UNCORRECTED TRANSCRIPT OF ORAL EVIDENCE
To be published as HC 782-i

House of COMMONS

Oral EVIDENCE

TAKEN BEFORE the

Health Committee

national institute for health and clinical excellence (nice)

Tuesday 27 november 2012

Stephen whitehead, laura weir, dr linda patterSon OBE and professor peter johnson

Evidence heard in Public Questions 1 - 66

USE OF THE TRANSCRIPT

Oral Evidence

Taken before the Health Committee

on Tuesday 27 November 2012

Members present:

Mr Stephen Dorrell (Chair)

Rosie Cooper

Andrew George

Barbara Keeley

Grahame M. Morris

Mr Virendra Sharma

Chris Skidmore

David Tredinnick

________________

Examination of Witnesses

Witnesses: Stephen Whitehead, Chief Executive, Association of the British Pharmaceutical Industry, Laura Weir, Chair, Patients Involved in NICE, Dr Linda Patterson OBE, Clinical Vice-President, Royal College of Physicians of London, and Professor Peter Johnson, Chief Clinician, Cancer Research UK, gave evidence.

Q1 Chair: Good morning. Can I first of all welcome you to the Committee? This is a brief inquiry that we are undertaking essentially as background because we are going to be doing a preconfirmation hearing for the new chairman of NICE when the Government recommend a candidate. We are doing this brief inquiry into the work of NICE because it seemed timely, given that there is a new chair about to be appointed, to assess where the organisation is and what its priorities should be for the future. That is the background to this hearing. We are grateful to you for coming to this first evidence session. Could I ask you to introduce yourselves briefly so we know who we are talking to?

Laura Weir: I am Laura Weir. I am head of policy and campaigns for the MS Society and I am also Chair of Patients Involved in NICE, which is a coalition of over 80 patient groups. We are independent of NICE and the pharmaceutical industry and act as a critical friend and respected and equal partner in shaping and developing aspects of NICE’s work. I am also a board member on the NICE Implementation Collaborative, which hasn’t quite been launched yet but is going to be there to help facilitate the implementation of NICEapproved medicines.

Stephen Whitehead: I am Stephen Whitehead. I am chief executive officer for the Association of the British Pharmaceutical Industry, which represents all the R and Dbased industry in the UK. 90% of sales go to the NHS. We are also the statutory body that negotiates with Government on behalf of the industry on pricing and reimbursement, as you know quite well, Chairman. I would also say that we work extremely collaboratively and closely with NICE and have done for a number of years. We think the work that Sir Mike Rawlins has done has been pretty superb in terms of establishing the leadership of health technology assessment. Like Laura, I sit on the implementation board for the NICE collaborative with "Innovation Health and Wealth", and I also sit on the implementation board for "Innovation Health and Wealth" with Sir Ian Carruthers.

Chair: Can you speak up a little bit as the acoustics in this room are not great? Do you want him to say it again?

Q2 David Tredinnick: I would. I was admiring your fluency, but would you just say it again?

Stephen Whitehead: Yes, of course, sorry. That is a recall question for first thing in the morning. I am Stephen Whitehead. I am chief executive officer of the Association of the British Pharmaceutical Industry, which represents the R and Dbased industry in the UK. We are also the statutory body that negotiates on behalf of the R and Dbased industry for the pricing and reimbursement scheme that operates with the UK Government, currently known as the PPRS and soon to be the valuebased pricing scheme as well. I also sit on the implementation board for "Innovation Health and Wealth" that is chaired by Ian Carruthers, looking at adoption and diffusion of innovation, particularly NICEapproved medicines within the NHS, and I also work closely with patient organisations on the collaborative around NICE introductions within the NHS in England as well.

Dr Patterson: I am Dr Linda Patterson. I am a consultant physician by background, working in East Lancashire, but I am the Clinical Vice President of the Royal College of Physicians and so I am responsible for quality, standards and safety of patient care at the college. The college has over 27,000 members, who are doctors mainly working in the hospital service representing over 30 medical specialties. These are the people who will be implementing NICE guidance in the clinical transaction with patients; so they are absolutely at the bedside. We also have a formal relationship with NICE in that the RCP hosts the National Clinical Guidelines Centre, which is contracted by NICE to prepare the national clinical guidelines, so we have a governance arrangement with NICE. That is the largest such guidelines centre in the world and produces over 60% of all guidelines that go into the NHS.

Professor Johnson: I am Peter Johnson. I am the chief clinician at Cancer Research UK and a practising oncologist. Cancer Research UK is the largest researchfunding organisation in cancer, independent of government, in the world and has several million supporters in the UK, who fund our research, and we don’t receive any direct funding from either governmental or pharmaceutical company bodies. I have taken part in several appraisals with NICE as an expert witness and have had occasion to comment on some of their particular singletechnology appraisals in the past as regard the access to cancer drugs.

Q3 Chair: Thank you. I would like to start, if I may, by asking some questions frankly for the purposes of clarifying what are the implications of approval by NICE of a particular drug or procedure. There is regular discussion about whether a drug once approved by NICE ought to be available, must be available and who is under an obligation. There was a piece of legislation passed in 2005 that is reported to have placed the NHS under a legal obligation to adopt, or to fund, NICEapproved drugs. What is your understanding of the precise consequence for the NHS of the approval of a particular drug or procedure by NICE?

Laura Weir: If a treatment is approved by NICE, it is judged to be both clinically and cost-effective. So, if a clinician says that a particular medicine is clinically appropriate for you as a patient with a particular condition and you meet the NICE criteria, then, by law, the patient should be able to access that medicine. That right is enshrined in the NHS Constitution.

It is also supported by a funding direction that was issued by the Secretary of State in 2003. I was at a conference very recently where a lawyer informed the attendees that the funding direction is lacking in meaning in that, in all of the court cases so far where PCTs have been taken to judicial review over not implementing NICEapproved medicines, the court has said that there has been a tension between the PCT’s legal obligation to balance its books and the policy that mandates it to prescribe these medicines. He said-this was his advice-that the PCTs can get away with not providing NICEapproved medicines so long as they do not have a blanket ban or a quota system. I am not sure to what extent that is a right in the eyes of the law, but there are certainly policy levers there that would support patient groups. I would say that the reason there have been so few judicial reviews is because PCTs will, in some cases, drag their heels and have certain delay tactics, but essentially, because of the funding direction, they wouldn’t have a case if it got to the court.

Q4 Chair: In principle, in the characterisation of the law you offered, if a clinician prescribes a drug, the PCT may drag its feet or not, but it is not in a position to refuse to fund. That clearly isn’t the experience.

Professor Johnson: I am not sure that there is an enormous amount of failure of implementation of NICE guidance. There are some cases and some instances, but our general experience is that, once a positive appraisal has been given, the uptake is reasonably broad and rapid, with a builtin delay, which is imposed by most commissioners, of a small number of weeks during which they have time to accustom themselves to the guidance financially. But, in general terms, the uptake has been pretty good.

Dr Patterson: I would agree with that. There are lots and lots of instances of PCTs failing to fund, as far as I am aware. I am sure there are some cases, but it is not a general issue as far as we can see.

Stephen Whitehead: I would take issue with that, Linda and Peter. The latest Metrics report that was produced by the Department of Health, in collaboration with the ABPI through the NHS Information Centre, very clearly demonstrated that the UK is both low and slow in terms of adoption even of approved NICE technologies, with huge regional variations. If you live in Leeds you may get something, whereas if you live in Lancaster you will not get something.

The UK is the slowest adopter of innovation in Europe against all DH standards-this is not just the industry talking but independent research that has proven this-and, although often medicines will go on the formulary, there is often not the freedom for the healthcare professional to prescribe the most appropriate medicine for the patient. This may be down to various issues such as silo funding or overfocus on the drugs budget compared to other costs. Although there are levers that can be pulled by active patient organisations and the NHS Constitution should guarantee the right for patients to have access to new medicines or new technologies, that is often not the case. This has been explicitly recognised by Government through the publication of "Innovation Health and Wealth", which says that the uptake of NICE medicines is a problem in the UK.

I can just talk from a medicines standpoint, but there are many other technology assessments that NICE do as well. It is recognised as a problem, and it is a problem that we must address. We have been very supportive as an industry in supporting NICE on this path, and Mike Rawlins himself-the current chairman-has said on record that, if these technologies are not being made available, then legal pursuit should follow. There clearly is a big debate round this, which demonstrates that there is a problem.

Q5 Chair: If I may make the rather obvious point, it is easier to address a problem if you first agree you have a problem.

Stephen Whitehead: Yes.

Q6 Chair: We have a conflict of evidence being presented to us.

Professor Johnson: Perhaps I could respond briefly to that. I absolutely agree that our rate of uptake of innovative treatment is lower in the UK than it is in some parts of Europe. I am not sure that I would necessarily place the cause of that at hindrance of uptake of NICE guidelines. We have a medical work force that is relatively more conservative than perhaps in some parts of Europe about the adoption of new forms of treatment. [Interruption.]

Chair: I apologise about the sound effects off.

Professor Johnson: It is the man from the drugs company trying to get out of the cupboard.

Chair: I interrupted you. You were saying we have a conservative medical work force.

Professor Johnson: The prescribing habits of UK doctors are relatively conservative perhaps by comparison with some of our colleagues on the continent. I think there are a variety of reasons why innovative treatments are not being taken up as rapidly as they are in the better-off parts of western Europe and I would not put the majority of the blame for it on obstruction of the implementation of NICE guidance.

