Cystic Fibrosis Gene Therapy Trial testament to the cf community as a positive, patient and persistent partner in research

I hope you will have seen or heard the news today of the positive results of a gene therapy trial for people with cystic fibrosis. And this is how The Guardian is reporting it.

Although it is early days and the improvement shown in patients only a modest one, it is nonetheless a very hopeful sign of how this approach may help people in the future.

I have no direct experience of the disease, but one of the most affecting and poignant patient stories I have witnessed was by a young woman with cf at an event at the political party conferences many years ago.  It has stayed with me ever since.  It has caused me to have a particular emotional interest – if I may call it that – in the condition.

The trial was conducted by the Cystic Fibrosis Gene Therapy Consortium which brings together scientists in the Universities of Oxford and Edinburgh with Imperial College, London.  They were formed in 2001 at the initiative and with the support of the Cystic Fibrosis Trust (CF Trust). The Trust has invested £40 million in the consortium since then and continues to do so (it announced a further £500,ooo funding just earlier this year).  But the funding environment for these sorts of projects is never easy and a few years ago the Consortium formed ‘Just Gene Therapy’ to bolster existing funding from the National Institute for Health Research (NIHR), Medical Research Council (MRC) and others.

So, the back-story of this important news today is one about the length of time it takes to do this sort of work, the importance of collaboration and the pioneering role that medical research charities have in supporting ground-breaking science.

But, above all, it is a testament to the cf community’s as a whole.  Their positive, persistent and patient backing and support through their donations.  The patients who took part in the trial, some as young as 12.  Without them, we wouldn’t be hearing this news today.

Comment: Jeremy in the Hunt for better meds behaviour #medicine

I have heard it from both sides of the Atlantic. So there must be some truth in it. We are entering the era of ‘retail Government.’ Call it the commoditisation of politics if you like. But the idea is that the bigger the numbers – £ or people – a Minister can shift with his or her decisions the better. We must get that deficit down after all.

There is something of this behind the Secretary of State, Jeremy Hunt’s, announcement yesterday that – from next year – prescription medicines costing over £20 will carry messaging about the indicative cost of producing it and a strap-line about how they have been ‘funded by the UK taxpayer.’ Although it also has a strong whiff of the pharma agenda behind it too.

If people are reminded of this cost they might be better at taking their meds. Or so the thinking goes. Estimates put the cost of medicines waste – that is the pills we don’t take even when prescribed – at £300M. However, whether this is all down to people’s
‘non-adherence’ (horrible term) is an interesting question in itself as pointed out in The Guardian article to which I have provided a link above.

I actually think the Secretary of State’s idea is not a bad one in itself. We should all become much more aware of the cost of that which we have perhaps taken for granted in the past. And not just in terms of medicines. Although I wouldn’t want it to be taken too far.

After all, what next, the local copper having his cost to the taxpayer printed on his truncheon or panda car? Or ‘This potholed motorway cost you…’ flashing up on roadside screens as you trundle along at 10mph? How’s the saying go about ‘knowing the cost of everything and the value of nothing?’ Is this the ‘shaming state’ going to be the new ideology? No more nannies or nudges?

The more important question we should all be asking is whether it will solve the issue of people not taking their meds as Mr Hunt hopes. On this, I am not at all convinced.

The Academy of Medical Sciences (AMS) held an excellent workshop on this issue just a few months ago. The published report of that discussion illustrates just how complex the problem is and how many-stranded our response must be.

We might – for instance – begin at a different point and actually ask our health research system and pharma companies to take notice of the meds that people need rather than those they think they want. The fact that we don’t reflect these patient priorities has to be a matter of concern to us all. It may be one of the root causes why public confidence could ebb away from medical research in the end. An issue that other parts of Whitehall have become concerned about.

Or we could support our pharmacies and pharmacists further to help patients understand their medicines and manage the side-effects. For we can not rely on our time-strapped GPs to do this any more.

Messaging on packs is not a new idea of course. We have had health warnings on cigarette packaging for a long time. I am not sure what the evidence says about their effectiveness. But if we are going to go down this route shouldn’t be trialling different sorts of messages. From ones that are health focused to those that are politically inspired? Otherwise it’s a bit of a shot in the dark isn’t it?

Which might work. But then again as Capt Marko Ramius says in the 1990 submarine film ‘Hunt for Red October: ‘Ryan some things in here don’t react well to bullets.’

Blog: I have ‘This Feeling’ – Could 9 out of 10 health apps be simply useless from a patient and carers point of view?

PatientView – the originators of the excellent directory – published an important piece of work this week entitled: ‘What do patients and carers need from health apps – but are not getting?’  You can download it here.

