to breathe is very different from being alive and
fully functional. Additionally, intermediate out-
comes (e.g. alleviation of pain or other symptoms
ofarthritis) are sometimes as important an outcome
as the final outcome.
The measurement of outcomes is critical to the
conduct of pharmaceutical research. Clinical out-
comes (efficacy and safety) are the hallmarks of
Food and Drug Administration (FDA) approval of
a product for marketing. Clinical outcomes are
necessary but no longer sufficient as a sole con-
sideration in weighing decisions, and for reimbur-
sement in socialized healthcare systems (where
reimbursement essentially governs marketability).
Patients have become more involved in their
own healthcare decisions, and economic consid-
erations have increased in importance. All have
contributed to the movement to extend outcomes
measured beyond the traditional clinical outcomes
associated with pharmaceutical research. Health-
care decision makers are pressed to know more
than simply the safety and efficacy parameters of
an intervention. It is important for them to know
how a specific intervention will impact budgets and
use of other resources, and how it will impact the
patient from the patient’s perspective.
Pharmacoeconomic information demands are
often not anticipated early enough in the clinical
development program. For example, several mil-
lion people in the United States are taking antihy-
pertensive medications to lower their blood
pressure, something we would generally think of
as good, as the medications can possibly extend life
by reducing the risk of stroke and coronary artery
disease. However, in some cases the potential ben-
efits of antihypertensives may not outweigh the
negative effects of the drugs on QOL; one study
reported that the health of a person treated with
antihypertensive medication is comparable to that
of an otherwise similar person 5–15 years older.
Clearly, trade-offs between the side effects and
benefits of the medications should be presented to
patients so theycan make informed decisions about
treatment (Lawrenceet al., 1996).
If a pharmaceutical company is developing a
new antihypertensive medication targeted for
chronic use, then preparing a submission with a
goal of having the drug prescribed is an accom-
plishment. But it is also necessary to convince
patients to take the drug on a regular basis, as
well as to ensure that patients understand the
pros and cons of taking the medication from their
quality-of-life (QOL) perspective. An astute phar-
macoeconomic researcher incorporates a QOL
component into appropriate comparative studies,
so that patient-derived and patient-reported aspects
of treatment are considered in addition to the man-
agement of physiological symptoms such as blood
pressure reduction.
Although clinical outcome is critical, it is no
longer the sole factor reviewed in making a deci-
sion to use an intervention. Just as the information
requirements increased from safety to safety and
efficacy in the 1960s, the bar has been raised once
again, and these requirements now include not only
clinical (safety and efficacy) but also economic and
humanistic outcomes. This paradigm shift has been
represented in a model termed the ECHO (eco-
nomic, clinical and humanistic outcomes) model,
described by Kozmaet al. (1993). Economic out-
comes include direct medical resources used to
provide a service or achieve an outcome, including
healthcare providers’ time, laboratory services and
diagnostic procedures. Patient productivity is also
an economic outcome. Humanistic outcomes
include health-related QOL, patient satisfaction
with interventions and patient preferences.
Under the new paradigm for decision making, all
decision makers will increasingly be forced to take
into account the perspectives of the other players
affected by their decisions. Prescribers will no
longer consider just the clinical impact, but also
the economic impact their decision will have on the
payer, and the QOL impact the decision will have
from the patient’s perspective. The payer and
patients will need to consider the impact of their
decisions on the rest of the system. Successful drug
developers now evaluate three-dimensional out-
come data as early as possible in the product devel-
opment life cycle. This information will also be
useful to investors who are making decisions
regarding the ultimate potential for success or fail-
ure of a newly discovered therapeutic product.
Table 23.1 provides examples of clinical, eco-
nomic and humanistic outcomes. Each outcome
type is not mutually exclusive, for example painNEW PARADIGM: THREE-DIMENSIONAL OUTCOME ASSESSMENT 293