A SPECIAL AFTERNOON

I had the privilege and the pleasure earlier this week of chairing a patient group meeting. You might think I have done a lot of that so why should it be so special this time. It is true, I have chaired and been part of patient groups quite frequently over more than 20 years but this one was special for a number of reasons.

First, it was of course a Zoom meeting – a screen full of unfamiliar faces, intriguing backgrounds, challenges with mute buttons, and some variable framing.

Secondly all those attending had been treated for a retroperitoneal sarcoma (RPS). This is where a sarcoma occurs inside the peritoneum. These tumours are rare, accounting for less than 300 cases a year in the UK. They can become extremely large, rarely causing pain until they put pressure on organs, thus often being diagnosed late. The main treatment is surgery which can be extensive, patients can lose organs compromised by the tumour. During the last ten years the surgeons around the world who specialise in this demanding surgery have come together as an international working group (TARPSWG), shared data, and have agreed that radical approaches are needed to deliver the best outcomes for patients.

I have only ever previously met one or two at a time of RPS patients. At this meeting there were 15, some initially treated more than ten years ago, a couple only treated last year and still getting to grips with their recovery.

And third, most of these patients had been treated by one surgeon – a member of the specialist surgical team based at the QEII Hospital in Birmingham. He was in the meeting too, along with members of his team and a research statistician.

This team are leading the development of a clinical study about follow-up. The TARPSWG group has recognised that there are no standards and it is impossible to identify the most effective approach to follow-up currently used around the world. For some patients it is probably unnecessarily intense, others may benefit from greater intensity – no-one is sure. Some patients have higher grade tumours (more aggressive), others are low-grade, and there are also variations in tumour histology.  Hence the work on the study.

The purpose of the meeting was to explain the background to the study, what is currently being planned, to get patient responses to some specific questions the study team have, and to see what issues the patient group raise and want to discuss.

Even though all of them were RPS patients the individual experiences were extremely varied. One person had had a primary tumour which weighed 35lbs (15.8kg), another described theirs as “large as a mango”. Several had serious organ loss – kidneys, spleen and other organs. A few had had recurrences and were in continuing treatment or active surveillance. It was a genuine cross section of lived patient experience. A couple shared their screen with a partner, both of whom also contributed valuable comments and ideas.

It was a lively meeting and the input from those assembled on screen was truly remarkable. I assured them at the outset that no question was ‘silly’, just give out whatever came to mind. Inevitably there were very individual issues expressed and we had to ensure that some of these were side-lined to become a personal call from the surgeon. But the readiness to understand concepts which are not in general public debate (eg randomisation, timing of questionnaires) and to constructively consider what intensive and less intensive follow-up actually meant for patients, was tremendous. I was especially encouraged by the willingness to grasp what ‘quality of life’ meant and how researchers go about assessing it.

I have always said, never under-estimate what an involved group of patients can bring to a study, and it was as true here as it ever was. The range of responses and issues raised will provide an invaluable input to the study team as they develop the idea further.

A truly special afternoon and I have to thank all those who took part for the privilege of meeting them and also the Birmingham team for the invitation to take part.

Sarcoma, COVID, science, BREXIT and Trump – what a year

I have not written in this blog since January of 2020. It has been some year and while I have been busy, the COVID-19 tragedy has been unfolding and our government has been deepening its self-created chaos around Brexit. Meanwhile the politics of the world have been changing. It is impossible to disentangle it all but I will have a go at summarising the year.

I have been busy because the world of sarcoma and canecr advocacy more generally doesn’t substantially change despite the pandemic. With sarcoma we have a disease which is rare, heterogenous (about 150 different histologies), and inherently complex. It is difficult to diagnose, requires specialist knowledge to treat and effective medical treatment options are scarce. There is limited access to research for patients and many studies of new treatments fail. Effective patient advocacy to help create change is desperately needed around the world but is largely unfunded. My life in the last four years has been about travelling Europe for meetings and events. That shuddered dramatically to a halt in March. I have not missed airports, life has been interesting on Zoom and Teams but I have missed the conversations over coffee or lunch.