Their report looks at the results of a survey they conducted of over 1,000 patients and carers across Europe about what they think about health apps.  It then highlights unmet needs in five therapy areas – cancer, diabetes, disability (pain management), mental health and wellness – that were identified in a follow-up workshop.  PatientView conclude the report with some recommendations for app developers about what they should be doing to ensure all their hard work and money is not going to waste!

The survey shows that ‘health apps’ are seen by patients and carers as a potential avenue for helping them understand and manage their condition, a source of practical support such as care planning, and a means of communicating with doctors and health professionals.  But people are confused by the number of ‘apps’ out there, find it difficult to find the ones that might be most useful, and are somewhat distrustful of who funds and makes them.

Interestingly this research also suggests that few doctors are suggesting ‘apps’ to their patients – a finding backed-up by a MedPanel survey in the US this week that showed only 15% of doctors are recommending mobile health apps to people.  Perhaps both doctor and patient are simply struggling to find the utility in this technology for treating and managing a range of conditions?

If you are a reviewer then it is likely you will have noticed a trend in the number of researchers putting forward applications and proposals to develop ‘apps.’  So I really like the fact that PatientView finish their report with ten recommendations for what developers and researchers should be doing if they want their inventions to be worthwhile to patients.  They are:

  1. involve patients: establish a transparent, fair and sustainable way to involve patients, patient groups and carers in app development
  2.  address unmet needs: identify and address truly unmet patient and public needs, and switch the balance from information-giving and trackers to tools that help patients put this knowledge and data into action, enabling them to self-manage
  3.  set up a one-stop advice shop for developers: in one place, provide them with definitive, up-to-date guidance on key issues such as regulatory requirements, clinical approval methods, best practice in involving patients, and horizon-scanning on technological developments, issues and opportunities
  4.  share best practice: identify, develop and communicate models of best practice, for example in involving patients and carers in identifying unmet needs
  5.  gain “air time” for quality apps: identifying ways for developers of quality health apps to market and differentiate themselves from the many products available
  6. find the business model: identify sustainable business models for apps, and make this funding transparent to browsing consumers
  7. demonstrate clinical rigour: confirm and clarify the clinical approval requirements which apply to health apps
  8.  bring apps into the mainstream: integrate them into wider and “real-world” healthcare solutions, rather than remaining fragmentary “add-ons”
  9.  future-proof apps: make them sustainable and adaptable to future changes
  10. enable informed choices to be made by patients and carers about healthcare apps, for example by raising standards of digital/app/mhealth literacy, and gaining clinician input on recommending and prescribing apps.

But let’s finish on a positive note.  Some people do strive to get it right. ‘This Feeling’ is an iPad app developed by Arthritis Research UK and the National Institute for Health Research Musculoskeletal Biomedical Research Unit with young people.   It helps children and young people with Juvenile Idiopathic Arthritis (JIA) describe their pain.  95% of the young people who were part of the project preferred the app and found it an interesting and engaging way to communicate about their pain.  You can see Professor Wendy Thomas talking about the work on youtube.

Have a great weekend everyone.

What next in identifying patient priorities for health research? #JLAevaluation

I spent yesterday morning at an excellent event looking at the James Lind Alliance Priority Setting Partnerships (JLA PSPs for short!). #JLAevaluation

Started in 2004, JLA PSPs have become a recognised and highly respected method for identifying shared priorities in health research among patients, carers and clinicians.  That they have is a testament to its founders but also the excellent JLA advisers and National Institute for Health Research (NIHR) support staff now working on them.

They are one of the success stories of UK public  involvement.  31 ‘official’ PSPs have been completed over the last ten years or so. 23 are underway. A further 86 wait in the wings for possible adoption. More than 26,000 respondents have been involved and over 350 treatment uncertainties identified in diseases covering the human body from head to toe.

Only this week, the National Institute for Health Research (NIHR) and British Society of Gastroenterology launched a PSP in alcohol-related liver disease.

Priority Setting Partnerships have undoubtedly contributed to the increasing cultural sensitivity across the UK health research system that it should be funding work of ‘relevance’ to patients and carers.  They are also becoming influential way beyond these shores. A colleague from Canada wrote to me this week to say that the Canadian Alzheimer’s Society is about to start a PSP. Others have taken place in Australia, Denmark, Sweden, Spain and the Netherlands.   They have entered the lexicon if not the rule book of public involvement across the globe.

But there’s no resting on its laurels. Yesterday’s meeting was about what comes next for PSPs.  Also on what needs to be done with regard to the wider challenge of identifying research priorities in any health research system. In my view there are a number of tasks ahead:

– As I said yesterday, collectively and individually PSPs are facing their ‘Strictly Come Dancing’ moment. In each disease area the resulting top ten treatment uncertainties or priorities take to the dance floor for potential funding by researchers and their institutions. Only the judges are predominantly scientists. No wonder few of them are left in the competition at the end. We need to work with funders and others to ensure that these patient-driven priorities are picked up.  That should not be the responsibility of JLA PSPs but it’s many partners and colleagues working across health research.

