Examination of Witnesses (Questions 240
WEDNESDAY 23 JANUARY 2002
240. May I just ask a question about Herceptin
because it is something I have been following fairly closely?
Correct me if I am wrong, but my understanding was that when the
Herceptin submission was put in, NICE were asked to look at two
things: Herceptin by itself and Herceptin in conjunction with
another drug. My understanding is that one of thoseand
I cannot for the life of me remember which way it isis
sitting there ready to go, the guidance is practically written,
but NICE will not release it because the other guidance is not
ready. What possible excuse could there be for delaying the guidance
that they are happy with?
(Mr Campbell-Morrison) That is my understanding as
well. As a fellow pharmacist, I defer to you because I know you
have quite a lot of knowledge about Herceptin. I do not have any
explanations. It does not seem rational to me to delay guidance
when you have it ready.
(Dr Jones) This is one of those very difficult situations.
This is a drug where there is incontrovertible clinical data that
it works. What happens is that there are many ways of treating
cancers: surgery, radiation and drugs. In these 25 per cent of
women who have over-expressed this particular gene, if you add
Herceptin on to a current treatment, particularly the taxanes,
you get dramatic extra benefit in terms of quality and length
of life. It is not a Beta-interferon situation where there are
different interpretations of the clinical potential of it: there
is incontrovertible data. There is a price on it. Many other countries
who have already licensed it are seeing the advantage of it coming
through to their patients. Is it then about the affordability
of this drug to the Health Service? If the answer is yes, that
to me is a political decision about whether this is a priority.
If it is about whether it is cost effective, why do we not give
the patient the benefit of the doubt whilst that is assessed in
the real practice of patients with this condition?
241. Why has it been referred to NICE? What
is the reason given for this?
(Dr Jones) I presume from the witnesses we heard before
our presence here, that it genuinely is the concern we all share
of how on earth you make decisions on priority of spend.
242. The reason I am pursuing this line is that
this is a particularly unusual case and the ones we argue about
nearly always are. How do we know for certain that a drug is performing
better than other existing treatments unless you have some procedure
like the NICE procedure. I know this one is unusual because I
have been to a demonstration and spoken to clinicians about it.
The reason for the whole process is to try to make sure not just
that something is better than nothing, but that it is better than
existing treatments with other competing drugs.
(Dr Jones) That is the role the Government has given
to the Medicines Control Agency, to ask whether a drug does work,
whether it is effective, safe and the right quality. This drug
can only be compared in a situation where you are showing the
extra benefit for these patients with this unique genetic expression.
The role of NICE is not to say whether it works or is going to
be effective. That is proven without doubt.
243. In this one case.
(Dr Jones) No, it is incontrovertible. The question
is whether the Health Service can see this as a cost effective
way of treating this particular sub-set who have this condition
and the oncologists would say it works. I think it is a clear
(Dr Patterson) The Department of Health has taken
a stand, through a thing called TAG, technology access group,
which sits almost entirely within the Department and has few or
no outsiders on it, that they are going to go with all new medicines
at the time of approval and try to do a NICE assessment. With
something like this where there is incontrovertible evidence,
it should be relatively simple, but it still takes one to two
years during which time nobody is prepared to pay for it to be
given to patients. Consequently and you have this problem you
have heard described. With many other cancer medicines, you have
evidence maybe of response of the disease but not comparative
data and maybe not survival data. Five years' survival data takes
five years. The issue there is that you cannot do a definitive
assessment at launch.
Dr Naysmith: I understand what you are saying.
My main point is to make it absolutely clear that here we are
picking incontrovertible evidence that it works and I do not want
it to be assumed that for every drug which comes up before NICE
it is the same situation.
244. One more question on Herceptin before we
move on slightly. I have also heard, I do not know whether you
have any evidence that this is the case or not the case, that
although there is a test available to determine whether someone
would benefit from this treatment they are not even having the
test because that raises questions of putting a moral responsibility
on somebody to provide and fund.