The COVID-19 pandemic has been tragic. Our government has consistently acted too late at the cost of lives, it has splashed money into the hands of incompetents and crooks (known as friends), ignored existing skills in the community (to benefit its friends), and its leader has lied, bumbled and blustered throughout. Ministers have ignored the importance of giving the public ‘understanding’ and relied on them to try to process information for themselves. Giving clear and simple explanations is not patronising, giving simplistic ones is. What we needed was the advice to avoid closed unventilated space, avoid crowds, avoid close conversation – instead we were told to wash our hands, wear a mask, and keep 2m away from others. One is about contexts of life and prevention and is simple. The other is about self-protection without any reason or context and is simplistic. No wonder you got crowds, people gathering indoors and politicians having unmasked close chats. In addition there has been an almost pathological addiction to big populist promises that cannot be kept, not least is the world-class test and trace disaster.

What stems from this could be an exciting opportunity for science, which has seen itself in the headlines more than ever before. The politicians mantra of “following the science” (which it soon became obvious that they weren’t) subtly morphed into “being advised by the science” (which allowed them to openly ignore it). Fortunately we do have a lot of honest scientists who will not be over-awed or bullied and who can communicate effectively. If we are lucky the demonstrations of the influence science can have should excite more young people.

The treachery of Brexit has also been a huge collection of extended promises. Lies built upon lies, reality ignored, vain hopes sold to the electorate which then evaporated, ideas of quick agreement became extended negotiations, irrational short term solutions disappeared as the real world emerged – it was predictable. The extended promises of 2016 were unfounded and sadly those who manufactured those lies came into power determined to deliver their desired outcome without any of the benefits they had similarly promised and causing a level of damage not done to the British economy and social-life since the 1940s. A Cabinet of self-serving Westminster cowboys, yes-men too lily-livered to stand up to bullying and admit to what was right. And now they are threatening to use gunboats in the English Channel!

Deepening the tragedy on the world stage has been the pantomime dame of American politics. The cringing performances of a coiffed man with a 10-year old’s vocabulary, the emotional sensitivity of a steam hammer and a view of the world that comes from one of those crazy mirror stalls at a funfair, moved us beyond laughter, to tears, to sending condolences to American friends. Welcome to the new 80-year old king, he will need to show steel to steal the show as the next act unfolds.

Oh dear, what a year. Well that’s it, I’ve had my say.

The search for patient benefit – a New Year challenge

If you have read this blog regularly you will know that I am not entirely comfortable with what is going on in cancer clinical research at the moment. I can appreciate innovation and recognise that scientific developments which may benefit patients need to be tested in clinical trials. However, stresses are showing.

When I started learning about cancer research, things were fairly simple. That wonderful teacher Derek Stewart defined patient involvement as learning to balance benefits and burdens. We needed to listen, question, challenge when appropriate, and when we had experience or information which was relevant, to make our voice heard, politely. These are fine principles and experienced patient advocates everywhere recognise them.

Targeted drug therapies have been challenging established models of research. The pinnacle of a drug’s journey to market used to be a Phase 3 Randomised Controlled Trial (RCT). Regulatory approval followed trial success and started the process of getting a new treatment into standard clinical practice. When involved with RCTs the concept of equipoise was an important one for us to understand; learning about endpoints, outcome measures, inclusion and exclusion criteria, side effects and their management was all do-able provided you were prepared to learn. Then you learned about ethics, you got into discussion about placebos, early stopping rules, systematic reviews – the list grew and grew. There were plenty of people happy to help with all the learning.

When you needed to look at research in a more strategic manner, as happens when you work on a programme of research or developing clinical guidance, you find that there are new issues. Questions can be harder to define and not always asked or answered in the systematic reviews. You need to ask questions in order to discover the real questions you need to ask. You find the professionals take certain knowledge for granted and may not see why it should be questioned.

So what is it which worries me about clinical cancer research today?

Results from Phase 2 studies now unlock registration leading to NICE opening up interim approval and funding for NHS use. The Cancer Drugs Fund (CDF), soon to be extended as an Innovative Medicines Fund, enables drugs which have not been tested in an RCT to be evaluated within standard clinical practice. Evidence on that experience can open up full regulatory approval. This journey is replacing the RCT.

Perhaps the most challenging current research is the work on genetically modifying T-cells, the body’s natural immunotherapy. There has been a lot of publicity for the technique, which has benefited a few patients, although the long-term benefits are completely unknown. CAR T-cell, as it is known, is a complex technology, very well explained on the NHS website if you need to know more. It is also very expensive.

