APPENDIX 1
Memorandum by Dr Andrew Bamji (NC 8)
SUMMARY
I do not believe that NICE presently offers
a fully credible assessment system for new drugs and health technologies:
1. The internal evidence on which it bases
judgements is open to question.
2. It cannot respond in a timely way to
new evidence.
3. Its cost-effectiveness judgements are
arbitrary and open to challenge.
4. There remains the potential for drugs
and procedures to be refused on cost grounds alone, even when
the evidence of clinical benefit is overwhelming.
5. Refusal may relate to population rather
than individual cost, which is unfair to individuals.
6. The process of submission from interested
parties is time-consuming and costly.
7. The appeal system causes delays.
8. It does not enable by default the prescription
of new remedies in a system of cost competition and limited budgets.
9. Its decisions have been questioned (and
modified) outside the standard system of evidence-based medicine.
10. NICE has no parallel within the European
Union, and thus decisions it makes may be at odds with decisions
of other EU countries and could potentially be challenged in the
European Court under Human Rights legislation. In the current
circumstances I believe that equity in prescribing will not be
achieved unless NICE or some equivalent body has pan-European
powers.
11. If NICE suggests that longitudinal data
must be collected to inform a decision, but then indicates that
it will not support the introduction of a drug or procedure until
such data is available, then post-marketing surveillance will
die in the UK.
12. If NICE proscribes a drug or procedure
which is later, by virtue of such evidence, found to be appropriate
one has to ask whether patients denied treatment would have a
case for legal redress. This is particularly likely if that drug
is clearly effective clinically, but has been turned down on cost
grounds alone. The costs of any judicial review, class action
and/or damages could be substantial.
13. If a decision from NICE is agreed by
all parties (clinicians, patients and government) then there is
no problem. In the case of a refusal the decision needs only to
be questioned by one of the parties for trouble to ensue. As I
have signed a confidentiality agreement I cannot reveal details
of NICE advice in draft to which I am privy, but the ß-interferon
saga is sufficient evidence; even though the clinical evidence
of benefit is weak, patient pressure has forced a change of attitude
by government which effectively overturns the NICE ruling. If
this can happen then NICE's position as an independent body is
fatally compromised.
Neither do I consider that there is any need
for NICE to develop clinical guidelines because other national
bodies (such as the Royal Colleges, specialty societies and other
professional and patient organisations) have shown that they can
produce robust guidelines. Any produced by NICE are likely to
duplicate effort
1. INTRODUCTION
1.1 I am grateful for the opportunity to
submit a written statement to the Select Committee. I do so as
an individual.
1.2 I am a consultant in rheumatology and
rehabilitation in a District General Hospital within the London
Region. I have a busy practice in the former (I have over 420
patients with Rheumatoid arthritis under regular follow-up) and
on the Elmstead Rehabilitation Unit I supervise the ongoing care
of 190 severely disabled patients, the majority of whom suffer
from multiple sclerosis (MS). Until April of this year I was Chairman,
Clinical Affairs Committee of the British Society for Rheumatology
and a member of the Society's Council. In that capacity I have
been responsible for or involved in the preparation and co-ordination
of submissions to NICE made either by the BSR or by the umbrella
organisation, the British League against Rheumatism (BLAR). These
have included:
— Participation in the development
of guidelines for the referral of back pain and osteoarthritis
of the hip and knee.
— Submissions to NICE on autologous
cartilage transplantation COX-2 selective anti-inflammatory drugs
TNF- blockade in rheumatoid arthritis (RA).
1.3 I remain Chairman of the BSR's TNF-
Register Committee and of the Data Monitoring Board.
2. OBSERVATIONS
ON THE
TERMS OF
REFERENCE
2.1 Is NICE providing clear and credible
guidance?
2.1.1 The documents produced by NICE have
from my experience been clearly expressed, but the guidance has
not always been credible, or usable. Two examples suffice:
2.1.2 The involvement of the British Society
for Rheumatology in the production of GP referral guidelines (vide
supra) occurred by chance; an enquiry to NICE by one of our officials
on another matter revealed that they were in preparation, but
representation had only been sought from GPs, orthopaedic surgeons,
therapists and patient groups. All of the conditions discussed
are frequently seen by rheumatologists and specialty involvement
should have been automatic. Similar omissions may have occurred
elsewhere and would clearly undermine any decisions. In this case
significant alterations to the draft proposals were suggested,
and agreed; but the late involvement of the specialty led to tensions
with other representatives who resented major changes being made
to documents that they had already agreed. Indeed the plethora
of groups involved resulted in guidelines that were so loose (to
accommodate differing points of view) that they added little to
existing material. I was clearly given the impression at the time
that the omission occurred because NICE was under government pressure
to deliver on a large number of targets and was simply unable
to cope with the workload. It may therefore be that my experience
was a "teething problem", but it will be counter-productive
to load so much work onto NICE that it either cuts corners or
fails.
