Examination of witnesses
(Questions 40 - 59)
THURSDAY 4 FEBRUARY 1999
PROFESSOR SIR
MICHAEL RAWLINS,
DR GINA
RADFORD and DR
TIMOTHY RILEY
40. Continuing my colleague's line of questioning,
Howard Stoate asked about the take up of advice and knowledge.
Do you think that issues about requirements for doctors to re-train,
keep up to date, be appraised, might have some bearing on their
willingness to take seriously the advice such as it is?
(Professor Sir Michael Rawlins) There is a lot of
discussion, as you are aware, about re-accreditation and one of
the criteria that is being contemplated is audit and whether people
can demonstrate that they have robust audit in place.
(Dr Radford) This is also answering Dr Stoate's issue
as well. NICE's role is but one piece of the jigsaw in terms of
the whole quality agenda, and you cannot expect just one piece
of the jigsaw to give you the whole picture. So continuing professional
development, self-regulation, performance assessments, looking
at comparative data through indicators, et cetera, are all part
of this jigsaw to really see whether the standards which are being
set by NICE and through the national service frameworks are being
delivered on the ground, and then a feed-back loop as to, if not,
why not, are there some issues where guidelines are not necessarily
user friendly, there are gaps in the knowledge, there are other
issues which need to be considered. So NICE by itself cannot do
everything, it is part of this overall jigsaw which, as you rightly
say, professional self-regulation, continuing professional development
are also parts of, to produce this overall improvement in the
quality of the care for patients.
41. Would you see part of your role as impinging
on this in a way, inasmuch as there are procedures, for instance,
which would be very clinically effective and cost effective if
done properly, but it is easy not to do them properly. I have
in mind a specific example of what became known colloquially as
keyhole surgery. It is not the keyhole surgery which is the problem,
it is the —
(Professor Sir Michael Rawlins) — it is the keyhole
surgeons.
42. — practitioners. Exactly. When things like
this come within your orbit, will you feel it is proper for you
to make that sort of caveat, that sort of reservation or recommendation?
(Professor Sir Michael Rawlins) I think at some stage
NICE and the Department would obviously engage in discussions
with the College of Surgeons and so on about how to carry these
new things through, particularly ones which require a lot of operator
skill and learning. I am not sure how we would resolve them but
I do not think there would be in principle any difficulties. We
would undoubtedly say that laparoscopic cholecystectomy is effective
if done in the hands of skilled operators, and then perhaps the
College of Surgeons would work out what constitutes a skilled
operator and how much training is needed.
Mr Gunnell
43. To go back to Howard's issue, first of all,
if one looks at the questions which we get in health questions
in Parliament, one of the ones which is frequently raised, which
is I think covered by this area, is the issue of Beta Interferon
for Multiple Sclerosis. It would seem to me, if I understand the
problem correctly, that there are some patients with Multiple
Sclerosis who can benefit from this, but there are many others
who cannot, therefore it is not sensible that we have got some
health authorities where the treatment is available across the
board and other health authorities where it is impossible to get
it. I would expect that NICE would be able to put forward guidelines
as to the type of patients who experience shows has benefited
from this, and then I wonder if you put forward such guidelines-I
do not know how clearly a line can be drawn-as to the sort of
sufferers who would benefit from this, would you be able to suggest
that health authorities ought to fund those particular patients
as opposed to either funding the lot or funding none?
(Professor Sir Michael Rawlins) You have put it, if
I may say so, very well, that is exactly the sort of thing we
would do. We would try and assess those patients who are most
likely to benefit and who achieve the greatest benefit in a priority
list. Sometimes it might be important to actually persuade the
manufacturer or the NHS R&D to look into further whether one
can predict which patients are going to respond and which patients
are not going to respond. So I think there will be a continual
feed-back between producing priorities and encouraging further
research.
44. So you would expect, for instance, that
is one of the issues you will look at fairly early on?
(Professor Sir Michael Rawlins) I would be very surprised
if it was not.
45. Would you have to go through the process
of getting the Department to agree if you could identify that
group of patients whom you felt were most likely to be helped
by the treatment? Would you be able to get the Department to have
some mechanisms whereby health authorities were not able simply
to say, "We cannot afford this"?
(Professor Sir Michael Rawlins) Yes, very much so.
