Examination of Witnesses (Questions 120
WEDNESDAY 16 JANUARY 2002
120. To be clear, you would support money for
cancer but not within that for cancer drugs?
(Ms Miles) I think there is a strong argument for
ring fencing the modernisation agenda across the NSFs and that
is about additional growth monies which you can trya nd spend.
There is a strong argument about, "Here is some more money
but what have you spent it on? How have you improved it and let
us see it done at patient level?". That is a cogent argument.
People do not think through the process of, "Let us have
some topics". The topic selection in itself will skew the
resource allocation in the end. Let us look at the assessment
of clinical evidence and do that pretty purely and scientifically,
and then let us have a think before we start about the impact,
the "what ifs", the actual costs, the pricing and whether
it is effective and whether we want to afford it. What do we afford
in relation to one thing versus something else?
121. In your evidence you also talked about
the blight issue. Would you recommend that in the interim, while
we are waiting for a NICE guidance, the decision should be taken
at PCT level or at individual commission level?
(Ms Miles) It is speed versus time to think and the
number of appraisals that NICE is resourced to do and at £10
million a year that it gets per year it is not going tobe able
to achieve very much very quickly. Yes, you do need to increase
the resourcing in NICE. There are existing processes for making
decisions about what is clinically effective at a local level,
and it is done by public health consultants, it is done by drug
therapeutic clinics; there is a range of organisations and advice
that comes through, and they should continue it. I think the DoH
has issued guidance, if not instructions, to PCGs, PCTs, that
they should do that. In particular, in respect to Glevec(?), the
drug for CML, recently, it was very strong. It said, "You
will have to pay for this drug irrespective of whether it is being
appraised by NICE and we will wait for the outcome of that appraisal".
122. I was interested in your evidence again
on the approach which has been made by the pharmaceutical industry
to help some patient groups with producing evidence for NICE.
Can you say a bit about how they may have already been compromising
patient groups or whether that is a theoretical worry, and whether
there is a way round that?
(Ms Miles) We have talked about the resourcing of
patient groups and it is again about why, what we want and what
do we define by the patient group and what we want from patient
groups. People are very confused about that. Patient involvement
is what I am interested in. It can happen at various levels in
various ways. It could be that the R&D committees could be
commissioning patient groups or research organisations to do patient
based quality research. In relation to supporting the input of
patient groups, clearly they cannot do this for nothing. Organisations
such as mine have not got the resources. We have already spent
a lot of time just to prepare for this process today. The industry,
I know, because they have phoned me, have offered support and
help, one, to share the information or their publicity about their
particular product with patients and patient groups, and I know
many of them have offered to go to meetings and hear the evidence.
That is probably sort of okay as long as we have the ability as
an organisation to robustly and critically appraise what they
tell us. Of course that is another time resource. When it slips
into, "Can we help you prepare your submission to NICE?",
which again is something that I have experienced, then it gets
into a compromising situation. It could be that that is a very
pure process and the organisations are both responsible for that,
but my board has chosen not to accept that offer because of the
risk of bias.
123. Mr Cayton, do you think the NICE guidance
has put inappropriate emphasis on drug treatment compared to other
forms of supporting Alzheimer's, and what about more general guidance
which puts drug treatment into its therapeutic place?
(Mr Cayton) This picks up in some ways what Dr Taylor
was thinking about in relation to MS and certainly again we would
want to say that you should not see drugs as either/or or separate
from the care that we provide. The problem that you pick up (quite
rightly) in relation to people with Alzheimer's is that these
drugs were the first drugs ever and therefore they were being
appraised in NICE's context against nothing. They were not being
appraised against other technologies because what we have currently
is primarily social care and diagnostic and supportive therapies
but not the kinds of therapies that NICE appraises. In some ways
there is a danger in the question because once we get on tot he
cost effectiveness debate we fall into this trap that the Health
Service budget is one budget, but all the other care, including
voluntary sector care, is not part of that cost base. Who costs
how much a carer saves from the person they care for having their
health improved? Who costs the savings to the Health Service because
the carer is not stressed? The whole system of cost effectiveness
and the ability that we haveand by "we" I mean
the whole of our research establishmentto make those assessments
is flawed. I think one of NICE's biggest problems was being given
this second task, which in a sense people have referred to as
really a political decision, of doing cost effectiveness as well
as clinical effectiveness which has dug NICE into a completely
different mire of shortage of adequate information and really
not very good techniques for carrying out those assessments.
124. Why do you feel that NICE should take into
account material such as patient views that have not been tested
in clinical trials? To what extent do they do so?
(Mr Cayton) I think this is terribly important, I
really do. Let me say first of all that I think that randomised
control trials are great. Randomised control trials are one of
the best ways we have discovered of objective appraisals of evidence,
but randomised control trials by definition always try and ask
a very narrow question, always try and have a very clear and focused
outcome measure, and always try and eliminate variables. Real
clinical life is completely different. Real clinical life is full
of variables, it is full of all kinds of odd outcome measures,
it is full of adverse events and difficulties. It is full of people
having different relationships. In my area diagnosis itself is
very difficult. The drugs for Alzheimer's were closely defined
by the FDA as being only for Alzheimer's disease. Actually we
do not really know how to diagnose Alzheimer's disease accurately
prior to death, so you have a drug that is appraised for Alzheimer's
disease and we are now being told it is not suitable for other
dementias and yet the clinical evidence is that it is particularly
good, for instance, for Lewey Body disease. What I would want
to argue is that we are now beginning to develop again good qualitative
ways of looking at patient perception and patient outcomes and
that those should be formally and very clearly partnot
allof the evidence. There is this curious idea that goes
around that somehow doctors are always objective and all they
do is read the BMJ and look at the manuals and they do exactly
what they supposed to do.
