Wonder drugs will play a part in transforming healthcare - but a roundtable of experts brought together by HSJ and the Association of the British Pharmaceutical Industry said implementing best practice could be even more important. Alexis Nolan reports

It started with a call to better inform the public about what research is being done in the private and public sector - and ended with a plea for the NHS to become more effective at implementing what we already know.

In between, 13 experts from the government and policy, clinical and patient organisations covered much ground in the complex area of clinical research at a roundtable discussion hosted by HSJ with the Association of the British Pharmaceutical Industry.

In the 60th year of the NHS and ahead of the Darzi review that will shape the future delivery of the health service, the event aimed to address clinical priorities. To help inform the debate, ABPI had commissioned Opinion Leader research to determine where the public believes clinical research priorities should lie, through a combination of survey and group discussions.

The initial findings discovered people’s biggest concerns are, in order of priority: cancer, heart disease and Alzheimer’s disease.

ABPI director general Richard Barker told the discussion he was pleased to see that the public’s view was reflected in the pharmaceutical industry’s development focus.

“The industry pipeline is not actually badly matched to these [priorities]. I think Alzheimer’s is proving to be the most difficult of the top three but, from memory, we have something like 42 products in the pipeline for Alzheimer’s, around 277 for cardiovascular disease and about 700 for cancer.” The industry, he said, is shifting focus “from medicines to treat millions of people to much more specialist medicines for life-threatening disease”.

Mr Barker added that there is a challenge for industry and government to improve communication, not just between themselves, but with the public about research priorities.

“As health minister, Lord Warner called for much more of this interchange between industry and policy makers about what industry is working on and indeed the ministerial industry strategy group has called for an annual review for its purposes of what the industry is working on,” he said. “But how can we relate to the public?”

He continued: “One of the things I’ve discussed with the Medical Research Council and Sally Davies [Department of Health director general for research and development] is about whether we should have an annual survey - an annual publication that puts some transparency into this for the general public.

“It would be an authoritative resource. There would be some resource challenges associated with that for all participants, but that might be a way of opening and maintaining a dialogue with the public about these issues, which are obviously vital for them as indeed they are for us.”

While there seemed to be a clear overlap between clinical and research priorities, there was a healthy debate about what drives them - and about the fact that they may not necessarily coincide.

King’s Fund chief executive Niall Dickson said research priorities “seem to me to have a rationale of their own and they are not necessarily identical to clinical priorities”.

Professor Davies went further: “If we look at clinical priorities, they are about prevention: there’s promotions, protection, disease treatment and we are in the process of trying to shift from a service that’s totally focused on disease treatment and management to one that has elements of the other parts,” she said.

“Inevitably if you are looking at clinical priorities, then there’s a disease burden as a measure to guide where we should be going. And clearly the press guides some of the noise in the system, because that is a marker of what’s coming up on the radar - and I was interested that Alzheimer’s is coming up at the moment.”

She added: “I think one could take the view that clinical priorities can be developed as they have been done. This is when ministers take advice on the burden of disease and advice on evidence-based best practice. And a lot of [junior health minister] Lord Darzi’s work about models of care and pathways is very much about delivering best care in what we know.

“If you turn to research, clearly disease burden matters. But we also have to think about tractability: is it possible to get an answer to this at this time? We get a lot of noise about motor neurone disease. I would love us to do more work on that, but actually at the moment we don’t have good outcome models.

“And if you just listen to babble, you miss some of the forward-looking things that we need to be concerned about. Who is raising their hand and saying we need a lot more research on novel antibiotics? Because as TB infection rates continue to rise and with MRSA and C difficile , we are going to need novel antibiotics and there is insufficient investment in that area.”

Professor Davies told the group she believes the funding streams of research also warrant further scrutiny. “You have to look at who is the research funder because we all work in different arenas. Pharma is looking to find, in general, medical responses to disease, prevention of disease or treatment of disease. The research councils do predominantly basic research and translational research. At the National Institute for Health Research we take the translated: ‘It looks as if it might work’ and [say]: ‘Look, does it truly work, what’s the cost-effectiveness?’ but also research into how best to deliver care. This is the stuff no one else would do: the cognitive behavioural therapy versus antidepressants.

“And we have to have a niche which supports improving health and treating ill health which no one else is filling. So clearly you can’t sit round this table and say there is just one set of research priorities, because people are working in different arenas with different objectives. It’s about improving the health of the population but in different ways.”

Removing barriers

Junior health minister Lord Darzi’s contribution to the debate began when he reminded the roundtable that his next stage review is focused mostly on models of care. These will be clinically and locally led, evidence based and built on principles of equality of access, personalisation of care, quality and safety.

The eight models of care go from cradle to grave: birth, children, staying healthy, long-term conditions, acute episode, planned care, mental health and end of life. Lord Darzi said the issues that have captured the imagination of the public in the ABPI research are particularly relevant to the long-term conditions, acute episode and planned care models.