(Mr Campbell-Morrison) That is something which has
a lot of moral implications. We have heard public health doctors
taking advice from lawyers. If a woman goes in and has a breast
tumour it is very traumatic. The doctor is going to perform a
biopsy. Whether she is treated or not with Herceptinand
it would obviously be better, if that were indicated, for it to
be treated if she has those markersshe is entitled to know
whether she has those markers because that indicates a more aggressive
disease. It is outrageous that people are not allowed to know
because physicians are scared about being put in this position
that you have this market, there is this drug which can treat
your condition, we are not allowed to offer it, that is a terrible
position to be in. I think that woman should have the right to
know that she has an aggressive tumour even if she is not being
treated, because then she can plan her life. She can make disposition
of property, what happens to children, whether she is going to
carry on working. I think that women with breast cancer in the
UK need to know that.
245. With this drug, although it is new to Britain,
is it being used in other countries around the world as a perfectly
acceptable, tested and considered safe drug?
(Dr Patterson) I am not an expert on Herceptin, but
I can tell you the answer is yes; recently introduced in many
other countries and no drug is absolutely safe. It was licensed
around the world around the same time over the last two or three
246. It is being used in other parts of the
(Dr Patterson) Yes, as part of the standard therapy.
247. And we are waiting for NICE to give their
appraisal which has been delayed by six months so far.
(Dr Jones) It is approved for use here in the UK by
the Government authorities.
248. The other question is on the numbers involved.
Mr McKinney, you mentioned a cohort of 5,000 and then you said
that it has been shown through testing that about 25 per cent
would benefit from the use of this drug. Is that right?
(Mr Fullagar) Different numbers. The total population
is higher than that. The company's estimate is that those who
would come into consideration for treatment are 5,000, that is
the proportion of the total breast cancer patients who would potentially
benefit from this treatment
249.would be about 5,000 per year.
(Mr Fullagar) Would be about 5,000 per year.
250. On the basis of the rough ballpark figure
of the cost per year for this drug, it has been worked out by
someone other than myself that it would cost about £7.5 million
a year which would extend and improve the quality of life of about
5,000 people. So we are talking possibly relatively small amounts
of money compared to the beneficial effects it would have on 5,000
(Mr Campbell-Morrison) Yes.
(Mr McKinney) Yes.
251. This sounds such a strong case; words such
as "incontrovertible" and "evidence" have
been used. This is medically safe as far as it is possible to
be. On a case as blatant as this has nobody gone to NICE to ask
them what on earth they think they are doing?
(Dr Jones) With respect, NICE have been given a task.
It is not NICE's judgement.
252. No, the words "inexplicably delayed"
have been used in the evidence. Has anybody asked for that explanation?
(Dr Jones) The process is that NICE is asked to make
an appraisal. There must be a time when it has to gather the data,
has to bring that to a committee, has to look at that data and
make a decision. It could say it did not need to do all that,
the evidence is incontrovertible that it works, off you go. That
is not an appraisal.
253. That is not my question. Has anybody sought
to ask why it has been delayed six months?
(Dr Jones) The question to ask, if I may say, is why
in the first place this process was raised. If you have breast
cancer or care for someone with breast cancer, what you want to
know is the best way to treat the condition and the guideline
in that direction, for example the Scottish SIGN approach or Royal
Colleges, is a valid way forward. NICE were asked, by a process
which is pretty intransparent, to look at this one and they are
doing their job. I do not think we ought to blame NICE for that.
NICE gets a lot of criticism when actually it is doing a pretty
good job in many ways. It has faults but the process which oversees
the selection and the timing of its work and the implementation
of its guidance are really the issues which are in front of us.
254. Whose fault is it if it is not NICE's?
Or is it no-one's fault but just the system?
(Dr Jones) Further to the issues we heard earlier,
before this blight which is occurring because of the issues of
appraisal, the Health Service managers and the doctors within
the Service made decisions on a case by case basis with the patient
in front of them as to whether it was relevant. I believe Herceptin
would be being used right now in a much greater preponderanceNICE
are not saying do not use itit would be being used much
more if the system we had before had been in place. In other words
people would have used their judgement to make decisions about
the patients in their own patch with their own costs and affordabilities.