Q7 Andrew George: The report that you referred to first is one which you all acknowledge-that comparisons across Europe as a whole show that in England and Wales, at least, implementation is slower than in other areas. You do accept that.

Dr Patterson: Yes. I think there is an issue about implementation, which is partly about practice-the clinical decision maker either implementing or not the guidance-and the drugs that are available. I thought what we were talking about was clinicians prescribing and then the PCTs saying that they weren’t going to fund. That was the bit about which I was saying that we have not heard a huge amount of noise. If we are going to talk about wider implementation, that is absolutely a debate we can have about how we encourage good practice to be implemented across the country without variation.

Q8 Chair: If that is right, the conclusion would be that the issue is slow takeup by clinical practice and that, if clinicians were quicker to respond to new treatments that worked for their patients, there would be a spending issue for the new CCGs that they would have to accommodate because there would be a legal right for the clinician to use the new treatment for the benefit of the patient.

Dr Patterson: Yes.

Stephen Whitehead: Could I add something there? [Interruption.] Can you hear properly? Bearing in mind-

Chair: Yes, but please do speak up because of the background noise.

Stephen Whitehead: It is very important to recognise, of course, that NICE undertakes value assessments. There are various outputs, which are obviously technology appraisals and clinical guidelines, which have various levels of legal applicability. Some of them have a legal followthrough and some of them don’t, depending on what process they have gone through. Having gone through a value assessment-proving the value of technology, be it a medicine, a device or whatever it is-NICE has made an assessment that it is a valuable addition to the healthcare system. Often it will change a patient pathway of care and make that patient pathway of care much cheaper.

For example, there is the introduction recently of new anticoagulant medicines that will replace warfarin, which is a very difficult drug to take; it includes a lot of hospitalisation. Warfarin is a very expensive, old medicine on the healthcare system because of its side effects. The new medicines are much cleaner to take. I think the cost is about £2 a day as a cost of the drug, but the overall cost of treating those patients comes down and the utilisation of the innovative technologies should free up other resources in the healthcare system. So an overfocus on the drug budget remains a problem in terms of patient access and wellbeing but also in terms of liberating the system to free up resources for other things.

Q9 Chair: Would the clinical witnesses agree with that?

Dr Patterson: I think it is right that you can overemphasise drugs and not see the oncosts of either giving or not giving a drug and, then, if the treatment pathway changes. Warfarin is a good example where people have to have regular blood tests and so on, whereas if the new drug is given they won’t have to have those oncosts: you don’t need the costs of the pharmacy, the technicians, the phlebotomists and the ambulance to take people out to the clinic and all that sort of thing. But, of course, trying to cost that in is difficult. That is really sophisticated costing, which we don’t do.

Q10 Chair: That was what the NICE process was supposed to do, presumably.

Dr Patterson: Yes. I am not sure it does it in such detail.

Stephen Whitehead: Linda is absolutely right. It is supposed to, but what we would like NICE to do is take into account broader societal assessment of value.

Q11 Chair: That is a different point.

Stephen Whitehead: It is, but it is an associated point.

Professor Johnson: I absolutely agree. There is a very tight focus a lot of the time in the way that NICE considers health technologies on the immediate costs and benefits and the rather broader consideration of what effect it might have in other parts of the patient pathway. A failure to take into account the broader value of particular healthcare interventions does restrict their vision of what constitutes "worth while" for the NHS.

Laura Weir: The reason why it is quite hard to make an assessment of implementation of NICEapproved medicines is because you need two sets of data. You need to know how many people are eligible for a particular drug versus how many people are actually on treatment. Currently, NICE try to do that in what they call a costing template, so when they make a decision on a medicine they will also break down by PCT how many people they estimate should be eligible for that treatment by PCT area. What the score card would do is take some of that data and give you a trafficlight colour, which shows, according to PCT, how well a particular medicine is being implemented. That would be fantastic for improving accountability because, currently, we don’t have that. There is no scrutiny or monitoring of NICEapproved medicines. There is no one body that is responsible for making sure that we do have much broader implementation of NICE guidance, not just restricted to medicines but to clinical guidelines and quality standards.

Q12 Chair: That, presumably, should be a function for the Commissioning Board, should it not, in the new world?

Laura Weir: The Commissioning Board has to have due regard to quality standards. I am not sure what that means. I think there is a role there for the Care Quality Commission potentially, together with Monitor, to assist the NHS Commissioning Board to carry out that role, to monitor clinical commissioning groups and to assist them in providing that scrutiny.

Dr Patterson: There is no funding stream for implementation at the moment. I don’t think it is for NICE to implement guidance. It is for the clinical community and the service to implement guidance. The professional bodies and the body of clinical opinion are there to be used to help in implementation. You can do that through audits, quality improvement projects, measuring what is happening and then looking for your improvement in how you are doing, particularly around the guidelines, I think. The use of drugs you could perhaps measure in a different way. At the moment it is a rather grey area as to who is responsible.

As to the issue about taking due regard for the quality standards, we are all slightly muddled as to what the status of the quality standards is going to be within the new system, how mandatory they are, whether they are advisory or aspirational, and how they really fit in with the clinical outcomes framework. Those of us who have struggled and tried to look at all this are a bit baffled. The ordinary clinician on the ground is going to be even more baffled, quite honestly, and I don’t think there is a clarity at the moment as to how the quality standards are going to be used. I don’t know if you would agree.

Professor Johnson: I would agree absolutely. We have a series of different forms of information and the quality standards appear to be predominantly aimed at commissioners rather than at clinicians in terms of their anticipated impact. We feel that an opportunity has been missed in formulating the quality standards that we have seen relevant to cancer to highlight the importance and the primacy of research in the NHS, which is absent at the moment from those.

Chair: We might come back to that, but we will move on.

Q13 David Tredinnick: I want to talk about patient views for a moment. There is concern that the views of patients are insufficiently taken into account when NICE takes its decisions on drugs or therapies. Can you give any specific examples of where this has happened?

Laura Weir: With regard to patient group involvement in technology appraisals, there is no clear role for patient groups. We are invited to give oral evidence when NICE are approving a medicine and we are also invited to give a written submission before then. It is not clear whether NICE want us to literally comment on the assumptions going into their model or if they want a case study. Do they want to know how this drug has impacted on someone’s life? Do they want us to produce more qualitative or quantitative evidence? It is very unclear where we fit in in the grand scheme of things.

Patient groups find technology appraisals quite an intimidating environment. It is not clear that our views are of equal value to perhaps others in that room. Unless I have got this wrong, I am not clear that there is a code of conduct either for committee members. I wouldn’t say that patient groups are not included in the process; we are, but it is just that our role isn’t very clearly defined. Yet patient groups will spend considerable time and resource in terms of inputting into that process.

Q14 David Tredinnick: How would you like to see the patients’ perspective included in future arrangements for NICE? We have a vision of the future, particularly now we have had these major health reforms.

Laura Weir: There are some simple things that NICE could do. It has been established for 12 years now, so it could look back at previous submissions from patient groups and tell us what we are doing well, where we need to improve and what is useful for the committee. It could help patient groups to understand what happens in a technology appraisal and what goes into an economic model. It could make better use of our expertise. For example, lay people on the NICE Appraisal Committee are invited to give a 10minute opening statement about the condition that a treatment is for, yet you have a patient in the room and someone from a patient group who could probably do as good a job, if not better, because they are the experts. So I think there are quite a few things that NICE could do to help.

Professor Johnson: An important point is touched on there, which is that the perspective of a patient with a condition is very different from that of a lay person in the healthy population. The transformation of the attitude and the approach to healthcare that takes place at the point at which somebody gets a diagnosis, particularly a serious diagnosis, is radical. NICE has relied heavily on lay opinion from the healthy population and rather less so on the patients.

Stephen Whitehead: In addition to that, clearly, in the operation of NICE, it has emerged that it has become much more important. We know, going forward, given its new independent status, that NICE will become even more important as an influence in provision of healthcare in the UK. There is very clearly, from the patient perspective, what we would call a democratic deficit. There is an overweight given to the health economists and less weight given to the clinicians, the patient organisations and even the industry. Within that context, going forward, the voice of the patient should be enshrined constitutionally in what happens to NICE because it is the patients, at the end of the day, that we are concerned about. Particularly, the voice of the patient concerned with the specific illness that is being assessed and looked at should be given greater clarity. That should be constitutionally enshrined rather than what we have at the moment, which seems to be somewhat ad hoc.

Q15 David Tredinnick: You say that, but what consideration have you given to the impact of HealthWatch and the Health and Wellbeing Boards? I put it to you that the whole health landscape is changing dramatically and that, for the first time, patients are going to have the statutory position that you have just asked for. Surely it is already there, isn’t it? It is coming forward.

Stephen Whitehead: It seems to be there at the local level. The role of NICE specifically, which we are addressing now, needs to embrace patient interests because they are going to be independent and much more powerful, much more driving standards across the NHS and the adoption of innovation. We need to look at this specifically within the context of NICE, where the patient needs greater clarity and greater presence.

Q16 David Tredinnick: I am saying that surely, with this huge Health Bill that has just become an Act, NICE has given consideration to the empowerment of patients through the Act. With these new consultative groups, which will be available in all local areas, we are going to see patient suggestions coming forward. Have you given any thought to an interface or to monitoring what is happening?

Chair: I think Dr Patterson is trying to get in.

