SECTION III
Special Populations and
Required Special Studies
Introduction
In 1993, the US Food and Drug Administration
(FDA), Europe’s Committee for Proprietary Med-
icinal Products (CPMP) and Japan’s Ministries of
Health and Welfare (MOHW) issued regulatory
requirements for testing and labeling in a ‘special
population’, namely the elderly. These were not
promulgated in isolation but after consultation
with academia and industry. In the United States,
initially this was done under the auspices of the
American Society of Clinical Pharmacology and
Therapeutics. Industry was allowed to participate
and was largely credited with aiding the process.
The First International Conference on Harmoniza-
tion (ICH) held in Europe (5–7 November 1991),
again involved the regulators and the regulated
and, for the first time, involved Japan as a
major contributor. As a result of pre-conference,
during-conference and post-conference discus-
sions, success was achieved. The ‘elderly’ drug
guidance was the forerunner of many future
tripartite agreements in the clinical area.
The special populations covered in the follow-
ing chapters include the four major demographic
segments: the elderly, women, children and major
ethnic groups; and although any smaller grouping
of people or diseases may be labeled ‘special’,
only renal and hepatic patients and orphan disease
have been included in this section. The four major
demographic segments were designated ‘special
populations’ because, despite the large size of
each segment (globally, women constitute 51%
of the population), pharmaceutical research has
been sparse in these groups. The basis for this is
multifactorial. Different responses to needs and
medicinal interventions, compared with that in
the White male population, have been only
sporadically addressed by the research, academic,
and industry pharmaceutical development
communities.
In general, globally and especially in the United
States, legislation controlling food and drugs
(including devices and biologics) has been stimu-
lated by therapeutic disasters. This, often in the
United States, caused the implementation of the
Food, Drug and Cosmetic Act of 1906, which out-
lawed the practice of embalming meat for con-
sumption. Further disasters triggered subsequent
multiple amendments to the Act.
In special populations, perceived omissions of
research and development have also resulted in
specific amendments to this Act. On occasion,
these amendments have been due to political pres-
sure from special advocate groups rather than a
specific therapeutic disaster.
Why did industry ignore these special popula-
tions, which represent major markets? First, the