Memorandum by the National Cancer Alliance
KEY POINTS RE THE WORK OF THE NATIONAL INSTITUTE
FOR CLINICAL EXCELLENCE
The NCA welcomes the formation of
NICE and the rationale behind its work and thinks following ongoing
reviews of its remit and work it can only improve.
We have some concerns regarding a
lack of clarity regarding NICE's overall remit and terms of reference.
This is reflected in the questions asked by the Health Committee
inquiry, which do not relate to what we understand to be the terms
of reference of NICE.
Topic selection, and the NHS implementation
of guidance, though not part of NICE's remit, need to be more
carefully thought through, particularly in relation to the impact
on NHS prioritisation of resources and the potential for sealing
in inequity of access, rather than addressing it.
We have a number of concerns regarding
some of its methods and evaluations processes for assessing Health
We are concerned about the cost-effectiveness
and economic evaluation methodology is not transparent or appropriate.
We are also concerned that linking
cost-effectiveness to clinical effectiveness in the one body evaluations
could lead to mistrust in the outcomes of both evaluations.
The purpose and type of patient involvement
needs to be clarified and properly resourced.
1. TOPIC SELECTION
1.1 The apparent criteria used to select
the Topics for appraisal or guideline development are opaque.
1.2 The adverse impact of the topic selection
process and outcome (which topics have and haven't been selected)
has clearly been under-estimated. This affects patients and the
NHS in two key ways:
It seems that many local health organisations
(Trusts, GPs, HAs, Commissioners) feel they must await NICE guidance
on a particular Health Technology Assessments (HTAs), or guideline
before agreeing to funding or prescribing (even if the appraisal
is due to take place some time in the future). This is clearly
an absurd stance to take. The DOH should issue firm instructions
to health ecomonies and providers that this is not an acceptable
(b) Skewing of NHS funding resources and
The Minister has instructed the NHS that from
January of this year, they have a statutory obligation to fund
NICE approved HTAsbut out of existing resources and cost-envelopes.
This is to be welcomed in some respects, as it deals with the
implementation and "NICE-But No Teeth" issue. However,
this ruling (inevitable though it has been) causes otherun-thought-through
scenarios and problems, most significant of which relate to the
skewing of funding priorities to those interventions which have
been "Nice-d". This means that other interventions or
service-improvements (whether for the same condition or different
condition) will necessarily get less funding. This is the case,
whether or not the NHS has received ring-fenced funding to support
the extra costs of the intervention.
1.4 An illustration
The government has promised additional monies
for cancer services modernisation. In 2001-02 an additional "hypothecated"
£255 million was allocated to Health Authorities for specified
areas of cancer services improvement. It was suggested by the
DoH that out of the £255 million, an indicative £170
million should be "sub-hypothecated" in order to cover
the possible costs of the 13 cancer drugs which NICE had been
asked to appraise as quickly as it could (in year). This meant
that only £85 million remained to fund all other major improvements
in cancer services, including meeting the very ambitious waiting
times target for people the GP suspected might have cancer; the
urgent need to increase the number of cancer specialist cancer
multi-disciplinary teams; the need to develop palliative and terminal
care services; etc.
The total costs of making these absolutely vital
service improvements would obviously be far greater than the costs
of the 13 NICE drugs, and would benefit many more patients in
a number of ways. However, the mandate was clearly laid out by
the DoH that HA's needed to ensure that they prioritised the funding
of these high-cost drugs, once approved by NICE. This is important
in that it effectively seals in inequity of resource allocation
in one disease groupeffectively for ever (the Saff agreements
for 2001-02 were required to specify the total projected spend
on the Cancer NICE approved drugs, both for 2001-02 and into the
future as carry forward sums).
1.5 The NCA, of course, is delighted that
hypothecated, or ear-marked additional and growth resources are
being allocated to fund improvement in cancer, and is also pleased
that 13 cancer drugs were put as a priority for NICE appraisal.
However, we would have liked there to be a more sophisticated
discussion about the fair and equitable allocation of resources
from the cost-envelope at both the national and local level.
1.6 So, should a person with lung cancer
have the choice of how to spend the £5,000 it costs for a
cycle of for the NICE approved drugs (which gives them an average
of three months extension of life). They could, for example, perhaps
choose to be able to ensure they have a nurse looking after them
at home, who knows how to manage the last difficult phases of
a lung cancer death (some people get this, most do not). But on
a population basis, the funding required to be "held back"
to fund Gemcitobene, etc has effectively meant that the appointment
of sufficient numbers of lung cancer specialists has had to be
given a lower priority.