Dr Patterson: Yes. I was going to put a more balanced view than that. What NICE does is something rather remarkable. It looks at academic evidence, clinical opinion, patient need, economic modelling and the views of the developers, research and so on, and brings that together into an appraisal through a process, and then out at the other end comes advice. Particular interest groups within that may not all be completely happy with every single thing that happens along the way. I have sat on a NICE appraisals group, so I know the process and know that sometimes academic clinicians have not felt that their voice has been heard as strongly as it should because the weight of research evidence is balanced against what patients want, what patients are saying is important, and then, finally, with an economic evaluation. We don’t believe that the economic evaluation always trumps everything.

What I am saying is that, on the whole, they do a pretty good job of bringing together all these particular interest groups. I am sure the voice of patients can always be strengthened and one would want to work to make that so. We have had experience at the RCP of very strong patient input into some of the guidelines, and really that is a very strong voice that has actually changed the way things have come out. Obviously, it is never enough, we could do more and should support patients to feel that they are able to vocalise what they want, but that is a voice that needs to be balanced across everything. On the whole we, as clinicians, have not felt highly critical of that process and have felt that it has come out in the end with a reasonably good job.

Chair: Before you move on, Grahame wants to come in on this point.

Q17 Grahame M. Morris: Briefly, it might be useful at this point if you could summarise some of the things you told us a little earlier. The written evidence that we have received as a Committee has been very supportive of the progress and the contribution that NICE has made, but most of the organisations that have submitted evidence say there is room for improvement. I have a question to each of you. Apart from the patients and clinicians having a greater say and speeding things up, is there one particular issue that you would care to highlight for the Committee that is of concern to your organisation?

Chair: Laura is nodding so we will start there.

Laura Weir: NICE does a fantastic job and produces some brilliant guidance. However, that guidance is only really going to help people if it is implemented. A lot of people will say, "What is the point of spending all this money on NICE if actually no one does what it says or follows what it says?" There is a real opportunity here for us to look at the role of the regulators. NICE was never set up to be a regulator; it has an implementation team that helps to facilitate and spread best practice, but it is not actually a regulator.

What can the CQC do, for example, to assist the NHS Commissioning Board? We are going to move from 152 primary care trusts to 212 clinical commissioning groups. It is going to be extremely difficult for patient groups to hold those clinical commissioning groups to account locally through HealthWatch and the Health and Wellbeing Boards that you have just mentioned if there is no accountability, if no one is offering that monitoring or scrutiny. For me, it has to lie with implementation. To add to that, one of the things that the Act is intended to do is improve the integration of health and social care services. NICE has a real opportunity here, I think, to produce guidance for commissioners that helps them to consider how they might improve the integration between health and social care by incorporating social care within its guidance. Those would be the two things from me.

Stephen Whitehead: I would make two points also. Clearly, once NICE has made a decision or a recommendation, it should ensure that it is followed and adopted within the NHS itself in England and Wales and where NICE procedures should be followed. Also, I think it is most important that NICE is linked to the life sciences strategy that the Government are pursuing so that it supports the growth agenda in life sciences that the Government are so keen to drive. Both this Government and the previous Government reflected upon the life sciences sector and saw it as fundamental to the future growth and economic prospects of the UK. What is pivotal is that NICE doesn’t undermine that in some of the recommendations and approaches it takes.

Q18 Chair: That is a theme we will come back to.

Dr Patterson: I would agree about implementation. I think there is a lot of expertise in the NHS and in professional bodies that can help that, and we have already discussed it. The other thing we would encourage NICE to do as it goes forward is to continue to be more flexible with the methodology. The gold standard is the randomised control trial and the research evidence that is within that. For many conditions, we don’t have randomised control trials so you have to have a "best fit" of what evidence there is available. Sometimes it is at the level of a professional consensus, and that is what doctors do all the time. They weigh up the evidence that there is around and come out with some sort of best decision.

NICE has begun to explore some of those methodologies. It has recently had a Delphi Exercise where it brings together and sifts out opinion in a way to come out with a best practice. Particularly as it moves into social care and interface issues between health and social care, the research evidence is not strong within certain academic disciplines. It is going to need to learn to sift evidence which is there in a different way, maybe more qualitative and so on. So there is a challenge about the methodology. We have seen a move on that. We have seen a much more flexible approach recently and we would consider that that needs to go forward.

Q19 Chair: This is a general question, so we will take Professor Johnson next and come back.

Professor Johnson: I would echo much of what has already been said. Cancer Research UK is very broadly supportive of NICE and feels that they have done an excellent job in a difficult environment. Where we see principal room for improvement is continued emphasis on the speed with which they carry out their appraisals. Although there has been a good deal of progress recently, we still feel there is room for improvement in speed and for the broader appreciation of what patients value in healthcare, which I think speaks to the point that Linda has already made. We welcome their increasing flexibility of input. Their concentration on measuring the measurable in the past has led them to diverge from what we would have regarded as a broad clinical international consensus on certain forms of treatment because they got themselves hung up on a particular methodological approach, and I think the broadening of their vision, particularly as they are asked to input into the process of things like valuebased pricing for drugs, would be welcome.

Chair: That is a nice leadin.

Q20 David Tredinnick: Yes, very much so, Chairman, thank you. I would like to move on to value for money. The Government have announced that they will move to a system of valuebased pricing for drugs-we just referred to it-in the NHS from January 2014. Do you support the move, please?

Professor Johnson: Yes. Broadly, we think we do. The difficulty we have is that at the moment the process is not especially transparent and we have rather little information to go on about how the notion of value is going to be derived. We would like to see patients very much involved in the design of that process of the derivation of value and recognise, obviously, that NICE needs to be at the heart of that system.

Dr Patterson: I would agree with that; it is not clear at the moment. There isn’t a "How it’s going to work".

Laura Weir: Patient groups have argued for some time that NICE should broaden what it considers as valuable. Currently, it takes into account costs to the NHS and personal social services, but to people living with a condition it matters if their child can return to school because they have autism and there is a medicine out there; if someone is living with dementia and there is a medicine there that will help that will reduce the burden on the carer, then obviously that matters. But those are all implicit rather than explicit parts of an appraisal.

Q21 David Tredinnick: May I suggest to you-going back to the previous question-that HealthWatch and Health and Wellbeing Boards are going to give patients a chance to articulate what they feel? What many patients do feel, I would suggest to you, is that they want a wider range of services. They are also very focused on cost. I would like to know how you would view the much less expensive but much older remedies such as traditional Chinese medicine, phytotherapy and herbal medicine. I see Mr Whitehead is trying to get in.

Stephen Whitehead: I am sorry. I was seeking to address your previous question and then I will seek to look at the other one as well, if I may. Clearly, on valuebased pricing, as with the rest of the people here, it is not completely clear how that will be introduced, but we do support the broader societal assessment of value. Also, through that process, as the valuebased pricing is developed as a methodology, we absolutely endorse and support the involvement of the patient organisations and the professionals in how that is looked at. It is fundamental that we address the issue of availability of new technologies into the system.

What we don’t want valuebased pricing to be is another hurdle we have to go through. Our preferred route would be that NICE does those assessments, looks at broader societal assessments and value, and takes into account the costs of carers and those sorts of things. I don’t think NICE-in fact I know NICE doesn’t-looks at traditional remedies. To be honest, my opinion is always based on "clinically proven" and I don’t know what trials there are on traditional Chinese medicines. I would probably not draw an opinion on them until I was aware of what trials there were.

David Tredinnick: I will help you with that.

Q22 Chair: Perhaps we should hear from the other witnesses on this point of alternative therapies.

Dr Patterson: I was going to agree that I think the evidence on traditional medicines, the number of trials and the validity of those trials could be looked at. That would be the basis upon which one would go forward and say, "These things are useful or not."

Q23 David Tredinnick: That is very helpful because, of course, the Department of Health is currently finalising proposals for the legislation to regulate herbal medicine practitioners, and we now have the European Directive on Traditional Herbal Medicinal Products, which has brought in standards. So the argument that we need a structure and more evidence, I would suggest to you, certainly in the case of phytotherapy, herbal medicine and traditional Chinese medicine, is not that strong; there are trials there. There is also the empirical evidence of 2,000 or 3,000 years of usage in China.

Going back to the costs issue, these therapies are a lot cheaper than many drugs that are available. Also, these practitioners very often reach the parts the other doctors cannot reach, to paraphrase the old Heineken advert. They tend to deal with patients who haven’t found that orthodox medicine has been able to treat them. I put it to you that this is something you should be interested in on two accounts: one, that we have legislation here; secondly, that it is costeffective.

Chair: Would you like to give the patients’ view?

Laura Weir: I guess it comes down to patient choice, but, given that this inquiry is about NICE and NICE bases all of its guidance on evidencebased medicine, it is very difficult when you look at homeopathy and complementary medicines because the evidence just isn’t there. I guess NICE has to make recommendations about how we should best use the money that we have and we want to use that on clinicallyeffective as well as costeffective medicines.

Q24 David Tredinnick: That is very helpful. I am going to finish on this, Chairman, because I know we need to move on. On the point you made about a research base for homeopathy, I have the Faculty of Homeopathy, which is the 150 medicallyqualified doctors, like you, Dr Patterson, who have also studied homeopathy. Their latest release on the research evidence base for homeopathy says, looking at the systematic reviews of randomised control trials of homeopathy, that four of five major comprehensive reviews of RCTs in homeopathy have reached broadly positive conclusions and that positive conclusions have been reported in 10 of 29 reviews of RCTs in specific categories of medical conditions, which are allergies and upper respiratory tract infections, childhood diarrhoea, influenza treatment, post-operative ileus-I hope I have pronounced that correctly-rheumatic diseases, seasonal allergic rhinitis and vertigo.

