- Established the Orphan Products Board within
the US government. - Allowed tax credits for certain expenses in clin-
ical trials. - Authorized a grant program that included med-
ical foods and medical devices, although med-
ical foods and medical devices cannot obtain
orphan designation. - Provided assistance to corporations and acade-
mic investigators by the FDA.
It is important to note that medical foods and
medical devices were not eligible for orphan
drug designation or for the marketing exclusivity
provisions of the Act. The Act was originally
designed for unprofitable and unpatentable medi-
cines only.
Amendments to the Orphan Drug Act
In 1984, an amendment to the Act changed
the standard for orphan drug designation from
profitability to prevalence, which was set at less
than 200 000 patients in the United States. The
requirement of unprofitability was dropped from
the Act.
In 1985, another amendment to the Act made it
possible for patented and patentable medicines to
receive orphan drug designation (a pre-marketing
classification) and orphan drug status (a post-NDA
approval classification).
In 1988, a further amendment established the
time period for filing for orphan drug designation.
This clarified that the designation must be prior to
filing the NDA.
In 1990, the US Congress passed a fourth
amendment that would have allowed shared exclu-
sivity for companies that developed an orphan drug
virtually simultaneously and to lose exclusivity
under certain conditions. However, this amend-
ment was vetoed by President G. Bush, in whose
judgment it was anticompetitive.
In 1991, a proposed amendment was proposed
that would have established a sales cap after which
an orphan drug would lose its exclusivity. This and
other amendments to the Act, proposed in 1992 and
1994, have not passed the Congress.
The benefits of the Act as it now exists actually
emanate both directly from the Act itself, as well as
from outside the Act.
Within the Act itself, the four major benefits
are (1) the period of marketing exclusivity,
which may be considered as a type of patent;
(2) the tax benefits on clinical trials between the
date of orphan drug designation and NDA
approval; and (3) the FDA’s Office of Orphan
Products Development grants to support clinical
trials on orphan drugs. A fourth benefit of
protocol assistance, from the FDA, was always
available for important new drugs (as well as
others) and is important, but not necessarily
new. Nonetheless, it is useful to call attention to
this provision.
The benefits from outside the Act (i.e. unofficial
benefits) are as follows:- Potential for more rapid regulatory review of
NDAs. This is considered extremely important
by many companies, but the author believes that
it is medical value of the treatment, rather than
the number of patients treated, that influences
the speed of regulatory review. - Enhancement of the company’s image by devel-
oping important orphan medicines. This often
can be parlayed into publicity that usually is
extremely important to the company. - Build a portfolio of products. Orphan drug ben-
efits may enable a company to develop and
market a new drug and help build a portfolio
of products in a therapeutic area of importance
to them. - Hope for a larger market. It is always possible
that a new use for an orphan drug may be found
that may give the medicine greater commercial
potential than originally believed. - Help fill important gaps in the company’s
development. There are often gaps in a com-
pany’s portfolio that orphan drugs could fill.
21.12 THE UNITED STATES ORPHAN DRUG ACT OF 1983 273