Examination of Witnesses (Questions 602
- 619)
THURSDAY 18 OCTOBER 2007
MR STEVE
WINYARD AND
DR RAFIQ
HASAN
Q602 Chairman: Welcome and thank
you for coming here to take part in the fifth evidence session
in our inquiry into NICE. Perhaps for the record you would give
us your names and the positions that you hold.
Dr Hasan: My name is Rafiq Hasan
and I am Director of Market Access at Novartis Pharmaceuticals
in the UK.
Mr Winyard: I am Steve Winyard,
Head of Policy and Campaigns at RNIB.
Q603 Chairman: We said to a previous
witness this morning that we tend to meet you on occasions in
Parliament. Whilst there is no direct financial interest we meet
you via back-bench committees and other ways to talk about the
issues that we intend to discuss today. I am not sure whether
that is a declaration of interest on my part, but I think I should
say that. Industry complains that in the UK the take-up of new
drugs is slow and variable and policymakers complain that clinical
guidelines are often ignored by practitioners. How can that problem
be mitigated?
Dr Hasan: As you have already
heard this morning, there is quite a lot of variation across the
country in terms of PCT uptake of new technologies. We heard this
morning about exceptional case committees that often put in place
what we see as hurdles and barriers in the way of patients getting
access to new medicines. If one takes the case of Lucentis, a
drug that is currently being appraised for wet AMD, the clinical
evidence suggests that over 70% of patients experience an improvement
in vision. Before that drug was available these patients had virtually
no other effective treatments that could have such a significant
impact. What we see is the whole concept of NICE blight before
NICE has made a decision. Once NICE comes out with some final
guidance we see slowness in uptake and implementation.
Q604 Chairman: We may want to cover
that a little later. Mr Winyard, do you have any general comment?
Mr Winyard: RNIB's involvement
in the NICE process is fairly limited. We have engaged in only
two appraisals and my comments will be based largely upon that
experience. Involvement in the work of NICE, however, is very
important. One of our royal charter objectives is the prevention
of blindness. We know that sight is precious to people. If you
ask your constituents nine out of 10 will say that sight is the
sense that they most fear losing. For a very long time there were
no treatments for wet AMD and that accounts for about half of
all new blindness registrations in the UK, so it is the big cause
of blindness. For RNIB, when new approved safe treatments have
become available in the past two years our trustees have tasked
us with making sure that they become available on the National
Health Service. That is why we are involved. The other general
point to make is that our primary relationship in all of this
is with the Royal College of Ophthalmologists and in a sense it
would be great to have the college here because it has the expertise
on these issues. The president of that college is on our policy
and advocacy committee which lays down what our policy will be
on different treatments. In terms of access, obviously there is
an enormous problem between the time a drug gets EMEA—European—approval
and getting NICE guidance. At the moment we are right in the middle
of that. Amongst the members of the Committee, we note that in
some areas—in yours, Chairman—patients will be treated
but in others they will be turned away. For example, in Staffordshire
there appears to be a blanket ban on treatment, and in many areas
patients have to go blind in one eye before the Primary Care Trust
will consider treating the second. This is fundamentally wrong
and we have to find a way round it. In the short term, together
with the Macular Disease Society we have established an advocacy
service funded by Novartis and Pfizer which supports patients
who contact us via our help lines and say they are going blind;
they have paid into the NHS all their lives and the first time
they draw on it they are turned away. They are told that they
cannot have funding. In those cases we support patients and engage
with the Primary Care Trust; and we also engage with MPs. We are
trying to apply sufficient pressure to ensure that patients get
treated. It is not an ideal situation but it is one that we face.
It all comes back to the prevention of blindness which is our
royal charter objective.
Q605 Chairman: If I may ask specifically
about patchiness in terms of the implementation of guidance, what
do your members think about it? Yours is a membership-based organisation.
Mr Winyard: We are indeed. There
is enormous anger and frustration that there is such wide variation.