Saying that there just isn’t any evidence has been used against homeopathy because of the recent campaigns by some of those who have very strong vested interests against the simple, cheap and effective treatment of homeopathy, which has been available in the health service since its inception, but the situation is that there is evidence. I would like to put it to you-I am very happy to leave this with you; in fact perhaps Mr Skidmore will be kind enough to pass it along so that you can look it up-that you should be looking at this and you shouldn’t buy into all the propaganda that is out there, because it is quite clearly not correct.

Chair: It is a debate, I suspect, that will run.

David Tredinnick: Chairman, can we maybe have a comment on that, please?

Dr Patterson: I practise in the way I was trained-to practise evidencebased medicine-and I do not practise homeopathy.

Stephen Whitehead: I would point out from the industry that we do not in any way disparage homeopathy or alternative remedies either, but we do look at evidence-based research and, if we have evidence, then of course we will look at it and assess it. But, as an industry, I represent evidencebased clinical research.

Q25 Chair: Professor Johnson, would you like to comment?

Professor Johnson: Yes. I think "demonstrably effective" is a very important starting point for any healthcare intervention. There is no doubt that there are some potentially valuable pieces of information to be dissected out from, for example, traditional Chinese medicine. There is a very effective antimalarial that has come out of traditional Chinese medicine. The difficulty is that sifting the evidence, up until now, has not been particularly successful. I think, as China itself opens up, there may be opportunities for collaborative research and for work in that area to enable us to pull out what is potentially of benefit for the future. But it is very clear, certainly in the cancer field at the moment, that there is an awful lot of work still to be done in assembling the evidence of usefulness.

Chair: Thank you. Those are clear answers. I think we will move on. We will take Andrew and then Virendra.

Q26 Andrew George: I have three what I hope will be quick questions and they may only be for my benefit. The first one is on valuebased pricing. As I understand it, if it is value based, then presumably the price of well-established life-saving remedies like Anadin will go up, will they? Those rather expensive-to-produce drugs that have only a marginal beneficial but significant effect for some people will be more expensive if they are value based. Is that right? Can one of you, at least, explain how you see valuebased pricing working?

Stephen Whitehead: It is a very interesting question, Mr George, and it is important to understand that only new medicines will be assessed; something like aspirin would not be assessed. It is only new medicines assessed from 2014 that would go through a valuebased assessment process. It is also fundamental to understand that, currently, any medicine that goes through NICE also goes through a value assessment. All we are talking about is an evolution from current assessment processes, hopefully with broader societal implications taken into account. We obviously do not want to see the price of branded prescription, new medicines go down as an industry because the UK already has the lowest price in Europe at the moment and the slowest uptake, I think it is important to understand, and the UK as a market typically does not recoup the R and D investment made in the medicines launched. It is a very difficult market for new medicines. We are hoping valuebased pricing will speed up the access and adoption of new innovation.

It is also important to understand that most medicine is iterative by its process. So you refer to little steps. Those little steps are fundamental to understanding and treating diseases. If you take something like HIV, thanks to the intervention, process and progress made by the pharmaceutical industry itself, it was three drugs that got us to a treatment. First, it was AZT, then things like 3TC and then the protease inhibitors. Taken together, they prevent the progression of the disease. It is iterative steps that get you to a solution and it is the same with colorectal cancer-three drugs together. So it is important to understand in medical discovery, in biopharmaceutical discovery, that it is little steps that get you there. It is iterative innovations. So long as valuebased pricing understands and accepts that process, then we would be happy to see its progression, particularly looking at wider assessments of value.

Q27 Andrew George: Also, in that and in relation to the second part of the question, those new innovations will be at their most expensive when they are first being rolled out and a value may be difficult to assess. A valuebased pricing mechanism may be very challenging for your industry.

Stephen Whitehead: It will be very challenging because, of course, at launch of the medicine you often don’t know its true potential. You often launch a medicine with quite a small indication and then subsequent data that we get, by working with the NHS through further trials and so on, gives us further indications for the medicine. Whatever valuebased pricing does, it must not interfere with that iterative process of working within the system to get more data. That is one thing.

Laura Weir: What your question helpfully highlights, Mr George, is that we are all speculating about valuebased pricing and there isn’t actually an informed debate because no one knows what value-based pricing will look like. It has not been transparent, and, as patient groups, we are outraged in fact that we are setting up a system that will benefit patients but we haven’t been involved in designing that system and we haven’t really had a say in what that system could look like. We would like to be involved in those conversations so that we can comment. Yes, it is technical, but it is not beyond us as patient groups and we have a role to play.

Q28 Andrew George: Dr Patterson, you have just heard Mr Whitehead complain again, if you like, or comment on low and slow take-up, but, on the other hand, there are others in medicine who clearly want to avoid a situation where patients become drug-dependent, as it were. There is often an emphasis on clinicians to reduce the prescribing budget. Are there two issues, if you like, running in conflict with each other there?

Dr Patterson: One aspect of NICE guidance that we have not articulated this morning is that sometimes it does talk about disinvesting. It says that there are certain investigations, drugs or ways of treating patients that are not valid even if they have been custom and practice and really should be stopped. There is another side to this. It isn’t all about doing new, doing more and giving new drugs and so on. Particularly, I think, in the guidelines-which are about effective best practice-we need to give patients the right treatment, but we also need to stop treatments that are ineffective. That is the clinical challenge, I think.

There are ways that we need to think about that. For instance, as the population ages, more and more people have more and more conditions. If each condition is treated as a single entity, you can end up with 10, 15 or 20 drugs to be taken every day, which may have interactions, side effects and which may in themselves cause some problems for patients. It needs a skill to be able to manage those multiple conditions alongside each other. What may be true if you just had the one single condition may not be appropriate for you if you have two or three others. That is a challenge that British medicine is coming to terms with because we are absolutely facing a change in population that we are dealing with.

Q29 Andrew George: Can I wrap my last two questions into one because of time? It relates to the effectiveness of procurement within the NHS and to ensure that the NHS doesn’t become beholden to a control that perhaps pharmaceutical and other companies have over them. NICE’s role, as you see it, is a universal price regulator through the NHS. When they establish a valuebased price for any remedy, are you content that both the existing and the new NHS structures are competent to ensure that those prices are universal through the NHS and that it doesn’t result in variations in pricing in different locations? I am simply aware of the fact that some commissioners of service perhaps aren’t as effective as others at ensuring that they get good value for money.

Dr Patterson: I think you have said that, yes, and we are going to assist them where we have an even greater number of potential commissioners; obviously, there will be variation in skills. I think that is something that we need to watch out for a lot. The question is, "Who is going to monitor that? How is that going to be kept an eye on?"

Q30 Andrew George: Do you see a role for NICE in introducing greater price transparency to assist commissioners in ensuring that they are getting their remedies at the right price?

Dr Patterson: I think, obviously, greater transparency. NICE is already trying to be transparent. It is on that journey and we would encourage more transparency. That is going to be a key thing. It is very pertinent that you are picking out these issues because I don’t think we do know at the moment what the system for valuebased pricing is going to be across the country. These are precisely the issues that need to be picked up and teased out. As to allocation of responsibility-"Is it commissioners? Is it the National Commissioning Board? Is it groups of clinicians? Is it NICE?"-we don’t know how that is all going to play together, I don’t think.

Professor Johnson: Some of the better examples of the success of NICE are in some of the access schemes that have been negotiated whereby pharmaceutical companies have managed, through such a scheme, to reduce the effective price of the drug without altering the list price. NICE has been handicapped in achieving those negotiations because it doesn’t have a direct responsibility for negotiating price at present. So a convergence of those two functions-the assessment of value and the negotiation of price-would be extremely valuable going forward and would build on a series of good examples of what it has done previously.

Q31 Andrew George: Before the two of you add your own comments on it, it has always struck me that the NHS is such a large and significant body that its procurement power, at least, if it is working in a co-ordinated fashion, should be able potentially to dictate market conditions rather than have them, as it seems, dictated for them by the fragmented way in which it seems to commission services.

Stephen Whitehead: If I could comment there, Mr George, the Department of Health has responsibility for pricing and undertakes negotiation on pricing every five years through the current scheme. That has secured the UK the lowest prices in Europe at this moment in time. Some of that may be currency fluctuations, but we are sitting in the bottom quarter within the European Union alongside crisis countries like Greece, Spain, Portugal and Ireland. Also, the other thing that we must recognise is that the DH represents the UK as a whole, whereas the NHS National Commissioning Board and NICE are technically only England. We don’t want to have differential pricing across the four nations that make up the UK. That would be a disaster for the industry. I also think the Department of Health try and balance in what they do through negotiations the health of the life sciences sector in the UK.

The NHS is a monopoly purchaser-we recognise that-and that is not always easy to deal with as an industry, you can understand, but also it is important to recognise that a negotiation goes on that is very arduous and difficult and has resulted in price cuts over the past 15 or 20 years. The last one introduced a price cut of 5%. This is just on the branded research-based medicines, not the ones that have gone through patent expiry.

Laura Weir: I have two very brief points. In terms of valuebased pricing, we already have a form of it. NICE take into account burden of illness and other factors that are not part of their statutory remit, but it is an implicit part of the process rather than made explicit, which is what valuebased pricing will do.

