National Institute for Health and Clinical Excellence - Health Committee Contents


Examination of Witnesses (Questions 602 - 619)

THURSDAY 18 OCTOBER 2007

MR STEVE WINYARD AND DR RAFIQ HASAN

  Q602  Chairman: Welcome and thank you for coming here to take part in the fifth evidence session in our inquiry into NICE. Perhaps for the record you would give us your names and the positions that you hold.

  Dr Hasan: My name is Rafiq Hasan and I am Director of Market Access at Novartis Pharmaceuticals in the UK.

  Mr Winyard: I am Steve Winyard, Head of Policy and Campaigns at RNIB.

  Q603  Chairman: We said to a previous witness this morning that we tend to meet you on occasions in Parliament. Whilst there is no direct financial interest we meet you via back-bench committees and other ways to talk about the issues that we intend to discuss today. I am not sure whether that is a declaration of interest on my part, but I think I should say that. Industry complains that in the UK the take-up of new drugs is slow and variable and policymakers complain that clinical guidelines are often ignored by practitioners. How can that problem be mitigated?

  Dr Hasan: As you have already heard this morning, there is quite a lot of variation across the country in terms of PCT uptake of new technologies. We heard this morning about exceptional case committees that often put in place what we see as hurdles and barriers in the way of patients getting access to new medicines. If one takes the case of Lucentis, a drug that is currently being appraised for wet AMD, the clinical evidence suggests that over 70% of patients experience an improvement in vision. Before that drug was available these patients had virtually no other effective treatments that could have such a significant impact. What we see is the whole concept of NICE blight before NICE has made a decision. Once NICE comes out with some final guidance we see slowness in uptake and implementation.

  Q604  Chairman: We may want to cover that a little later. Mr Winyard, do you have any general comment?

  Mr Winyard: RNIB's involvement in the NICE process is fairly limited. We have engaged in only two appraisals and my comments will be based largely upon that experience. Involvement in the work of NICE, however, is very important. One of our royal charter objectives is the prevention of blindness. We know that sight is precious to people. If you ask your constituents nine out of 10 will say that sight is the sense that they most fear losing. For a very long time there were no treatments for wet AMD and that accounts for about half of all new blindness registrations in the UK, so it is the big cause of blindness. For RNIB, when new approved safe treatments have become available in the past two years our trustees have tasked us with making sure that they become available on the National Health Service. That is why we are involved. The other general point to make is that our primary relationship in all of this is with the Royal College of Ophthalmologists and in a sense it would be great to have the college here because it has the expertise on these issues. The president of that college is on our policy and advocacy committee which lays down what our policy will be on different treatments. In terms of access, obviously there is an enormous problem between the time a drug gets EMEA—European—approval and getting NICE guidance. At the moment we are right in the middle of that. Amongst the members of the Committee, we note that in some areas—in yours, Chairman—patients will be treated but in others they will be turned away. For example, in Staffordshire there appears to be a blanket ban on treatment, and in many areas patients have to go blind in one eye before the Primary Care Trust will consider treating the second. This is fundamentally wrong and we have to find a way round it. In the short term, together with the Macular Disease Society we have established an advocacy service funded by Novartis and Pfizer which supports patients who contact us via our help lines and say they are going blind; they have paid into the NHS all their lives and the first time they draw on it they are turned away. They are told that they cannot have funding. In those cases we support patients and engage with the Primary Care Trust; and we also engage with MPs. We are trying to apply sufficient pressure to ensure that patients get treated. It is not an ideal situation but it is one that we face. It all comes back to the prevention of blindness which is our royal charter objective.

  Q605  Chairman: If I may ask specifically about patchiness in terms of the implementation of guidance, what do your members think about it? Yours is a membership-based organisation.

  Mr Winyard: We are indeed. There is enormous anger and frustration that there is such wide variation. If you are fortunate to live just over the border in Cheshire and Merseyside both eyes will be treated, whereas if you are in Staffordshire you will be turned away. There is anger, frustration and a sense of real unfairness. That has been reflected in the very high level of media interest when local and national cases emerge.