2.1.3 Guidance on the use of COX-2 selective
agents is clear but unworkable. These agents are used to treat
the pain and inflammation of arthritis, being part of a larger
group of drugs known as non-steroidal anti-inflammatory agents
(NSAIDS) which have a propensity to cause bleeding within the
gastro-intestinal tract. COX-2 selective agents have a much lower
risk, but are more expensive than older drugs. NICE has set criteria
for prescription related to age and risk of bleeding, but indicates
that they should not be co-prescribed with anti-ulcer drugs (such
as H2 antagonists or proton pump inhibitors). However such guidance
does not take into account the clinical situation of a patient
who has been unable to tolerate standard NSAIDs with or without
such drugs, gets good relief from a COX-2 selective agent but
gets indigestion on it. I doubt that any consultant rheumatologist
would not co-prescribe an anti-ulcer drug in such circumstances
and on that basis the NICE recommendations are being ignored.
If they were to become mandatory, as has been suggested, then
specialists would have to consider the risk to themselves of breaking
the guidelines set against the risk that their patient might sue
them if they conform when the clinical situation suggests they
should not.
2.2 Has NICE ended confusion by providing
a single national focus?
2.2.1 Certainly not. While a drug is still
under consideration by NICE, Health Authorities and Primary Care
Trusts have prevaricated over introduction. The assessment process
takes at least 11 months, longer if there are disputes or appeals,
and NICE does not always decide on assessment immediately a new
drug or health technology is launched. Thus a drug may be available,
but cannot be funded because purchasers invoke a reference to
NICE as an excuse to delay a decision (notwithstanding the original
provision of HSC (99) 176 and interventions in the House of Lords).
Once NICE has deliberated, then government interference may anyway
subvert its decision. The classic example of this is the case
of ß-interferon in MS, where a preliminary decision was
leaked, with awkward consequences, and the final decision (being
unpalatable to patient groups in particular) being effectively
overruled by a decision that the drug could be freely used but
that the NHS would only pay if it worked, leaving the drug companies
to pick up the bill for patients where it didn't. If NICE is to
be independent then this sort of thing must not happen.
2.2.2 A further issue is that of the level
of cost effectiveness that NICE deems to be acceptable, and on
what basis the cost effectiveness calculations are made. When
NICE was established its aim was to judge clinical effectiveness.
However it appears from recent preliminary and final rulings that
even if a drug is found to be effective it may yet fall foul of
NICE by being too expensive; at present the "cut-off"
figure appears to be about £30,000 per patient per year.
The derivation of this arbitrary figure is unclear. There also
appears to be a differential applied depending on the number of
patients likely to benefit. Thus a drug used on a short term basis
in small numbers of cancer patients may pass, while an equivalent
in cost per patient terms but suitable for long term use in large
numbers may fail. Furthermore while NICE has questioned some submitted
cost analyses and found them wanting, it has then applied its
own analysis which may be no less suspect.
2.2.3 NICE have, in one draft judgement,
expressed the view that there may be a need for large scale studies
or surveys (over and above those conducted as part of any standard
post-marketing surveillance programme) to assess both longer term
benefit and risk. However it is unreasonable to expect that such
data could be collected by what effectively is an indefinite clinical
trial at the expense of the drug company, particularly if the
company is not required to do this elsewhere in the world. Some
rare side-effects may take years to be appreciated while long
term benefits from short or medium term treatment, for instance
in the field of arthritis, may take decades to quantify. For example,
it is recognised that the development of joint damage from RA
will result in a need for later joint replacement surgery; also
the life expectancy of RA sufferers is reduced. Any new drug which,
by stopping early inflammation, may reduce these risks will not
demonstrate this within 10 years. If NICE proscribes a drug then
the research it requires to approve use will necessarily be driven
overseas while British patients may be denied treatment freely
available elsewhere.
2.3 Is NICE providing guidance that is locally
owned and acted on in the right way?
2.3.1 Experience so far suggests that clinicians
have questioned some NICE conclusions (in particular those that
deny or restrict a drug's use) while purchasers use the guidance
to block prescription. The guidance on uncemented hip replacements
was widely condemned by orthopaedic surgeons on the basis that
the evidence had been inadequately reviewed. However there are
local variations, and because of this postcode prescribing still
occurs. In some instances the guidelines have been generally accepted,
and clinicians have worked with purchasers to devise rational
prescribing strategies.