We also have patient access schemes, so, if a drug is clinically effective but the cost is so high that NICE may be looking at a no decision, there is an opportunity for companies to work with NICE to maintain their list price but offer their drug with an NHS discount. So we are already negotiating value and price there. We feel that NICE should be at the heart of any new system of valuebased pricing. One of the good things about NICE is that it has used the same methodology, applied it fairly and consistently across all condition areas allowing for comparisons. Will we be able to say the same about valuebased pricing? I think that is an important question for the Committee to ask.

Q32 Chair: Before we go to Barbara, can I be clear on this? Accepting entirely that "valuebased pricing" is a phrase with very little substance attached to it at the moment, you seem to be speculating that the effect of this proposed change is to enhance the role of NICE as a direct price negotiator for the introduction of new compounds. Instead of a new compound being proposed and a judgment being made, there would be just a straight negotiation about the price to be applied to it. Is that a correct interpretation of what you are saying to us?

Laura Weir: I am not sure to what extent it will enhance the role of NICE. From what I have heard, it will be a different committee that will make the final decision. One of the great things about NICE is that it is evidence-based; it is objective. Also, if you are introducing another layer, if you are introducing another system on top of NICE, what does that mean for the time it takes to appraise a medicine? Are we potentially increasing the levels of bureaucracy?

Dr Patterson: I don’t know.

Q33 Chair: You don’t know.

Stephen Whitehead: Obviously, the Department of Health is not here, Chairman, but it has gone through a consultation process and is continuing the consultation process as this system is designed. We are part of that consultation process, obviously, and then it will be subject to negotiations, we hope, with the industry. There should be as much stakeholder feed into that process as possible, so it is not simply done in closed rooms and we are conscious that the consultation process on VBP is going on.

Q34 Chair: It is about the process. It is not about the way prices and decisions are made on individual compounds.

Stephen Whitehead: We don’t know how that will be designed at the end of the valuebased assessment process at this moment in time.

Chair: "Don’t know, guv." Right.

Q35 Barbara Keeley: Hopefully people might have an opinion on the next question. The Cancer Drugs Fund will come to an end in 2014. How effective has it been and should the principles underlying that fund be used in the new system?

Professor Johnson: I think the Cancer Drugs Fund has been immensely effective in removing the very difficult and heated discussions that went on previously for access to novel treatments. There is no doubt that it has speeded up the access to treatment for somewhat above 20,000 patients in the UK. I think we are still awaiting a detailed assessment of what value has been gained from that and it is important that, in evaluating the effects of the Cancer Drugs Fund, we get some good data from the Department of Health about how the money has been spent and what the benefit has been.

Even if we move to valuebased pricing, there will be a tail of drugs that we will still need to pick up in some way, and I think some continuation of that specific form of funding would be very effective. Certainly in talking to European colleagues, from having felt rather sympathetic to the UK cancer treatment community, they are now slightly envious of our much improved access to novel drugs as a result of the introduction of the Cancer Drugs Fund. So it has done a great deal of good in terms of patients’ access to treatment, but at the moment we don’t have the data on what the outcomes and the impact have been on quality and duration of life.

Dr Patterson: I would agree about the importance of having some good evaluation of the data that we have to see how useful it is.

Laura Weir: In my role as Chair of Patients Involved in NICE I don’t just represent the cancer charities but quite a number of conditions. I think the Cancer Drugs Fund was sold to us as something that would help speed up access to cancer medicines and make sure that people were able to access the medicines they needed. I would say that that is applicable across all condition areas. I would like to remind the Committee that the Cancer Drugs Fund costs £200 million. That wasn’t new money; it came out of the NHS. I have read-I think it was in the BMJ-that it is estimated that the diversion of resources will displace something like £480 million from other patients using the NHS. There has also been evidence, which has been published in academic journals, to say that the policy itself is based on a perceived societal value and not an actual societal value. So there is not strong evidence to support that society values cancer patients more than other patients. In fact, if you read the Department of Health impact report on the Cancer Drugs Fund, which is a very interesting policy document, it says, "It is possible that society values health benefits to patients with cancer more highly than to patients suffering from other conditions." So it is saying, "It is possible." There isn’t really the evidence there to support that.

Stephen Whitehead: Certainly the industry did not lobby or push for the introduction of the Cancer Drugs Fund, but, now it is here, we have to recognise that probably a transitionary process towards moving it on post the introduction of the new valuebased scheme is appropriate. We also need to remember, critically, that this is not UK-wide and it does cause real tension, particularly if you are living on the border of Wales and England where there are some patients who can get access to medicine and some who can’t. That does create a quite specific tension that we need to recognise.

Laura Weir: I think there are some lessons there for valuebased pricing because, whatever our definition of "value" is, it must fairly and consistently apply across all conditions.

Dr Patterson: One other point we could look at is the very rare diseases because they have often been left out of all the evaluations where perhaps there is not great research evidence. NICE has limited resources and does its evaluations on its topics, and there are rare conditions that have often been left out. Therefore, there is then variation of access to medicines across the country. One of the things we have suggested is that NICE maybe should bring together a rare diseases group to look at some of the treatments for really rare diseases.

Laura Weir: To follow on from that point, there was a separate system for appraising medicines for rare diseases. It was called AGNSS. AGNSS is going to be no more very soon and NICE will instead take on the function of appraising those medicines for rarer diseases. I know that patient groups who represent the rarer diseases want to know when NICE is going to be consulting on what that process looks like, for it to have more transparency and to be involved in that.

Stephen Whitehead: Yes.

Q36 Barbara Keeley: Obviously, Members will be lobbied by people with rare conditions, but the difficulty is that their numbers are small and they must feel that they don’t carry the weight of, say, patients in the Cancer Drugs Fund.

Can I take us back to another aspect of costeffectiveness in a general sense, because ABPI have argued that NICE has focused disproportionately on cost-effectiveness when assessing drugs therapy? Realistically, given the state of the economy, is that not something that is at the heart of NICE’s remit and absolutely bound to continue over the next period? It is a criticism that is made and I know that we have had a lot of points on this around value and the wider value judgments-I understand that-but is that not just going to be a fact of life?

Stephen Whitehead: I think the industry accepts value assessment; without doubt, we have to. Clearly, there is always a tension-and it is a healthy tension-between proving our value and them criticising the value. That is obviously something that should go on and we have no issue with that. Our concern would be balancing input and interest in that so that the weight of consideration is not always given to the health economists and there is a broader assessment of value undertaken looking at broader societal implications, taking into account the patient, the clinician and the broader stakeholder interest. The other issue also is looking at the value of a medicine not in a silo economic context but across a pathway of care. The utilisation of medicines can, in many cases, reduce the cost of treating a disease and free up resources elsewhere.

Laura Weir: If I could add to that, it is a common misconception about NICE, probably fuelled by the media, that it weights costeffectiveness more heavily than clinical-effectiveness. The truth is it has to see them both together; those calculations have to be done together, and that is why we have the QALY-the quality adjusted life year. It will weigh the cost of a medicine against the quality and length of life that a treatment will give. Where medicines only offer a marginal clinical benefit but they cost a lot, that is when patient access schemes will often come to the fore and where patient groups will lobby companies to reduce their price, because, after all, if the price is lower, there is a higher chance that patients will be able to get that medicine on the NHS.

Stephen Whitehead: The other issue to address, which Laura has covered very eloquently, I think, is to look at recognising that medicines are, by their nature, iterative and, if we don’t adopt and use them, then the next process of discovery cannot happen, because we get greater understanding by usage. It costs a lot of money to develop a medicine; about £1 billion was the figure that we use and we have justification for that, but you need to-

Q37 Chair: That is not an entirely satisfactory argument to use with medicine, "We are using this medicine on this patient because a better medicine will come down the road as a result."

Stephen Whitehead: But, Chairman, clearly you would only use it if there was evidence. If you talk about progression of diseases such as colorectal cancer, it is the combination of medicines that get you to an effective treatment if you have early enough diagnosis. You will not get a medicine through without evidence.

Q38 Chair: You have to judge it step by step, don’t you?

Stephen Whitehead: Everything is step by step and should be assessed step by step because you don’t have "big bang". That is the issue I am saying in terms of treatment.

Q39 Barbara Keeley: Sometimes there are issues that work the other way. Take the case of stockpiling, at great public expense, of Tamiflu. Clearly now it seems there was evidence about the lack of effectiveness of that that was not released. There is a feeling that sometimes decisions are made in a rush or in a panic and the effectiveness information is not available. This is not a perfect picture.

Stephen Whitehead: I am very cognisant of the issue, as you can imagine, given the furore over a book that has been published recently. Bear in mind, of course, that any medicine has gone through a quality, safety and efficacy approval process by the regulator, so it has already demonstrated its approval before cost-effective-

Q40 Barbara Keeley: Did they have all the information they needed? That is the question I am trying to ask. Sometimes it seems that we talk about this as if it was a perfect world with all the information available, but in a pandemic or suspected pandemic situation, if information is held back, or if information is not available, the right decision isn’t going to be made.

Dr Patterson: The RCP has called for all clinical trials to be registered and for all results to be published, because, at the moment, if you have negative results then in fact many trials are not published. In fact, it might be really useful to know that something is not working.

Q41 Chair: Surely there is still a professional and ethical obligation on somebody conducting research to make it available with reasonable dispatch.

