|
Corinna Sorenson MPH, MHSA, LSE Health, London School of Economics, London, UK; Michael Drummond PhD, Centre for Health
Economics, University of York, York, UK; Panos Kanavos PhD and Alistair McGuire PhD, LSE Health, London School of Economics,
London, UK
Does The Work Of The National Institute For Health
And Clinical Excellence (NICE) Have Any Relevance For
The United States?
Introduction
The growing emphasis on evidence-based decision-
making in health care, especially regarding
health technologies, has generated notable
debate and discussion in the U.S. around establishing
a more formalized process or system for
conducting comparative effectiveness research.
In the eyes of many observers, this is quite similar
to the HTA programs existing in a number of
European countries. The National Institute for three types of guidance, including technology
appraisals, clinical guidelines, and interventional
procedures. In mid-2005, NICE also assumed
the responsibilities of the Health Development
Agency (HDA), which provided the Institute with
authority to develop guidance on public health
interventions or programmes.
Regarding its role in health care decision-making,
NICE guidance essentially serves a quasi-law
function in the National Health Service (NHS) and
broader health care system. Beginning in January
2005, technology appraisals are supported by
mandate, in that the NHS in England and Wales
are now legally obligated to provide funding for
medicines and treatments recommended by
NICE. Specifically, if NICE guidance supports that
a particular technology be made available by the
NHS to a certain patient group(s), then associated
health care organizations are obligated to
implement such recommendations. Moreover,
NHS organizations are required to do so within
three months of the date the guidance is issued.
Topic Selection and Prioritization. To prioritize
assessments and initiate the guideline development
process, NICE receives suggested topics
from a number of sources. In general, the
Department of Health (DH) commissions NICE to
develop clinical guidelines, guidance on public
health, and technology appraisals, while topics
for the interventional procedures program are
submitted directly to NICE, usually by clinicians.
However, topics for potential NICE guidance are
also derived from health professionals; patients;
the general public; manufacturers; and, within
NICE itself. NICE compiles and maintains a list of
all submitted topics, which aids in ensuring
transparency.
Guidance Development. Guidance development
entails a number of key processes, from topic
selection to systematic review of evidence and
consultation on draft guidance. Several aspects
inherent to NICE's procedures lend themselves to an effective deliberative process, many of which
are focused on ensuring the highest degree of
transparency and the participation of a wide
range of stakeholders. NICE's engagement of a
broad representation of stakeholders, from multiple
sectors and disciplines, serves to introduce a
variety of perspectives into the appraisal and
decision-making process. This is particularly
helpful when reaching consensus on conflicting
evidence or recommendations; making such
judgments typically requires knowledge of the
scientific literature, realities of clinical practice,
and underlying social values. Moreover, as there
is a paucity of scientific evidence about patient
treatment preferences and viewpoints on issues
such as equity and fairness in health care, it is
important to involve a variety of stakeholders in
the process to elicit such perspectives. At the
implementation stage, a high level of stakeholder
involvement increases public and professional
ownership in the guidance, which enhances the
likelihood that it will effectively guide decisionmaking
and clinical practice.
Guidance Dissemination and Implementation. NICE employs different mechanisms to disseminate
its guidance, from publication on its website to
distribution to local decision-makers. Adequate
and timely communication and implementation is
crucial, as guidance is only effective if it is indeed
used by decision-makers to make health policy
and/or provision decisions. This is especially true
in the case of NICE, whereby its guidance carries
significant weight, in terms of NHS funding of and
access to health technologies. However, existing
evidence suggests that the uptake of NICE guidance
is often slow, patchy, and without adequate
incentives for implementation. There are several
factors that are considered to impact whether or
not NICE guidance is fully implemented, including
local political drivers, lack of provider support, deficient
knowledge and understanding of the assessment
process, media and patient group pressure,
limited mechanisms for accountability, and poor
financial planning of local decision bodies.
