4.Unprofitable drug for a common disease. This
category of drug is described in the orphan drug
legislation and could refer to tropical diseases
that are not prevalent in the United States or to a
drug that may be medically important for a
subset of patients with a common disease but
would not be expected to recoup the company’s
investment. Few drugs of this category have
been developed.
5.Variable commercial potential for both a rare
and common diseases. Virtually every pharma-
ceutical company that develops an orphan drug
hopes that the drug will be found useful in
treating a more common disease, but this sel-
dom occurs.
21.5 The interested parties
Eight groups involved in orphan drug development
and use are briefly discussed. These include
patients as well as both public and private institu-
tions. These parties have a variety of motives for
their interest in this area.
Legislatures
National, provincial and potentially other levels
of legislatures can become involved in orphan
drug development, primarily through creating
new legislation. They primarily influence devel-
opment through incentives that provide tax
benefits or grants, or otherwise incentivize phar-
maceutical companies to develop and market
such drugs.
Regulatory authorities
Their motivation is usually to improve and protect
the public health of the community they serve. This
is most obviously apparent through the approval of
orphan drugs for marketing. These authorities are
primarily motivated by their perception of the
drug’s medical value and less by whether the
drug is considered to have orphan drug designation
or the potential to help only a small number of
patients.Patient associations
These groups focus primarily on one specific dis-
ease or one type of disease process (e.g. inborn
errors of metabolism, muscle disease, glycogen
storage diseases, autoimmune diseases) or serve
as umbrella organizations representing the inter-
ests of their specific rare disease association mem-
bers [e.g. National Organization of Rare Disorders
(NORD)]. Their goal is to stimulate the discovery
and development of new treatments for their
specific diseases. Another important function of
many of these groups is to provide patient
information to their members, and often to the
public as well.Pharmaceutical companies
The motivation of these organizations is not
solely profit oriented in most cases, as they
usually accept social responsibilities for the
patients they serve with their more profitable
drugs. In addition to the small amount of profit
they may make on orphan drugs, there is an
enhancement of the company’s image, which is
becoming more and more important in our criti-
cal society.Trade associations
Professional trade associations representing phar-
maceutical companies or other groups are con-
cerned with the image of the industry, as well as
providing social benefits through publicizing the
products of their members. The Pharmaceutical
Manufacturers Association had a ‘Commission
on Drugs for Rare Diseases’ that focused on issues
relating to orphan drugs for many years.268 CH21 ORPHAN DRUGS