Dr Patterson: Yes, there is. I agree there is an ethical imperative there, but we know that a lot of the negative research-the poor results-have not been published and therefore cannot then form part of your evidence base when you are looking for efficacy. I think there is a link there with the way that research is registered and published, and there is certainly a big drive at the moment in the academic community that research should be more widely published.

Stephen Whitehead: I would add that negative trials often give you as much information that is helpful as positive trials.

Dr Patterson: Absolutely.

Stephen Whitehead: They should be published and made available. The big issue, looking at the industry, which conducts a lot of the trials that take place-but not all-is that often there is a problem getting negative trials published, which is why we have developed repositories like clinicaltrials.gov.

Q42 Barbara Keeley: But when the Government were about to make a decision on stockpiling very large amounts of a drug, it would not have been a difficulty to get negative results published, would it, really?

Chair: There seems to me a very serious issue here, if I can link back to something Dr Patterson was saying earlier, about, first of all, the importance of disinvesting when treatments aren’t effective and the importance of the principle of transparency, and link that with the third point that I just made about the professional obligation to make available evidence about efficacy to the general clinical community, subject to peer review. When we then read that, in truth, none of those three principles have been observed in an important piece of research activity, there is an important issue of public policy there, is there not?

Stephen Whitehead: Yes.

Professor Johnson: As a funder of probably the largest number of cancer trials in the UK, Cancer Research UK puts an obligation on those whom we fund to do the research to put it in the public domain, to make it publicly available, whether published in other forms or through the cancer trials registry. Certainly the academic side of research funding is absolutely clear that it is an obligation on us as research funders to make this data publicly available in all cases, to have open access and open publication. That is absolutely the standard to which we should be aspiring, both in the academically-funded research that we do and also in the commercially-funded research, which is obviously the business of the pharmaceutical industry.

Q43 Chair: Is it an issue in which you think there is a role for NICE, which, after all, has a responsibility for clinical excellence-that is what is in its title-and, where drugs are in use and research evidence is not being published, that ought to be an issue for NICE, oughtn’t it?

Stephen Whitehead: I would say that it is the responsibility for the regulator that looks at quality, safety and efficacy. That is not NICE’s remit. They take data post that and make costeffectiveness decisions. There is no issue-

Q44 Chair: They should be satisfying themselves of the principle of transparency, which is one that Dr Patterson was emphasising.

Stephen Whitehead: That would be the issue of reverse creep in terms of authority and it would be an issue between the regulator and NICE. The EMA is looking at the transparency issue. As we know, there was an evidence session last week and obviously the MHRA is subject to the EMA. We do support the EMA process absolutely on this, but it is a regulatory issue rather than a NICE issue.

Laura Weir: I would like to get to the bottom of your question, Mr Chairman, because it seems bizarre to me that certain data is available to the MHRA, for example, but isn’t available to NICE. Maybe that is a question you would want to put to NICE in a future meeting.

Q45 Chair: Could you enlarge on that? What are the circumstances in which information is available to the MHRA and not NICE?

Laura Weir: I think Dr Ben Goldacre implies that in his book. I can’t remember off the top of my head whether there are any examples, but I think where there are issues for NICE we would like to explore them as Patients Involved in NICE. In fact, we have written to Dr Ben Goldacre to invite him to discuss some of the issues he raises and to look at where we can make a practical difference as a group. We are very concerned that we are seen as being in the pockets of industry and that that discredits patient groups when they give evidence to bodies like NICE. We want to do something about that. We have not been very vocal in the debate so far, but we certainly want to be able to look at what practical things we can do as a sector. For example, could we adopt a patient charter?

Q46 Rosie Cooper: Might I make a very brief comment? Mr Whitehead, you do your industry no favours whatsoever when you say you can’t get negative results of trials published or that there is difficulty with that, when, in the recent past, we have had so many pharmaceutical companies fined for hiding results of trials that have not been to their liking. I have said to you in other places that the pharmaceutical industry is one on which we all depend so much, but comments and behaviour like that mean that people like me don’t have a great trust in the pharmaceutical industry. You do yourself and the industry a great disservice by going down that line. You are not getting fined for nothing.

Stephen Whitehead: I am sorry, Ms Cooper, that is not what I actually said. What I said is that I do believe in greater disclosure and transparency; that is my first point. We absolutely support the EMA in their progress and the process they are making towards disclosure of data.

Negative trials are disclosed under clinicaltrials.gov. The industry goes beyond seeking just to get them published in publications. Negative trial data, as I say, is as helpful as positive data in furthering understanding of science. I apologise if I was unclear in my statement there because we do make every effort and there are international standards on disclosure. We are on a progressive journey and that journey has to involve all stakeholders. A lot of the data is confusing. For example, do people want the data we submit to the regulator or do they want the data post the regulator assessment, because it is subject to different meta-analysis? So it is a complex debate that we need to have with all stakeholders.

I would hate to do my industry a disservice because we are on a strong and progressive journey on this. We don’t have anything to hide. The regulator is very, very tough in terms of assessing, and rejecting quite often, data that comes in and re-evaluating. It is a conversation that it is very hard for the industry to have on its own. It has to have it involving the regulator, possibly discussing with people like you, about the process we go through.

What has been called into question by The Guardian journalist Ben Goldacre in his book is not just the industry but the regulator and the healthcare professionals themselves. Everybody is being called into question. I hate the way the debate has been so polarised because it has become "us and them". I represent an industry that has launched 90% of medicines in the world and has transformed and saved lives. To sit and be accused of things wrongly by Mr Goldacre I find personally deeply upsetting. As an industry, we are a slowmoving and easy target for someone like Mr Goldacre to go for. I accept some of the criticisms he makes, but we are seeking to address them as rapidly as possible.

Q47 Rosie Cooper: I am not talking about Mr Goldacre. I am talking about the general public’s view of the pharmaceutical industry. You have just talked about the data being confusing. It is not confusing to other medical professionals and I think to mess about in that area is really silly. Medical professionals will have a view, given the data. There may be arguments about it, but they will have the pure data to deal with. Frankly, when a member of the public sees their medical professional, they want a judgment about whether this will help them or not. That has to be based on the best evidence available. So I hear what you are saying, but it is obviously not what is going on everywhere, and I think that is the big danger. I am really sorry if I misheard, but I am not sure about that. The bottom line is that you won’t have difficulty getting negative information published to medical professionals who need to know whether a drug they are using their clinical judgment to prescribe works or not. They make that decision based on the information you give them. If you are not clear, then we are all in trouble.

Stephen Whitehead: I absolutely agree with you and we do seek to get all data published. We are seeking that. There are international standards we align with. There is clinicaltrials.gov. There are a lot of things that we are trying to move towards and the regulator is pushing this agenda as much as we are. The medical profession needs to have as much data as possible, good and bad. There is the Cochrane Library that does a lot of these assessments that are undertaken. The data is heavily scrutinised already, but I believe and accept that there is more to be done.

Q48 David Tredinnick: Last week we had a very interesting presentation in the House-not to this Committee-by Penny Brohn from Cancer Care, which operate in the Bristol area. They use something called "the Bristol Approach", which is described as a "unique combination of physical, emotional and spiritual support designed to help" patients "live well with the impact of cancer, at any stage of" their illness. It sees the patient as a whole and so it treats not just their physical health but their mind, feelings and relationships and the deepest parts in them. As part of their treatment, they use counselling, psychotherapy, group work and creative therapies like art and music to help express feelings. Do you think that this kind of holistic therapy should be highlighted in NICE guidelines, please?

Professor Johnson: If you look at NICE practice guidelines rather than the technology appraisals, there are elements of that which are present already. There is probably more that can be done to reflect the broad approach to health, which is critical to the delivery of proper healthcare. In general terms, these kinds of supportive treatments are rarely the subject of the clear commissioning decisions that the more technological-based treatments are. It brings us back, if you like, to the debate about the wider notion of what constitutes value in healthcare. The difficulty for NICE is to circumscribe their remit. I would be sympathetic to them if they felt that this was outside the bounds of their expertise and capability, but that is not at all to detract from its vital importance to what patients want out of their healthcare system.

Dr Patterson: When clinicians deal with patients, we would want that experience to be positive, not just in clinical effectiveness but in their experience and, as you say, that feeling of people being treated as a whole person and their needs being treated in the round. That often involves using multidisciplinary teams, using a wider approach, a service approach. Some of the NICE guidance at the moment absolutely advocates that. Particularly the guidelines-not the technology appraisals-will talk about the need for a multi-professional assessment, engagement with the patient, promoting better selfmanagement and better support for patients. The interesting thing is how you evaluate that in terms of outcomes and the methodology for evaluating multidisciplinary assessment. It can be done, but it is complex and there isn’t a lot of it about in the literature.

One of the problems is to say that there is a sense in which, on a humanity level, of course this is a better way to treat people-of course-but we don’t always have an academic evaluation of that. Whether that should matter within good patient care I think is a moot point because good clinical practice would take forward exactly what you have said, which is bringing together different professions and different support for patients.

Stephen Whitehead: My sister is a palliative care nurse in the Yorkshire Dales and she has been with the NHS for 30 years on and off, also working for Marie Curie. She is a big believer in other treatments that go alongside traditional treatments, including massage, talk and counselling. I do believe that some of this should be included in guidelines. I have no question on it because patient wellbeing, particularly end-of-life situations, is not always about medicine. It is often about other things that make the people feel whole and better, cared for and loved. In the context of certainly what we see of some of the scare stories in old people’s homes and hospitals, some of that approach needs to be revisited. If it requires stronger guidelines in that, then they should be welcomed.