“What we are trying to improve, to integrate here, is the outcomes. Within that envelope there is a lot of interest in how we are setting the scene over the next decade in meeting some of the challenges of improving quality of care based on new drugs. How do you integrate the latest research and development and redesign the service around some of these innovations?”

The Health Innovation Council, set up in October last year, will help in this, he said. He added that he sees both the council and his review as enablers, “removing some barriers which might be in the way of introducing either a new drug or, more importantly, a new treatment protocol”.

The need for a focus on outcomes was picked up by National Institute for Health and Clinical Excellence chair Sir Michael Rawlins. “What we need to do is ensure that the NHS continuously improves the outcomes of patients and that is partly a structural problem - that’s Ara’s [Lord Darzi’s] problem.

“It’s also partly to try to ensure that clinicians generally practise medicine that has been shown to reliably improve patient outcomes; in a sense, that’s my job at NICE. The third bit is where we have gaps in the evidence, where we need R&D and that’s Professor Davies and the pharmaceutical industry’s problem,” he explained.

“These things are, of course, very highly inter-related and from our point of view, we are looking for quality of care, but it has to be quality of care that we can afford. We cannot do everything we might want. We just do not have enough money - no healthcare system in the world has enough money - and every healthcare system is facing exactly the same tensions as we are.”

Sir Michael argued the public has a good grasp of this tension and annual surveys in recent years showed that 70-80 per cent think NICE is doing a good or very good job.

“There is some understanding. Individual patient groups, of course, can’t understand why they can’t access their particular drug, but nobody dies as a result of not having anti-cancer drugs. Sadly, many of them produce increases in survival that are very modest - we are talking one, two or three, months.

“I quite understand those two or three months may be very, very precious, but the health service has to look after everybody who seeks their care and we have to try to devise ways of doing this fairly in a way that everybody’s interests are looked after.”

Folate and fluoridation

But Patients Association head of special projects Vanessa Bourne thought appealing to financial constraints was a potentially weak argument with the public. “While there is a perception that there is waste in the system in all sorts of ways, there will always be that pressure to say to NICE: Well, why can’t we do everything? We could if we weren’t wasting money on this, that and the other.”

Moving on to the importance of prevention - and the importance of acting on existing evidence-based research, Sir Michael cited the example of [vitamin derivative] folate. “In 1976, evidence started to emerge that folate would prevent neurological disease. By 1981 the epidemiological evidence was really quite powerful and the MRC put in place a randomised controlled trial, to its huge credit, which reported in 1991 that you could reduce neural tube defects by about 40 per cent. That was taken up by, curiously, the US and Canada, who both fortified their flour and have since seen substantial reductions.

“In the UK the advice is to take folate preconceptionally and that’s OK, except that half of babies are born as a result of unplanned pregnancy, so it’s not really effective public health advice.”

But still in 2008 we do not fortify flour in Britain. “Nicholas Wald [director of the Wolfson Institute of Preventive Medicine] thinks that results in 400 babies a year born with spina bifida - 400 a year. It is 20 years since we had the evidence and we are talking about thousands of babies with permanent and very disabling conditions as a result of a failure of public health. But the row publicly of fortifying, ‘adulterating my flour’ and so on is very difficult to get over. This is a political and public health problem that we have to resolve, but it requires courage.”

In a similar vein, Mr Dickson gave his support to health secretary Alan Johnson for recently reopening the debate on fluoridation of water and pointed to the success of the smoking ban as two examples where preventive action could make a big difference. He also raised the sometimes tricky question of national policy direction versus local priorities, something he feels is “critical” to the role of NICE.

“To what extent and how do we make priorities at local level? What space is there for people to make choices of priority at local level after you’ve set those national ones?” he asked.

But if there is tension between national and local priorities, there is a similar need to look not just at single disease areas but multiple ones, said Royal College of GPs president David Haslam.

“With single diseases, we are inevitably talking about prioritisation and I’m completely with Lord Darzi on the concept of personally focused care - most long-term patients have got multiple problems,” he said. “Someone might have not only heart disease and diabetes and hyperlipidemia, but also osteoarthritis and, not surprisingly, depression, as a combination, so the personal requirements for them are actually quite different from the single disease issues.

“One of the areas that we desperately need research into is how guidelines work together, how you work out how to care for patients with co-morbidities, because all the research obviously excludes patients with other conditions, whereas most patients have got other conditions.”

Media interest

This issue can be particularly relevant for older people, but not in disease terms. Dr Finbarr Martin, consultant physician at Guy’s and St Thomas’ foundation trust and an adviser to the DH on older people, pointed out that elderly people are most concerned about dependency and institutionalisation.

