Select Committee on Health Minutes of Evidence

Examination of Witnesses (Questions 101 - 119)




  101. Can I welcome our last group of witnesses and ask you to briefly introduce yourselves. In doing that could you if possible say how many NICE inquiries you have been involved in and submitted evidence to as it is not clear from the submissions that we have?
  (Ms Miles) I am Becky Miles. I am Director of the National Cancer Alliance. You have had information about us. We are an alliance of ordinary people with cancer, an individuals' alliance if you like, and the health care professionals. We formed in 1994 as a membership charity campaigning for improvements in services. Our involvement in NICE has been limited in some ways, particularly in relation to the receipt of large quantities of paper which I have not been able to find the time to contribute any cogent thoughts on. Increased involvement in NICE in a peripheral sense has been through the National Guidance Development Programme which is now under the auspices of NICE chaired by Professor Bob Howard. The NCA has been commissioned to find a way of getting proper patient input (ordinary folks with cancer and occasionally their carers) into the beginning phase of the development of the guidelines. That is different from the HTO assessment process. We have produced some thoughts on those patient experiences, patient views, patient recommendations and so on. We have been commissioned and paid for that process.

  (Mr Cayton) I am Harry Cayton. I am Chief Executive of the Alzheimer's Society. We have been involved solely but extensively in the appraisal of anti-colonosterase inhibitors for Alzheimer's disease.
  (Mr McDonald) Glynn McDonald from the MS Society. We have been involved in a single appraisal of disease-modifying drugs for MS for some two and a half years which is longer than most appraisals last. We are also involved in developing guidelines for MS services which come under the auspieces of NICE as well.
  (Ms Rule) Joanne Rule from CancerBACUP. We have been involved in 11 appraisals and two appeals, so extensive involvement throughout the process.

  102. Can I begin by asking a number of questions mainly to Mr Cayton and Mr McDonald? I am not sure in whose evidence I read it, but it was one of the two of you, where you talk about the difficulties in evaluating the impact specifically of treatments on long term conditions. Obviously that is of concern to both your organisations and others as well. Can you tell me a little more about that, whether you have any thoughts on how that might be overcome within the kind of framework that we have with the NICE regime?
  (Mr Cayton) It may well have come from both of us. The particular point that we made is that some of the new health technologies that NICE is having to appraise are absolutely on the borderlines of what health technologies can do, and particularly around the neurological diseases—MS and Alzheimer's being two of them—which are now seen by the World Health Organisation as a major cause of morbidity in ageing populations across Europe. The technologies that are being developed are particularly difficult to appraise and particularly uncertain in their effect. I think that creates real problems because NICE is having to do a job on the boundaries of where medical science and research can currently take us.
  (Mr McDonald) I would agree with what Harry says in terms of improving our understanding of how our input is used and how the process rolls forward. We have made comments in our recommendations. I will give a couple of examples. I will start by saying that there have been some improvements as NICE's processes have developed but there is still room for further improvement. If this was a meeting of NICE's Appraisal Committee Harry or I could come along and make a presentation, we would be asked questions by the Committee members. We would then be required to leave the room while discussion took place and that is then completely opaque to us because what comes out of that is some minutes which are published but which do not contain much about the discussion that has taken place. They tend to focus on the issues were covered and the outuput.

  103. That is still the procedure now? Somebody has just said that is how we work. What are the issues about transparency then, to answer that question, is working out what happens behind closed doors.
  (Mr McDonald) Yes.

  104. I have no problem with people coming in and listening to our meetings that we have in private. We have discussed this in the past.
  (Mr McDonald) It has a general effect on the way the organisation is perceived. It has an effect on organisations like ours because there is a cost to us of being involved and we do not know in what way our work is used.

Jim Dowd

  105. I presume this is what you meant by NICE not being open and transparent, that they will not let you take part in their deliberative sessions, simply the informative sessions. Are there other elements where you say they are not open and transparent and, if there are, what particular difficulties has this presented for any of you?
  (Mr McDonald) You have to look at the openness and transparency which is open and transparent to the general public as well as to consultees. We have certainly faced issues about openness and transparency in relation to our role as consultees. To summarise, that has limited our ability to comment and become actively involved in various parts of the process. I have mentioned that if there is no audit trail of decision making we do not know whether our input is worthwhile. In terms specifically of our experience of the appraisal we have moved to a situation where the appraisal system is basically re-run on beta interferons. We were told that that was because there were gaps in the original economic modelling and that there was going to be new economic modelling. What we then needed to know is what are the gaps so that we can start to think about the process. We were told that an explanation of those gaps would be contained in the minutes of the Appraisal Committee meeting. We waited three months to receive the minutes of that Appraisal Committee meeting. Those minutes arrived two days before we were due to have a meeting to discuss the new economic modelling with the Institute, which makes it very difficult for us to participate fully. What happened when the minutes came out was that they had a very sketchy explanation of what were the gaps in the original economic modelling which again disadvantaged us because we had waited three months and had two days before a meeting and did not have time to request more information.