Q49 Chris Skidmore: Isn’t the problem with valuebased pricing that you are going to open a Pandora’s box? Essentially, there will always be a continuing debate around the definition of "value" and the goalposts will always continually move. Once you create a valuebased pricing structure, there will always be cause for that value to be reassessed and changed. How are you going to cope with that pressure? We live in a world where there are increasing expectations on the NHS, and the Cancer Drugs Fund has raised that bar somewhat. So when the Cancer Drugs Fund disappears you are going to be left also with a medicine cliff edge, as it were, because people are not going to be able to get all the drugs they used to be getting. At the same time, how are you going to cope with this constant call for redefining value? Will you stick to your guns or will you constantly look to reassess it? You have calls from David here about homeopathy and all this sort of stuff, but it is entirely subjective, it seems.

Stephen Whitehead: There does need to be a decommissioning of many things when assessing the value of something new, because otherwise money will continue to be spent on things that aren’t as relevant. From the pharmaceutical industry perspective, of course, it is only pharmaceuticals that go through real health technology assessments. 8% of the NHS budget goes on medicines, which is 0.9 % of GDP, yet it is the most rigorously assessed part of the budget. Other parts, which David raised, are not looked at in that context, and value in the rest of the system is not proven in that way. It is often difficult as an industry-and I clearly have to fight a lot of battles on behalf of the industry-and it is ours that is most rigorously assessed and looked at. I do think, going to your point about valuebased pricing, that it needs to be a continual and iterative reassessment as you go through a medicine’s life because it will bring more value.

Chair: I am conscious that Health Questions start on the floor of the House in exactly half an hour and we have a number of questions we still want to get through, one of which comes from Mr George.

Q50 Andrew George: Moving from price to quality, as you know, the Department of Health sees that the primary purpose of NICE’s quality standards is to provide a clear description of what high-quality health and social care services look like. To that extent, how do you see NICE going forward, managing the tension between what inevitably is going to be looking at individual remedies and, as Mr Whitehead described, an iterative process, even with each of those individual remedies, and the need for the integration of those services? Do you think that NICE, in its role, is going to assist in the process of seeing each of those remedies in the context of an integrated solution to patient care?

Dr Patterson: I think it is starting to. The trouble with the quality standards is that NICE is doing standards based upon its own guidelines that it has, which of course have been rigorously tested to the best evidence. There is other guidance around. There is professional guidance, and specialist societies put forward guidelines for good practice and so on. One of the debates there has been with NICE is as to how much they should take into account that broader area of guidance and not just their own guidelines when they are putting out the quality standards. There has been quite a lot of debate about that and putting that together.

The quality standards and the guidelines need to be set in a context of us trying to look at more integrated care between secondary care, primary care and social care now. It would be good to see social care and healthcare being evaluated together rather than social care going to be evaluated somewhere separate, because that is not how people experience it. They experience their life and their services across the boundary of health and social care. It is a real challenge for NICE to do that well, but it is the holy grail that we are all working towards at the moment, which is perhaps a more integrated seamless care. Patients have told us they want care to be joined up; they want it to make sense. Anything that can help us to do that would be good.

Q51 Andrew George: You are almost implying that in fact NICE don’t really have a significant role in this. Clinical guidance, commissioning groups and others seem to have a more significant role when it comes to the integration of the whole pathway of care.

Dr Patterson: Except they could, I think. The quality standards ought to be looking at a whole pathway. At the moment, they tend to be looking at parts of it because they have to be quite focused. Of course the rehabilitation pathway would be huge. There are real methodological challenges in that, but I think that is the direction in which NICE is moving and will have to move, particularly if it is taking on social care. There is no point at all in evaluating social care interventions without having regard to health interventions alongside them.

Q52 Andrew George: When it comes to medical quality and guidance, you, as a Royal College, set clinical guidance.

Dr Patterson: We do.

Andrew George: To what extent are you content that NICE might then be, to a certain extent, duplicating the work that you are doing? Would it not be better either to feed your proposals to NICE and they take the lead, or for them to feed their proposals to you and then you take their advice into account?

Dr Patterson: I think you are absolutely right. Certainly NICE can use some of the work that is already being done in the professional bodies as another source of evidence. It may not meet the gold standard of randomised controlled trials because that evidence is not always available, and guidance that the colleges have given has perhaps been on conditions that NICE have not picked up. They have been either rarer or a smaller focus and so on and have brought together as much evidence as they can, but that needs to feed into the NICE process.

Q53 Chair: Can I pause on that? Is it right that the relationship between NICE and the Royal Colleges-"Who leads the process?"-is something that needs to be thought through more clearly? Does it not concern you, as a representative of the Royal College and the professional leadership that represents, that that relationship as to who owns responsibility for defining what "good" looks like, to put it crudely, is not as clear as it needs to be?

Dr Patterson: Yes. I think we both have a role because the processes and the scope are different. NICE has a huge resource. As I say, the National Clinical Guidelines Centre produces 60% of the guidance going into the NHS and is the biggest centre in the world.

Q54 Chair: But you have every physician in the country registered in your membership. That is not a bad resource either.

Dr Patterson: It is not and in fact we do that. For instance, one of the things that NICE has just been commissioned to look at is sevenday consultant working. Our own college has already done some work on that. There is some evidence about that. The Academy of Medical Royal Colleges is just about to publish some standards on that. It would be crazy for NICE not to build on those things. We have had a positive relationship with NICE and that needs to be built on and taken forward. We would welcome more clarity, but it hasn’t been a major problem of late because NICE has begun to change its methodological stance, which has incorporated other sources of evidence.

Q55 Chair: Can I bring in Professor Johnson as well?

Professor Johnson: Yes. This is not an area where Cancer Research UK has particularly made a submission to the evidence as to the expanded remit of NICE. The organisation of effective cancer services is extremely multi-disciplinary: it crosses providers and crosses many of the boundaries that are present in the Health Service. Having our overarching practice guidelines standards is extremely helpful for holding the ring in what looks to become an increasingly fragmented service. That is an area where NICE potentially can play a very valuable role and we would like to see that strengthened. I am not sure we have a particular view on the relationship between NICE and the Royal Colleges, but, as you say, it makes sense to have as much alignment as is possible between the broad body of expert opinion and the guidelines that NICE is producing.

Laura Weir: I have a very quick point. NICE is committed to developing 180 quality standards. It was originally to be by 2015 but it has now slipped to 2019. I would argue that this is a very pertinent debate because NICE is at risk of being sidestepped. The health reforms rely on commissioning guidance that balances quality and efficiency, and, if NICE can’t deliver that in a timely way, then the NHS Commissioning Board will look to the other professional bodies to produce that guidance.

I would also like to add that quality standards are only one part of the story. If you are a patient or a carer, you rely very heavily on the clinical guidelines, which, if they are kept up to date, reflect the evidence base and treatments that people are entitled to and should be almost like a bible, if you like, for patients. Those are very important. It is important to recognise that because the quality standards are aimed at commissioners, whereas the guidelines are for patients and carers.

Q56 Grahame M. Morris: Can I follow up on that because it leads in nicely to what I wanted to ask? Could I ask Dr Patterson a question in relation to the clinical guidelines? Professor Johnson was saying how robust they are, although he expressed some concerns about methodologies being too tightly defined on occasions. Why, if the clinical guidelines are recognised as being excellent, are they not universally applied?

Dr Patterson: It brings us back full circle to where we started this morning, which is talking about the challenge of implementation and how to ensure good practice. It is an ongoing challenge. It is about education and training, about a medical profession that is open to learning new things, to learning how to do things in a different way, and also the fact that professional leadership can help this by articulating that this is important and that these are helpful to clinicians in practice. The thing about the guidelines is that they are aimed at absolutely best practice in all possible areas and may in fact be aspirational in some areas rather than being able to be completely delivered in a standard way. Of course, local circumstances, in the way a service is set up, may mean that some things may not be able to be delivered in exactly that way. But we, as a professional body, have said that clinical guidelines are helpful and that it behoves a good professional to be building on that.

Professionals need help to implement them. There are an awful lot of guidelines out there. How do you keep on top of everything and keep up to date and so on? It goes across the whole professional development of doctors, and there are other professionals of course, but I am speaking for doctors.

A powerful lever can be patients themselves. If patients are aware of what guidelines say, that can be a dialogue between patients and doctors and is often very helpful for doctors, who may be holding a whole raft of things and the patient will just have one particular thing they are interested in. That can be very helpful.

As I said, there are some tools. NICE does produce some tools to help with implementation and I think the implementation arm could be strengthened. The professional bodies can help implementation of good practice through measurement, audit, quality improvement, teaching, training and so on. So it is a whole raft of things.

Q57 Grahame M. Morris: Is it part of your members’ regular appraisal whether they follow these guidelines?

Dr Patterson: It in fact is, yes. They should be able to demonstrate evidence that at least they know about the relevant NICE guidance and they should be able to give some evidence of how they are going about implementing it.

Q58 Chair: It is something to do with revalidation.

Dr Patterson: Indeed, and this may very well be an iterative process, which in fact is strengthened over time.

Q59 Barbara Keeley: You touched on social care. We are very interested in that issue and we have an inquiry ongoing on that and particularly in integration. I have a couple of points. Do you think, with its widened remit, that NICE will have the capacity and expertise to produce guidance on social care, and is joint guidance an effective way to encourage integration in your view? I know it is a big question.

