Evidence Submitted by Dr Imogen Evans,
Mrs Hazel Thornton and Sir Iain Chalmers (NICE 21)
We welcome the opportunity to submit comments
to the Committee's Inquiry into NICE. [169]Our
expertise encompasses medical journalism/ethics (IE), independent
patient advocacy (HT), and research and research evidence appraisal
(IC). In 2006 we wrote a book for the general public—Testing
Treatments: better research for better healthcare—in
which we emphasised the need for unbiased research to assess the
effects of treatments, and we highlighted the key role of NICE
(Evans et al. 2006). We therefore hope that this submission
will be helpful to the Committee. In particular, we wish to address
the following interlinked issues: NICE's evaluation process; public
confidence in NICE; and recent challenges to NICE decisions.
NICE'S EVALUATION
PROCESS
1. The cost of healthcare weighs heavily
on all national economies, and the need to set priorities is inescapable.
NICE was established to provide independent guidance on the clinical
and cost-effectiveness of health technologies for use in the NHS,
partly because the drug licensing system is unable to provide
this information. The NICE approach to evaluation has been commended
by an external review conducted by the World Health Organisation,
has found favour among other European countries, and has even
aroused serious interest in the USA.
2. In its investigation of NICE in 2002,
the Health Committee stressed the importance of transparency as
a precondition for credibility of the Institute's appraisals and
guidance. Paragraph 40 of the Committee's report read as follows:
" . . . much of the information on which
NICE appraisals are based is unpublished, as it is supplied to
NICE by manufacturers in confidence. Sir Iain argued that "while
this state of affairs persists NICE cannot be expected to achieve
the credibility that is essential for it to earn the trust of
the public." We recommend that all information which NICE
uses in its decision making process is made available for public
scrutiny. If industry or others have previously unpublished data
which they want to use to support their case then this should
no longer be presented to NICE subject to confidentiality."
3. The Health Committee reiterated the need
for greater transparency two years later in the recommendations
in its report on the Influence of the Pharmaceutical Industry.
Despite this, NICE's transparency was further eroded last year
when the Institute switched from independent assessments of scientific
evidence to sponsor-produced evaluations of effectiveness and
cost-effectiveness. We urge the Committee to continue to press
for greater transparency, which we continue to believe is a precondition
for public confidence in NICE.
PUBLIC CONFIDENCE
IN NICE
4. From the outset, the rationale for NICE
has been undersold to the public, and NICE's decisions have been
undermined by political interference. A passage we wrote in Testing
Treatments illustrates the immense difficulties that NICE
faces:
"Because the annual cost of treating each
[multiple sclerosis, relapsing and remitting type] patient with
an interferon was over £10,000, NICE . . . concluded that
using these drugs . . . would not be a responsible use of limited
resources. Many patients with this debilitating disease, and especially
the organisations lobbying on their behalf, were outraged. They
were angry that the NHS could deny patients drugs that appeared
to hold out some hope. Yet did they fully realise the extent to
which the available evidence was far from convincing? It was based
on partial release of the relevant research results, outcome measures
of dubious relevance, and follow-up over only two or three years
in a disease that usually lasts at least two decades. The government
caved in under pressure. ... But this effectively ended the possibility
of learning whether [interferons] are helpful to patients."
5. It is highly unlikely that the scheme
agreed between the Medicines Division at the Department of Health
and the companies making interferons to make interferons available
to patients will yield reliable information about the impact of
these very expensive drugs on important issues such as whether
they delay dependence on mobility aids, or the moment when people
with multiple sclerosis become bed bound. Provision of interferons
in this way means that unbiased assessment of their effectiveness
becomes virtually impossible. Moreover, for ostensibly inexplicable
reasons, the contract for analysing the data has been moved from
the University of Sheffield to Parexel (the contract research
organisation associated with the TGN1412 disaster); this does
little to promote confidence in the government's reasons for overruling
NICE's guidance.
6. Uncertainties about the value of the
interferons in multiple sclerosis have been handled in a much
more responsible way in Italy. The Italians have begun a randomised
comparison of interferon with azathioprine, a dramatically cheaper
drug, which existing evidence suggests may be just as effective
as interferon beta (Sudlow and Counsell 2003). It is worth noting
that the costs of the Italian trial are being met from a fund
of 35 million Euros derived from a 5% tax on the marketing budgets
of pharmaceutical companies to support independent drug research
(Chalmers In Press).
