could be a clinical or a humanistic outcome, but
only needs to be measured once in a study.
23.3 Pharmacoeconomics
in development programs:
advantages, disadvantages
and challengesPharmacoeconomic tools will notmakea decision,
but are useful as an aid to decision makers regard-
ing the appropriate use of a product. While typi-
cally considered to aid the end user, pharma-
coeconomic data also have great applicability at
the drug development level. This is not an entirely
altruistic concern for the pharmaceutical company:
if incorporated early into the development of a
drug, a strategic advantage due to a more complete
package of outcomes information is available at the
time of product approval.
Pharmacoeconomic tools can also assist in
selecting an area of preclinical exploration, choos-
ing which drugs should moveforward into humans,
and whether to progress a drug from phase II to
phase III research. An understanding of the current
burden of the illness or condition, in terms of its
natural history, resource use and QOL profile, can
help a research team put the estimated develop-
ment costs and the desired return on investment in
proper perspective. A drug that ‘cures’ an illness
that is common but not very debilitating is not
likely to be seen as worthy of a premium price by
many formulary committees. This does not mean
that the drug should not be developed, but the
expected return on the drug must be put in the
appropriate context. Early research can also iden-
tify targets for comparative studies – a must under
the new paradigm. If research is conducted in the
most severe patients with a particular condition,
but they constitute only 5% of the treatable popula-
tion, then the perspective of those patients needs to
be put in context with other patients who suffer a
less severe form of the same condition. This will
help to demonstrate how patients with less severe
forms of the disease might respond to treatment
and todeterminewhat the impactof theconditionis
on their QOL.
Must pharmacoeconomic research delay devel-
opment programs?A perception exists that disad-
vantages of incorporating pharmacoeconomic
parameters into the development program neces-
sarily delays the filing of the new drug/product
licence application (NDA/PLA), while gathering
data that will not be ‘useful’. First of all, the same
statements could apply to anyclinical measure, and
every efficacy end point is carefully considered
before incorporation into a study or program. Simi-
larly, not every program requires all conceivable
pharmacoeconomic components. Acute treatments
(e.g. antibiotics for otitis media) may not require
QOL components; ‘me-toos’ (e.g. a newb-blocker
or a NSAID) may only require a simple cost com-
parison study.
Drugs for chronic use should be considered as a
prime target for pharmacoeconomic study. If a
disease is not going to be cured, and the patients
are expected to take a product for the rest of their
lives, there should be some message that can be
provided to the patients that will support their use
of the product in a compliant fashion for a number
of years.
It must be remembered that the ultimate objec-
tive of the pharmaceutical company is the success-
ful launch of a worthy new product. If phase III
studies are already completed by the time pharma-
coeconomic components are considered, the like-
lihood of having any outcomes data beyond
traditional safety and efficacy at product launch is
small and/or delay to gather such data is obligatory.
A strategic advantage for the product will haveTable 23.1 Examples of outcomes
Type of outcome Examples
Clinical Symptoms, diagnosis
Adverse events
Drug interactions
Economic Hospitalizations
Physician visits
Prescription drugs
Productivity
Humanistic QOL
Satisfaction with treatment
Preference for one
treatment versus another
294 CH23 PHARMACOECONOMICS: ECONOMIC AND HUMANISTIC OUTCOMES