– We heard an excellent example yesterday from a team in Australia who had tried to do ‘JLA in a day.’ There have long been thoughts about developing a ‘JLA Lite’ version. I understand the concern of the methodologists and they are right to urge caution. But if JLA PSPs are now the skyscrapers of priority setting, we must also recognise the need for a village hall option.  My observation would be that JLA PSPs do not tend to close off the debate about priorities.  Rather they set the agenda for a more informed debate.  Smaller, more localised exercised that follow-up or are a presage to this work may have a place.

– Which leads me onto another possible innovation. PSPs have almost exclusively been concerned with specific conditions or diseases. But could we do this with local communities or neighbourhoods? To identify public health concerns for instance?  Similarly I would be interested in a focus on care and services and not only treatment.

– From an NIHR point of view we should ensure reviewers are aware of PSPs relevant to the programme or scheme to which they are applying. Applicants should be guided to explain in their application how their proposal will address them. And if not why not. For there may be good reasons.

– Finally, when I replied to my Canadian friend I said that I thought that a PSP was the single most important strategic intervention in research that disease specific charities and patient groups could make in research.  Working with patients, carers and professionals they would not just be informing their own funding strategy, but the whole field of science relevant to the disease area as well.  It would be good to see the Association of Medical Research Charities (AMRC) make this a recommended action of charities when – for instance – they are developing their research strategy which is an AMRC membership requirement.

Capacity, capability and quality will be issues that need further attention in the coming months and years.  It is great that JLA PSPs are homed in NIHR’s NETSCC organisation.  But they need to be part of the mainstream in how we think about the value of research.  In the same way that we now do about systematic reviews.  As I hope my thoughts about the future make clear, achieving this will be as much down to research funders, patient groups and other partners as to our excellent JLA colleagues.

It’s all about relevance.

From A to B on using medical records to help people get better

If the day has a ‘y’ in it then I guarantee that policy-makers and experts somewhere across Whitehall and Westminster are meeting to plot a way through the stalemate otherwise known as the ‘use of health data.’  That is: how we are going to use my and everyone else’s medical records to make people better now and in the future?

This afternoon, sitting in my third meeting on the subject in as many days (yes, I know, I lead an exciting life don’t I?), I did wonder – and not for the first time – if I was about to disappear down a very deep hole; there are so many organisations, working groups, initiatives, Boards, committees, panels, all working on the issue. I might just make one up for the hell of it.

Talking of which, if we had placed moles at High Barnet, Morden, Lewisham and Upminster and asked them to create the London tube, the resulting mess wouldn’t be far off where we are now.  A maze of tunnels that mean something to the owner but no one else.  A complex system that you would only know was there because of the small mounds of earth on otherwise pristine lawns.  Everyone is trying to do something and with the best of intentions.  But the overall purpose of this activity remains frustratingly nebulous.  We can’t even agree how to express why we want to do it.

Maybe the task ahead on data is essentially an engineering feat akin to Crossrail.  We need to know the starting point, the end point and then have a bloody large tunnelling machine to hand.  We might cause a few edifices to lean.  Some egos might vanish down the odd sink hole or two.  A few people will miss out completely on a connection to the finished product.  But if we want to get from A to B, that’s the price we will have to pay.  Some will no doubt say that the National Information Board meeting last week is the start of this.  I hope so.

There are rumours Government is ruminating on a new health challenge for its next five years in office.  Like dementia in the last parliament.  I’ve heard obesity, diabetes and mental health all being mentioned as possibilities.  Which probably means it’s all just a pointless rumour.  But, if not……I hope that the Prime Minister picks none of these.  I’d rather he went for something like ‘health data’ which would benefit all of us.  It probably needs that sort of ‘oomph’ and clarity of purpose.  Critically, it probably needs this sort of leadership to help us get to the other side.

And what of the public?  Well, you’ll note they are conspicuously absent from my opening line.  Need I say more. In the meantime we have all become amateur experts on the subject of public confidence and trust.

It might be heresy to say it but I suspect we know as much as we need to about public attitudes.  We certainly don’t need any more surveys or polls or focus groups.  We also knows what works in public engagement.  Not just in general but on the specific issue of data use as well.  We just need to get out there and talk it through with people in terms they can connect with.  The same with our GPs and other health professionals. Because it will take time to build a compact on this issue.  And it will be too late if we don’t start soon.

So, there you go, a blog with no real ‘A’ nor ‘B’.  But this is just a blog.  Not about helping people to get better.

Is public involvement in UK health research a danger to itself?