2.3.2 But evidence may continue to accumulate
following a NICE ruling, and that evidence may have a fundamental
effect on the decision. For example, initial reports suggested
that one COX-2 selective agent might predispose patients who took
it to an increased incidence of adverse cardiac events, while
later research appears contradictory. Such shifting of ground
inevitably means that much time could be wasted at a local level
in continual review of evidence, and I doubt whether NICE has
the capacity to run rolling reviews.
2.4 Is NICE actively promoting interventions
with good evidence of clinical and cost-effectiveness so that
patients have faster access to treatments known to work?
2.4.1 NICE promulgates its advice most comprehensively;
paper documents are circulated widely and material is available
on the Internet. However the promotion and introduction of new
and expensive treatments does not in my experience involve NICE
at all, but is driven by local discussions between clinicians
and purchasers on the one hand and by political pressure by patient
groups on the other. When new treatments are clearly of benefit
and cost-neutral then no problems arise. When they are expensive
(though potentially just as beneficial) then if they fail on cost
grounds the patients may not get treated.
2.4.2 Further, in a limited budget the funding
of an expensive new treatment may require the diversion of funds
from elsewhere. We have had local experience of just such an episode;
Bexley & Greenwich Health Authority, when given extra funding
of some £9 million two years ago, chose to place £6
million with Greenwich Hospital to "bail out" a financial
crisis, almost £2 million with the local mental health Trust,
just £150k to my own hospital and £1.5 million for two
patients (that is £750k for each patient) with haemophilia
who required a special preparation of Factor VIII. In such a situation
the movement of money to fund TNF- blockade in RA (which we estimate
to be about £200k for Queen Mary's) is going to cause extreme
stress in the financial system.
2.4.3 The only way for NICE recommendations,
if expensive, to be implemented with equity is for all necessary
funds to be ring fenced and delivered to providers on top of their
normal allocation.
3. THE NICE PROCESS;
IS IT
EFFECTIVE?
3.1 I have been involved in the preparation
of three submissions to NICE in my capacity as an official of
the British Society for Rheumatology. I have not been the designated
witness for the Society in any of those three, but estimate nevertheless
that I have spent a minimum of three hundred hours in meetings,
discussions, teleconferences and document reviews in the last
18 months. In two cases the review process has involved over 20
people. This expenditure of effort is unpaid and interferes significantly
with clinical work.
3.2 I have been party to some pharmaceutical
company submissions. The time and cost of these submissions is
substantial. I have also seen some of the officially commissioned
evidence on which NICE has based its cost-effectiveness decisions.
These too are lengthy and expensive; those produced by clinicians
must necessarily interfere with clinical work, but not all have
been underpinned by the input of a health economist.
3.3 I have several concerns about the process.
1. There is much duplication of effort (thus,
professional submissions necessarily repeat much of the evidence
base quoted by NICE's advisers, but are essential inclusions for
clarity and completeness).
2. It far from clear whether all of the
official evidence is robust (in other words I would question the
competence of some of it).
3. It is lengthy and expensive.
4. The usual terms of reference make it
difficult if not impossible to include data on non-NHS costs or
savings. Thus assessments of potential changes to Social Services
spending, or the effects of keeping patients in work by providing
treatments are not addressed fully.
4. COULD WE
DO WITHOUT
NICE?
1. It is already apparent that political
pressure can subvert or even overrule a NICE decision, which raises
questions about its independence.
2. Decisions may require review in the light
of new evidence, and I do not believe that NICE has the capacity
to maintain a comprehensive review system.
3. Decisions on cost-effectiveness are apparently
arbitrary, do not have any internal comparison and may fly in
the face of both clinical evidence and practice in other countries.
It makes it impossible for clinicians to manage patients who know
that a drug that is unavailable on the NHS may be available in
Europe or the USA and this simply results in a shift of the discussion
from the medical to the political arena. It seems to me that then
the "anything for a quiet life" philosophy takes over.
4. In the absence of resources a NICE decision
to recommend a new and expensive treatment does not deliver the
needed resources to the prescribing clinicians, who still have
to negotiate locally, placing their bid against other competing
requests. Local differences in priorities lead to postcode prescribing
on that basis.
5. I have no doubt that the NICE process
has to some extent informed the cost-effectiveness debate. However
such debates have taken place at a local level for decades and
have resulted, for example, in the development of drug formularies
by hospitals and prescribing committees that recognise the different
costs of preparations that do the same thing, allowing economies
by avoidance of expensive drugs and bulk buying others.
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