Examination of Witnesses (Questions 101
WEDNESDAY 16 JANUARY 2002
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
(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 diseasesMS and Alzheimer's being
two of themwhich 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
(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
(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.
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
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.
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 sayand
this is a note from Sir Michael Rawlingsis 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
Dr Taylor: Another example of lack of openness.
We are getting many points of the things we need to raise. Thank
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
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.
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
(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
(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
separatelya drug or an intervention or laparoscopic surgery
for colorectal cancer, etcoutwith 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
(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