Examination of Witnesses (Questions 320
WEDNESDAY 30 JANUARY 2002
320. I am interested in the set up of NICE which
said NICE is required to be "sympathetic to the longer term
interests of the NHS in encouraging innovation". What do
you understand by innovation?
(Professor Sir Michael Rawlins) I think there are
a number of aspects to it. The first is that we would be very
anxious for innovative new treatments that are clinically cost
effective to be made available to patients and that the patients
could benefit from them. I have to sayand I do say with
some regret as a clinical pharmacologistthat British doctors
are probably the most conservative in the western world and are
the slowest to take up new treatments. There is a therapeutic
conservatism in British medicine that goes back 100 years to William
Osler and previously and we are unquestionably almost the slowest.
There has been evidence for this recently published in the Wanless
report and the PICTIF report and so on. One of the things we wish
to do is really for new innovative treatments to become available
to patients as quickly as possible. I think that is the first
part. The second part of it is that we are anxious to ensure that
new treatments are looked at in a way that, if there are any doubts
about it, we should default in the interests of patients. There
are a number of individual appraisals, which we could go into
if you wish, where there has been some doubtit has not
necessarily been beyond reasonable doubt but on the balance of
probabilitiesthat patients would be advantaged by having
access to a particular medicine, and so I believe that in encouraging
innovation we should encourage that sort of use of treatments.
The third thing is that there are a number of occasions when we
have felt that innovative new technology needed further studies,
and so we have promoted clinical trials or other sorts of investigations
to make sure that the potential new treatment really does fulfil
321. You said in there that there were some
cases where it was not absolutely clear but you thought in order
to benefit patients you would recommend that particular treatment.
That is also, in benefiting the patient, benefiting the particular
company that is pursuing that treatment. Could it also be argued
that having that particular recommendation there that you have
to encourage innovation is intrinsically building in a benefit
to drugs companies, to other companies, to develop new products.
(Professor Sir Michael Rawlins) I think it would and
I think if the company produces a new product that is clinically
cost effective then it is perfectly reasonable for it to have
the rewards of its original investment. It applies not just to
pharmaceutical products but it also applies to procedures and
surgeons who have introduced new procedures, for example.
322. Can you say whether or not having that
criteria has changed any of the guidance that you have given out,
that had that criteria not been in there you would have had a
(Professor Sir Michael Rawlins) Can I ask Professor
Barnett, who chairs the Appraisal Committee.
(Professor Barnett) I think innovation is only one
component. It is certainly very important. It is very important
obviously for new innovative therapies to be made available, if
they are clinically cost effective, as soon as possible, but innovation
also means innovation in the way which we apply the guidance.
An example would be total hip replacement. There is a huge number
of possible hip replacements. We have heard evidence from a variety
of individualsclinical experts, patients themselves, from
manufacturerson the wide range of possible hips that are
available. It seemed to us that the guidance encompasses the concept
of innovation by setting a benchmark which has never been set
before on the basis of clinical effectiveness. A new hip achieves,
if you like, its licence on the basis of the fact that it has
certain criteria in its manufacture, not because it reaches a
benchmark in clinical effectiveness, which is what patients want
(namely, that it lasts long enough, it is not too difficult to
put in, "It's effective for me"). So the NICE guidance
identifies a benchmark which new hips, new innovative ideas, would
have to achieve: to be as good as that which we already have.
There is an example of innovation, if you like, for something
which is kind of old.
323. Can you be absolutely clear: When you encourage
innovation, it might be a drug, it might be a new technique, but
it might also be an old technique, like physiotherapy or something
(Professor Sir Michael Rawlins) It could easily be.
It could easily be, yes, most certainly.
324. And you are comfortable with those criteria
being your guiding criteria.
(Professor Sir Michael Rawlins) Yes. I think, as Professor
Barnett says, it is just one and has to be taken in the context
of the overall judgment that needs to be made.
325. I was struck by something Mr Dillon said
in the earlier part of that exchange. We were talking about the
fact that the resources could be redistributed in the National
Health Service if current treatments and so on were demonstrated
not to be effective. Now most of the examinations that have been
carried out by NICE have ended up recommending virtually every
technology that you have examined. I was under the impression
when NICE was set up, I hoped it would be, that many existing
techniques would be examined and found to be wanting and replaced
with better techniques. The best example I can find of that that
has happened is impacted molars, in saying that they should not
be removed if they are pathology free. I remember myself 25 years
ago having an argument with a dentist about my impacted molar,
saying it was not giving me any trouble, and he was very anxious
to do something about it. I won in the end: I have still got my
impacted molar and it has never given me any trouble since. That
is the kind of thing that is going on in a number of other areas
of the National Health Service. What can we do to encourage more
examinations by NICE of these kinds of thing? I know you are not
supposed to make the recommendations but perhaps you can give
us some advice on how to seek to make that happen.
(Mr Dillon) We would genuinely like more of those
topics to come through to us. We think the best way to do that
is to engage actively with health professionals and those who
support them because those are the people who know, either directly
because of the work they have done in their overall health communities
on the way in which interventions are used or because of what
they have read, of the potential topics that we could look at.