1.7 The skewing of resource allocation because
of the NICE process will of course be as polarised across the
conditions and service-types as within them.
Topics selected has already had an
adverse effect on NHS priorities and access to equitable services.
In some ways it is sealing in inequity - rather than improving
THIS IS NOT the FAULT OF NICE - but crucially
relates to it's work programme.
The criteria and process of topic
selection needs to be transparent and should cover a wide spectrum
Guidance should be issued to to the
NHS that it cannot await NICE guidance before making interim decisions
on appropriate interventions. Business as usual should apply
The number of topics addressed by
NICE needs to be dramatically increasedat least in the
2. THE PRODUCTION
2.1 There seem to be three main areas of
activity for NICE: Health Technology Appraisals, Guidelines and
2.2 However, there seems to be ongoing confusion
about the differences and similarities between these strands of
work, how they link together and more importantly about the processes
and methodolgies used and their final status.
The process and methodologies used
2.3 For the final guidance (HTA or Guidelines)
to be credible, whether with the public, patients, or the NHS,
the process of assessment or development needs to be highly robust,
transparent, and if at all possible, above criticism.
2.4 It is recognised that NICE has been
"learning on the job", and should be congratulated for
sharing its learning and being open about the changes and developments
it has made, and continues to make, to its appraisals and guideline
2.5 However, there remain a number of concerns
about the process and methods used by NICE in both its appraisal
process and guidelines development processes. In particular:
(1) a general lack of clarity about the methodology
and process used in the appraisals and the guidelines development.
(2) allegedly inappropriate differences in
approach to assessing and weighing the evidence and the impact
on the final decision-making process between different appraisals
(3) the reportedly inadequate quality of
the evidence-base available, how NICE handles this when making
a final recommendation, including the availability of relevant
evidence (whether they be high-quality RCTs, or high-quality quality
of life measures)
(4) the lack of clarity and perceived differences
in the handling and impact of the patient-"witness"/
representative "qualitative input, including taking into
account "patient-values", quality of life (usually never
systematically and objectively measured).
(5) the difference in approach to "patient-involvement"
between the guidelines and the HTAs.
(6) the lack of clarity and transparency
regarding the whole methological approach and relevance to the
cost-effectiveness appraisal-side of the workand the impact
of this on the final guidance.
(7) the lack of transparency and perhaps
honesty regarding the approach to the links to having to assess
"value-for-money", costs to the NHS, cost-effectiveness
assessment (sometimes using QALYS, sometimes notoften with
questionable outcome), and affordability.
(8) the impact of the influence of the different
stakeholders, whether government, industry, patient-groups, academics,
the NHS itself, on the process of evaluation remains unclear.
This links also to the topic selection issue.
3. STATUS OF
TO NHS IMPLEMENTATION
3.1 The difference in "status"
between NICE issued/ recommended HTAs and Guidelines in terms
of the requirement of the NHS to implement NICE's recommendations
(ie HTAs approved by NICE have to be funded by Health Authorities/soon
to be PCTs, whilst Guidelines do not) is questionable, confusing
and could indeed increase inequity of access to best quality care.
3.2 Why are guidelines not going to have
to be statutorily implemented in the same way as HTAs? Is hints
loudly at this really a matter of affordability in disguise. It
makes no sense, especially when the guidelines are far more relevant
to the overall costs and comprehensive management of a particular
problem or condition.
4. MIXING CLINICAL
4.1 Although this is currently NICE's remit,
mixing the "does it work, or work-enough?" evaluation
process with the "is it worth they money/ is there something
equally helpful for less money/ can or should we afford it"
causes problems to the process and outcomes of both elements of
4.2 Firstly, so-called cost-effectiveness
measures are still very crude, hotly debated can only be used
in some, restricted circumstances. So-called "cost-effectiveness"
gets quickly entangled with "value-for money, and affordability
issues and the broader rationing and ethical prioritisation debate.
4.3 Patients, patient-groups, clinicians
and indeed the NHS, think that the cost-effectiveness assessments
will have an impact on the final outcome of the NICE approval
process, either positively or negatively. So it is widely assumed
that if an intervention is deemed to be "too expensive"
(and it is unclear what this "too-expensive" is and
how it is arrived at, as QALYS are used in only half the assessments,
and in some cases perhaps inappropriately so), then even if it
proven to be "clinically effective" (ie has more benefits
than dis-benefits for patients), then it still won't be approved.
This means that people are suspicious of the
purity and objectivity of the process.
