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.

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.

Sarcoma diagnosis – a new challenge emerges  

Conversations at the recent British Sarcoma Group conference in London (an excellent event) brought up a growing problem with a recent change in the guidance for GPs considering potential cancer symptoms. The idea is that when a GP has suspicions about a soft-tissue lump he can order a direct ultra-sound scan.  When I questioned the chair of the NICE Guideline Group during its development I was assured they had considered all outcomes and my concerns were dismissed. As far as I am aware no specific sarcoma expertise was consulted during the development process.

The question which needs answering is whether the new approach is working for the benefit of patients and/or the NHS.

Prior to the change three years ago a GP was advised to make a 2-week referral to a specialist centre if certain clinical symptoms existed.  More patients were actually being diagnosed from non-urgent secondary care referrals or other routes than from the 2-week system. Hence the change, which made direct access to x-rays (for suspected bone sarcomas) and ultra-sound (for soft-tissue tumours) available to GPs so they can diagnose non-malignant tumours or determine uncertainty which requires expertise.

The problems arise because sarcomas are rare, diverse in their histology and can appear in any location on the body – in fact ‘typical’ is not an adjective applicable to sarcoma. The ratio of benign soft tissue tumours to malignant sarcomas is over 100:1. The rarity means ultra-sound imaging technicians have little familiarity with sarcoma although for bone tumours X-rays can be definitive.

We are finding that ultra-sound imaging reports will err on the safe side. Some tumours are clearly benign but if they have any doubt they are likely to say that they cannot be certain.  So what do GPs do with this uncertainty?  Their uncertainty boundary has shifted so they make a 2-week referral to a specialist centre, even for a tumour which under the old guidance would have been recognised as benign.

Thus specialist sarcoma centres are being overwhelmed by urgent referrals for ‘uncertain’ tumours. Diagnostic expertise is now being exercised by nurses operating triage clinics. This allows their consultant colleagues to spend longer with the fewer potentially malignant cases who come through the triage.  The people with sarcoma who would benefit from urgent attention are being hidden in a cloud of benign conditions until a late moment in the process. GPs are allowed to opt out of responsibility for diagnosing the benign condition, hiding behind the ‘uncertain’ ultra-sound. Those with benign conditions suffer a journey to a regional specialist cancer centre only to be told by a nurse that their highly qualified GP must re-consider their case.

Patients are not being respected. The problem is not the principle of the new guidance, but the implementation which has shifted the workload inappropriately. Those picking up the pieces are the specialist centres overloaded with diagnostic work. The new guidance was intended to help them and it is doubtful whether this is leading to earlier diagnosis of malignant tumours either.  The whole approach needs review in the light of the experience the sarcoma centres are reporting and any review must be in collaboration with them, rather than imposed by a committee led from primary care.

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.

 

 

SOME THOUGHTS ABOUT THE NHS LONG TERM PLAN

I do not suppose that I am alone in finding that as far as cancer is concerned the NHS Long Term Plan, published on Monday, is admirable if somewhat underwhelming and, to be fair, cancer is only a small element in the whole. Its primary focus is on diagnosis.  There is no mention of the loss of valuable skills caused by Andrew Lansley as Secretary of State for Health in 2010-12, although work on Cancer Alliances to mend the damage proceeds.

The paragraphs on cancer in the Long Term Plan start with the following:

3.51. Cancer survival is the highest it’s ever been and thousands more people now survive cancer every year. For patients diagnosed in 2015, one year survival was 72% – over 11 percentage points higher than in 2000.

As a general point that should be borne in mind, even if NHS idiosyncrasy was not taken into consideration, evolution in clinical practice would account for much of that improvement. Indeed it can be argued that if progress on the 2007 Cancer Reform Strategy had not been politically diverted we would be showing even further improvement.  Things have obviously moved on and while not all of the 2007 ambitions were achieved the NAEDI National Awareness and Early Diagnosis Initiative, led by Cancer Research UK for the last 10 years, has been critically important. It identified many of the factors which result from the behaviour of patients, GPs or the wider NHS, in the diagnostic process and it underpins what is now planned.

The NHS Long Term Plan has these promises.

Assuming that full funding for these developments is forthcoming in practice and that staffing issues for radiographers, radiologists, pathologists, laboratory staff, nurses and oncologists of all kinds are addressed (given the effects of the B word) this will be a very welcome development and will go a long way towards meeting the cancer survival ambitions.

There is however a shadow, identified in the Health Foundation report Unfinished Businesspublished shortly before Christmas. This is the role of GPs as ‘gatekeepers’. Those with undiagnosed cancer rely on a GP having suspicion and initiating tests (the right tests) or making a referral. That will not alter although the frustrated will be able to self-refer to a Rapid Diagnostic Centre. These will certainly pick up some of the otherwise undiagnosed but not all. Indeed there will be some who attend a Rapid Diagnostic Centre who may not be diagnosed early – a small percentage of false negatives come from almost any test, there are always interpretation issues and some rare cancers have barely discernible characteristics at their early stage.  So from a patient viewpoint this is a welcome step forward but quantifying its potential is massively uncertain and success will rely on the skills levels available in the Diagnostic Centres, especially those located at a distance from a major cancer centre.