“If we look at things like quality of life and loss of functional ability in older people, only about 50 per cent of it is actually disease attributable,” he explained. “So there is a lot there which is related to more subtle co-morbidities or frailty-type issues which are important in determining the outcomes for individuals.”

But focusing on specific disease areas also highlights individual challenges. In cancer, for example, national cancer director Mike Richards questioned whether there is the right balance between prevention, screening and early diagnosis on the one hand and treatment on the other.

“There will always be a lot of media interest in an individual patient - are they getting a treatment or not? - but far less media interest in the thousands of patients who may benefit from bowel cancer screening, from stopping smoking, whatever it may be.

“Getting that balance right is difficult,” Professor Richards argued.

“Our second problem is that we are still somewhat slow in taking developments from research and development back into practice. And that doesn’t only apply to drugs. If you look at something that Ara [Lord Darzi] is an expert in, laparoscopic colorectal surgery, only 10 per cent of surgeons in this country are still actually trained to do it and so only a minority of patients are able to benefit, despite the fact NICE has said it’s a good thing. We need to find faster ways of making progress and that’s where the Health Innovation Council may have things to say.”

For national director for heart disease Professor Roger Boyle, there remained the question of how reliable public opinion about priorities is - and how much it has been influenced by marketing.

“What we are seeing here is the success of some of the charities in selling their wares. We know that the cancer community has been absolutely excellent at that,” he said.

NHS Confederation policy director Nigel Edwards warned of “the danger of availability bias: the things I have seen are the things I remember”. A crucial question is how people view trade-offs between disease areas, he adds.

The “success” of cancer charities in raising the profile of the condition was reinforced by director of policy and public affairs at Cancer Research UK Richard Davidson, who reminded the roundtable that a Cancerbackup survey last year showed that, unprompted, 77 per cent of a sample of the public said cancer should take priority over other diseases in the NHS.

Definite disapproval

“There has been less competition from the cardiovascular and mental health charities over time and you could hardly miss the emergence of obesity and diabetes. You would have to stop reading every single piece of media to be untainted by the opinions of others,” said Professor Boyle.

“But for the public to ignore stroke, which is the third biggest killer and by the far the biggest cause of disability, when a third of all those strokes actually happen under 65 and when more than half the population do not know the difference between heart attack and stroke, then we are starting with a knowledge base which is way back beyond all of this.

“What it does require is a lot more public education and understanding and making information accessible to the public. Doing this in a meaningful and simple way that gives a balanced view about what these diseases mean for our communities could lead to more of a public debate about this.”

This view was supported by Long-Term Conditions Alliance chief executive David Pink, who called for a better match between the priorities of the public and priorities made purely on the basis of the burden of disease.

“We could have a perfect evidence-based health service which nevertheless collapses because the population it serves lose faith in it because their priorities are different,” he said.

“Maybe we should openly put into our equations a new term for the opinions of the public. I think that’s a frightening area to go into and like many other people, I would be worried about some of the things that might come out of it, but there’s no point in us pretending that they aren’t there.”

Nigel Edwards said he had been struck by comments made by US surgeon and writer Atul Gawande that health services research will make more impact on the burden of disease than anything in the current pharmaceutical pipeline.

“In other words, if we apply what we already know and pay attention to the way that systems of care work, we may produce much better outcomes,” he said. “Many of the interventions that you talk about in stroke and cancer care are actually often organisational interventions, almost management interventions. There is a point where you actually end up doing something clinical, but I think there’s a question here about priority.

“If you were prioritising and wanted to make the biggest impact, the public are unlikely to say that health services research is any area they want to put money in because it sounds suspiciously like management, and they definitely disapprove of that.

“If you ask them, the public often reply that the actual care they get is quite good but everything around it, the organisation, the way people co-ordinate it, is really rather poor.”

Lord Darzi on the discussion

The roundtable has been extremely useful. I probably should say this wearing both hats, with my new ministerial hat but also as a clinical scientist. What I have heard today is the science of discovery. There will be all sorts of different views about how we should invest in different areas of discovery, but what has come out very strongly, which I couldn’t support more, is that it’s the science of implementation of evidence-based care.

That is probably the most frustrating thing because you constantly believe that it is what’s happening out there. In matter of fact, that is not what’s happening out there, whether it’s the folate example, the obesity example, or the stroke example.

One thing we are not short of in this country is guidelines or standards. We probably have the best engine producing this through NICE and others, but not necessarily the best system in which we can implement these. And you need to ask why.

It’s for all sorts of complicated reasons, but I think the one agent which could be the most powerful is patients’ and the public’s understanding of what is a good quality outcome. The bit where we are behind is how do you empower patients with the knowledge base? We can only empower the patient if we actually have a more transparent way of measuring the quality outcomes being monitored, and they have to be high-impact issues.

