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Hello. This is Dr.
Steven Hirschfeld of
the Uniformed Services University
of the Health Sciences.
We are going to discuss
Pediatric Research
Laws and Regulations.
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We will begin with the evolution
from protection of children
to advocacy children.
In 1962, the US
Congress in Public Law
87-833 established
the National Institutes of
Child Health and Human
Development (NICHD).
A few years later,
Dr. Harry Shirkey invented
the term "Therapeutic Orphan"
to describe the situation
where sick children were
deprived of use of medicines
because they had not been
tested in that population.
In 1972, former FDA
Commissioner Charles Edward
noted that medicines
commonly prescribed
for children were
based on empiric grounds
and not scientific studies.
The same year, the National
Academy of Sciences
published a report describing
the different responses to
pharmacological agents based on
age and physiologic maturity
noting that plasma
concentrations
vary based on these factors.
Much as John Snow, in
London, observed about
two centuries earlier.
In 1973, the FDA commissioned
a report from the American
Academy of Pediatrics
on guidelines for the
evaluation of drugs to be
approved for use
during pregnancy
and for the treatment of
infants and children.
The FDA adapted the
report and published
it as an FDA guidance
document in 1977.
The same year Dr.
John Wilson found
that only 22% of
prescription drugs
had information in
the product package
describing how to use the
product to treat children.
Continuing our evolution from
protection to advocacy in 1979,
the FDA published an
important regulation
which introduced the concept of
pediatric use as a subsection in
the product package insert
in the section
called precautions.
1983, US Congress in Public Law
97-414 known as the
Orphan Drug Act
established the principle of
using government incentives to
promote product development in
an area of public health need.
The incentives consisted of
extending the period of
marketing exclusivity by
two years and providing
other means of support for
the development of products
that met the requisite criteria.
1988, Dr. Franz Rosa working at
the FDA surveyed
product package inserts
of drugs that were used to
treat children and found similar
to what Dr. Wilson found
several years earlier that
only 25% had information
that considered them
safe and effective.
In 1994, the FDA
issued a revision
of the pediatric use
subsection permitting the use
of efficacy extrapolation if
the disease in adults and
children were
sufficiently similar.
The use of extrapolation will be
explored in an additional
lecture, in greater detail.
In 1994, the NICHD,
the NIH Institute Dedicated
to child health research
established a research network
to study pediatric pharmatology.
Toward the end of the 20th
century, specifically 1997,
the Food and Drug
Modernization Act
extended the principle
of using incentives
from not just orphan
products, but for
the study of pediatric use
and selected drug products.
The following year,
the FDA issued a
regulation mandating
pediatric studies under
defined circumstances.
The regulation was
invalidated by
a federal court decision in
2002 based on the principle that
the FDA lacked the authority
to mandate studies in
a population that a drug sponsor
did not intend to market for.