We would tell the Department it might cost them £10, £20
million a year, and that resources would need to be found. If
the Department accepted that, then it is implicit that the resources
would be found. Have I said the wrong thing, Dr Radford?
(Dr Radford) No.
46. I am interested that you had to check that.
If you were able to come to some clear guidelines so you could
advise health authorities and know whether the treatment would
be funded or not, what sort of ruling would you be able to have
over the private sector and the independent sector? Because a
person could say, "I cannot get this through the public sector
because it is obviously not thought to be particularly beneficial
for me ...", though they might not take it that far, "...
I will just go to the private sector and get it." What jurisdiction
would you have over that?
(Professor Sir Michael Rawlins) NICE quite clearly
does not have any jurisdiction over the private sector, nevertheless
the private sector has indicated very clearly, at least the private
sector limited by the private health insurance agencies, they
would wish wherever possible to adopt the NICE guidelines and
NICE appraisals. Nevertheless, individuals might still wish outwith
health insurance to purchase Beta Interferon or something and
it is a licensed product on the market and that would be their
privilege to do so.
47. So then it would be a position of advice
rather than instruction, if you like?
(Professor Sir Michael Rawlins) Absolutely. I might
add that private insurance companies are not very keen on providing
Beta Interferon.
48. I am sure they are not. I wonder if you
could look at another aspect that sometimes occurs within the
private sector? Are they not sometimes advertising or suggesting
that certain treatments which might be relatively expensive are
likely to be effective whereas they are not? Can you give negative
guidance for the private sector?
(Professor Sir Michael Rawlins) That would be difficult
but there are other mechanisms for handling those sort of issues.
If they are claiming that a pharmaceutical product is effective
when it is not, then the Medicines Control Agency have an army
of enforcement officers who will jump down their throats. So there
are other mechanisms which can get rid of most of those. Dr Radford
may be more familiar with this type of problem.
(Dr Radford) Clearly NICE has been established to
provide guidance to the NHS about the services provided in the
NHS. As Professor Rawlins has said, if the private sector wish
to consider that in terms of the way it practises, fine, but we
have to be quite clear what NICE's role is, and it is not within
the private sector directly or specifically.
Mr Lewis
49. I want to talk about the structure of the
organisation and therefore I suppose how accountable, transparent
its decision-making processes will be. I think the word "independent"
also comes to mind in the discussions we have just begun to have.
First of all, how will the members of the Board and the Partners'
Council be appointed and what will their respective roles be?
(Professor Sir Michael Rawlins) It is envisaged in
the Establishment Order that there will be seven non-executives
and four executives. The seven non-executives would be appointed
by the Secretary of State, the chief executive would be appointed
by the Secretary of State, the other executives would be appointed
by the NICE Board. The Partners' Council again would be appointed
by the Secretary of State, and in fact already a large number
of letters have gone out to various organisations proposing individuals,
so those names will come up from a wide range of organisations
concerned with health care, not just doctors, nurses but other
organisations too which have an interest in health care.
50. How do you see that relationship between
the Board and the Partners' Council and how serious will the relationship
be or will it be a tokenistic one?
(Professor Sir Michael Rawlins) I very much intend
it should not be tokenistic. It should not be like an annual shareholders'
meeting once a year. I think the Establishment Order does propose
that it should meet once a year to receive the annual report.
If that is all it does, to be honest with you I think it needs
to be more than that because it could be very, very useful and
very important. Not only do I hope that it will comment on what
NICE has done in the past but also I hope it will comment on future
plans. I very much hope that it will be a sounding board for ideas
that have not fully crystallised. I very much hope that individuals
in it will be engaged in NICE and help us on individual issues,
for example like the patient associations that Mrs Wise was talking
about earlier. I think there is a big role for the Partners' Council.
I think the constructive criticism from the Council to NICE is
also very important too. I expect a dialogue, I expect it to meet
more often than once a year and I expect it to do much more in
reality than merely review our annual report.
Chairman
51. You are due to come into effect this spring,
how soon do you anticipate you can really get into your remit
and what are the early issues that you feel you will be addressing?
(Professor Sir Michael Rawlins) We will come into
being on 1 April and I would hope that some of our early outputs
would materialise between four to six months. It will take that
amount of time. We have no premises, we have no bank account.
