23.1 Outcomes, health economics
and pharmacoeconomicsOutcomes researchis the study of the end results of
medical interventions: Does the healthcare inter-
vention improve the health and well being of
patients and populations?
The field of outcomes research emerged from a
growing concern about which medical treatments
work best and for whom. Outcomes span a broad
range of types of intervention, from evaluating the
effectiveness of a particular medical or surgical
procedure to measuring the impact of insurance
status or reimbursement policies on the outcomes
of care. Outcomes research touches all aspects of
healthcare delivery, from the clinical encounter
itself to questions of the organization, financing
and regulation of the healthcare system. Each of
these factors plays a role in the outcomes of care or
the ultimate health status of the patient. Under-
standing how these factors interact requires colla-
boration among a broad range of health service
researchers, such as economists, sociologists, phy-
sicians, nurses, political scientists, operations
researchers, biostatisticians and epidemiologists
(Foundation for Health Sciences Research, 1993).
Health economicsoffers basic tools and criteria
with which to analyze these issues of efficiency and
the distribution of healthcare. These tools include
techniques of optimization and the determination
of equilibrium situations (e.g. predicting change in
demand for services). The set of criteria is used to
determine whether someone is better or worse off
as a result of a particular action. Health economics
tools are often used to evaluate how much money
should be allocated to a healthcare program or
service. To the extent that health economic ana-
lyses can clarify the costs of alternative medical
treatments and make the values underlying those
alternatives explicit, it is a useful approach to the
study of medical care (Feldstein, 1983). Health
economics focuses on all aspects of healthcare
and as such can be very useful for generating data
to make policy decisions involving multiple
healthcare programs and systems. While some
health economists take a ‘big-picture’ or macro-
view and focus on issues involving healthcare
policy, others may focus specifically on pharma-
ceutical industry issues such as drug pricing, or the
cost of drug development.
Pharmacoeconomicsis defined as the science
that identifies, measures and compares the costs
and consequences of pharmaceutical products and
services (Bootmanet al. 2005). As such, pharma-
coeconomics focuses primarily on pharmaceuticals,
and attempts to evaluate the economic and huma-
nistic impact of drug therapy. Pharmacoeconomic
toolsarederivedfrom avarietyofsources, including
the fields of economics and outcomes research.
Quite often, the pharmacoeconomist will bring to
thedevelopmentteamskillsandexperienceinasses-
sing QOL, patient satisfaction and other patient-
centered measures. Health economists and pharma-
coeconomists differ (while the terms are sometimes
used interchangeably), in having stronger back-
grounds in the theoretical and applied aspects of
health economics, respectively. A researcher with
solid pharmacoeconomic skills may not be a very
good health economist andvice versa. When hiring
pharmacoeconomists or health economists, first
determine what they will do, then evaluate their
skills and experiences to make sure that they will
be able to deliver what is needed for your specific
drug development program.23.2 New paradigm:
three-dimensional
outcome assessmentHealthcare used to be constrained mainly by the
technologies available to assist in delivering care.
As technology becomes increasingly sophisti-
cated, its cost is potentially outpacing the resources
available to pay for such care. Which patients
should get which treatment? How should health-
care be allocated, or in some cases rationed?
Health outcomesare the measured end results of
a medical intervention. They represent what hap-
pened to patients. Being cured of an illness is an
outcome, as is succumbing to it. However, this
radimentary, epidemiological distinction tells us
very little about the current functional status of
the patient. Being alive but relying on a respirator292 CH23 PHARMACOECONOMICS: ECONOMIC AND HUMANISTIC OUTCOMES