On evidence-based implementation, the one area where there is a gap is long-term conditions. The science of stroke is easier because there is probably more clarity on the end points, but the science of how you manage long-tem conditions is probably the most challenging over the next 10 years. If you ask where am I going to put my money or where do you invest in the science of evidence-based implementation, it has to be in long-term conditions, whether that’s primary prevention or secondary prevention.

That is what we are trying to achieve through the next stage review, but that science is not held centrally; it should be at a local level, engaging clinicians and disseminating the knowledge base and its implementation.

Lord Darzi is a surgeon and junior health minister.

Cancer is by far the biggest concern for public

Cancer and heart disease are the disease areas that top the public’s wishlist for spending and research, according to a poll carried out by Opinion Leader for the Association for the British Pharmaceutical Industry.

Nearly three in four (72 per cent) in a nationally representative sample of 2,014 people in a survey in November identified cancer as a priority. This was some way ahead of heart disease (33 per cent for spending, 25 per cent for finding new treatments) and Alzheimer’s disease (11 per cent and 12 per cent respectively). These were followed by diabetes, HIV/Aids, arthritis and mental health.

“There were very few demographic differences, which in itself is quite interesting. One issue that did emerge is that people tend to be more concerned about diseases which affect their age group,” says Opinion Leader researcher Katie Shearn.

A follow-up event in January with 30 participants from across the UK confirmed the survey research but also found that people were concerned about a wide range of diseases to which they had a personal connection.

Overall, the research also found a strong consensus that individuals are responsible for their own health. There was an allied concern that today’s teenagers might be the first not to live as long as the previous generation because of poor diet and lack of exercise. It also found an underlying current of fear around mental illness.

Medical intervention to prevent diseases was viewed as a priority and there was a desire for the government to provide education.

There was limited understanding about who funds medical research and about the progress being made.

There was also concern over conflicting information about research, particularly around treatments and how people should be looking after their health and a feeling that GPs were a declining source of information.

Richard Barker on making the most of consensus

For all the talk of joined-up thinking in policy circles (especially health policy circles), it is often just that: talk. Different players frequently prioritise their own agendas and only pay lip service to holistic approaches to solving the big health challenges the UK faces.

However, the Opinion Leader research that helped to inform this HSJ and ABPI roundtable debate has uncovered a first - an area where there is genuine join-up between public desire, government action, academic research priorities and industry investment.

The surveys and discussions outlined in these pages show unanimity across society as to what should be the priorities for clinical research - cancer, heart disease and Alzheimer’s are the number one priorities. Public, NHS, academia and industry all agree.

Not only are the public and voluntary sector efforts on clinical research (as co-ordinated by the Medical Research Council) focused on these disease areas but the pipeline of the UK pharmaceutical industry is, too. In total,£3.9bn is being spent on all medicines research in the UK. Much of this effort is focused on providing treatments for these diseases, which are likely to be the cause of many of the challenges facing the NHS in the 21st century.

However, for all this agreement, there is a problem. Many are unaware that all this effort is going on. The public wants to know what is being developed on their behalf and all health stakeholders want to know what treatments are likely to be developed in the years ahead. There is an information gap and it needs to be tackled.

There is a clear role for industry to work in partnership with government to ensure greater public understanding of developments in both prevention and treatment, and increased accessibility of immediate information on treatments.

In response to this research and the discussion with experts outlined on these pages, the ABPI wishes to work with government and other key stakeholders to produce a survey of all clinical research that is under way in the commercial and non-commercial sectors. Building on the UK Clinical Research Collaboration report, this “state of the nation” report would be by therapy area, and would analyse a number of indicators which could include financial resources, people involved and number of compounds/treatments being developed.

By pioneering a way of tracking clinical research, the industry will lead in showing the value that all of the UK research community - across public and private sectors - is providing.

Richard Barker is director general of the Association of the British Pharmaceutical Industry.

Clinical priorities roundtable members

  • Richard Barker, director general, Association of the British Pharmaceutical Industry

  • Vanessa Bourne, head of special projects, the Patients Association

  • Professor Roger Boyle, national director for heart disease

  • Lord Darzi, junior health minister

  • Richard Davidson, director of policy and public affairs, Cancer Research UK

  • Professor Sally Davies, director general for research and development, Department of Health

  • Niall Dickson, chief executive, King’s Fund

  • Nigel Edwards, policy director, NHS Confederation

  • Dr David Haslam, president, Royal College of GPs

  • Dr Finbarr Martin, consultant physician, Guy’s and St Thomas’ foundation trust, and DH specialist medical adviser (older people)

  • David Pink, chief executive, Long-Term Conditions Alliance

  • Sir Michael Rawlins, chair, National Institute for Health and Clinical Excellence

  • Professor Mike Richards, national cancer director

  • Richard Vize, editor, HSJ