Relevance of Nice To The
United States
In the U.K., NICE is constituted as a Special
Health Authority within the NHS. Therefore, it has
an 'arms-length' relationship with government,
although all its funding comes from the DH.
Experience from the U.K. and elsewhere suggests
that an appearance of independence is
important for HTA agencies or entities, as the
findings of HTA reports are often controversial. A
survey of general practitioners in the U.K.
showed that NICE was perceived as being independent
from industry, but not independent from
government. Also, when NICE guidance is considered
by the media, the Institute is normally
referred to as 'the government's health watchdog'
or, occasionally, as 'the NHS's rationing body'.
In the U.S., the primary decision affecting the
governance, funding, and organization of any
HTA entity would be where it is located. That is,
should it be a new Federal agency, part of an
existing agency, or outside of government?
Regardless of the governance arrangement(s),
any public HTA entity will likely end up informing
decisions of a variety of payers, even if the entity
is only charged with guiding the decisions of
the Federal Government. Indeed, currently the
HTAs generated for the Medicare Coverage
Advisory Committee (MCAC) decisions are posted
on the CMS website and are available for consultation
by private health plans.
Remit for HTA Agency. On the international level,
NICE is probably in a minority among HTA entities
in having such a clear remit to consider costeffectiveness.
The recent debate in the U.S. has
been conducted using the term 'comparative
effectiveness'. For many commentators, the
study of comparative effectiveness would involve
consideration of clinical outcomes only, usually
through the conduct of clinical trials, comparing
relevant technologies in a real life (i.e., routine
practice) setting. On the other hand, some commentators
acknowledge that an assessment of
comparative effectiveness could also consider
costs.
Scope of Analyses. As the debate about comparative
effectiveness progresses in the U.S., the
breadth, or restriction, in the scope of the
required analyses will be a critical issue. At one
end of the spectrum, the HTA effort could focus
solely on the funding and conduct of clinical trials
to compare alternative technologies. Unlike
most of the trials currently funded by industry
(e.g., Phase III for drugs), these trials are likely to
compare two or more widely-used therapies,
enroll large numbers of patients, and have longterm
follow-up. They are also likely to be quite
costly, so their number will be limited, even with
the budgets currently being proposed for the
comparative effectiveness initiative, which at
present range from $4-6 billion a year. The current
funding level for NICE is more modest
(around $50 million), but crucially the Institute
does not commission primary research, such as
comparative clinical trials. Rather, it relies on
systematic reviews of the existing literature, in
addition to economic modelling. The main reason
for this is the emphasis on the timeliness of
the assessment. That is, since a decision has to
be made (on the appropriate use of a health technology),
the principal need is to develop the best
possible guidance given currently available data.
If, in the U.S., the emphasis in 'comparative effectiveness'
assessment were to be on large, longterm,
controlled trials, this would have to be
developed under a scheme similar to 'coverage
with evidence development', since the technologies
would have to be approved for funding in
order to allow such trials to take place. Under
'coverage with evidence development', funding for
the technology to be studied is contingent upon
participation in the clinical trials. Therefore, more
discussion of the design of such schemes,
including study requirements, funding and risksharing
arrangements (if any) is urgently required.
Setting Priorities. Priorities for assessment by
NICE are set by the government in the U.K.,
according to published criteria. If any HTA entity
in the U.S. were also servicing the decision-making
needs of the Federal Government, a similar
process could apply. For example, the topics
could relate to those technologies for which coverage
decisions are required. If an HTA entity in
the U.S. were seeking to be relevant to a wider
range of health care decision-makers, the nature
of the process for setting priorities is less clear,
although many private health plans also cover
Medicare enrolees.