3.52. This Long Term Plan sets a new ambition that, by 2028, the proportion of cancers diagnosed at stages 1 and 2 will rise from around half now to three-quarters of cancer patients.

In 2007 we could not have stated an ambition in those terms. This target comes from the work that has been done on data, initiated in 2007. The National Cancer Registration & Analysis Services is a world leader in cancer data. In each tumour type the push to record staging data at diagnosis in the initial cancer registration is resulting in a much more accurate picture of prognosis at diagnosis. This can be compared with the eventual treatment outcome and also provides for targets such as this one.

The NHS remains excellent at creating ambitions while overlooking some of the issues. There are two things evident in the NHS Long Term Plan. First, it is not a plain English document.  Second it did not involve independently minded patients in its development but we have now come to expect that. Patient involvement has been reduced to tokenism in NHS cancer service provision. It cannot be described as patient-centred any more and a key weakness of the whole structure is that the voice of end-users is missing. The charities, good as many of them are, are not a substitute.

On the matter of creating ambitions while overlooking some of the issues I find it hard to believe that a faster diagnosis standard for sarcoma will emerge without resourcing specialist sarcoma units to handle it. Pathology skills, one example, are already at a premium. Over 50 primary diagnoses, with many variants by location, histology and surgical feasibility, suggest that words like “all” and “full”, which appear throughout the new plan, could be unrealistic. The case for genetic profiling to take over the challenge has no evidence base and apart from profiling the whole population I cannot see it working for sarcoma. I should like to be proved wrong.

IN SEARCH OF CLARITY

There have been some thoughtful blogs recently on the subject of PPI in research from Simon Denegri (headless chickens) and Jim Elliot (mis-selling PPI), and an excellent editorial in the BMJ on evaluating PPI in research.  It may be time to put forward some of my thoughts about what is not happening and perhaps should be.

Patient and Public Involvement is too generic, the term is too imprecise, to allow it to be other than a mantra for ignorant management to follow.  It is difficult to implement unless those charged with actually doing it really understand what it involves.  As a result Jim’s dislike of the PPI abbreviation is widely shared by cognoscenti and those organisations which thoughtlessly perceive it as a route forward find themselves in Simon’s headless poultry farm. To cap it all after twenty years the research community still questions its value.

So if it is too generic how do we break it down? The immediate thing to do is to separate ‘patient’ and ‘public’.

Being an involved patient means bringing the “lived experience” of a disease or treatment to the table where it is relevant to have that experience available and expressed. In research this need is easily identifiable and a general public involvement is not required. For some roles you need a patient, nothing else works. Simple. Hmmm …

When you go beyond research into the more general arena of healthcare the public voice is important but the involvement of patients is also a need.  What that means is that a large overall part of generic PPI activity is actually about public involvement and the involvement of patients where a research focus is not present. However having patients involved is a management mantra (“look how clever we are, we are patient centred”) so there are contradictions and political expectations to be met.

The National Standards published last year call it all ‘public involvement’.  That distorts it. Research has specific needs .  Sadly these Standards point towards lowest common denominator corporate behaviour, the NIHR ignored my consultation suggestion of having a standard about senior management being actively engaged (over and above signing it off). That is what Simon is now calling for. Evaluation plays no real part in the Standards and that need is well described in the BMJ editorial.

So we have management mis-understanding, inappropriate pressures on front-line managers, lack of terminological precision, lack of ambition, conflciting purpose, no meaningful feedback about performance. Yet, we want more of it. Confused?

The general healthcare need for public involvement is about engaging the community, whether geographical or a community of interest which the organisation serves.  Recruiting, training and supporting public members is different from the processes for recruiting patients to be involved in research.  If there is a specific need for involving patients outside research (eg developing a new therapy suite) these can usually be easily handled because there is a local community involved.

My personal wish is to see the research issues sorted out. PPI in research is focussed on the “lived experience”, first-hand experience of a disease or treatment, and importantly patients are sought for what they can bring as individuals, not just because they belong to a defined community. They can be harder to find and, for example, ensuring that a rarer disease is represented can be all but impossible.  Inevitably this means that there are roles and occasions where a more generic experience is valid but putting in a member of the public solely because they are a member of the public is not appropriate.

We must manage these separate needs separately even when all these requirements may co-exist in one organisation. It must be recognised that they are different and should be managed in different ways.

I am sure that understanding this difference and recognising separate approaches according to need could help Simon’s chickens grow into fine oven-ready birds. Clear ideas about evaluation would probably help. As for a replacement acronym for PPI, assuming we cannot have an acronym-free environment, I give up. Identifying these different purposes will perhaps give focus to evaluation efforts, allowing us to develop involvement with a clear idea of how value can be recognised in all its different purposes.

Footnote: I recognise that there are some situations where the only way of tapping into the “lived experience” is through those who care for or lived with a patient. In these instances the carer’s “lived experience” is valued just as the patient’s would be.

 

https://simondenegri.com/2018/12/11/public-involvement-is-beset-by-headless-chicken-syndrome-in-too-many-organisations/

http://www.drakesyard.co.uk/have-you-ever-been-miss-sold-ppi-why-it-matters-how-we-refer-to-public-involvement/

https://www.bmj.com/content/363/bmj.k5147