The NHS centres conducting the CAR T-cell studies on diffuse large B-cell lymphoma (DLBCL), have been charged by NICE with reporting on their work by March 2021. An initial report was presented at the recent ASH Convention in Florida and the results so far are disappointing. Sadly trials in a lot of targeted drugs and immunotherapies are disappointing.

This is where questioning comes in. This is the pinnacle of personalised medicine. These patients have advanced cancer which is untreatable with standard therapies. An assessment of how long patients survive and what other treatments achieve are questions which need an answer, together with a proper appraisal of quality-of-life. There are further questions about biomarkers which could predict response, predict side effect problems, even perhaps predict a cure. All these are questions which the scientists involved know about and will try to answer without an RCT.

But there are bigger questions. Should these therapies be tested at an earlier stage of disease? Would similar sums spent on maintenance therapies which hold relapse at bay deliver better and more patient-centred care? Never mind value-for-money. Are we being dazzled by our own cleverness?

We have seen that with these new treatments a truly prospective RCT may not be feasible. We need a new rigour in our system which can unlock patient benefit. I believe the answer lies in the NHS itself. We have 150 cancer hospitals using the same data standards for recording cancer clinical performance. We must engage the real world of clinical practice more widely in the research challenge. There are many cancer treatments where research in the real clinical world could add value for both patients and the NHS. We can gather data in a structured manner on every new treatment. Consent for treatment allows your data to be used for research.

This is an environment where we can go further. We could construct prospective studies, without relaxing our research consent practices. We should be looking at the sequencing of new drugs where more than one is available for any condition, accruing patients into the studies from any hospital. We should be looking at studies of combination therapy, new-with-new and new-with-old. We should be looking at the ‘minimum effective dose’ of a drug rather than blindly accepting (as we do) the developer’s recommendation based on ‘maximum tolerated dose’. We could be looking at studies using known effective drugs earlier in the course of disease, or as maintenance therapies when there is no disease present. We could look at new indications for approved drugs. We should build studies like these with patient involvement, questions defined by patients should be answered. Patient Reported Outcomes should be at least co-primary endpoints, we might even get ‘patient preference’ accepted as a standard outcome.

Industry has no real interest in running studies of this kind. Their interest is commercially driven despite high-sounding words about better patient outcomes. Their real world studies that lead to converting interim drug approval into full and funded NHS approval are run in their commercial interest. Their studies receive funding through the Cancer Drugs Fund, guaranteeing the manufacturer an income, but there is no automatic funding for real world NHS research with a patient-led focus. That funding has to be found. As therapies are already funded through CDF or NICE approval treatment would not be a study cost. A study will all be about prospectively gathering and analysing data. The NHS is capable of doing it and is in the NHS’s interest to get such results.

A focus on high technology in cancer research is important but it must not be the primary focus. It is great for creating headlines and makes wonderful TV but we have seen that very few patients benefit and costs are phenomenal. I am not saying we should not do it but I am saying that we are failing to do the other things which the NHS is uniquely capable of delivering. Cancer research is doing a dis-service to everyone, not just to cancer patients, if it does not shout about this challenge.

We need routine research within clinical practice, we need real world evidence, we need it to be driven by patient reported outcomes. Given the growing cost of drugs I believe that every penny invested in such research could be returned in value to the NHS.

A Busy Autumn

Five events, two months, some important messages and valuable feedback. It has been a busy autumn this year. Preparing a presentation takes time so these trips have taken out a huge chunk of time, although to be fair two of these events did not involve a presentation. In one case I chaired a panel and another involved a dinner and a Board meeting. Each event also gave opportunities to meet and discuss matters with other patient advocates in both informal and more public settings.

Its worth looking at each of these events and seeing if we can draw some conclusions. Its also worth noting that a patient is often included in a programme a bit reluctantly so ensuring that what we say is well grounded in evidence that cannot be ignored is an important step to take. The character of the organisation running the event is therefore also an important background consideration.