  106. So it was not that you did not get the information: you did not get it in a timely fashion?
  (Mr McDonald) We did not get it in a timely fashion and when it arrived it was very sketchy which meant that we could not then participate fully in a meeting which was due to discuss this. If I can give one further example which is closer to where we are now, we have a sister organisation, the MS Trust, that conducted some research on quality of life issues, which was used in the economic re-modelling at a fairly late stage in the process. It was unclear to all of the consultees how that information had been used in the modelling. Straightforward questions from the Trust which we would also find useful to have answered were turned aside by the Institute and consultees were told that the only way of finding out how that information had been used was through NICE's formal appeals process, which we did, but we could not actually make any critique of how that information had been used because no explanation was given of how it had been used.
  (Mr Cayton) Another problem is the lack of transparency about who is providing evidence and whether evidence that has not been specifically sought by NICE but received by them is taken into account. In terms of process I am not sure that it matters on one level whether the evidence has or has not been given. I think what matters is that we should know that if someone has submitted evidence NICE has made a decision either to exclude it or to include it in their deliberations and that evidence should be accessible to other people who are trying to review their processes and their decisions.

  107. Are you saying that it is the origins of the information which is used or that you want more information about rather than the conclusions they draw from it?
  (Mr Cayton) We need to know as much about the NICE process as we can. One of the problems that we have, certainly in the appraisal that we were involved in, is that we were aware of high quality evidence that was submitted by certain research groups. We do not know, because that evidence did not appear in lists of evidence received by NICE, whether or not they had received it, whether they had chosen to exclude it and so on. That whole business in terms of when you come to the process of saying, "How excellent is NICE at doing its job", means that we need to see that the processes that it is using are very important because there is also so much public concern both from the patients' side, from the industry side and from the medical side. Transparency is a fundamental building block to public confidence.

Dr Taylor

  108. Mr McDonald, I think you were sitting there when beta interferon came up in the discussion, so I think we have probably covered that, but what we would like to know is, in a shopping list of things that the MS Society would like for sufferers with MS where does beta interferon fit on that? What other priorities do you have?
  (Mr McDonald) What we have to do is look at these drugs not as an alternative to service provision but as a component of an overall service offered to people with MS. That is certainly what we see in the centres of excellence like the Walton Neurology Centre in Liverpool, that these drugs are regarded as one component of a system of care which people will use in different ways over the 30 or 40 years during which they may have this disease. I would hate to have the issue portrayed as an either/or but the drugs are only of benefit to about ten or 15 per cent of people with MS. Those people are identified up front. You do not have to try everybody to find who those people are. There is clearly a need for services for those people who will never benefit from these drugs. That is why we are very pleased to be co-operating with NICE on the development of guidelines for services for people with MS. What we would like to see ideally is for these drugs to be included in the guidelines which come out as a component of the care and not as an alternative to other services.

  109. Have you any comments on the trials that are being set up?
  (Mr McDonald) Perhaps I can explain for the Committee's benefit exactly where we are at at the moment. There is no decision from NICE at the moment. A final determination has been produced which is negative but that is at the moment subject to appeal. It is also important to be clear that NICE does have the capacity to say things other than just yes or no. What it has said in this case is no but it has made a recommendation to the Department of Health that they should pursue ways of seeing whether the drug can be obtained cost effectively. What I am very pleased about is that the Department of Health has shown some real imagination about that recommendation and in discussions (which are only on a contingency basis at the moment because we still do not have a decision form NICE) is designing a scheme which would see the drugs prescribed to all of those who meet the clinical criteria for their use. Drug companies would be required to effectively nail their colours to the mast and say, "These are what we think are the logn term effects". If those long term effects were not found to be realised the companies would have to reduce their prices. I think that is an imaginative solution which is still under development by the Department.

  110. Can I come on to quality adjusted life years because you have those in your submission of evidence? You say that there was no consultation about using those, that it was the Appraisal Committee itself that just decided it was going to use those.
  (Mr McDonald) Yes. What I am talking about there is the creeping establishment of a threshold for cost effectiveness. That has emerged from a mist. It has been the subject of no directions from the Department of Health or the National Assembly. It has been the subject of no public consultation. It has emerged solely by the custom and practice of NICE's Appraisal Committee. The only reason we actually know it is in place apart from the experience we have accrued during our experience of the appraisal is because it was contained in papers which were given to the public at NICE's annual public meeting. They have not been placed on the website but they are effectively in the public domain. What they say—and this is a note from Sir Michael Rawlings—is that in future the Committee should provide very clear reasons for recommending technologies where the cost per life year gain for QALYs is in excess of £30,000. That effectively becomes the threshold of cost effectiveness. What we would argue is that that is a decision of such importance to the Health Service is that it should be taken only after open public consultation.