If you are fortunate to live just over the border in Cheshire
and Merseyside both eyes will be treated, whereas if you are in
Staffordshire you will be turned away. There is anger, frustration
and a sense of real unfairness. That has been reflected in the
very high level of media interest when local and national cases
emerge.
Q606 Dr Naysmith: Dr Hasan, you suggest
in your written evidence that the quality of work carried out
by some of those who produce assessment reports and others who
may review the evidence is both questionable and variable. That
is quite a serious accusation. Can you give us some examples?
Dr Hasan: I think it comes back
to the whole discipline of health economics which is embryonic
relative to clinical medicine, randomised controlled trials and
evidence-based medicine. When we assess health economic outputs
we must understand that there is a high degree of uncertainty.
The point we made in our written submission was that when NICE
commissioned independent assessment groups to run some of that
evidence generation there was a lot of variability in the quality
and standards between different evidence groups. Often there are
errors in those models and the reports generated. Subsequently,
addenda must be issued. Sometimes one questions the assumptions
that go into some of the models. We would welcome a broader debate
about some of the assumptions that go into those models and the
emphasis placed on the health economics and the concept of cost
per QALY in the appraisal committee decision-making process.
Q607 Dr Naysmith: One of the things
NICE may say in response is that often there are lots of mistakes,
even arithmetical errors, in the submissions made to it by drug
firms. Is it not a fact that in this area some mistakes are made?
You still have not given me any examples of what you mean.
Dr Hasan: I can give you the example
of the Alzheimer's disease appraisal. One assumption was that
30% of the costs of institutionalisation was not borne by the
NHS. That may be based on some evidence that perhaps is not as
current as we would expect. We know that some of the costs put
into the economic models were perhaps a little out of date. Where
one has the opportunity to have those discussions one can identify
some differences between what maybe we would put into a model
versus what one of the assessment groups would do.
Q608 Dr Naysmith: You argue that
the conflicts between yourselves and NICE might be reduced if
there was a clear rationale for how NICE reached its decisions
if it was provided regularly. How much explanation do you believe
is given at the moment either in private or public, and how would
you like to see it made more explicit?
Dr Hasan: We would like to see
some form of debate around how important the health economics
are in the decision-making. We see an over-reliance on cost per
QALY. I know that Professor Stirling Bryan has presented himself
before the Committee. Only last year he published some research
he had done into the NICE decision-making process. I almost reiterate
some of the evidence you heard earlier today. There was a feeling
among clinical experts and patient representatives on the NICE
committee that they were sometimes out of their depth when there
was a discussion about health economics. There was one quote in
one of the papers about some of the clinical experts. You can
see the way they are thinking. They start with the cost per QALY
and then work backwards. The question we ask is: is that the right
way to make a full and broad assessment of the clinical benefits
of a particular technology that is delivered as well as the patient
experience? Another example is Professor Anthony Goldstone who
presented himself at one of the NICE appeals. Subsequently he
wrote a letter to The Times in which he said he felt put
down, that his opinions were not heard and that 30 years' experience
treating cancer patients was largely ignored. He believed that
decisions were being made by people who had never seen a case
in their lives. We believe that there is much scope for improvement
in terms of a broader debate on the real decisions that are being
driven within that process. How much weight is being given to
health economics? Surely, there must be some weight attached to
that, but what weight is being given to clinical and patient experience?
Q609 Dr Taylor: Therefore, you agree
with the previous witnesses that the right experts are not necessarily
used by NICE?
Dr Hasan: Absolutely. One other
point that is highlighted is: have they had the right training
in health economics to be able actively to debate it?
Q610 Dr Taylor: Last week some of
our expert witnesses criticised the quality of data that some
pharmaceutical companies passed on to NICE: it could be incomplete,
partisan or of poor quality. What is your response to that? Is
that fair, or do you believe that the information you pass on
is always absolutely superb?
Dr Hasan: I can speak only on
behalf of Novartis. We believe that our submissions are of very
high quality and we have a track record to demonstrate that.
Q611 Dr Taylor: Would it make a difference
to pharmaceutical companies, not necessarily your company, if
the eventual price of drugs reflected the quality of the evidence
given?
