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.

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.

Government pushes forward with review to speed up availability of innovative medicines and treatments

Two posts in one day!  Ah, those were the days when we got two Post Office deliveries a day….

Just to say the Government has published the full terms of reference for its Innovative Medicines and MedTech Review to be headed up by Sir Hugh Taylor who is currently Chair of Guy’s and St Thomas’s.  Yes, that’s the very same review I said this morning would be out any time soon.  Only it happened yesterday!

The remit is comprehensive and the door is very much open to patients and patient groups to give their views.  The specific para of relevance here is as follows:

‘Consider how to ensure patient trust in the regulation and assessment of medicines and medical technology, and recognise the importance of the patient voice and role of medical research charities as both sponsors and beneficiaries of innovation.’

The review is being supported by the Wellcome Trust – in other words they are doing the legwork – and there is a short blog on their website here.  They haven’t announced the other members of the review panel nor details of how it will work. We will learn more in due course.

I think Hugh Pym at the BBC has written a good piece here on the significance of the review for the future of medical research and speedier access to medicines for patients.

Also this from law firm Pinsent Masons which highlights the review focus on digital technologies.

 

Spend £1 on cancer research and get 40p back every year: what’s not to like?

Today the All-Party Parliamentary Group for Medical Research holds its summer reception in parliament. This annual event has become an important fixture on the health research calendar. The ‘great and the good’ assemble to focus on a key issue of the day and to network. At some point in proceedings they will be addressed by the Science Minister, David Willetts MP, or Health Minister, Earl Howe, or both.

This year’s event is headlined ‘healthy futures’ and aims to bring to light how investment in research has long-term economic and social benefits. This is all with an eye on the forthcoming General Election next May and a subsequent Comprehensive Spending Review (CSR).

In recent years, research funders have sought to understand better the impacts arising from the money they spend on research. Often this impact has been expressed qualitatively. But is has proved more difficult to place a financial value on impact. The first report to do so was published in 2008 when I was the Chief Executive of the Association of Medical Research Charities (AMRC).

The report looked at the economic returns arising from government and charity funding of cardiovascular research, also testing the methodology to a more limited extent on mental health research. The study, undertaken by the Health Economics Research Group at Brunel University, RAND Europe, and the Office of Health Economics, found that for every £1 invested by the government and charities in cardiovascular research, a total annual return of 39p was generated each year in perpetuity. The figure for mental health research was 37p per £.

Now the study – commissioned by Cancer Research UK, Wellcome Trust, the Academy of Medical Sciences and the Department of Health – has been repeated in cancer and published as a paper in BMC Medicine to coincide with the All-Party Parliamentary Group on Medical Research reception. You can read the paper here:
http://www.biomedcentral.com/1741-7015/12/99

The key findings in the study which are also published in report form as ‘Medical research: what it’s worth,’ are as follows:

The British public has funded £15b of cancer research over the 40 years to 2009 through taxes and charitable donations.

The time lag between investment in cancer research and it’s eventual impact to patients is around 15 years. This is similar to the figure obtained in the earlier study, and shows the long term nature of the payback from research funding.

Key cancer treatments and interventions have delivered the equivalent of £124b og health gains for UK patients in the 20 years up to the end of 2010.

The proportion of these benefits attributable to UK research was 17%.

Taking the spend, and allowing for the time lag and the proportion of benefits attributable to UK research, each £ invested in
cancer-related research by the UK taxpayer and charities generates returns of around 40p in every following year. Of this, 30p is the estimated benefit from research to the wider economy; the direct health benefits amount to 10p per annum for every £ spent. The overall annual rate of return is estimated to be 40%.

I am sure the study will be challenged by some and so it should.,Only through ever-greater scrutiny are we going to improve these
socio-economic models about research. But they seem of enormous value to me in trying to quantify the impact of money spent today, on society tomorrow. And if it makes policy-makers and newly-minted Ministers put their red pen away then all power to its elbow.

A brief update on NIHR’s review of public involvement in research

We have just passed the mid-way point in the ‘evidence-gathering’ phase of NIHR’s strategic review of public involvement entitled ‘Breaking Boundaries.’  So here’s an unofficial Chair’s view.

The announcement of the review on 31st March, its terms of reference and our initial call for views can be found on NIHR’s website here

People can at the moment choose to respond to the review using the downloadable questionnaire on the website, or using the online survey which is available here.  So far, almost 200 people have responded to the online survey.  61% have said they are a service user/patient/consumer/carer.

In addition there is now a facilitator guide as well as some PPT slides available for people who are planning to hold a group discussion with patients, carers and colleagues before responding.  Our sense is that quite a few organisations are planning to do this as part of their response.  Anyway, the guide and the slides can be found at the end of this post.

Also over the last month we have held discussion sessions at four meetings where the excellent RAPPORT study of public involvement was reported. These events took place in Manchester, Bristol, London and Cambridge and reached about 200 people, again mostly patients, carers and members of the public.  Thinking about these sessions and the sorts of comments we are getting through the written and online survey evidence, some key issues and questions.  Thematically, these might be headlines:

  • The purpose and value of public involvement
  • The infrastructure needed, national, regionally and locally, to deliver public involvement
  • The resources necessary to deliver public involvement that works
  • The importance of relationships to the success of public involvement
  • The learning (knowledge, skills, experience) needs of all partners and how to support them
  • The need to tackle the impact and outcomes agenda

We have also held two ‘international’ sessions by phone with colleagues from Canada, USA, Denmark, Australia and the European Union.  It was clear from these conversations that the UK is held in very high regard for what it has achieved in public involvement; we are the recognised leader in many respects. Also, that this leadership is closely associated with the work of INVOLVE over the years.  Nonetheless, we have much to learn from them.  We are all battling with the need to prioritise and find models suited to the future not the past or present.  The case for an international alliance or association where we can share our ideas and challenges is growing in strength.