  111. I am obviously not up to date with this but when QALYs came in, and I think they were suggested by York University years and years ago, I thought that they had gone onto a back burner as being discredited.
  (Mr McDonald) There are some questions about whether the QALY, which is a general measure, is appropriate for different conditions. That is perhaps a discussion which could be had as part of an open debate on where the threshold would be applied. Our real concern is that that decision has been taken behind closed doors effectively.

  Dr Taylor: Another example of lack of openness. We are getting many points of the things we need to raise. Thank you.

Sandra Gidley

  112. The QALY seems to me to be a bit of an imprecise science. A lot of people have raised this threshold of £30,000 and I thought the latest drugs being approved had a QALY of about 38 which seems to knock that one on the head.
  (Mr McDonald) It is not saying that £30,000 is an absolute cut-off. I do not think any of us have ever understood that that is the case. What it is saying is that £30,000 per QALI is a threshold and if the Committee wants to recommend drugs which are more expensive than that it has to give additional reasoning than it would otherwise have done.

  113. But drugs have been recommended? I think that needs to be put on the record.
  (Mr McDonald) Yes.

  114. Surely there is this affordability question. If we do not use QALYs, which seem to me to be flawed and quite difficult to get your head round, what do you use? It is a bit of a dilemma.
  (Mr McDonald) It is a dilemma. The dilemma is really between using a generic measure which may have flaws and which may have more relevance to some conditions than others as opposed to using measures which are more relevant to individual conditions but which may make it difficult to assess relative cost effectiveness. I do not particularly have answers but I would like to see the issues debated in an open manner.
  (Ms Miles) The end of your sentence is what I was going to say. I would like to support what somebody said earlier about the separation of the clinical effectiveness evaluation and assessment from the cost effectiveness and that we can start to look at what is an appropriate measure, whether it can be a QALY or some other form of cost effectiveness measure that will then be useful for us to determine how the NHS then implements these recommendations.

  115. There seems to be an agreement that that should be a consultative process.
  (Ms Miles) Yes. We do not know the answer, you do not know the answer. I do not think NICE knows the answer. Dr James Rafftey, who is at the Birmingham Health Services Centre, looked at the NICE appraisals and only half of the appraisals had used QALYs or any cost effectiveness measures and it is unclear to me what is going on. That is why we need to stand back and say, "How do we do this stuff? How do we separate out cost effectiveness from the affordability question?" I do not think we can.
  (Mr Cayton) There is a lot of research going on into quality of life measures. In all areas of medicine people are beginning to recognise that qualify of life is measurable but these usually have to be disease specific. NICE is going to need some way of taking into account the new research in those areas and of bringing it together and weighing it up. The point that was being made in the earlier session about patient determined outcomes is fundamental to the position that all of us inthe patient organisations take, that the pharmaceutical industry has no regard to patient determined outcomes and so there is a cascade of problems for us that flow from the way in which these products are developed in the first place.
  (Ms Rule) Can I pick up on that point because in a way the NICE process has dramatised some of the lack of quality of life data. There are a number of determinations which have made very specific references. These have been positive ones which have actually recommended particular cancer treatments but they have said time and time again particular emphasis should be placed in future trials on specific quality of life measurements, further good quality studies are needed to confirm the impact or quality of life. You can go through and pick those out. In the longer term we have to ensure that these quality of life issues, and that means for people with cancer the ability to go about your ordinary life free of pain and other symptoms of cancer, are picked up in randomised control trials from the beginning, that they are commissioned through the NHS R&D programme. We must take these longer term measures but in the short term if it is only the voluntary sector who are doing it, commission it properly; give it some clout. It is not about throwing money at lobbying. It is actually about how we can in the short term fill in some of those gaps by commissioning quality of life data in a systematic way. In the longer term NICE is throwing down the gauntlet and I believe that some of those changes will happen because of the processes highlighting the lack of this data.