  Q606  Dr Naysmith: Dr Hasan, you suggest in your written evidence that the quality of work carried out by some of those who produce assessment reports and others who may review the evidence is both questionable and variable. That is quite a serious accusation. Can you give us some examples?

  Dr Hasan: I think it comes back to the whole discipline of health economics which is embryonic relative to clinical medicine, randomised controlled trials and evidence-based medicine. When we assess health economic outputs we must understand that there is a high degree of uncertainty. The point we made in our written submission was that when NICE commissioned independent assessment groups to run some of that evidence generation there was a lot of variability in the quality and standards between different evidence groups. Often there are errors in those models and the reports generated. Subsequently, addenda must be issued. Sometimes one questions the assumptions that go into some of the models. We would welcome a broader debate about some of the assumptions that go into those models and the emphasis placed on the health economics and the concept of cost per QALY in the appraisal committee decision-making process.

  Q607  Dr Naysmith: One of the things NICE may say in response is that often there are lots of mistakes, even arithmetical errors, in the submissions made to it by drug firms. Is it not a fact that in this area some mistakes are made? You still have not given me any examples of what you mean.

  Dr Hasan: I can give you the example of the Alzheimer's disease appraisal. One assumption was that 30% of the costs of institutionalisation was not borne by the NHS. That may be based on some evidence that perhaps is not as current as we would expect. We know that some of the costs put into the economic models were perhaps a little out of date. Where one has the opportunity to have those discussions one can identify some differences between what maybe we would put into a model versus what one of the assessment groups would do.

  Q608  Dr Naysmith: You argue that the conflicts between yourselves and NICE might be reduced if there was a clear rationale for how NICE reached its decisions if it was provided regularly. How much explanation do you believe is given at the moment either in private or public, and how would you like to see it made more explicit?

  Dr Hasan: We would like to see some form of debate around how important the health economics are in the decision-making. We see an over-reliance on cost per QALY. I know that Professor Stirling Bryan has presented himself before the Committee. Only last year he published some research he had done into the NICE decision-making process. I almost reiterate some of the evidence you heard earlier today. There was a feeling among clinical experts and patient representatives on the NICE committee that they were sometimes out of their depth when there was a discussion about health economics. There was one quote in one of the papers about some of the clinical experts. You can see the way they are thinking. They start with the cost per QALY and then work backwards. The question we ask is: is that the right way to make a full and broad assessment of the clinical benefits of a particular technology that is delivered as well as the patient experience? Another example is Professor Anthony Goldstone who presented himself at one of the NICE appeals. Subsequently he wrote a letter to The Times in which he said he felt put down, that his opinions were not heard and that 30 years' experience treating cancer patients was largely ignored. He believed that decisions were being made by people who had never seen a case in their lives. We believe that there is much scope for improvement in terms of a broader debate on the real decisions that are being driven within that process. How much weight is being given to health economics? Surely, there must be some weight attached to that, but what weight is being given to clinical and patient experience?

  Q609  Dr Taylor: Therefore, you agree with the previous witnesses that the right experts are not necessarily used by NICE?

  Dr Hasan: Absolutely. One other point that is highlighted is: have they had the right training in health economics to be able actively to debate it?

  Q610  Dr Taylor: Last week some of our expert witnesses criticised the quality of data that some pharmaceutical companies passed on to NICE: it could be incomplete, partisan or of poor quality. What is your response to that? Is that fair, or do you believe that the information you pass on is always absolutely superb?

  Dr Hasan: I can speak only on behalf of Novartis. We believe that our submissions are of very high quality and we have a track record to demonstrate that.

  Q611  Dr Taylor: Would it make a difference to pharmaceutical companies, not necessarily your company, if the eventual price of drugs reflected the quality of the evidence given?