4.4 It may be that there should be a separation
between the assessment of "clinical-effectiveness" and
of cost-effectiveness", the former undertaken by NICEsystematically,
objectively appraising and assessing the clinical and scientific
evidence base for interventions, guidelines, etcwhilst
another body or organisation should be established to undertake
the work on costswhich should include a more robust understanding
of what what the costs and/or prices are and why, whether they
represent value for money, what they are worth (financially and
in health-benefit terms), etc.
4.5 This separation of the two processes
or functions of NICE, would mean that hitherto hidden and opaque
decision-making processes regarding funding and resourcing of
health care interventions, (including costs, links to effectiveness
and affordability, etc) would be brought into the open, shared
4.6 Currently, the mixing up of the clinical
and cost-effectiveness processes pollutes the outcomes of both,
especially when the methods are unclear or questionable.
5. ECONOMIC ASSESSMENT
5.1 Apart from the above point on the need
to separate the clinical evaluation from the cost-effectiveness
assessment, we are concerned that the approach to health economic
assessment in the NICE process has not been consistent. A review
needs to be undertaken of the methodologies and measures used
by NICE, and the applicability and use for different circumstances.
6. PATIENT CONSULTATION
6.1 The establishment of the new patient-involvement
"unit" is to be welcomed. However, this does not really
address the key problem, namely that small voluntary organisations,
especially those made up of, or run by "real-patients",
rather than patient-representatives or health professionals, simply
do not have the resources or time to be able to properly contribute
to the NICE process.
6.2 NICE needs to resource the relevant
involvement of real patients and carers and those organisations
or groups which truly represent patient-views and experiences.
6.3 NICE should also be much clearer about
whom it is "consulting" in relation to the "voluntary
sector" or so-called patient-advocacy groups and for what
purpose. Many large national health-related charities have no
formal patient-constituency and have no formal mechanisms in which
to hear the views or represent the views of patients. Many raise
money for research and support health professionals' research
work (often in partnership with the pharmaceutical industry).
Other's are effectively "service-providers" in their
own rightbe that professional information and advice services,
or other more direct care-provision. These are therefore not "patient-groups".
6.4 Many of the smaller patient-groups (and
probably some of the larger) have received offers of help and
support from the pharmaceutical industry in the formation of their
submissions to NICE. Some have turned these offers down, others
may have accepted help in tangential ways. This draws into question
the legitimacy and value of the patient-input and the independence
of the overall consultation and submission process.
7. HAS NICE ENDED
7.1 The NCA greatly welcomes the establishment
of a national body undertaking the national development of clinical
guidelines, protocols and evaluation of interventions. This, amongst
other initiatives aimed at addressing inappropriate variation,
and inequity of access to relevant and best treatment is to be
7.2 The eventual outputs of NICE, having
a national focus and producing a national body of work, with and
on behalf of local health professionals, will, in theory, be able
to help address inequity.
7.3 However, there are a number of limiting
factors already alluded to above, including the number of topics
being appraised and the rate of appraisals being so limited as
to increase inequity; the problems associated with the need to
be constantly updating assessments and guidelines.
7.4 The NHS implementation issue and the
differing status of the guidelines and HTAs has been discussed
above. NICE is working on improving its dissemination and publicity
process but it does not have the remit or role of overseeing implementation.
However, if the Guidelinesarguably the more important part
of NICE's workare not given equal status (in terms of statutory
implementation obligation) there is danger they will remain in
the territory of Confuciuswise thoughts from the mountain!
Topic selection needs to be reviewed,
as does the spread of topics, number and rate of appraisal
The NHS should be given clear guidance
of how to handle the situation whilst awaiting NICE guidance
Guidelines should have equal implementation
status as the HTAs
The nature and type of the clinical
evidence needs to be "fit for the purpose"ie
RCTs are important in some circumstances and qualitative evidence
is A Grade evidence in others
The weighting of the evidence needs
to be more transparent.
The overall methodology, process
and above all rationale needs to be clearer and more transparent
The cost-effectiveness assessments
need to be undertaken by a separate body. NICE should concentrate
on the clinical-effectiveness agendawhich is hard enough
A different body should be set up
to use the clinical effectiveness information and work in a different
way on the cost-effectiveness agenda and the links to NHS costs
and affordabilityeven rationing.
There should be a review of the cost-effectiveness
measures used and how they can and cannot influence final cost-effectiveness
ones should be given resourced properly to be able to input effectively
to the process
"Patient-groups" need to
be honest about who they represent and how they do thisthe
may not be real patient groups. NICE needs to be sure about which
groups it works with for what purpose.