research is often referred to as phase IIIb, if con-
ducted prior to or after dossier submission, but
prior to marketing approval. Other approaches
include the design and execution of clinically
important trials whose results are disseminated
principallyviatheir scientific publication to more
or less targeted, broader clinical audiences. These
approaches have in common the necessary require-
ments to adhere to principles of good clinical prac-
tice (GCP) and clinical trial ethics. Clinical trials
for any reason need to comply with the principles
outlined by the Belmont Report, the Declaration of
Helsinki and the International Council for Harmo-
nization (ICH) guidelines, and thus are hypothesis-
driven studies that appropriately take into consid-
eration the risk and benefit of clinical trial partici-
pation, and assure adequate statistical power to
maximize the likelihood of obtaining a result that
is scientifically rigorous and interpretable.
Other objectives of later phase clinical research
include addressing health authority commitments
after the drug has been approved (phase IV com-
mitments) which can focus either on safety or
efficacy questions that were not fully addressed
in the registration package, or the establishment
of patient registries to exclude the possibility of
very rare adverse events that require a very large
population-level exposure to reliably exclude spe-
cific adverse outcomes. Clinical trials of this sort,
in the phase IVenvironment, are pursued to provide
further information to health authorities and clin-
icians as to the long-term safety and efficacy of
drugs that otherwise have been demonstrated in
earlier clinical research phases in trials of generally
shorter duration. This includes the provision of
data showing efficacy and safety in pediatric popu-
lations, if this had not been the focus of the original
registration of the drug. The process, thereby,
accelerates the availability of valuable new medi-
cines, while at the same time fostering design
and execution of additional research to further
categorize the drugviathe conduct of these later
stage clinical trials or monitored use programs.
Additional research efforts conducted at the
Medical Affairs divisions of pharmaceutical com-
panies include the study of Health Economics and
Clinical Outcomes Research, which examines the
effect of drugs on the cost of healthcare delivery, as
well as effects on patient-reported outcomes such
as quality of life. In addition, useful summaries of
efficacy and safety can result from data mining of
pooled databases, conducted by clinical and statis-
tical personnel in Medical Affairs divisions, often
in collaboration with external scientific advisors, to
provide further information concerning the safety
and efficacy of marketed drugs.37.2 Phase III and phase IIIb
studiesClinical trials falling under the rubric of phase IIIb
are those that are performed with all the rigor of the
phase III registration program, but for a different
purpose. Most commonly, such studies, designed
while the phase III program is nearing completion
and often initiated prior to NDA submission and
conducted during agency review, are performed for
the purpose of data dissemination during or just
after launch of the new drug. The type of study that
is often performed is a randomized double-blind
comparator study to assess the safety or efficacy of
a drug, compared to another of its or another class
which is indicated for a specific medical condition.
Hence, these trials usually employ the same out-
come measures used in the phase III program.
Many other types of designs can be employed
including, but not limited to, single-blind studies,
open-label trials, crossover studies and other types
of clinical trials that aim to provide additional
efficacy or safety information that would assist
clinicians in assessing the value of the new drug
in their medical practice. Importantly, this is not
the same as increasing potential market uptake of
pharmaceuticals through ‘experience’ trials which
are not scientifically driven, but rather represent
hypothesis-driven clinical trials with clear scientific
rationale that are prespecified and adequately pow-
ered for, as documented in final study protocols.
The form of planned data dissemination will
often influence the type of trial conducted. In the
past, such choices could have included methods of
communication from simple data sharing at invited
scientific symposia to disclosure of researchvia
publication in peer-reviewed scientific journals. As
a response to the increasing scrutiny of medical520 CH37 MEDICAL AFFAIRS