The story of Concorde – the supersonic airliner that graced our skies for thirty years – was once a totemic example of the UK’s inability to turn great innovation into commercial success.

Lots of factors were said to cause its undoing. Concerns over its environmental impact. US protectionism. But it was its lack of commercial viability that prevented it dominating the skies. Fact is that it went into service with a high price tag just at the time millions of us were beginning to enjoy cheap air travel.

I worry that the British model of public involvement and engagement in health care is following the same flight path as Concorde.

Two recent trips to Toronto, Canada, a delegation of Swedish dentists, conversations with European neuroscientists and an event at a European meeting of periodontologists have all focused my mind. Our
international colleagues are learning, adapting, adopting and improving on the UK’s approach. The question is: are we learning from our own mistakes, are we slipping into a ‘British is Best’ complacency as the world passes us by.

Nowhere was this brought home to me more than in Toronto. Our Canadian colleagues are wonderfully flattering about what the UK has achieved in public involvement. But I also sense some things that may give them the edge over us during the next decade. Chief amongst them are:

– They understand the power of consumerism and are ready to embrace it in ways we are too prudish to contemplate. They will use it to drive change in their health care systems.

– They have the scale, enthusiasm and open-mindedness to overcome barriers. People may regard it as sickening positivity. But it seems a better starting point for change. Here we shoot ideas down too quickly by listing all the barriers in its way.

– They have a more grown-up attitude to data. Both professionals and patients. Meanwhile we sulk in our respective rooms like teenagers. I think we probably have 5 years to sort out a new social contract on data in the UK. If we don’t do it in that time then we will be in danger of seriously undermining the quality of health care people can expect from their NHS.

– They are less obsessed with ‘self’ in public involvement and care more about community engagement. 90% of our time and effort (and possibly money) in public involvement here is consumed by an obsession with getting people on committees, groups etc. All good stuff. But essentially inward looking. What if we devoted only 50% to this in future and the rest to reaching out to our communities.

– They see developing their partnership with the public as a potential competitive advantage in research. We don’t. Again, we are reluctant to think in those terms.

No one is more proud of what we have achieved in public involvement in the UK than I. But we must change our mindset if we do not want to become just another part of our heritage. Glorious, worthy of a blue plaque perhaps, but essentially grounded and no longer airworthy.

Going commando in public involvement in health research

Its probably not the done thing for a National Director to applaud direct action.  So I won’t.

But I couldn’t help but warm to the recent appeal by Professor Luke Clements for carers to get in touch with their militant side.  Prof. Clements advised the parliamentary committee that looked at the Care Act.  In his recent talks around the country he’s been saying that the legislation does a great deal of good but essentially appeals to our aspirational side; rather than dealing with the nitty, gritty of what urgently needs to be done.

Half-way through Carers Awareness Week last week – and having listened to a host of very powerful carers stories – I did wonder whether we are letting people off the hook with a good and well-intentioned, but essentially cosmetic, tear in the eye or shallow platitude.

By constantly labelling carers ‘heroes’ do we inadvertently undermine their case by implying that they will go on whatever happens? Has the urgency of their struggle been whitewashed out with soft-focus public relations?  Have we made it easier for people to walk past our ‘heroes’ with the rallying but essentially patronising cry of:  ‘Keep caring, and carry on?’   When what we need to do is stop people in their tracks.

Aye, there’s the rub.

Organisations, systems, cultures, are all good at findings ways to accommodate and essentially smother the radical or the challenging until it becomes acceptable.  It’s no different in public involvement. The well-crafted advisory group with an isolated patient voice.  The agenda or conference programme with a patient on at the end (when everyone has gone home to get an early train).  Choosing to work  only with the corporate voice of patients – a charity CEO who is supposedly  ‘representative’ – rather than bringing a variety of voices together to reflect on their experience.  I think half the time, it’s not even a conscious decision.  And then again….

I try to tell the groups of researchers I speak to that public involvement is at its best when it is allowed the time and space to challenge what they are doing?  In the same way that they would expect their colleagues to challenge them.  And it is at its worst – in fact, worse than useless – when restricted, confined, expected to conform to their rules.  As someone once said at a recent conference I attended:  It must no longer be about how people should serve the system but how the system serves people.

A number of recent campaigns are about prising open some of the long-standing bastions of the science community, not least where and how it meets to talk about our health – yes, when I say ‘our’ I mean you and me.  Like Patients Included which is about ensuring patients are involved in the organisation and delivery of healthcare conferences.  Although I prefer the name of the more irreverent Patient Commando intiative in Canada which has a similar intent.  Creating space for the patient voice to be heard and to challenge.  Raiding the cultural norms around medical research and health care.

Now there’s a thought: ‘going commando.’  But then I know some people will think it’s all fur coat and knickers.