The first of the five meetings was the ECCO Cancer Summit. ECCO has evolved as an organisation developing a clearer focus than hitherto on influencing policy within Europe. Within its structure it has a Patient Advisory Committee which makes significant contribution to the work which ECCO undertakes. The PAC is chaired by Ian Banks, a patient from Northern Ireland, and includes in its membership a varied and experienced group of advocates from all across Europe. The Cancer Summit was created to focus on some key issues and to agree how they should be represented. It also gave the chance to debate some issues at an earlier stage in their development, when clarity about what is involved is necessary. Patient integration in the Summit was excellent.

I co-chaired one of these debates with Dr Denis Lacombe of EORTC. The issue is the growth of short-term clinical studies resulting in regulatory approval and the loss of the scientific rigour which comes from Phase 3 Randomised Controlled Trials and long-term studies post-regulatory approval. The demand for evidence of value from Health Technology Appraisal (HTA) agencies which approve funding for national healthcare systems, and from patients wanting better quality information, creates a need for evidence from routine clinical practice (Real World Evidence – RWE) in the absence of prospective evidence. There are, as yet, no structures of any kind to support such studies, which take place ad hoc, have no consistent standards, are open to bias and dis-information.

What did our debate conclude? There is a need for industry, academic and regulatory organisations to work together to set RWE standards and create a route for accountability. EORTC’s Manifesto on Treatment Optimisation provides a strong foundation for a such collaboration. The ball is now in ECCO’s court to move this forward.

Then came ESMO, an event of such scale that describing it is almost impossible. Close to 30,000 delegates, up to 12 parallel conference streams, the largest cancer focussed exhibition in Europe, altogether astonishing. I followed streams on sarcoma, quality of life and PROs, patient involvement in clinical care and research, survivorship and trials methodology. Quite difficult prioritisng because there were many schedule clashes.

I spoke in a session on ‘Surviving a Cancer Diagnosis with a Good QoL’. Its a very open topic and the speakers covered a wide range of issues. A highlight was the work of Isabelle Lebrocquy from the Netherlands on cancer and employment. She has created a charity, oPuce, which helps get people with cancer being refused employment because of a cancer diagnosis, into jobs that they want.

The sessions on sarcoma had many important revelations – treatment of GIST, mifamurtide 10 years on, sorafenib for Desmoid tumours, radiation sarcomas and developments in precision medicine for sarcoma. The Patient Involvement session in the Nursing stream was very disappointing, poor quality information, nothing new or exciting, although there was an excellent case study from the Royal Marsden. The Patient Track in the programme included an excellent debate, in memory of Hans Keulen, about going the extra distance to extend life when terminal. Kathy Oliver chaired this superbly. Other standouts included a talk on Survivorship from Australia, and discussion on Fatigue – which revealed just how little we actually understand about this long-term side effect.

Then it was off to Rotterdam and the annual meeting of the European Society of Surgical Oncology (ESSO). The session I was speaking in here was led by Prof Gerard van Oortmerssen, my colleague on the board of Sarcoma Patients Euronet. I looked at Patient Involvement in the context of an emerging partnership with professionals, taking the most strategic view I could think of. For me the conference highlights were hearing sarcoma surgeons describing and debating key issues which impact on them. They are very different from the oncology issues which tend to get a wider exposure. Although I must say that when Andrew Hayes (Royal Marsden) asked me to (effectively) summarise from a patient viewpoint an excellent debate he had with Marco Fiore (Milan), with no notice, I really had to think on my feet.

A week later and it was off to Madrid for SPAEN’s tenth anniversary. SPAEN hosted a dinner for the meeting of the EORTC Soft Tissue & Bone Sarcoma Group (STBSG) to thank them for the support members have given us over those ten years, for presentations and discussions at meetings, round table consensus consultations, and many one-to-one advice and information calls. We also had a board meeting and set out some of the challenges we have for the coming years.

The Dinner was held at the Madrid Casino, a beautifully decorative building. It featured the presentation of Lifetime Achievement Awards to four of our leading specialist doctors, Ian Judson (London), Peter Hohenberger (Mannheim/Heidelberg), Jean-Yves Blay (Lyon) and Paolo Casali (Milan). They have been unstinting with their support and advice during our ten years and this was a powerful way of saying thank you. They also embarrassed me with a similar award so I have included a picture of it.

Then came the NCRI Conference in Glasgow. The National Cancer Research Institute is the UK’s partnership of major cancer research funders and provides a forum for collaboration on strategies and developments which over nearly 20 years has proved very successful. An important element of its structures is the part played by patients and carers with ‘lived experience’ who come together in the NCRI Consumer Forum.

