  It goes on to add :“By addressing the justifications for patient and public involvement up front and acknowledging that there are contexts in which some types of involvement are inappropriate, a future version of the Standards or another guidancedocument could provide researchers with the overarching clarity they need.”   Here is some real value-added.

Both papers are addressing the need for better understanding of PPI based on good evidence. I would maintain that we must not be confined by traditional evidence-based approaches.  This is not about medicine but is about people.  We need to develop research ideas through creative understanding of what is happening and what has happened in the world of PPI in research, through understanding the people involved.  If we are to move forward effectively we need those creative ideas to help researchers identify where evidence about PPI can add value, to help develop the new ‘frameworks’ – they will start conceptual but require practical experience so they can become real.

That will keep up the momentum which PPI is developing.

The two papers referenced:

Synthesising conceptual frameworks for patient and public involvement in research – a critical appraisal of a meta-narrative review    David Evans, Noreen Hopewell-Kelly, Michele Kok and Jo White   BMC Medical Research Methodology (2018) 18:116    https://doi.org/10.1186/s12874-018-0572-0

National Standards for Public Involvement in Research: missing the forest for the trees     McCoy MS, Jongsma KR, Friesen P, et al.     J Med Ethics Epub ahead of print: [accessed 12thOctober 2018]      doi:10.1136/medethics-2018-105088

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.