Not all of them may be suitable for the kind of appraisal that
we do but clearly some of them will be, and so what we have been
asking the Department of Health and the National Assembly for
Wales to do is to expose the process of selecting topics to the
NHS and to engage actively with the service. We genuinely believe
that if that happens then two things will result. One is that
we will get more of those sorts of topics coming through. Secondly,
I think the service will be more confident that the guidance that
we eventually produce will be on topics that they regard as being
important in their local health communities, that, regardless
of the array of other priorities they have, what the service in
effect which owns NICE will be asking them to do will be the things
it regards as being important.
326. How did the pathology-free impacted molar
study get onto your agenda? Have you any idea?
(Mr Dillon) When NICE was first established we published,
the first list of technologies that was given to us, the Department
and the Assembly wanted it to display the range of potential technologies
that NICE could look at and, indeed, apart from aspects of health
promotion, which might be regarded as a technology, they were
all present in that list. But the reality was at the time we were
established that there was a significant backlog of pharmaceuticals
that were already subjected to substantial geographical variation
in use, and it was clear that the Department and the Assembly
wanted us to deal with those as quickly as possible, which is
what we have done.
(Professor Barnett) With respect, the concept that
it is all or nothing is a narrow view of what we do, and I know
that we discuss in committee in great detail the issue about targeting
an innovation or an intervention because by so doing you actually
do, if you like, identify the correct use of the funds and therefore
reduce inappropriate expenditure. That does not perhaps come out
quite so clearly because part of the appraisal will be to target
the technology appropriately.
327. You say you are doing that all the time.
(Professor Barnett) Absolutely.
328. The Committee has received a number of
submissions which worryingly describe NICE guidance as being flawed
or even perverse. I refer particularly to the submissions on the
Drugs and Therapeutics Bulletin, which is highly regarded in medical
circles, which highlighted six technologies where they felt the
guidance had been flawed or perverse in some way. Does this not
actually give you cause for concern, that such a well regarded
body is picking quite large holes in the guidance that NICE provides?
(Professor Sir Michael Rawlins) I am disappointed
in how they perceive us. There are both general points that I
would like to make and more specific ones. The general points
are that we do not believe our guidance is flawed. It is constructed
to a very robust process, probably more robust, open, transparent
than any analogous type of activity anywhere in the world. I have
been involved with the licensing of drugs: I chaired the Committee
on the Safety of Medicines for six years and was vice-chairman
for the previous six years, I have been involved in the European
Medicines Evaluation Agency; and the World Health Organisation,
and the robustness of the way NICE goes about it and the way it
is open and transparent is very different from, for example, the
regulatory authorities. We also challenge and dispute that we
contradict the Summary of Product Characteristics. We do not.
All our guidance to date has been entirely in accordance with
the indications in the Summary of Product Characteristics that
has been produced by companies but with the approval of the regulatory
authorities. So we would dispute that. It has also been suggested
that we should incorporate a complete account of the use of a
particular technology within each appraisal guidance. We do not
expect in our guidance to replicate what is already in the Summary
of Product Characteristics for the pharmaceutical industry any
more than we would expect in our guidance to instruct an orthopaedic
surgeon on how to fit a new hip. That information is quite separate.
For drugs it is contained in the Summary of Product Characteristics
and that is the proper place for it. Nevertheless, we do refer
to specific sentences from the SPC where it is appropriate. The
other point I think is that sometimes our guidance should have
covered another technologyand that particularly applies
to the criticisms of our appraisal of methylphenidate. We are
of course asked to look at particular technologies in relationship
to current best practice and methylphenidate we were asked to
look at it because there was a lot of controversy about its use
and that was the whole reason for its referral to us. So, in general,
I reject the notion that our guidance is flawed, and we have prepared
a detailed commentary on all the individual pieces of guidance
that are criticised by the Drugs and Therapeutics Bulletin. If
you want to question us on those, that would be very welcome,
but we have prepared it in written form and we will send it to
you early next week.
329. So you contest every one of their assertions?
(Professor Sir Michael Rawlins) Certainly, Chairman.
330. Every one of them.
(Professor Sir Michael Rawlins) Certainly.
Sandra Gidley: It would have been useful to
have had that here today, actually, I have to say, so that we
could have looked at it before we spoke to you. Why was that not
provided? We have had all this load of stuff which I doubt
all of us have had a chance to read.
Chairman: Have you not read it? What have you
been doing today!
331. What would have been useful would have
been that rebuttal.
(Professor Sir Michael Rawlins) We were advised that
we should send it after we had given our evidence.
332. Who advised you of that?
(Professor Sir Michael Rawlins) I think that is right.
(Member of the audience) We made our submission on
the 4th, which is the information you have there, in line with
333. I am not sure we can take evidence from
a member of the audience. I am sorry about this.
(Mr Dillon) We are sorry, Chairman. We had prepared
it. If we have been misinformed, then I apologise for that.
334. OK. But you are prepared to supply us with
the detailed information.