  Dr Hasan: We are certainly strong believers that the price should be reflected in the value that a particular medicine delivers. We believe that through these types of appraisal processes we are able to demonstrate the value of our medicines.

  Q612  Dr Taylor: NICE seems to be more influenced by quantity rather than quality of life. Mr Winyard, do you think NICE has underestimated quality issues particularly for people who are at risk of losing their sight?

  Mr Winyard: Without a doubt. I am not an expert on QALYs and health economics, but the system does have a bias towards quantity of life rather than quality issues. In the two appraisals in which we have been involved what has been frustrating is that they have been some of the longest in the history of NICE and each time NICE has first come out with its consultation document it has got it disastrously wrong. In the first case, in relation to PDT we would have gone to appeal but NICE withdrew the ACD because there was such an enormous outcry over a policy that said a person had to go blind in one eye before he or she could be considered for treatment. We went into the process this time for the anti-VEGF treatments full of hope. What happens? It does exactly the same thing. Of course, there is an enormous outcry. No one supports this; it is wrong and against good clinical practice. You will not find a single ophthalmologist who says that you should first let the patient lose sight in one eye. If you let that happen you do not know whether or not you can save sight in the second eye. NICE has got it disastrously wrong. The only thing the appraisal committee seems to be interested in is cost per QALY. It says in the literature that it is interested in the impact of the condition on people's lives; it wants to know what it is like to be blind. What would be the impact on the quality of life if one could keep one's sight? We present that evidence and there is absolutely no indication that it has been taken into account in any way. It is frustrating. Inevitably, that leads us to reflect the anger and frustration of our members and the wider society and then it is a very high profile public debate which is not ideal. I can see problems with that.

  Q613  Dr Taylor: Does industry provide evidence on quality of life issues, or is that outside your remit?

  Dr Hasan: We submit an economic case for all our medicines that captures the quality of life benefits, as well as the quantitative benefits, of those technologies, so we submit a cost per QALY model based on the evidence generated in our randomised controlled trials.

  Q614  Dr Taylor: Is there hard evidence that elderly people, for example, value quality rather than length of survival?

  Dr Hasan: As part of the process of generating that evidence often we undertake the right type of research to elicit what is called utilities, that is, a quality of life measure. NICE's own reference case suggests that that is done with members of the public. Therefore, members of the public are asked to quantify how much they value sight versus other things. That will capture those quality of life benefits. One point I should like to pick up from the RNIB is that if, for example, NICE's final decision is that an individual should be treated only for the better seeing eye, has it captured the impact on that patient having to be told by his or her doctor that treatment cannot be offered and the patient must return when the other eye is affected by the disease? What about the detrimental impact that has on the quality of life of that patient? These are the types of debates that we believe should take place at the appraisal committee so that there is a broadly informed understanding of how a decision is reached.

  Q615  Dr Taylor: It is fearfully difficult, is it not? Do you believe that NICE takes any account of non-health costs?

  Dr Hasan: Part of its remit is to focus very much on NHS costs only. Wet AMD and blindness is a very good case in point in that there are broader societal costs that should be taken into account. The challenge that we always get back from Sir Michael Rawlins is that he needs almost statutory permission to be able to go down that road and capture the broader costs. We know that in Sweden, for example, the LFN, which is almost equivalent to NICE, does capture broader societal costs. It does an economic evaluation taking into account benefits in terms of getting people back to work and off benefits. We believe that that is an area where that debate should be included.

