This would provide a wide variety of benefits to
a company, including the stimulation of staff
who recognize the medical importance of the
product.- Have a new message and product for sales
representatives to give to physicians.
This may be an entry of importance to the
company.
21.13 Unintended consequences
of the Orphan Drug ActWhen Congress passed the Act, therewere several
different factions and intentions of those who
wanted an Act passed. All sides had to make
compromises, but everyone knew that some
incentives had to be given to pharmaceutical com-
panies in order for the Act to have any impact or to
influence behavior. Nonetheless, the incentives
worked too well in some people’s opinion in that
some of the Act’s loopholes were exploited or
were found to benefit companies in unintended
ways.
For example, companies with orphan drug pro-
tection sometimes charge very high prices, which
raised questions of appropriateness. In situations
where the medicine had a reasonably strong patent,
such as with Retrovir (zidovudine, azidothymi-
dine), the orphan drug designation and exclusivity
was not of consequence to the company, at least not
for market protection. For drugs such as growth
hormone, erythropoietin, pentamidine and Cere-
dase, orphan drug designation and resultant exclu-
sivity on FDA approval were essential. To some
people, the high price of the drug represented an
abuse of the Act.
An important issue for politicians was that the
Medicare and Medicaid programs had to pay large
sums of money for protected medicines. One final
issue to some people outside the pharmaceutical
industry, and also a few companies affected by the
issue, was the inability of a second company to
market a drug with an approved NDA. Of course,
this was clearly known for the entire history of the
Act and was the obvious consequence of marketing
exclusivity.
A current trend is that more biotechnology pro-
ducts (see Chapter 2.11) are applying for orphan
drug designation. The main reason for this phe-
nomenon is that biotechnology patents are so diffi-
cult to obtain and orphan drug protection is
valuable, while the inventors wait to see if a strong
patent will issue.21.14 Establishing prevalence
or incidence of a diseaseThe FDA recognizes any authoritative evidence to
support the prevalence of less than 200 000 patients
in the United States. The major sources of evidence
include the following:- Peer-reviewed literature.
- Textbooks.
- Surveys by patient support groups.
- Data from the National Disease and Therapeu-
tic Index. - Hospital discharge data based on ICD codes or
other clear classifications. - Data from the Centers for Disease Control.
- Data from the National Center for Health
Statistics. - Data from IMS or other reliable market data
organizations. - Sales data of companies.
- Testimony of a few experts, based on evidence
from their (or other) hospitals or practices. - Theweakest data is testimony of experts based
on personal experience unsupported by hard
evidence.
274 CH21 ORPHAN DRUGS