Memorandum by Dr Martin Duerden (NC 117)
1.1 My background is that of a General Practitioner
with a particular interest in prescribing and therapeutics. After
seven years as a full time principal in general practice I worked
for several years as a Health Authority Medical Adviser, with
a particular remit for prescribing advice. Following a period
spent training in public health medicine I subsequently worked
for three years as medical adviser/medical director of the National
Prescribing Centre. In that role I was closely involved in identifying
and evaluating drug innovations that had potential to change clinical
practice. Over the last year I have worked as a medicines policy
adviser at University College London Hospitals Trust. From the
beginning of this month I moved to work as a clinical author for
PRODIGY (a computerised decision support system for GPs).
1.2 I continue to work as a part time general
practitioner. I also am involved in therapeutics training for
GPs and other health care professionals.
1.3 The viewpoint I give is my personal
view as a result of these experiences. I do not represent any
particular organisation. I will comment specifically on drug technology
appraisals and the guideline development roles of NICE. There
are political and philosophical issues in relation to the rationing
role, and tensions with the media, drug companies and patient
groups that I have not covered.
2. MANAGED ENTRY
2.1 Over the last decade or so the mechanisms
to allow the use of new drugs within the NHS have appeared poorly
co-ordinated and fragmented. There has been a popular perception
that new drugs are necessarily better and older drugs are "old
fashioned" and also that new drugs can cure many ills previously
not amenable to therapy. The licensing process has been designed
to strike a balance between enabling innovation, encouraging a
strong drug industry and protecting patients from adverse drug
reactions. Pressure has been applied by drug companies, interested
clinicians and patient representatives to rapidly assimilate new
drugs into clinical practice. The dilemma is that in many instances
new drugs do not necessarily represent major advances and that
at the time of licence the safety profile of the drugs has not
been fully evaluated. Of particular concern is that drug trials
required to get a drug licensed do not necessarily reflect the
information needed to decide the place of that drug in clinical
practice. As a response to these dilemmas and as a result of budgetary
constraints many Health Authorities developed mechanisms to rationalise
uptake and funding of new drugs, usually through evaluation by
their Public Health departments. This has resulted in different
decisions being arrived at in different localities, so-called
postcode prescribing. Many felt that the only way to improve on
this lack of co-ordination was to have a central process for assessing
the value and clinical worth of new drugs. For this reason the
concept of NICE was positively received by many involved in prescribing
advice, including myself; there was considerable expectation that
NICE would avoid duplication of effort and produce credible guidance,
satisfying commissioners, prescribers and patients, and avoiding
inequities in provision.
3. USE OF
3.1 Alongside varying access to drugs there
has also been concern about variation in practice in hospitals
and general practice for relatively common interventions. This
concern has been alongside emerging evidence about variation in
outcome. An obvious solution to this problem is the development
of evidence-based guidelines to guide practice for commonly encountered
complaints. Also the concepts of clinical governance encouraged
by A First Class Service help to drive quality practice.
One problem that has arisen from development of guidelines, often
supported and promoted by drug companies, has been the sheer multiplicity
of guidance that has emerged, often of varying quality. The role
of NICE in developing clear, consistent quality national guidelines
has also been eagerly awaited.
4. VIEW ON
4.1. The intention was that NICE would provide
guidance on use at the time a new drug came to market. Possibly
as a result of the time taken for NICE to develop its infrastructure,
this has certainly not been achieved and this has proved a major
problem for those agreeing drug use on a local basis (now commonly
called "NICE blight"). A vacuum is created where it
has been difficult to provide local interim guidance in case it
is subsequently contradicted by NICE; also simply advising "no
use" in anticipation of guidance has been deemed by the Department
of Health as unacceptable. In many respects this is worse than
the previous arrangements where at least on a local basis, following
due process, firm decisions could be reached. As a result new
drug use has become less controlled, more subject to idiosyncrasies
and is also less well informed. A good example of this was seen
with the anti-inflammatory drugs, the COX-2 inhibitors (rofecoxib
and celecoxib). Hopefully the appraisals will become more timely
as NICE evolves.
4.2. In mitigation, the expectations placed
on NICE have been tremendous and there was a perception that the
appraisal work and guideline development could happen immediately.
As NICE has started off the sheer scale of work has become evident,
and there has been increased recognition of the cost of such processes.
The original intention that the Institute should be "cost
neutral" has clearly been unrealistic (it was expected to
draw on funding already available for drug evaluation), and I
suspect even with its current level of funding, it is not capable
of performing its role of new drug appraisal in a timely manner.
With hindsight it might have been better to clearly separate the
catch up role, where central guidance was required for
drugs already on the market, being used subject to local vagaries
(for example, beta-interferon for multiple sclerosis) from the
need to horizon scan and have guidance available for drugs at
point of licence (even if the latter was an interim appraisal).
Also it might have been better, rather than expecting technology
appraisals on established therapies (as part of the catch up
role) to occur in isolation from overall disease management guidance,
to have included these appraisals as part of the guideline function.
4.3 Similar problems exist in relation to
national guideline development. The guideline programme suggests
that many needed guidelines will not be available for several
years. Ideally National Service Frameworks should be underpinned
at launch by clear national policies on best practice, provided
by NICE. Lack of these clear guidelines has been deemed "guideline
blight". To a certain extent this has been circumvented by
good guidance provided by professional Colleges; that produced
by PRODIGY and the exemplary guidelines produced for Scotland
by SIGN. Again I suspect that expectations of immediate solutions
have been unrealistic but there has been lack of clarity about
what to do in the interim.
