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Diana Brixner PhD, 2007-2008 ISPOR President and Associate Professor and Chair of the Department of Pharmacotherapy and
Executive Director of the Pharmacotherapy Outcomes Research Center at the University of Utah College of Pharmacy, Salt Lake
City, UT, USA
Comparative Effectiveness: What Are We Comparing?
A new term -- “comparative effectiveness” --
has been introduced to our outcomes
vocabulary, and many of us are struggling with
the definition. The reason for this uncertainty is
most likely a combination of perspectives,
national experiences, and roles--researcher, policy
maker or payer. As an international organization
of health care researchers and policy makers
from various settings including academia,
research organizations, industry, health insurers
and payers, we need to collectively embrace our
differences in perspective, and therefore the
alternative ways we may each define and utilize
the terms of our science.
The United Kingdom has a well recognized
approach for evaluating health technologies by
the National Institute of Health and Clinical
Excellence (NICE) [1], whereby the outcomes of
using all technologies are translated into a common
denominator based on the Quality Adjusted
Life Year (QALY), and judged by the incremental
cost per unit of health gain. This approach is
inherently a societal approach, focused on the
indirect benefit of quality of life and considered
across all therapeutic areas equally. Comparative
effectiveness in this context includes quality of
life as an effectiveness measure, and the costs to
gain that benefit.
Contrast this with the recently proposed
approach by the Institut für Qualität und
Wirtschaftlichkeit im Gesundheitswesen (IQWiG)
[2] in Germany, where the focus is on the benefit
vs. cost of a new therapy within a therapeutic
area only. Although the principle of not restricting
any benefit of therapy because of cost was
initially intended, the recognition of the non-sustainability
of such an approach has led to the
development of a system for the economic evaluation
of new drug therapies and other technologies.
The determination of value across therapeutic
areas is left to the decision making bodies
designated by law. In the German system it is
also important to note that the perspective is narrower
than society at large, and instead represents
that of the community of citizens insured
by the Statutory Health Insurance of the country.
In this case, effectiveness measures would be
the clinical outcomes, e.g., blood pressure lowering
for hypertension or A1C lowering for diabetics,
and the direct costs of technologies in
each of these areas to the insurance provider.
The listing of reimbursed medicines for
Australian citizens is called the Pharmaceutical
Benefits Scheme (PBS). The Pharmaceutical
Benefits Advisory Committee (PBAC) also considers
both cost and effectiveness, among other
factors, when reviewing an application to list a
new medicine on the PBS, referred to as a “value
for money” assessment. Once a medicine has
been recommended by the PBAC, it is referred to
the Pharmaceutical Benefits Pricing Authority
(PBPA) for consideration who then advises the
Health Minister on the pricing of medicines on the
PBS. Recommendations are referred to the
Minister, or to the Cabinet, if the estimated annual
cost to the PBS is
greater than AU$10
million in any of the
first 4 years of listing,
for approval. In this
system, comparative
effectiveness again
takes a broader view,
clearly including both effectiveness and cost.
In the United States we have been engaging in
ongoing discussions about the establishment of
a comparative effectiveness program intended to
provide better health outcomes and value nationwide.
However, the diverse system for delivery
and payment of health care in this market is not
conducive to a single approach in combining outcomes
and costs collectively in decision making.
Health care spending in the United States currently
accounts for 16% of the gross domestic
product (GDP). Despite the significant higher
level of spending-both as a % of GDP and in real
per capita terms-in the U.S. vs. other developed
countries such as Australia, Germany, and Great
Britain (where spending ranges from 7-10% of
GDP), there is little evidence of improved health
outcomes in this country [3, 4]. A formal comparative
effectiveness center will provide an
opportunity for clinical effectiveness and observational
data to complement currently available
information from selective clinical trials in controlled
environments. Collectively, these data can
enable clinicians to improve performance, consumers
to make more informed decisions, and
payers to set medical policies that improve quality
and value. In the United States, however, there
is an active debate about whether cost should be
an integral part of comparative effectiveness
assessments performed by a national body.
Perhaps the best approach currently for the
United States is for comparative effectiveness
research to explicitly inform considerations of
cost-effectiveness. At a minimum, recognizing
that comparative effectiveness should not focus
only on clinical outcomes, but should also consider
important measures of effectiveness such
as patient-reported outcomes, including healthrelated
quality of life, patient satisfaction, activities
of daily living, and work productivity as relevant
to the various U.S. stakeholders. In a letter
to the Congressional Budget Office, ISPOR leaders
have pointed out that if we appropriately execute
comparative effectiveness studies, then the cost-effectiveness analyses will improve
based on a more accurate denominator. We recognize
the diversity across the United States in
health care budgets, practice patterns, and cost
levels, and we acknowledge and recognize the
necessary flexibility in the interpretation of comparative
effectiveness and cost-effectiveness by
health plans, employers, government payers, and
policy makers. Therefore, the varied perspectives
of the U.S. prohibit a country-specific or
country-wide definition of comparative effectiveness-
and certainly cost-effectiveness.
Regardless of our individual or country perspectives,
the expertise of ISPOR's membership in
outcomes research, economics, and health policy
can assist in the ongoing discussions of these
important issues. For example, we must continue
to debate the appropriate scientific methods of
incorporating economic factors in assessments
in order to provide relevant information for decision
makers across perspectives. Clinical effectiveness
will be defined by data from randomized
clinical trials, retrospective and prospective
observational data, and other sources. Cost considerations
by patients, payors, providers, and
society as a whole will be incorporated by individual
stakeholders as appropriate to assure a
foundation for informed health care decisions.
As a collective membership of health care
researchers, policy makers and payors around
the world, where both comparative effectiveness
and cost-effectiveness information are integrated
into the health care decision making process in
various ways, we can all learn from our diverse
experiences.
References
1. http://nice.org.uk/
2. http://www.iqwig.de/download/08-01-24_Methods_of_
the_Relation_of_Benefits_to_Costs_Version_1_0.pdf
3. Orszag PR. (2007). Challenges of Health Care Costs,
Congressional Budget Office.
4. Wilensky GR. Developing A Center For Comparative
Effectiveness Information. Health Aff 2006;25: w572-85.
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