255. A lot of health authorities who are ultimately
responsible for the funding of health care at the moment at local
levels will saytake beta-interferon for examplethat
they will not fund it because they are awaiting the NICE appraisal.
So it is not really fair for you to say that people can use this
and health authorities can fund it, because I will bet your bottom
dollar, given the pressures on Health Service budgets throughout
the country, that if a health authority can avoid it, particularly
if they can find someone else to blame for taking that decision,
they will say they are very sorry but they cannot afford this
sort of level of expenditure because NICE has not done its appraisal
yet. It just is not realistic out there in the Health Service
today to say that health authorities should be providing the funding
because they will hide behind the NICE appraisal or the awaiting
(Mr McKinney) We have been very critical of the way
that whole process has worked. We had a statement from Lord Hunt
in the House of Lords, we have also had the Health Service circular
two years ago to say that health authorities must not or need
not wait for NICE guidance before funding drug treatments. But
I think you are right, this is the reality of what is happening
up and down the country. They will not do it. This is one aspect
of the two prongs of NICE blight. We have been very critical of
health authorities. In fact we wrote to the Chairman of every
health authority on colorectal drugs and other general cancer
drugs to remind them of their duties and responsibilities to allow
the clinicians to prescribe these drugs. No change there. Not
256. I have some questions which arise out of
the GlaxoSmithKline evidence. Further to one of the things which
is said in itand given the exchange which has gone on already
once I have asked you this question we may decide there is no
role left for NICEwhy do you think that NICE should not
be involved in assessing affordability of technologies, drugs
and so on?
(Mr Gray) We would concur with the answer we heard
earlier. Clearly different bodies have been established to ask
separate questions, MCA for safety and efficacy. Clearly there
is a brief for NICE to ascertain cost effectiveness through individual
appraisals. In a situation where NICE deems any particular medicine
or other technology to be cost effective for the system, as we
heard very eloquently from the last set of witnesses, that sets
up a whole set of funding questions and challenges. Do they have
enough total funding to absorb this recommendation? Do they have
to shift funding from elsewhere to cover the recommendation from
257. Do you think NICE should not be involved
in that sort of thing?
(Mr Gray) We think those issues of funding are strictly
the responsibility of the Minister.
258. What is NICE's role? We have heard some
critical comments from the other end of the table. What do you
think NICE should be doing?
(Mr Gray) We would concur with NICE's goals absolutely.
It was set up with goals to try to address quality assessment
either through guidelines or assessment of appraisal of medicines
and technology, to try to do something about what was called post-code
prescribing and to try to foster faster access to treatments.
What we have heard from the evidence today, and certainly it is
our experience, is that there is not a great deal of progress
towards those goals. From our point of view, we feel that the
concentration on appraisal as opposed to guideline may well have
contributed to that. Going forward, looking to put greater emphasis
on setting quality standards through guidelines within which local
clinicians and local authorities can start to address decisions
to achieve those best practice guidelines is more likely to deliver
the result that NICE was set out to do.
259. Quality was one of the things you were
critical about in your submission. You mention the Appraisal Committee
and say it does not have the appropriate calibre with regard to
clinical expertise in evidence assessment and the appraisal process.
You are saying it is not up to scratch basically.
(Mr Gray) No, I think we may have made a slight error
in the written evidence. It is not the Appraisal Committee; that
seems very high standard. Where we do see problems is a great
deal of variability in the assessment groups and the original
assessments they provide for the Appraisal Committee to consider.
To be fair, NICE themselves acknowledged that at recent board
meetings. They have themselves been talking about the difficulties
in recruiting enough high calibre individuals in those centres
to do these assessments. Certainly obviously we know nothing about
the recruitment issue, but where we notice it is in the variability
of those assessments and indeed a rather high percentage of factual
errors in those assessments which are obviously indicative to
us of the problem which NICE have recognised.