  Mr Winyard: There are two recent cross-national studies of the impact of sight loss and AMD on quality of life: one by Professor Giselle Soubrane and one by Professor Frank Holtz. Both demonstrate the major impact of AMD and sight loss on quality of life. As to the whole professionalisation of the assessment process, there does not appear to be an acceptance that QALYs are not scientific. There is a claim that they are scientific, but clearly they embody value judgments to a very great extent. Whom do you ask? You want to measure the importance of sight to quality of life. Does one ask the sighted public, or people who are in the process of losing their sight, or do you ask those who lost their sight five years ago, for example? You will get different answers from those different groups. Which questions does one ask? Do you use a generic questionnaire, the EQ-5D which is very limited, or something much more specific to AMD like the MacDQoL that has been developed by Professor Clare Bradley at London University which captures much better the full impact of AMD? If one goes in the direction of specific measures it becomes much harder to compare different disease areas. Basically, there needs to be a recognition that QALYs are not scientific and lead the whole assessment process in the direction of great complexity, and it is excluding. This week we have sat down with the Royal College of Ophthalmologists to look at the additional data provided by NICE following the very negative response to the appraisal consultation document. Clinical experts—the leading retinal specialists—and the patient group cannot understand it. We cannot make sense of the data that has been provided because it is in a very special language. One probably needs a master's degree in health economics before one can begin to make sense of it. It cannot be right that it is such an excluding process.

  Q616  Dr Taylor: Do you have any suggestions for an alternative?

  Mr Winyard: In a sense, it is there already. In the Secretary of State's Directions to NICE it is pretty clear that wider things should be taken into account: the broad balance of benefit and cost and the degree of clinical need of patients. If the appraisal committee can take rather better account of those things QALYs will be part of that process, but there is a wider picture and in the end you have to make some tough decisions. Is sight important? Everything tells me that it is. Your constituents will tell you that it is terribly important, and that must be captured.

  Q617  Dr Taylor: How do you balance it against survival of an extra few months with a cancer drug?

  Mr Winyard: I am not an expert on running health systems. I agree that that is a problem. Obviously, in one part of America the public has been asked about pretty well every sort of treatment and ranked them. Fortunately, that is not my problem. You need a system that recognises that there is no science that will deliver precise answers to these questions. One needs to take account of QALYs but in the end one must recognise what is important and what is less important.

  Dr Hasan: I think there needs to be recognition that the QALY is an estimate. With most things there are wide confidence intervals around that estimate, but added to that it is almost a prediction. We are doing economic modelling sometimes with time horizons of 30 or 40 years. We are saying that what we know today will be replicated in 40 years' time. We also know that there are slight changes in assumptions which may be based on differences in clinical opinion. Therefore, one group of experts will say that a particular assumption is correct but another may have a completely opposite opinion. That can change the cost per QALY significantly. It could take it above the perceived threshold or even down to a negative value, ie that product dominates other products. There should be recognition of the strengths and weaknesses of QALYs. We suggest that part of the appraisal committee process is to say that in this disease area when assessing the health economic benefit, quality of life and cost per QALY these are the strengths of the assessment and these are the weaknesses. Let us have that debate and see whether we can come to some agreement.

  Q618  Mr Scott: Do you believe that lower drug prices can be the answer to this problem?

  Dr Hasan: I think we need more clarity as to the cost per QALY versus the price of the drug. You can have a very cheap drug that may not be very effective but has a very high cost per QALY because it is driven by the effectiveness of the product, whereas you may have what you perceive to be a high priced product that delivers significant benefit. An example, which I am happy to discuss, is that the SMC has already appraised Lucentis and in one scenario within the economic modelling the cost per QALY of that drug versus Visudyne being used with photodynamic therapy is about £5,000 per QALY. Therefore, at face value one may say it is a high priced product but when the economic modelling is done one sees that it delivers a significant amount of value and is seen as very cost-effective. On that basis it was approved by the SMC in June.

  Q619  Mr Scott: Would not another option be a high quality product but a slightly lower profit margin?

  Dr Hasan: One can debate profit margins versus price but that is probably a slightly different discussion. If one asks how to quantify that value one can use things like cost per QALY to show that a lot of products represent good value for money for the NHS.


 
previous page contents next page

House of Commons home page Parliament home page House of Lords home page search page enquiries index

© Parliamentary copyright 2009
Prepared 28 October 2009