It is a conference dominated by science and advances in research, with crucial forays into advanced clinical issues where research and patients come together. The strong patient/carer presence is felt most as speakers address questions raised by their audience. Some of the toughest questions seem to come from patients and the challenges this can raise make this one of the most rigorous conferences there is. I had been asked to offer a patient view on developments in clinical trials and I faced the opposite of this situation with questions from two of the conferences earlier speakers with endless experience, Professor Mark Ratain (Chicago) and Professor Ian Tannock (Toronto) – very flattering.

The NCRI Consumer Forum held one of its three annual meetings at the conference and heard a exemplary joint presentation from Dr Helen Matthews (research biologist) and Max Williamson (a medical student) both of whom are cancer patients, about work they had done together. We also heard about plans to expand the role of the Consumer Forum from chair Emma Kinloch.

For the first time the Conference looked at a topical issue. Rather than a debate it was a discussion with presentations from four viewpoints to start it off. The topic was Assisted Dying and Cancer Care, chaired by Professor Sam Ahmedzai. I had the role of presenting a patient viewpoint, with the brief to be personal not detached or theoretical. It was an interesting challenge to take on and a privilege to be asked. The input from the audience was exceptional, with additional personal experiences from patients and doctors, a medical student explaining his reservations, and palliative care expertise from experienced nursing and medical professionals. This was a first for NCRI and indicates a way in which the conference might evolve valuable new approaches.

So, after seven weeks of seeming endless travelling and exploring the depths of airport terminals, conference centres and large hotels, how do I sum up what I experienced?

As a speaker I enjoyed the challenge of getting some very different messages across and I think my efforts were appreciated. As an attendee and delegate I learned a lot, especially about some of the advanced research now coming into clinical use – CAR-T cell being the obvious example. The evolution of clinical trials and adoption of RWE research is an issue which is becoming critical and needs greater attention. It was valuable to listen to other patients make presentations, and to hear presentations about patient issues, although I wish that presentations about patient involvement were given by patients, maybe together with a relevant professional, rather than by professionals alone. I was pleased with the advances being made in treating sarcoma, although this is slower than in most other cancers, something which may reflect the different underlying biology. And I was humbled to receive an award although I have to say it is nice to be ‘stroked’.

Writing Patient Consent Information for a Clinical Study

This was initially written to advise trainee oncologists on a training workshop.

Patient Consent Information

There is good guidance available on how to construct a Patient Consent Information leaflet. This includes templates you can follow.  Have a look at the following:

This guidance from the UK has been developed through involvement of patients, ethical committee members and researchers.

Some advice about style (as opposed to content):

  • Use simple language – guidance would be to aim for age 12 capability
  • Use short sentences and short paragraphs
  • Do not join different concepts – abolish “and” if you can
  • Use short words – no more than three syllables in English, if you possibly can
  • Avoid technical jargon – if you have to use them, acronyms MUST be spelled out
  • Do not assume that the reader knows anything about research
  • Do not make reference to other documents unless they are freely available

Within the structure and the constraints of content:

  • Try and tell it a bit like a story – invite the patient to read along with you
  • Start from something the patient understands
  • Remember that all patients are different – you can repeat the story a different way
  • Explain what the study is aiming to demonstrate and why
  • Explain what will happen to them in the study – show empathy
  • Answer the obvious “what if” questions in your text (eg. what if it doesn’t work?)

You take your pick on formatting advice, however:

  • Use diagrams and tables to show issues clearly eg. schedule of hospital visits
  • Use side-boxes to explain issues separately from the main flow of the story if necessary
  • When formatting the sheet make it look inviting and easy to read
  • Put a line space between paragraphs, don’t crowd text tight to the page edge

In the back of your mind:

Remember that the patient will only take one pathway through the study so complex issues like cross-over between arms in an RCT, or choice of successive therapies driven by response, need careful handling. A diagram can often help.