We need to create some sort of infrastructure to support it. That
will all come into being on 1 April. The advertisements for the
non-executive directors have already been placed in the press
and there was a massive response. There were over 2,500 interests
and there have been over 500 applications. The advertisement for
the chief executive has also been placed in the press and there
have been hundreds of telephone calls and letters of interest
but I think it is a bit early to know how many applications.
(Dr Riley) It is certainly heading towards three figures.
(Professor Sir Michael Rawlins) We are getting on
with the process but it will take time. I hope you will accept
that I cannot do miracles.
52. You must have in your own mind some early
agenda other than setting up the fabric of the organisation.
(Professor Sir Michael Rawlins) Quite clearly we need
to get on with the appraisal process particularly of the most
urgent things with the Department and we need to start producing
the guidelines. In a way the most important thing is for us to
gain the confidence of the professions, of the public, of Parliament,
of ministers, of the Department as a whole. If we can do that
and do that early on then I think that is the most important thing
to do. We will do that by the quality of the technical scientific
work we do, by being transparent in so far as is humanly possible
and in making sure that we can consult and disseminate the output
as widely as possible.
Julia Drown
53. Can I just go back to the appointments.
I noticed in the consultation paper where you are talking about
the appraisal group, that one of the things you are asking for
views on is whether the members of the appraisal group should
be kept anonymous to protect the integrity of the group. That
would rather go against the goals of openness and transparency
that you talked about earlier. Could you just say a bit more about
why you think it might be justified to keep the members of the
appraisal group anonymous?
(Professor Sir Michael Rawlins) I think there are
two sides to it. On the one hand, if they are known then they
might get all sorts of people ringing them up and contacting them
morning, noon and night. I think that is perhaps one reason why
it has been put that way. On the other hand, I am a member of
the Committee on Safety of Medicines and only once have I ever
been contacted by a member of the public and it happened many
years ago. That was once in 20 years. I do not think that this
is too much of an issue. I think it is for consultation and discussion.
Mr Austin
54. Both the pharmaceutical industries and the
health care industries have given a cautious welcome to NICE.
I would be interested to know what discussions you are having
with them about some of their reservations and how you are going
to prevent NICE from becoming a bureaucratic barrier to patients
benefiting from new medicines and new technologies?
(Professor Sir Michael Rawlins) There are several
points here. First of all, I very much hope that we do not become
a bureaucratic barrier otherwise we would impede patients benefiting
from new developments. I think by the process that Dr Radford
described earlier, horizon scanning and starting the process well
in advance of a new intervention being launched should assist
that enormously. It is very important for the Institute to develop
good relations with the pharmaceutical industry. I say this very
genuinely and I have said it to them in person. First of all,
they are the people who invent new technologies, academics like
myself do not for practical purposes. Not only that but they are
extremely good at developing them. As someone who has sat on the
CSM for 20 years I know that pharmaceutical companies can deliver
effective clinical trials much, much more effectively and rapidly
and rigorously than academics can. In the CSM my heart always
used to sink when it was a new drug that had been developed by
an academic body, usually in the United States, who made a complete
mess of the whole thing and you never knew where you were. We
need to make sure that that expertise in the industry is harnessed
together with the sorts of research questions that NICE will want
for the future and at present. We will need to engage the industry
in how to ensure that we can be reasonably satisfied that new
interventions are both clinically effective and cost-effective.
Industry does not really object to that, it realises that it cannot
just assume a market and income without having to demonstrate
that you are getting value for money. We also need to join in
with industry in the further research that may be necessary, or
at least the R&D but proposed by NICE, in order to ensure
that the maximum benefits are gained.
55. Can I pursue that because Howard Stoate
earlier asked the question as to whether it is possible to make
truly evidence based decisions at the time of launch. You are
now talking about cost-effectiveness. One of the issues that the
pharmaceutical industry have said is that the introduction of
cost-effectiveness trials is inevitably going to lead to an increase
in the cost of the drug. They have also pointed out the difficulty
in the licensing arrangements where they have to show the safety
and the efficacy of the drug compared with other things that are
on the market for treating that condition. If you are then going
to require evidence about cost-effectiveness how easy is it to
do that based on clinical trials rather than on the naturalistic
use of the product?