This week we will be meeting with representatives and members of the Association of Medical Research Charities (AMRC) and Association of the British Pharmaceutical Industry (ABPI) to get their views on public involvement in research.  Heading into June there will be a range of events where we will be holding ‘consultation’ sessions including North West People in Research Forum on 30 June and the Health Services Research Network (HSRN) conference on 19/20 June.  And, finally, some yet-to-be scheduled tweet chats and social media activity.  Please remember that the hashtag is #PPIBreakingboundaries

Please keep the responses coming in.  I know I speak on behalf of all the review panel when I say they are insightful, helpful and hugely important in their own right.

The  previously mentioned guide can be found here: 

  And the slides can be found here:

The medical marathon; a piece to mark the #LondonMarathon2014

What is, I wonder, the equivalent of Noel Coward’s lyric ‘only mad dogs and Englishmen go out in the midday sun,’ which might encapsulate the phenomenon that is the Virgin Money London Marathon?

This morning tens of thousands of runners will set out from Greenwich Park as they do every year with high hopes of finishing two, three, four or more hours later at Buckingham Palace.  It will be the 34th London Marathon.

People from all ‘runs of life’ including patients, carers, doctors, nurses researchers, scientists and charity fun-runners will have trained long and hard these past few months so that they can take to the Capital’s streets and know that, with every pounding foot, money is being raised for their favourite cause. And medical research dominates those causes.

It is difficult to say precisely how much of the £660 Million raised for charity since the London Marathon started in 1984 has gone to medical research.  But my reckoning is that, out of 54 charities who have been honoured to be the official London charity of the year since then, 20 have been medical research charities.  The British Heart Foundation leads the pack having been chosen four times, the Anthony Nolan Trust is not far behind.  Including this year, it has been the official London Marathon charity on three occasions.  There’s a page dedicated to the history of the London Marathon and its links to charities which is worth a look if you are interested.

The first of these medical research charities was the Middlesex Hospital Research Fund in 1986, the hospital being notable for being a significant cancer research centre then and now I believe. Also, for being where I was born!

There are many, many more charities who simply enter runners into the event of course.  I calculated that, of the 91 who I assume pay for the honour of being included on the main listing provided by Virgin Money, 38 are known for their work in supporting medical research.  Yet this only skims the surface.  Trawl further through the A-Z listing and many household names pop up who also do much to fund medical research.  And who knows how many local research charities barely merit a mention beyond their local newspaper if that.

And what of the runners themselves?  I have a feint recollection of turning up at Trafalgar Square in 1993 or 1994, and being marshalled by the wonderful Paula Dawes – the Alzheimer’s Society’s then head of fundraising who did more for staff and volunteer motivation with her gigantic fundraising thermometer on the wall than any excel spread-sheet produced for a committee – to cheer on just over 20 runners on a dreary Sunday morning.  Last year, the number of people running for the Alzheimer’s Society was nearer 400 I believe.  That seems a pretty incredible rise in the numbers.

The links to medical research do not end there.  For 31 out of the last 34 years, there has been a London Marathon Medicine Conference which looks at the medicine and science of marathon running.  It is also proving to be a teaching and research opportunity for sports scientist students.  However, I would be interested in knowing whether anyone has published a scientific paper about, or run a clinical trial involving, London Marathon runners?  Forgive me for not knowing that, if there has been.  Perhaps the only thing we lack is a few more significant medical research landmarks along the route; The Crick in 2016?  It might be one for the public engagement team there to think about.

Philosophically I wonder whether there is something deeper that draws together the modern penchant for running marathons and our commitment to, or support for, medical research?  The metaphor of the marathon certainly serves us well in health conjuring up images of commitment, camaraderie and the joy of reaching the finishing line.

For example, my good friend and colleague, Derek Stewart, has written before about the similarities between his progress as a runner and his journey in public involvement in research.  I also remember from my time at the Alzheimer’s Society an article written by a carer, comparing the years spent tending for their loved-one to that of running a marathon.  It became a very popular piece among fellow carers and supporters.  How many times have we also heard scientists and researchers talk about the long process from scientific discovery to viable treatment of therapy?

For this writer (and I am not a runner) the act of doing a marathon is a symbolic nod of respect, a doffing of the cap or handshake of support, in recognition of what it takes to live with ill-health and to find a remedy.

And we shouldn’t forget that the London Marathon is just one of many such events that take place up and down the country throughout the year – a good few in the park behind my house in South London – where people are going that extra mile for research.  Indeed, considering  the UK is second only to the Netherlands (70% versus 78%) in Europe terms of the number of people saying they have donated money to fundraising campaigns for research – the Europe-wide figure is 39% – that’s one gutsy performance by our marathon runners.

It also reinforces what I think is the key message that emerges from the recent IPSOS Mori/Department for Business Public Attitudes to Science report [by the way this is a superlative piece by Alice Bell in The Guardian about itHe blog is worth checking out too] . Namely, that you may be able to make an argument that the British public’s understanding of science leaves much to be desired.  But you can’t fault their gut instinct in support of it.  And what better symbol of that, than their staying the course over 26.2 miles.

Good luck to them all.  And, if any should be reading this afterwards while nursing aching limbs and sore feet, thank you.