Dr Hasan: We are certainly strong
believers that the price should be reflected in the value that
a particular medicine delivers. We believe that through these
types of appraisal processes we are able to demonstrate the value
of our medicines.
Q612 Dr Taylor: NICE seems to be
more influenced by quantity rather than quality of life. Mr Winyard,
do you think NICE has underestimated quality issues particularly
for people who are at risk of losing their sight?
Mr Winyard: Without a doubt. I
am not an expert on QALYs and health economics, but the system
does have a bias towards quantity of life rather than quality
issues. In the two appraisals in which we have been involved what
has been frustrating is that they have been some of the longest
in the history of NICE and each time NICE has first come out with
its consultation document it has got it disastrously wrong. In
the first case, in relation to PDT we would have gone to appeal
but NICE withdrew the ACD because there was such an enormous outcry
over a policy that said a person had to go blind in one eye before
he or she could be considered for treatment. We went into the
process this time for the anti-VEGF treatments full of hope. What
happens? It does exactly the same thing. Of course, there is an
enormous outcry. No one supports this; it is wrong and against
good clinical practice. You will not find a single ophthalmologist
who says that you should first let the patient lose sight in one
eye. If you let that happen you do not know whether or not you
can save sight in the second eye. NICE has got it disastrously
wrong. The only thing the appraisal committee seems to be interested
in is cost per QALY. It says in the literature that it is interested
in the impact of the condition on people's lives; it wants to
know what it is like to be blind. What would be the impact on
the quality of life if one could keep one's sight? We present
that evidence and there is absolutely no indication that it has
been taken into account in any way. It is frustrating. Inevitably,
that leads us to reflect the anger and frustration of our members
and the wider society and then it is a very high profile public
debate which is not ideal. I can see problems with that.
Q613 Dr Taylor: Does industry provide
evidence on quality of life issues, or is that outside your remit?
Dr Hasan: We submit an economic
case for all our medicines that captures the quality of life benefits,
as well as the quantitative benefits, of those technologies, so
we submit a cost per QALY model based on the evidence generated
in our randomised controlled trials.
Q614 Dr Taylor: Is there hard evidence
that elderly people, for example, value quality rather than length
of survival?
Dr Hasan: As part of the process
of generating that evidence often we undertake the right type
of research to elicit what is called utilities, that is, a quality
of life measure. NICE's own reference case suggests that that
is done with members of the public. Therefore, members of the
public are asked to quantify how much they value sight versus
other things. That will capture those quality of life benefits.
One point I should like to pick up from the RNIB is that if, for
example, NICE's final decision is that an individual should be
treated only for the better seeing eye, has it captured the impact
on that patient having to be told by his or her doctor that treatment
cannot be offered and the patient must return when the other eye
is affected by the disease? What about the detrimental impact
that has on the quality of life of that patient? These are the
types of debates that we believe should take place at the appraisal
committee so that there is a broadly informed understanding of
how a decision is reached.
Q615 Dr Taylor: It is fearfully difficult,
is it not? Do you believe that NICE takes any account of non-health
costs?
Dr Hasan: Part of its remit is
to focus very much on NHS costs only. Wet AMD and blindness is
a very good case in point in that there are broader societal costs
that should be taken into account. The challenge that we always
get back from Sir Michael Rawlins is that he needs almost statutory
permission to be able to go down that road and capture the broader
costs. We know that in Sweden, for example, the LFN, which is
almost equivalent to NICE, does capture broader societal costs.
It does an economic evaluation taking into account benefits in
terms of getting people back to work and off benefits. We believe
that that is an area where that debate should be included.
Mr Winyard: There are two recent
cross-national studies of the impact of sight loss and AMD on
quality of life: one by Professor Giselle Soubrane and one by
Professor Frank Holtz. Both demonstrate the major impact of AMD
and sight loss on quality of life. As to the whole professionalisation
of the assessment process, there does not appear to be an acceptance
that QALYs are not scientific. There is a claim that they are
scientific, but clearly they embody value judgments to a very
great extent. Whom do you ask? You want to measure the importance
of sight to quality of life. Does one ask the sighted public,
or people who are in the process of losing their sight, or do
you ask those who lost their sight five years ago, for example?