(Professor Sir Michael Rawlins) You can get a lot
of information from clinical trials, particularly if economic
appraisal is taken at the same time and particularly if you develop
quality of life measures. You may have to resort to some sort
of modelling and that actually is remarkably effective. As you
know, doctors have been treating high blood pressure for 35/40/45
years and all we knew for many years was that it just lowered
the blood pressure and we assumed that was a good thing. It was
not until the 1970s, 20 years ago, that we had real good evidence
not only that the lowering of blood pressure with drugs was actually
lowering blood pressure but also preventing myocardial infarction
and stroke and so on. You can actually model that, you can see
what happens if you lower blood pressure, what is likely to be
the clinical outcome and for a time one may have to do that and
one's cost-effectiveness when you are using a surrogate marker
may have to be based on modelling of what you know of what happens
because, as you say, it could take many years. In the case of
hypertension it took 20 years before you knew you were getting
real clinical benefit in terms of reduction of myocardial infarction
and stroke.
56. It is only at the end of the day by the
use of medicines?
(Professor Sir Michael Rawlins) Years and years out,
yes.
57. One of the points that one of the pharmaceutical
companies has made in the evidence that I would like your comment
on is: "It is not possible to undertake Naturalistic Studies
prior to launch and indeed clinical trials cannot easily be used
or changed to generate naturalistic clinical data pre-launch."
They go on to say: "Ethics Committees regard Naturalistic
Trials on individual products as unethical." Do you see a
problem there?
(Professor Sir Michael Rawlins) No, I would not really
accept that. I do not think they are talking about naturalistic
trials, I think they are talking about giving it to some people
and seeing what happens to them. That is not the same thing at
all and you do not actually get decent, good economic evaluation
out of that, you have to do it in the context of one group receiving
one treatment and another group receiving another treatment and
random allocations to them. Otherwise, you really do not know
where you are, it just does not work like that.
58. Can I then turn away from medicines to devices
and other technologies? One of the points which the Association
of British Health Care Industries has made is that the appraisal
process does not recognise the full complexity of introducing
health technologies. What they said in their evidence is "it
is not until users have had experience of a new technology, and
seen how others have been able to use it, that best practice can
be developed: this process may take some years before the ultimate
benefits are realised."
(Professor Sir Michael Rawlins) Yes, we recognise
there is a particular problem with devices often made by relatively
small sponsors, manufacturers, and of course the regulation of
devices is not quite the same as it is of medicines and it is
really safety which is being looked for. Nevertheless, I think
that it is not unreasonable for the National Health Service to
expect some sort of return in terms of benefit on its investment
when it purchases devices, and if a device seems like a good idea
then that is the moment perhaps when the NHS engages through its
R&D programme with the sponsor to try and evaluate whether
it really is of benefit or not. So there is a special problem
with devices and I think there perhaps needs to be more debate
and discussion between the research and development part of the
NHS and device manufacturers. Tim, I know this is an area you
have been worrying about.
(Dr Riley) Yes. I think it is worth saying that fortunately
we are not starting from a completely blank sheet, there has been
a history within the Department of Health of supporting evaluations,
in practice using protocol-driven research on various devices,
particularly devices from small companies. Indeed there have been
examples of that in terms of ventricular assistance devices and
so on. So we have in fact got quite a foundation on which to start
up a productive debate with the sponsors of those devices to actually
ensure there is protocol-driven research we can put in place and
bring alongside that economic evaluation informing the deliberations
of NICE.
59. But in order to carry out your appraisals
you are going to need data and information from the manufacturer.
Ultimately you may have to decide, or you may decide, that it
is a product which should not be available on the NHS. Bearing
that in mind how co-operative do you expect the manufacturers
to be and would you therefore see any need to introduce legislative
measures to help you obtain their co-operation if it were not
forthcoming?
(Professor Sir Michael Rawlins) I can understand they
would be disappointed, probably more than disappointed, but if
NICE had determined that it was not effective or not cost effective
and had given its advice to health authorities that that was the
case, then I do not think health authorities would take it up.
So I would not have thought there was a need for legislation.
(Dr Radford) Could I just check the question you are
asking? You are talking about the industry in its totality and
getting information from industry at an early stage to help inform
the appraisal process?
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