Dr Patterson: Yes. I think it is a challenge for NICE to be taking on this huge next piece of work. It needs to be properly resourced. The danger of any organisation that gets too big with lots and lots of different functions is that you get silo work. There is difficulty with knowledge management and so on, so I think they are going to have to take care and pay attention to that. We would support the work, but, as an organisation, they are going to have to tackle that properly. I am sorry, your last point was social care and whether guidance-

Q60 Barbara Keeley: We are interested in whatever contributes to integration, but is joint guidance the way to do it?

Dr Patterson: It will be one of the things that help. I don’t think in and of itself it is going to be the magic bullet that brings together health and social care, but if anything can promote that active thinking that would be helpful. The real levers are going to be around commissioning, bringing together social care and healthcare on the ground, and moving towards that. I don’t think it will hinder; I think it will help.

Laura Weir: It does have to be integrated, to answer your question. The dementia guideline and the quality standard that have been developed are good examples of that. It is a real travesty that the clinical guideline for MS that will be published in 2014 won’t incorporate social care. That is a longterm fluctuating condition where, if your social care needs are not met, you can’t live independently; you cannot live a normal life.

It is going to be a real challenge for NICE to take on the function of social care. It is going to have to think very differently and be more flexible with the evidence base. It is going to have to approach it very differently. As patient groups, we would not term it "social care". We would talk about co-ordinated care or personcentred co-ordinated care. You might say that is semantics, but actually it points towards what NICE should do in relation to evidence and guidance. In a way, it needs to let go of the clinical emphasis and rebalance that with evidence regarding the personal outcomes and benefits that service users and carers want. It might want to look at measures associated with wellbeing or the responsiveness of services, for example, and I think the "Making it Real" campaign is a good example of how it could include service user statements to help frame some of the guidance.

Stephen Whitehead: I would agree with all the panel. There does need to be a greater integration. We call it "pathway of care" because it is not all about a single medical intervention; it is about how the patient is looked at throughout the whole treatment process, including in their home. If you look at the NHS reforms that have gone through and the changes in England, what we see is the possibility of the greater emergence of diversity of treatment and standards across England. NICE should be one of those bodies that is able to guide and lead, and there should be enforcement and obligation with CCGs to follow NICE guidance on social care standards.

Q61 David Tredinnick: On Mr Whitehead’s point there, my understanding is that the Department of Health trials on personal budgets have shown that patients are benefiting from being able to use a wider range of treatments and also carers in many instances are now able to go out to work when they have not been able to do so before because the funds are being applied differently. The second point is that patients are hugely responsible when they take on their budgets and the increasing choice is benefiting the Health Service in the sense that it is reducing costs but also, of course, giving patients better treatments. I don’t know whether you want to comment on that but that is the information we have.

Stephen Whitehead: Without doubt patients prefer to be treated at home. Healthcare at home is the direction of travel, greater healthcare in the community and greater focus on primary care clearly in delivery, because it is the most costeffective way and also what patients want. They don’t want to go into hospital. They don’t want to be treated as sick. They want to be in their home and live their lives as best as possible. Often patients will choose different things in that context, but the healthcare system should provide an integrated care pathway for that patient. Patients may choose to add things on themselves. From a NICE perspective, they will look at clinical effectiveness and clinical evidence to make decisions about what can be provided by the state and what cannot; that, I think, is the issue.

Q62 Chair: There are two big challenges for NICE in this, aren’t there? One is to think of the system, as Mike Farrar puts it, as "a care system with a medical adjunct rather than a medical service with a care adjunct". That requires a change of mindset on the part of NICE. The second challenge flows from the first, which is to rethink the relationship between the care service provider and the patient or service user so that it is enabling people to maximise the benefit from their own lives rather than simply providing a service to them. Both of those thought processes require a considerable learning curve within NICE. Is that a fair way of putting it? Perhaps we can start with Dr Patterson.

Dr Patterson: It is a challenge for the whole of the healthcare system, so NICE, being part of that, absolutely.

Q63 Chair: Given that the bit we are focusing on this morning is NICE, it is just about thinking through how NICE develops its definition of clinical guidelines, quality standards, technical innovations and so forth, into a world that is less medically dominated and accords a larger role to the individual patient.

Dr Patterson: Yes. I certainly agree about the larger role to the individual patient. We know from what Wanless said 10 years ago that we need to be on this trajectory for patients to be better selfmanaging their conditions. To do that, patients need information, and they need good information. I am absolutely in favour of patients accessing the NICE appraisals in a way that they can use them, they should know the guidelines and they are absolutely part of it. Their role, not just in formulating them but in using them to improve care, is really positive, undoubtedly.

Stephen Whitehead: I absolutely agree with Dr Patterson on this one. There is also an urgency to this because, obviously, NICE are assuming the remit. There is secondary legislation going down under the Health and Social Care Bill on the changes that NICE has to adopt and adjust to. What I would say within that legislation that must be reflected, if NICE is to do this properly, is greater consultation with stakeholders of relevance to make sure that these things are developed appropriately under the right governance and standards so that we are very clear about what they are doing and what they are delivering in the joinedup healthcare that Mike Farrar so well explains.

Laura Weir: We have evidencebased medicine but we don’t necessarily have the same for social care. That is going to be the real challenge for NICE. In terms of the personal budget question that you raised earlier, it raises quite a few questions. As a patient you might want to be able to choose anything that you think might help you. In the context of NICE, NICE will want to recommend something where there is evidence that something works or that it delivers value for money. Therefore, there is going to be a real tension with personal health budgets.

Q64 Chair: I understand that, but there also needs to be a greater respect, doesn’t there, within NICE for patientreported versions of their own outcomes?

Laura Weir: Absolutely.

Stephen Whitehead: Yes.

Chair: That is not the same argument necessarily. In some cases it will be, as with alternative medicines.

Q65 David Tredinnick: Hinckley, which I represent, in Leicestershire in the middle of England, has been a forerunner or a big supplier in producing aromatherapy oils and, through Penny Price and people like that, running courses on aromatherapy for nurses from the Leicester Royal Infirmary, in using oils to help relieve stress, pre-operative and post-operative, and for dealing with palliative care, helping with patients towards the end of their life, using simple remedies, such as lavender; I am not going to go into the whole range of them. They are also used at the big George Eliot Hospital A and E in Warwickshire, where they are helping doctors to do more operations because the preparation has become easier by having aromatherapists on duty there. This is recognised and you can look it up on their website. We are probably talking the same language, but, with personal budgets and more patient choice, we are going to have to look more sympathetically at the range of treatments that people want and let them make decisions.

Laura Weir: You mentioned patient choice and I think that is at the crux of this. NICE will give recommendations based on the evidence, whereas what I understand has happened in the personal health budget pilots is that patients have had no information and it has been for them to go out, find that and make their own decisions. I am not sure whether that is patient choice.

Stephen Whitehead: You need information.

Laura Weir: Yes; information is absolutely key.

Q66 Chair: We are running out of time. Are there any points that anyone on the panel wishes to draw out that you think might have got twisted or you wish to put a concluding thought to-a catchall question?

Laura Weir: I will start, if I may. NICE is doing a really good job, but I am concerned that it is responding to where there is an evidence base for things, so it will develop guidance where there is an evidence base. I am concerned about those areas where there might be wide variations in outcomes for patients and service provision, but there might be fewer patients who are affected, or the patient group might be smaller and therefore not be as adequately resourced, and that evidence base is not there. Therefore, they are not prioritised.

I am also concerned that NICE perhaps isn’t adequately resourced to take on this new remit. Its remit seems to be forever expanding, but I am not sure that its resources have. There is a huge expectation out there. But we do want to work with NICE as patient groups because we want the best evidence for the people that we represent. We have not had a good relationship with NICE in the past, but we do want to be trusted. We appreciate that NICE has a difficult job to do, but it does it reasonably well and we want to support it.

Stephen Whitehead: I think Sir Mike Rawlins has led NICE through a very successful period. The challenges facing us are very significant and quite difficult. I suspect that NICE’s extended remit can’t be met within its £70 million, realistically, if it is to meet time frames and delivery for the healthcare professional. Going forward, it needs greater stakeholder engagement-patient, clinician and industry-and I think also there needs to be some partnership collaborative agreement with all parties to get the proper governance in place as NICE assumes its very additional and more independent role. What we don’t want to do here is to have a democratic deficit. There has to be full stakeholder involvement as it moves forward under its new structure.

Dr Patterson: The college has been supportive of the work NICE has done. Clinicians, on the whole, have found it helpful. We would support that going forward. There needs to be a recognition-and I think there is from NICE-that the college holds a huge repository of clinical expertise, which in fact NICE uses. Many of their experts are drawn from our ranks. That needs to be built on. The use of the clinical community, both in terms of academic research and also practitioners, is really important.

The final thing is that we will have to see how the clarity plays out between the quality standards and the commissioning outcome framework and so on, because we don’t really have a clear view about how that is going to work.

Professor Johnson: I echo the fact and we recognise that NICE is highly expert and recognised internationally as a standard for the evaluation of efficacy in the Health Service. What we need to see in the future going forward is more bottomup input from the patient constituency so that we are not codifying paternalism in the health service and NICE is telling people what is effective and what is available and not what they want.

Chair: That is a very good way of putting it. Thank you very much. We can get out before the carpenters start again. Thank you very much indeed.