Conclusions and recommendations
1. We
note that it is not the role for Ministers to directly or indirectly
seek to influence the NICE decision-making process. (Paragraph
68)
2. It is clear that
the environment in which NICE operates has changed considerably
since the Institute was established in 1999. It is also clear
that there is a vital role for NICE in the rationing of healthcare
and in encouraging best clinical practice. In the future the role
of NICE will be ever more important and demanding with new expensive
drugs and a slower rate of growth in NHS expenditure. There remains,
however, concern about aspects of how NICE does its job. (Paragraph
79)
3. It seems to us
appropriate that topics are selected for interventional procedures,
clinical guidelines and public health guidance. It is not appropriate,
however, to limit technology appraisals to selected, often new
and expensive, products. Instead, as we recommend below, all new
drugs should be assessed. (Paragraph 94)
4. Witnesses were
concerned that NICE's focus on acute treatments, in particular
medicines, could skew NHS spending towards selected new and expensive
(NICE-approved) drugs for acute illness. (Paragraph 95)
5. In our previous
report we recommended that NICE give more emphasis to examining
old technologies to encourage disinvestment. This the organisation
has failed to do as fully as we expected. Its statement that few
interventions have absolutely no benefit may be true but is irrelevant.
Many treatments currently used are not cost-effective as many
studies attest. NICE should adopt a similar standard of cost-effectiveness
in assessing such treatments as it uses in its technology appraisals.
The organisation must now give more emphasis to disinvestment.
One approach would be to undertake more MTAs, which would reveal
the existing treatments that provide poor value for money. (Paragraph
96)
6. We heard much criticism
of the use of QALYs. Some of the criticisms seem to be the special
pleading of disappointed parties. It is vital that a method which
allows comparison of the benefits and costs of different treatments
for different conditions is used in cost-effectiveness evaluations.
However, it is also vital that the system is accurate and reflects
the real costs to society and the benefits to patients. We recommend
that:
- Research is undertaken to follow
up specific guidance to see whether the predictions of the cost-effectiveness
analysis are borne out in practice;
- Wider benefits and costs, such as costs borne
by carers and social care services, be more fully incorporated
into NICE's assessment. We were told that this would have to be
a decision for Parliament. (Paragraph 120?)
7. NICE
does not have all the information it needs to assess and compare
treatments. First, while access to EMEA documents and other changes
have improved NICE's access to information, it still does not
have access to all the relevant information which is available.
Secondly, clinical trials undertaken by pharmaceutical companies
understandably focus on generating data about the drug's efficacy
and safety, which is required for the licensing process; such
trials are not usually designed to generate the type of data on
cost-effectiveness which NICE requires. Third, in some areas,
without commercial sponsors, notably public health and many physical
and psychological therapies, there is little research about the
cost-effectiveness of different interventions. (Paragraph 143)
8. We recommend that
NICE be granted the right to see all the evidence the MHRA uses
when making its decisions. We appreciate that this would mean
that there would be some commercial-in-confidence material that
NICE could not make public when it published its guidance. (Paragraph
144)
9. We welcome the
fact that both NICE and drug companies are aware that they need
to collaborate closely to ensure that clinical trials are undertaken
with the needs of NICE appraisal in mind. The Government should
encourage all countries in which large-scale clinical trials take
place to adopt a similar policy. We support the mandatory registration
of all clinical trials so that the results of all negative trials
are accessible. We recommend that NICE assesses and reports the
quality of the research it receives. (Paragraph 145)
10. More publicly
funded research should be undertaken to assist the development
of public health guidance and other areas without commercial sponsors.
(Paragraph 146)
11. Many witnesses
thought that too few experts with the relevant detailed expertise
were involved in the process of producing guidance. Since they
have a permanent membership, Appraisal Committees are unlikely
to have such experts. They do consult experts, but this is unsatisfactory
because such experts appear for the day alone. We therefore recommend
that Appraisal Committees appoint specialist advisers, without
voting rights, to work with the Committee throughout consideration
of a technology appraisal or clinical guideline. This will improve
guidance and ensure public and patient confidence in the system.
Decisions about which experts should be appointed should remain
the responsibility of NICE following consultation with the appropriate
clinical bodies. (Paragraph 156)
12. The wide consultation
which takes place during the development of NICE guidance is greatly
valued. While we agree that it is difficult for some organisations
to respond within the often brief time limits, we recognise that
a long consultation period would slow the guidance production
time further. Nevertheless, the situation would be improved if
NICE were to give interested stakeholders greater warning of forthcoming
consultations, to allow them to organise their resources in time
to respond effectively. (Paragraph 168)
13. Some consultees
complain that their views are ignored. We understand that NICE
does not have the resources to respond individually to each consultee.