Ethics reviewers will want to see that you:

  • Be clear about side effects – not just short term but any possible long term issues
  • Be clear about reporting and managing side effects, including self-management

Dont rush:

  • Give yourself time.  Draft, read and re-draft. Read it back 24 hours later
  • Get others to read it, especially patients
  • Have you been balanced in your explanation? Maintain equipoise
  • It does not matter if key issues are repeated
  • Remember that anything which appears to incentivise patients to enter the study will be unethical

Other issues:

  • Does the study have a website? Give the URL
  • Explain how to contact the research team – both email and telephone
  • Explain how to act in an emergency, with at least two telephone numbers
  • Have a card or sheet with contact details on it which patients can put on the kitchen noticeboard

Document management:

  • Use document management to control versions until ethics approval is finalised
  • Number the pages

 Passing ethical review:

Every ethical board has its own view of what makes a good patient consent information sheet. Almost everyone is agreed that page after page of legal statements should be at the back and that the first two or three pages is all that a patient will actually take in. Much of the rest will be read by family or friends. These first few pages are where the heart of the story must be told, this is where to place the diagrams they may need to refer to, this is where contact details should be (probably repeated right at the end). My personal view is that the first 2/3 pages should be like an executive summary, this is followed by the full story, then the statutory material. At the very end is a repeat of the contact details so that they can be easily separated from the main document as an aide memoire.

Roger Wilson                                                                                 

Short Term v. Long Term – dichotomy or opportunity?

One of the challenges of patient advocacy is developing a balance between addressing immediate patient needs and creating a future which addresses the issues which patients see as important but which take time to change.

A good current example of the latter is the demand for earlier diagnosis. We have been voicing the need for twenty years to my knowledge and now it is government policy, cancer charities support sit, Public Health England has taken note, and the NHS is starting to consider how it can be done. It will take another five years before technologies, manpower and systems are fully in tune with the concept. One of the bigger changes involved is shifting the behaviour of some GPs.

When we discuss patient involvement we do not draw any distinctions between patient advocacy for the short term and the longer term.  The opportunities for involvement in research range from reviewing patient information for a simple research study to providing patient perspectives on multi-million pound strategic projects, with just about anything in between.

Lets be clear, the review of information about a ‘simple’ research study is important and valuable, just as informing those making multi-million pound decisions is valuable. Similarly the support for a patient having problems accessing a new treatment is valuable. These are all roles in the whole context of patient advocacy.

There is no scale of value in this but we do need to understand the differences and the implications that these differences bring with them. The main ones are the ability of the advocate, the training they have had and perhaps most importantly, their life experience prior to being involved in work as a patient. We do not assess these when we embark on involvement, and perhaps we should not do so as many people will grow into roles which are initially unfamiliar and their skills will rapidly develop given the chance to do so.

So I can hear you ask, why are you raising this issue?

There is one area where we are approaching a crunch. The dichotomy between patient advocacy viewpoints of short term issues of access to new treatments and the long term issues of the value of new treatments. We want a patient who believes there is value in a new treatment and whose doctor agrees, to have access to that treatment. At the same time we want to see the evidence of value in that treatment so that it can be appropriately used in the clinic in the future. Regulation is moving fast to accept data from short term Phase 2 trials with ‘surrogate’ endpoints, rather than favouring randomised Phase 3 trials (RCTs) offering statistical significance of effect based on an Overall Survival endpoint (OS). A short versus long term conflicting view.

Seeing new treatments come faster into clinical use meets the demand of most patient advocates. However most of them also fail to consider the cost in terms of long term understanding, the increase in risk which is implied, and the inability to control the cost of treatment through a meaningful understanding of value. Many advocates also welcome the reduction in the numbers of RCTs with placebo comparators; the placebo is generally disliked and there are questions about using data from such trials in the wider treatment context, something which information technology is increasingly enabling.

How do we move forward through this dichotomy? Quick and easy access versus long term validity and value.

The route being proposed by professional bodies, supported it must be said by many patient groups, is to develop means and methods to gather ‘real world evidence’. The idea is that a treatment can be given early access to clinical use. Data gathered from that use is analysed and used to review value, changing authorisations, pricing etc as seems fit. Many patients are stunned when told that does not already happen, so making it happen could be best described as ‘common sense’.

The means and methods do not currently exist. Let us see whether policy makers and regulators can make it happen. Maybe there will be subtle resistance from the pharmaceutical industry which currently gets it own way in such decision-making. I believe it is in patient interests to make it happen. 

That is a long-term advocacy project.

 

 

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.