125. I do not think anybody here thinks that.
(Mr Cayton) I do not know a single doctor who does
not prescribe on anecdote.
126. You said they should be part of the clinical
(Mr Cayton) Yes.
127. Should they not be part of the clinical
(Mr Cayton) Yes.
128. Not something separate?
(Mr Cayton) The point goes back to this patient determined
outcomes. One of the faults of clinical trials, and I can only
speak particularly in my own area,
129. I accept what you are saying about trials,
but if you have got other evidence that this drug is doing good
things assessed by patients then it should be part of the clinical
(Mr Cayton) Yes.
130. It is not assessed by doctors, not assessed
by scientists, but if you have some method of measuring this clinical
trial which includes what the patients think of it, then that
is what you should have.
(Mr Cayton) Yes, but the problem is that clinical
trials tend to be statistical, quantitative measures and we are
talking about qualitative measures. That is a research methodology
that needs to be enhanced and developed for, if you like, the
more subjective patient evidence to become more objective.
131. But if 100 patients say that it made them
feel better, ten patients say it did not make them feel better,
then that converts qualitative evidence into statistical evidence
and that is what we are looking for.
(Ms Rule) But it is a question where we would not
start from here because the reality is that most of the RCT data
is not including these quality of life issues, so we are all filling
132. I agree with the basic thesis that it should
be assessed, but not separate from clinical trials.
(Mr Cayton) We have got to get right downstream and
that means getting downstream of the pharmaceutical industry.
The pharmaceutical industry do not start with the patient. With
respect, they start with two things. They start first with science
because they think, "Ooh, is this a way we could get a rational
drug treatment and make money?", or they start with the doctors
because that is who they talk to and that is who they sell the
133. When I had a proper job I was a scientist,
but you could include that in the clinical trials. I agree with
you that if the drug companies are not doing that you have to
find a way of doing it.
(Mr Cayton) As has been said, one of the benefits
of NICE is that it is beginning to force the pharmaceutical industry
to face up to the poor quality of its evidence in these particular
134. I will play Devil's advocate for a moment.
I have the same problem as Doug in a way, that the scientist in
me wants to see the factsand the evidence and all signed, sealed
and delivered, and it makes the decisions much easier. The politician
in me thinks that the patient should have some sort of voice.
I accept your point entirely about good qualitative evidence,
and I was quite impressed with what you had done in your submission,
but the danger is that not all organisations approach matters
in quite that sort of way. If we do more work on the quality of
life issues which were highlighted earlier we may come to a position
where that is the norm. Having said that, is there a role for
the wider patient voice? I become alarmed at this stageI
am not going to mention whose submission this iswhen I
read a submission saying, "allegedly inappropriate differences
in approach", "reportedly inadequate quality evidence
base", "perceived differences in the handling",
"lack of transparency and perhaps honesty", without
any real back-up for those statements. That is the scientist in
me saying, "Fine, but where is the evidence?" It is
down to the patient groups if you like to provide good qualitative
evidence, but should the taxpayer fund that? Where you have got
health priorities, and we talked about this at length earlier,
where you have got a lot of pressures on the health budget, should
we be giving huge chunks of money to patient groups so that their
(Mr Cayton) I would absolutely want to say that I
am in favour of giving huge chunks of money to patient groups,
though not necessarily for that purpose. We have touched on an
important issue which is the very varying capacity of patient
groups. There is absolutely no doubt about that. We have very
different levels of resource, we have very different levels of
skills, we have very different depths. Some of us have our own
major research arm so that we can come at these things with a
team of our own researchers. Others are real family associations
struggling, especially with the rarer diseases, to write their
submissions on the kitchen table at home. I take your point but
I am not sure that in a way we are saying the patient groups have
to give NICE the evidence. We are saying that NICE must have high
quality evidence of what patients think. If NICE cannot get that
from the patient group in a particular appraisal it should be
commissioning that research just as we did. We commissioned independent
research and so have other patient groups who have been involved.
NICE should not say, "Oh, well, there is no patient group
here" or, "There is no-one competent to give us the
patient perspective". They should commission the research
if there is a gap in the essential evidence that they need.
(Mr McDonald) Could I comment? There are two issues.
One is the issue that Harry has been talking about of delivery
of patient related information, which need not come from patient
organisations but could come from other sources. It could be funded
by NICE but not involve giving money to us. However, there is
a much lower level of support which is needed. I do not mean to
be pejorative but the kitchen table charities are very small organisations
which are facing real choices about "Do we do this and stop
doing other things that we currently do?" At the moment there
is no financial support for those organisations at all. NICE is
about to start making very generous grants of £400 to those
sorts of groups. What is interesting and what I would like to
see developed is that NICE has a patient involvement unit which
helps organisations like ours, or small organisations particularly,
in their involvement with the development of guidelines on services.
I would like to see that extended to offer similar support to
small organisations who struggle not to deliver questionnaires
to 10,000 people but who struggle to even have any voice in the
process at all.
Chairman: Are there any further questions from
colleagues? We could go on but there is a health debate at seven
o'clock that some of us want to participate in, so can I thank
you for what has been a very helpful session.