7. Many vested interests contribute to the
public's belief that expensive new treatments are better than
existing, less expensive treatments. The truth is that new treatments
(assessed in randomised trials) are as likely to be inferior as
they are to be superior to existing, standard treatments (Kumar
et al. 2005).
8. Although selling the rationale for and
principles of NICE to the public was never going to be easy, this
essential public relations challenge appears to have been entirely
overlooked by the government when NICE was set up. It is sorely
needed now, not least to explain why the criteria for licensing
a drug are far less stringent than the criteria for establishing
the clinical and cost-effectiveness of the self-same compound.
RECENT CHALLENGES
TO NICE DECISIONS
9. Pressures from the pharmaceutical industry,
and from patient lobby groups often funded by industry challenge
a National Health Service such as ours, which is based on the
principle of shared risk and equitable distribution of limited
resources. Because of the lack of public appreciation and ministerial
support for NICE's decisions, it is unsurprising that the Institute's
decisions have been contested, and that the public has come to
see it as a spiteful rationing organisation. Almost all the media
coverage of NICE is negative, and this impression is made even
worse when, as with interferons for multiple sclerosis and more
recently herceptin for early stage breast cancer, ministers leap
in and overturn or pre-empt NICE's recommendations. Most recently,
NICE's guidelines on the use of anti-cholinergic drugs for people
with dementia have been challenged by a manufacturer of these
drugs, and the Institute is currently the subject of a judicial
review.
10. The current situation with anti-cholinergic
drugs for dementia would probably have been avoided had the Institute
been making greater use of its option to recommend that a treatment
be used only within the context of evaluative research. When there
is uncertainty about the effects of a treatment, this is the proper
way forward—as an ethicist once commented, in these circumstances
"the trial is the treatment". Yet again, the importance
of public backing is crucial. And this will only come about when
the public is engaged widely in the clinical research process,
and when there is general appreciation that patient participation
in research is a risk-limiting strategy and not a risky endeavour.
11. NICE has used this `only-in-research`
approach in only 15 out of its 400 appraisals between 1999 and
2006. When used, however, it has made an important contribution
to generating the additional evidence needed, and has led to supportive
recommendations when the original guidance was reviewed. The benefit
of the "only-in-research" approach is faster accumulation
of evidence to inform practice and make shared decisions. It also
exposes to the public the need to reduce uncertainties concerning
interventions in common use, as well as uncertainties about the
effects of new treatments. Importantly, the "only-in-research"
approach can reduce the risk of having to reverse previous decisions,
as has happened with the anti-cholinergic drugs for dementia.
12. We suggest that promoting public partnership
in the "only-in-research" approach should become a key
role for NICE's Citizens' Council, and that the expertise of Council
members be used to promote NICE more widely.
CONCLUSION
13. As a 2006 Guardian leader commented,
NICE embodies "the least bad way of tackling an impossible
job". The Institute should be strengthened, freed from ministerial
whims, encouraged to engage more with the general public, and
allowed to do its work for the benefit of all who seek treatment
in the NHS.
Imogen Evans, Hazel Thornton and
Iain Chalmers
March 2007
REFERENCES
Chalmers I (in press). When does clinical science
cease to exist? Science in Parliament.
Evans I, Thornton H, Chalmers I. Testing treatments:
better research for better healthcare. London: British Library,
2006.
Kumar A, Soares H, Wells R, Clarke M, Hozo I,
Bleyer A, Reaman G, Chalmers I, Djulbegovic B (2005). What is
the probability that a new treatment for cancer in children will
be superior to an established treatment? BMJ 331:1295-8.
Sudlow C, Counsell C (2003). Problems with UK
government's risk sharing scheme for assessing drugs for multiple
sclerosis. BMJ 326:388-92.
IC has been a member of NICE's Research and Development
Advisory Committee since its inception; addressed NICE's Citizens
Council on 26 January 2007 on `Uncertainty and the `Only in Research`
Option`; and has contributed a witness statement in connection
with the judicial review of NICE's guidance on the use of anti-cholinergic
drugs for people with dementia.
HT was invited to attend a meeting of the NICE Citizens
Council 26 January 2007 as a Question Time Panel Member on day
2 of their deliberations on "Uncertainty and the `Only in
Research' Option".
169 From Dr Imogen Evans (IE), Mrs Hazel Thornton (HT)
and Sir Iain Chalmers (IC), co-authors Testing Treatments:
better research for better healthcare, British Library 2006. Back
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