Assessments vs. Appraisals. NICE makes a
clear distinction between assessments, where
only the technical analysis is undertaken, and
appraisals, where the evidence is evaluated and
the decisions made. The Institute relies heavily
on expert committees in its decision-making
processes (e.g., Appraisal Committee, Guideline
Review Panels) and this adds somewhat to the
appearance of independence. The experts are a
mixture of academics (across several disciplines
including medicine, statistics, and economics),
NHS decision-makers, and patient representatives.
There is no reason why a similar approach
should not work for an HTA entity in the U.S.,
although it may be more of a challenge to secure
adequate representation from the various decision-
making groups.
A more fundamental issue in the U.S. context is
whether an HTA entity would have a decisionmaking
role at all, given the diverse nature of the
health care system. It is possible, indeed more
likely, that the responsibilities of any entity in the
U.S. would cease at the assessment stage.
Assessments could then be made publicly available,
for the various payers to use (or not use) as
they see fit.
Summary and Conclusions
Although the increased use of HTA in the U.S.
may lead to a more cost-effective use of health
care resources, it will also increase the burden on
industry to produce data. In addition, to the extent
that HTA is linked to decisions about the pricing
and reimbursement of medicines, it will lead to
greater controls on the prescribing and use of
drugs. However, these wider considerations are
beyond the scope of this project. Although there
are considerable differences between the health
care systems in the U.K. and the U.S., several
important lessons can be drawn from the U.K.
experience with the NICE model:
Establish an Independent HTA Entity. The governance
and organization of any HTA entity is
critically dependent on whether its role is to serve
the decision-making needs of one major payer or
the needs of many decision-makers in a diverse
system. However, whatever its governance and
organization, it is important that any HTA entity is
as independent as possible.
Consider All Health Technologies. In order to
make the broadest impact, an HTA entity should
consider all health technologies (not just drugs).
Priorities for topics need to be set in an explicit
manner and assessments should be rigorous and
transparent, conducted in accordance with a
clear set of methods guidelines.
Involve All Major Stakeholders. Any HTA entity
should make strenuous efforts to involve major
stakeholders in the development of methods
guidance, the scoping of individual assessments
and in commenting on the results of studies.
Early and consistent involvement of technology
manufacturers is particularly important, as they
play a major role in conducting
the studies on which the assessments
are based. Ideally, assessments
should be carried out by
independent research groups,
under the general direction of the
HTA entity, and should be as
transparent as is possible.
Broaden Types of Evidence and
Improve Methods Synthesis.
There needs to be a debate in the
U.S. about the pros and cons of
focusing the HTA effort on the
conduct of additional large, longterm
randomized controlled trials,
versus investing more effort in improving the
methods of evidence synthesis using available
data. There also needs to be discussion of how
the timing of the production of more evidence on
health technologies is linked to decision-making,
perhaps through a system of 'conditional reimbursement',
or 'coverage with evidence development'.
Avoid Narrow Focus on Clinical Outcomes.
More discussion is required about the ways of
incorporating economic factors in assessments,
in order to provide relevant information for decisionmakers
in the U.S. context. A focus on clinical
outcomes alone is inappropriate as it may
exclude important advantages and disadvantages
of health technologies, such as impacts on quality
of life and savings in other health care
resources. On the other hand, calculation of a
single (incremental) cost-effectiveness ratio may
not be very helpful, given the great diversity
across the U.S. in health care budgets, practice
patterns, and cost levels.
Provide Evidence Interpretation, Not Guidance.
Finally, the most appropriate focus for an HTA
entity in the U.S. is on undertaking high quality
assessments (i.e., interpretation of the evidence),
rather than appraisals (i.e., the production of guidance
for decision-makers). The objective should
be to produce high quality assessments that will
enable various decision-makers to undertake an
appraisal from their own perspective.
References
[1] Sorenson C, Drummond M, Kanavos P, Mcguire A. National
Institute for Health and Clinical Excellence (NICE): How does it
work and what are the implications for the US? Report for the
National Pharmaceutical Council. Reston, VA, USA. Available at:
www.npcnow.org. |