(Mr Dillon) Absolutely.
335. I just want to come back on a couple of
the points you raise. You mentioned methylphenidate, which is
perhaps a good example of something we covered earlier. The fact
that it was not looked at in comparison to dexamphetamine has
been picked up as problematic. With all technologies would it
not be useful for the future to look at them in a broader sense?
Is it not actually causing problemsand it is not a problem
necessarily NICE is makingthat everything is looked at
in such isolation?
(Professor Sir Michael Rawlins) When it came to methylphenidate,
we did actually consult with professionals as to whether we should
extend the guidance to dexamphetamine and they said, "No,
we hardly use dexamphetamine. We do not regard it as an appropriate
form of treatment for most patients and what we really need to
know about is your views on methylphenidate (Ritalin) because
it is a very highly controversial topic. There have been a lot
of adverse comments from some sources and we would really like
a robust and authoritative guidance on its use in our practice."
That is what we did. The health professionals involved have been
very pleased at having such authoritative and robust advice from
us and have made comments positively, as have many patient groups
(Professor Barnett) Can I just concur with that. The
appraisal process meant that we had to engage with clinical experts,
with patient care organisations, with in fact the parents of/the
carers of patients with the problem and the use of Ritalin. We
had to deal with the evidence base that was available and the
lack of, if you like, head-to-head comparisons directly in the
evidence base between dexamphetamine and methylphenidate. In addition,
what is perhaps most important and which has not been referenced
in this particular appraisal is the comparison between the use
of Ritalin with non-pharmacological approaches to the control
of this condition. It is pretty obvious that the evidence base
suggested quite dramatically that actually behavioural therapy
did not stand up too well against the drug therapy. If you will
look at our advice, it was, as the evidence was presented to us,
that waiting for behavioural therapy before commencing Ritalin
was actually an inappropriate thing to do. So we did make comparisons
to the alternative approaches to the management of this condition.
Leaving out dexamphetamine was a separate component of that.
336. What about the allegations that, say, for
the anti-diabetics you have actually recommended a use which was
not in the data sheet at the time.
(Professor Barnett) I do not think our guidance does
go contrary at all to what the data sheet says. What our guidance
is intended to do is to give pragmatic and practical advice based
upon the licensed indications
337. You actually recommended a use that was
not licensed at the time, according to the Drugs and Therapeutics
(Professor Barnett) I refute that. We have given advice
based upon the licensed indication for medication in a practical
way to achieve what the experts requested of us as to how they
should best use this medication in clinical practice. We could
go into the details if you wish, but I would suggest that might
be better in a written form.
338. It would be useful to have that. If I could
just go back to this robust, open and transparent. You are very
bullish about this in your submission to us. Quite a few of the
submissions have said that there seems to be an inconsistency
between the six different centres that are performing the assessments.
There appears to be certainly a very strong feeling out there
in the medical community that you are not necessarily getting
the same standards from the six centres. How would you answer
that allegation that has come forward?
(Mr Dillon) By looking at how those assessment reports,
as we describe them, are commissioned. I think it is the assessment
reports that are provided to us by one of now six universities
in the UK that form part of the evidence base that the Appraisal
Committee looks at. They are commissioned on our behalf by the
National Centre for Health Technology Assessment, which is part
of the NHS Research and Development Programme. They are not commissioned
directly by NICE but we set out what it is that the Appraisal
Committee needs. Once we do that, the commission is done by the
National Centre for Health Technology Assessment and the National
Coordinating Centre for Health Technology Assessment. They commission
the reviews from independent academic department. Those departments
themselves produce reports to standards that they agree jointly
with NCCHTA. Those assessment reports are peer reviewed before
they are received by NCCHTA; they are reviewed by the Centre for
Health Technology Assessment before they come to the Institute;
they are reviewed again by the Institute's technical staff. In
the new process that we have got, they are then sent out to all
the consultees, who have the opportunity to read them and to comment
on them and to correct any factual errors which from time to time
do, perhaps inevitably, despite all of those quality checks, creep
into the report. They are then subjected to another, in effect,
peer review process, because they go to the Appraisal Committee.
There is then a further opportunity for consultees to comment
again if there is anything left in the documents that they feel
unhappy about. So there is a whole series of stages in the process
that these documents go through where they are exposed to clear
opportunities for all consultees to identify any errors, any missing
evidence or anything that might mislead the Committee. I cannot
think of a process that parallels that in the NHS that secures
quality in the same way.
339. Has that always been the case?
(Mr Dillon) Yes, that which I have just described.
The only thing that did not exist until recently, when we put
our new process in place and, only applies to appraisals that
were initiated after 6 February 2001, the only element that did
not exist was that it was not until the new process came in is
that we send the assessment report out to consultees before the
Appraisal Committee sees it. But all the other checks that I mentioned
have been in place right from the start.
(Professor Barnett) The Appraisal Committee themselves
are a highly political bunch, and we spend a lot of time looking
through the assessment documents and in our committee discuss
the concerns that may have been raised. We are very grateful for
the quality checks that go on.