You will get different answers from those different groups. Which
questions does one ask? Do you use a generic questionnaire, the
EQ-5D which is very limited, or something much more specific to
AMD like the MacDQoL that has been developed by Professor Clare
Bradley at London University which captures much better the full
impact of AMD? If one goes in the direction of specific measures
it becomes much harder to compare different disease areas. Basically,
there needs to be a recognition that QALYs are not scientific
and lead the whole assessment process in the direction of great
complexity, and it is excluding. This week we have sat down with
the Royal College of Ophthalmologists to look at the additional
data provided by NICE following the very negative response to
the appraisal consultation document. Clinical experts—the
leading retinal specialists—and the patient group cannot
understand it. We cannot make sense of the data that has been
provided because it is in a very special language. One probably
needs a master's degree in health economics before one can begin
to make sense of it. It cannot be right that it is such an excluding
process.
Q616 Dr Taylor: Do you have any suggestions
for an alternative?
Mr Winyard: In a sense, it is
there already. In the Secretary of State's Directions to NICE
it is pretty clear that wider things should be taken into account:
the broad balance of benefit and cost and the degree of clinical
need of patients. If the appraisal committee can take rather better
account of those things QALYs will be part of that process, but
there is a wider picture and in the end you have to make some
tough decisions. Is sight important? Everything tells me that
it is. Your constituents will tell you that it is terribly important,
and that must be captured.
Q617 Dr Taylor: How do you balance
it against survival of an extra few months with a cancer drug?
Mr Winyard: I am not an expert
on running health systems. I agree that that is a problem. Obviously,
in one part of America the public has been asked about pretty
well every sort of treatment and ranked them. Fortunately, that
is not my problem. You need a system that recognises that there
is no science that will deliver precise answers to these questions.
One needs to take account of QALYs but in the end one must recognise
what is important and what is less important.
Dr Hasan: I think there needs
to be recognition that the QALY is an estimate. With most things
there are wide confidence intervals around that estimate, but
added to that it is almost a prediction. We are doing economic
modelling sometimes with time horizons of 30 or 40 years. We are
saying that what we know today will be replicated in 40 years'
time. We also know that there are slight changes in assumptions
which may be based on differences in clinical opinion. Therefore,
one group of experts will say that a particular assumption is
correct but another may have a completely opposite opinion. That
can change the cost per QALY significantly. It could take it above
the perceived threshold or even down to a negative value, ie that
product dominates other products. There should be recognition
of the strengths and weaknesses of QALYs. We suggest that part
of the appraisal committee process is to say that in this disease
area when assessing the health economic benefit, quality of life
and cost per QALY these are the strengths of the assessment and
these are the weaknesses. Let us have that debate and see whether
we can come to some agreement.
Q618 Mr Scott: Do you believe that
lower drug prices can be the answer to this problem?
Dr Hasan: I think we need more
clarity as to the cost per QALY versus the price of the drug.
You can have a very cheap drug that may not be very effective
but has a very high cost per QALY because it is driven by the
effectiveness of the product, whereas you may have what you perceive
to be a high priced product that delivers significant benefit.
An example, which I am happy to discuss, is that the SMC has already
appraised Lucentis and in one scenario within the economic modelling
the cost per QALY of that drug versus Visudyne being used with
photodynamic therapy is about £5,000 per QALY. Therefore,
at face value one may say it is a high priced product but when
the economic modelling is done one sees that it delivers a significant
amount of value and is seen as very cost-effective. On that basis
it was approved by the SMC in June.
Q619 Mr Scott: Would not another
option be a high quality product but a slightly lower profit margin?
Dr Hasan: One can debate profit
margins versus price but that is probably a slightly different
discussion. If one asks how to quantify that value one can use
things like cost per QALY to show that a lot of products represent
good value for money for the NHS.
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