NICE could, however, issue a standard response to inform every
consultee how it will respond and setting out how the system works.
(Paragraph 169)
14. We
note the pressure to change the grounds for appeal, but consider
changes might cause more problems than they solved. Allowing additional
evidence at the appeal stage would extend the process significantly,
and might discourage companies from producing high quality trial
data at the time of first assessment. It also might risk more
"gaming" appeals. We make recommendations in the next
section which we expect will lead to fewer appeals being brought
in the first place. (Paragraph 184)
15. A shorter, less
in-depth initial evaluation of medicines at an early point would
be useful. It is important that clinicians have access to independent
information about new therapies as soon as they are available.
However, a quick, in-depth, fully consultative evaluation for
all new medicines by the time of launch is not possible. We therefore
recommend that NICE should examine all new medicines for their
indications as set out in the marketing authorisation. Assessment
should be carried out during the period between licensing and
launch. It should be brief and published prior to, or at the time
of, launch. There should be no formal appeal process and only
limited consultation. These brief assessments should be followed
by a larger scale multiple technology appraisal for selected products
(an MTA or STA as appropriate) at a later date, when more evidence
is available. The technology appraisal should include current
levels of consultation. The guidance issued at this later stage
should be definitive, over-riding that issued earlier. (Paragraph
200)
16. Since providing
an evaluation of all drugs at launch will be a more rough and
ready process, it would be inappropriate to use the same threshold
range as the full assessment. One of the aims of the new process
is to ensure that treatments which are obviously cost effective
are available at an earlier stage than at present. We therefore
recommend that a threshold below the current range be used in
these early assessments. This could be raised for individual products
in special circumstances, for instance where no other treatment
exists. At the time of the full assessment, the cost per QALY
threshold could increase. (Paragraph 201)
17. The threshold
or ceiling NICE employs (measured in pounds sterling per QALY)
to decide whether a treatment is cost-effective, and so should
be available in the NHS, is not based on empirical research. Nor
is the threshold directly related to the NHS budget, since the
threshold has remained constant while the budget has increased
hugely since 1999. (Paragraph 239)
18. The threshold
used by NICE does not take into account the funding decisions
made by PCTs generally. For interventions not assessed by NICE,
PCTs appear to use thresholds which vary from treatment to treatment
but for the most part seem to be lower than the NICE threshold.
(Paragraph 240)
19. Many PCTs struggle
to afford to implement NICE technology appraisals, as well as
clinical guidelines. As more interventions are evaluated it is
feared that the position will become unsustainable. Funding is
essentially ring-fenced for technology appraisals, leaving PCTs
little room for manoeuvre in their budgets to reflect local needs
and priorities. (Paragraph 241)
20. A
number of steps were proposed by witnesses to alleviate the situation.
To improve coordination between NICE and PCTs, we support the
wider use of implementation consultants, who would provide information
both from NICE to the PCTs and from the PCTs to NICE. (Paragraph
242)
21. There must be
incentives for clinicians to be very careful about the use of
expensive drugs. We recommend that current exclusion of high-cost
drugs from the payment by results tariff be reviewed. (Paragraph
243)
22. It is difficult
for individual PCTs to decide which areas to prioritise and in
which to reduce spending when their expenditure rises as a result
of new NICE guidance. In the absence of NICE guidance on disinvestment,
we recommend that groups of PCTs should work together to determine
appropriate areas of spending in consultation with the public.
Such groups should also examine existing treatments to determine
which are not cost-effective. (Paragraph 244)
23. While the measures
listed above would mitigate the problems PCTs face, the fundamental
problem which has to be addressed, according to several witnesses,
is NICE's cost-effectiveness threshold. Given the uncertainties,
for example about the thresholds used by PCTs, we are not in a
position to decide authoritatively whether the current threshold,
or threshold range, is appropriate. We recommend that more work
similar to that undertaken by Professor Smith and colleagues at
York University takes place on the thresholds used by NICE. We
are encouraged that NICE has commissioned its own research in
this area. (Paragraph 245)
24. During the inquiry,
doubt was cast on whether NICE alone should continue to determine
the level of the threshold. We consider the present situation
is unsatisfactory. We recommend that a separate body, with representation
from NICE, the Department, PCTs and others should set the level,
or range, to be used. NICE's threshold should be closely linked
to that used by PCTs. The threshold should also relate to the
size of the NHS budget. The new body should decide whether orphan
drugs continue to be treated differently from other treatments.
(Paragraph 246)
25. Demand for NHS
services will always exceed the ability to meet it. Not every
treatment can be provided to every person. NICE has a vital role
to play in the rationing arrangements and, working with Government,
should make clear to the public how and why such decisions are
made. (Paragraph 247)
26. There need to
be additional measures to improve the implementation of clinical
guidelines. There should be more help for PCTs to implement guidelines.