4.4 Another problem that has arisen with
new drug appraisals by NICE are concerns about the technical integrity
of some of the guidance. Sometimes the guidance seems to be at
odds with the supporting Health Technology Assessment (for example,
riluzole for motor neurone disease). Up to a point this can be
accepted in that a fine judgement is required to balance a pure
evidence based medicine (EBM) determination against social needs
and practical delivery of care, but the balance seems to be overly
weighted against EBM, which should be the prime driving force.
There also appear to be technical mistakes or omissions. For example
the zanamavir revised appraisal (Relenza, `the flu drug)
recommended use in patients with asthma or chronic lung disease,
whereas the Summary of Product Characteristics (SPCspecifications
that lay down the licensing recommendations which are designed
to ensure safety) advises caution with this group of patients
and also clearly states that there is no evidence of benefit in
this group. Another example is that the sibutramine guidance failed
to state that the SPC advises that the drug should be used by
"under the care of a physician skilled in the treatment of
obesity" This is a key point and recommendations for the
use of the drug hinge on the definition of this group. In future
it might be worth involving drug information pharmacists who are
highly skilled and trained to identify these points. I am aware
that confidentiality has been an issue, but this should not hamper
appropriate professional involvement.
4.5 From a GP perspective there are concerns
about how realistic and how useful the guidance, so far issued
has been. For example, the advice on the use of proton pump inhibitors
seemed too general to change or refine practice. This guidance
also omitted a crucial issue; that of concomitant prescribing
of PPI with anti-inflammatory drugs to reduce risk of adverse
gastrointestinal events (it has been estimated that as much as
20 per cent of PPIs are used in this context). Another area of
contention was the advice on the new anti-inflammatory drugs,
the COX-2 inhibitors, that appeared to sanction widespread use
of "black-triangle" drugs (those that are still under
intense surveillance by the Committee on Safety of Medicines to
determine their adverse effect profile). Another technical point
is that again it would have been useful to have some comment on
how COX-2 inhibitors compare with the use of PPI plus conventional
anti-inflammatory drugs in reducing risk of adverse gastrointestinal
events. Also this appraisal included two drugs which were not
strictly in this class, and where the evidence base was less securemeloxicam
and etodolac. Why these latter drugs were included is unclear.
4.6 The real problem that has challenged
the credibility of NICE with GPs has been the change in guidance
for use of zanamavir (Relenza, the `flu drug) without an
obvious change in the evidence base to support this approach.
This could have major implications for GP workload at the time
of a flu epidemic and it is unclear how practical this advice
is. There is a concern that there was insufficient involvement
of GPs at the time of this appraisal.
4.7 A major current issue is the recent
directive stating that NICE recommendations should be funded within
three months of release. This creates major conflict with local
priority setting. The problem is that the guidance recommended
may be relatively unimportant alongside other local issues. It
may divert resources away from other important projects, particularly
diverting manpower (the NHS seems more personnel and resource
strapped than cash-strapped, at present). At present it seems
perverse to progress straight from the situation of "NICE
blight" to rapid implementation at the point of release;
this does not enable strategic planning. One solution here is
to carefully demarcate where guidance is intended to reduce postcode
prescribing; for example certain high cost low volume drugs, which
are deemed to require immediate action, from those other appraisals
where service development has greater importance (for example,
drugs in Alzheimer's disease). There should be greater flexibility
allowed in local implementation.
4.8 A concern already alluded to, is the
development of advice on using a drug outside the context of a
disease management framework. The two anti-obesity drugs, orlistat
and sibutramine give an example of this. These clearly should
be used within a carefully constructed and agreed obesity management
guideline. Taken in isolation it is difficult to know how best
to use these; for example, which should be used in preference
and in what circumstances. Part of this problem is that NICE appears
constrained by the very narrow focus they are given for their
appraisal questions. It would seem better to make this more flexible
so that the appraisal could have greater range, where necessary,
which would make the guidance more workable.
Apart from those mentioned earlier in my report,
some suggestions that may improve the process are:
5.1 Greater independence from political
processes. This may be possible if decisions on clinical effectiveness
are separated from decisions on affordability. Also prioritising
of topics for NICE should be more closely focused on the NHS service
needs, rather than driven by media interest or political choice.
5.2 NICE seems to have a very large agenda.
Is this realistic for one body? One suggestion is to divide this
into separate functions; for example, a National Cancer Institute,
a National Guideline Institute, and a National New Drug Advisory
5.3 Enhance funding of NICE to enable more
robust processes and speed up the work. At present there is a
concern that much support is provided on the basis of good will
and this may be overstretched.
5.4 Ability to be more reactive to urgent
needs. The advent of the drug imatinib for chronic myeloid leukaemia
was sudden, but the appraisal will not be until later this year.
The NHS needs urgent guidance in cases like this.
5.5 Greater involvement of the service;
involving prescribers and drug information specialists more closely,
(hospital doctors, GPs, nurses and pharmacists) and seeking views
of health care commissioners in the consultation process. Also,
it might be better to have a greater mix of these people on the
various committees. I was quite perturbed to hear from NICE, at
a recent conference, that there is no mechanism of feedback or
appeal from within the NHS.
5.6 Mechanisms to enable local priorities
to be incorporated within plans to use the appraisals and guidelines.
Maybe the output could be stratified so that subjects for immediate
implementation are highlighted (rather than a blanket requirement).
5.7 In conclusion, my view is that NICE
should be welcomed and when it has fully developed and evolved,
will be very useful and improve equity of health care provision.
If the process becomes timelier it will enhance and speed up access
to effective treatments. The main impact of NICE will be seen
with the further development of national clinical guidelines.