Actual use studies are often called ‘slice-of-life’
studies. In the real world, what will this OTC
product do? It helps when inclusion and exclusion
criteria are minimized, as they are in the super-
market or pharmacy. Every effort should be made
to simulate the way in which patients will actually
use the drug. Eliminating large segments of this
population by strict admission criteria will simply
give a result that is irrelevant. In some cases, it may
even be necessary to even have patients pay for the
drug, in order to assess the motivational factors
associated with a purchase (they can be reimbursed
post hocand without their prior information).
In the same philosophical vein, it is important to
design the study for minimuminteractionwith the
patient. He/she must be left free to act, guided only
by the labeling. Intervention by the investigator
will only distort the results.
These types of studies are not unscientific. Even
if lacking well-matched placebo-controls among
others, there is still a hypothesis under test, and
these studies are addressing different sorts of ques-
tions. At the stage where a drug is being considered
for a switch, the umbrella question is, ‘What
impact will this drug have on the public health as
it will really be used by the lay public’? – the
central question that the Regulators and the Spon-
sor need to be answered.
Real-world studies are tests of the labeling as
much as they are tests of the drug itself. It is
essential that the combination of the drug and its
OTC labeling work closely together to enable
patients to self-treat effectively. Not only is a
great deal of creativity necessary in developing
effective labeling but appropriate label compre-
hension studies are also important in ensuring
that the best labeling is obtained. The labeling
may, in fact, make all the difference between
approval of the Rx-to-OTC switch.
Research has shown that patients by and large do
read labeling and they do heed it, particularly when
they are using OTC products that are unfamiliar to
them. Prior to any program being advanced to the
stage of the definitive clinical studies, it is wise to
develop a variety of different versions of the pro-
posed labeling, so that these versions can be tested
in label comprehension studies. These studies are
sometimes organized by the medical department
and sometimes they are carried out as market
research, as they need not involve actual ingestion
of drug. They consist of comparative studies in
which patients in a realistic setting read the pro-
posed labeling and then are quizzed on their com-
prehension of it. In this way, it is possible to see
whether they understand how the drug ought to be
used and whether they have understood key pre-
cautions. It is best to check both short-term and
long-term comprehension to see how well the
patients are able to remember what they have
learned. This sort of pre-screening of labeling can
be absolutely essential to success and it has saved
many careers by avoiding disasters in large-scale
definitive studies. Note that Institutional Review
Board/ethics committee approval may still be
required even when a drug is not being swallowed
because, at thevery least, therewill still be issues of
informed consent and confidentiality that must to
be accorded to participants when documenting
their experience of disease.14.6 Market support studies
The market support study is the second major class
of study that is used commonly to research OTC
products. These often involve active comparator,
head-to-head clinical comparisons between alter-
native formulations or against competitors. Only
authentic differences will emerge as successful
claims at the end of the study process.
Locating such possible advantages for quantifi-
cation in market support studies can be done
through usage and attitudes (U and A studies)
studies, usually performed by marketing depart-
ments. Focus group sessions can be invaluable in
discovering the possible existence of advantages
for a particular formulation over its competitors, as
well as individual interviews, and these are dis-
cussed elsewhere in this book.
Carefulreviewandsurveillanceoftheliteratureis
another way in which differences can be identified.
Theterm‘literature’shouldbeinterpretedloosely;it
should include the academic journals, newspapers,
magazines, patients’ newsletters and any and all
ephemera associated with the disease or drug of
interest. Even small differences may be quite188 CH14 THE UNIQUE ROLE OF OVER-THE-COUNTER MEDICINE