We recommend that the Department ensure that PCTs are aware of
the assistance that is available and develop other ways of helping
PCTs to plan and prioritise clinical guidelines. (Paragraph 294)
27. Better measurement
of guidance implementation is also needed. Self-assessment is
not enough. We recommend that the Healthcare Commission conduct
more in-depth inspections of this element of practice. (Paragraph
295)
28. Improvements to
the system of evaluating medicines and greater involvement of
experts in the technology appraisal and guideline development
processes should also result in guidance that is more acceptable
to clinicians. (Paragraph 296)
29. We also recommend
greater involvement of Royal Colleges and other professional organisations
in ensuring implementation. For instance, the approval of trusts
as training organisations could be linked to uptake of guidance.
Elements of clinical guidelines, particularly those covered by
technology appraisals, such as risk assessment of VTE patients,
should be mandatory. (Paragraph 297)
30. To combat public
confusion over the status of technology appraisals and other types
of guidance, we recommend:-
- Recommendations made following
technology appraisals should be referred to as 'NICE directives';
and
- Everything else should be referred to as guidelines
or guidance. (Paragraph 298)
31. Greater
involvement of PCTs in NICE assessments and a re-examination of
the NICE cost per QALY threshold, which we recommend above, would
produce guidance which NHS organisations find more affordable.
(Paragraph 299)
32. Given that discussions
between the Government and the pharmaceutical industry on future
drug pricing arrangements are already underway, we do not make
any firm recommendations on how a future system should operate.
However, we agree with the Government that better mechanisms are
needed to ensure that the NHS pays a fair and affordable price
for medicines. Any change to the system of medicines pricing is
likely to have profound consequences for NICE and the Institute
should be involved in any changes that might affect how it works.
Moreover, it should be funded for the alterations in practice
it might be required to make. (Paragraph 343)
33. We recommend that
risk-sharing schemes be used with caution. They should not be
used as a catch-all in cases of uncertainty over a drug's benefit.
The Department must bear in mind the evidence that will be foregone
in such cases. Uncertainty would be better addressed by the careful
design and performance of a publicly funded randomised controlled
clinical trial. Better use should be made of NICE's 'only in research'
recommendation in this regard. (Paragraph 344)
34. The short evaluation
of all medicines at launch, which we recommended earlier, could
be established in such a way that negotiations on drug pricing
could be incorporated into the process. The NICE evaluation process
could also take account of potential improvements in subsequent
data about clinical and cost effectiveness, and its consequences
for product pricing. (Paragraph 345)
35. To improve the
evaluation process, we recommend that:
- All drugs be assessed at the
time of licensing, so that clinicians can prescribe useful and
cost-effective products as soon as they are launched;
- There be more emphasis on disinvestment;
- Our last report on NICE recommended that the
legislation be changed to accommodate the need to ensure that
assessments of products take account of the wider benefits to
society; we make the same recommendation here;
- NICE have access to the same material used by
the licensing body, clinical trials be registered and there should
be closer working between NICE and the industry to enable these
early assessments to take place. (Paragraph 349)
36. The
affordability of NICE guidance and the range, measured in cost-per-QALY,
it uses to decide whether a treatment is cost-effective is of
serious concern. The threshold it employs is not based on empirical
research and is not directly related to the NHS budget, nor is
it at the same level as that used by PCTs in providing treatments
not assessed by NICE, which tends to be lower. Some witnesses,
including patient organisations and pharmaceutical companies,
thought NICE should be more generous in the cost per QALY threshold
it uses, and should approve more products. On the other hand,
some PCTs struggle to implement NICE guidance at the current threshold
and other witnesses argued that a lower level should be used.
However, there are many uncertainties about the thresholds used
by PCTs. Accordingly we cannot authoritatively at this stage recommend
a change in NICE threshold. Nevertheless, we recommend that it
be reviewed. We do recommend that an independent body determine
the threshold used when making judgements of the value of drugs
to the NHS. (Paragraph 350)
37. To improve the
implementation of NICE guidance we recommend:
- More help for PCTs to implement
guidance;
- Better assessment of the level of uptake;
- That PCTs should play a larger role in the development
of guidance;
- Better use of experts in the development of guidance;
- A change in the terminology used by NICE, to
clarify to patients what they can and cannot expect by right from
their local NHS organisation; and
- That some elements of clinical guidelines should
be made mandatory. (Paragraph 352)
38. We
recommend that risk-sharing schemes be used with caution. They
should not be used as a catch-all in cases of uncertainty over
a drug's benefit. Uncertainty would be better addressed by the
careful design and performance of a publicly funded randomised
controlled clinical trial. Better use should be made of NICE's
'only in research' recommendation in this regard. (Paragraph 354)
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