Julia Drown

  116. I should like to ask the witnesses from The National Cancer Alliance and CancerBACUP first of all whether on balance you might say that it is too early to say you think that NICE is beefing up the availability of access to effective treatments. We have had evidence from CancerBACUP about NICE blight, as it is called, in relation to treatments for breast and colorectal cancer. I should like to know from you how you see an alternative policy to this because presumably you do not want anybody else to try and create their little mini NICEs to make decisions in advance of NICE officially making a decision? Particularly for the National Cancer Alliance to address, you said earlier about how we can separate ultimately effectiveness from affordability. Would the proposal to separate it out and have a separate body or committee looking at affordability actually mean further delays and that blight goes on potentially for longer?
  (Ms Rule) To pick up on the speeding of availability first, the answer is yes, in part. Of the appraisals overall, and remember that each appraisal can include more than one treatment, nine cancer treatments have been recommended by NICE and without doubt in implementing its guidance there is a time lag. Implementation is a massive issue but you begin to find that they are more available. In part of course NICE was set up because of concerns about postcode prescribing specifically on cancer treatments. We get people phoning us up and they are increasingly saying, "Is there a national guideline? What is the standard?" There is a real expectation of the process. In part it has sped up. The implementation is slow. There is a real time lag between a recommendation and its actual implementation, so there is an issue there. Nine have been recommended, eight are in process, many of them from the end of 2000/very early 2001. They are the ones that there are problems around what we are calling NICE blight. It is colorectal drugs, breast cancer, and non-Hodgkins' lymphoma. It is precisely those ones that have just been going on and on. What happens is that we will have patients phone us up who say, "My cancer doctor believes that I could benefit from X but is unable to fund it unless and until NICE recommends it." Almost invariably they then say, "But I can have it if I go private", and of course if something is not available you also begin to have higher expectations from that specific treatment than might otherwise be the case. We had an example last week of somebody phoning up one of our specialist nurses to say, "Should we go private? Should we spend our savings on this treatment?" The blight issue is real and partly you deal with it by speeding up appraisals.

  117. Where is this document? Is that at PCG level?
  (Ms Rule) No, no. This is the actual technical appraisal process. Oh, you mean the health authority?

  118. Yes, it is the health authority.
  (Ms Rule) You might for example get a particular cancer centre where patients from one health authority are able to have a treatment and patients from another are not, in other words precisely the set of circumstances that NICE was set up to resolve, but it is happening, and that is partly a cause for speeding up the process. On one of the appeals that we took part in successfully last May, the Committee met again to reconsider its guidance in November and it still has not come to the end of the process. That includes a treatment which could offer potentially curative surgery. Another case study I have is in that category. Speed it up, I would say, and also allow clinical judgement for treatments during the appraisal process because the treatments we are looking at in the cancer category are for advanced cancer. We do not have time for these very lengthy processes. Those are the two points I was asked to consider and I know my colleague will pick up the others.
  (Ms Miles) I do not think it is an issue of speed. I think it is an issue about access to effective treatments that I am more concerned about. The answer to your direct question, are people getting better access, improved access, to clinical effective interventions, is that it is probably a mixed bag. There is an issue there which is quite complex and I am going to struggle to explain it. I had a go in the submission and I think I did not do it very well, so I will have another go now. It is on the relationship between the topic selection in the NICE appraisal process and the implementation process and the impact on resourcing the recommendations. NICE is not responsible for the first one and the third one. It is not responsible for the implementation impact. However, the implementation impact and the prioritisation of the appraisals is skewing the resources and the prioritisation of resources within the NHS now, and therefore it is increasing inequity of access. That relates to an earlier point, and I am quite pleased that other people have made this point, about the whole care pathway. If health technology assessments are considered separately—a drug or an intervention or laparoscopic surgery for colorectal cancer, etc—outwith the whole patient pathway it again skews the resource allocation. I come back to the status of the guidelines versus the status of the HTAs. If the HTAs have the status such that people are going to have to fund those statutorily, again you skew resources. Particularly on ring fenced funding for the cancer modernisation agenda, where the regional offices and the health authorities have been told (and it is not an optional thing) that you will out of the £250 million you have for this financial year hold back £170 million on funding the NICE appraised drugs as and when they come through, once again you skew the prioritisation for the broader service for individual groups of cancer, because of course cancer is many diseases. I am sorry that is a complicated point.

  119. No, you did explain it in your evidence but the difficulty then is what we as a Committee make as a recommendation for the way round it because we have a lot of evidence from other people saying that of course this should be ring fenced for the drugs.
  (Ms Miles) It should be.
  (Ms Miles) But you have exactly pointed out the problem with it because it can skew the treatment away from what might be a patient priority.
  (Ms Miles) What has happened is that the ring fenced money for cancer development and improvement, £255 million rising to £570 million, has gone to the health authority base lines, of which there is a sub-hypothecation which then is effectively skewing it so that it is effectively not then £570 million to cancer; it is minus £170 million for 13 potential drugs. That is the point. People then have to choose at a health economy level, health authorities, PCTs and others, how they make those prioritisation decisions and that is very difficult because some of it has already been imposed.
  (Ms Rule) Perhaps it is under-funding rather than skewing. That is another way of looking at it.
  (Ms Miles) The result of the process is that it is skewing priorities at the moment because you have to choose, do you not? You have to say, "Do I spend £5,000 on drugs for lung cancer patients or do I buy a specialist nurse for lung cancer patients?"

previous page contents next page

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

© Parliamentary copyright 2002
Prepared 8 July 2002