Introduction. Drugs used for treatment of rare diseases are known worldwide
under the term of orphan drugs because pharmaceutical companies have not been
interested in ?adopting? them, that is in investing in research, developing
and producing these drugs. This kind of policy has been justified by the fact
that these drugs are targeted for small markets, that only a small number of
patients is available for clinical trials, and that large investments are
required for the development of drugs meant to treat diseases whose
pathogenesis has not yet been clarified in majority of cases. The aim of this
paper is to present previous and present status of orphan drugs in Serbia and
other countries. The beginning of orphan drugs development. This problem was
first recognized by Congress of the United States of America in January 1983,
and when the ?Orphan Drug Act? was passed, it was a turning point in the
development of orphan drugs. This law provides pharmaceutical companies with
a series of reliefs, both financial ones that allow them to regain funds
invested into the research and development and regulatory ones. Seven years
of marketing exclusivity, as a type of patent monopoly, is the most important
relief that enables companies to make large profits. Conclusion. There are no
sufficient funds and institutions to give financial support to the patients.
It is therefore necessary to make health professionals much more aware of
rare diseases in order to avoid time loss in making the right diagnosis and
thus to gain more time to treat rare diseases. The importance of discovery,
development and production of orphan drugs lies in the number of patients
whose life quality can be improved significantly by administration of these
drugs as well as in the number of potential survivals resulting from the
treatment with these drugs.
Incentives for Pharmaceutical Companies to Develop Treatments for Rare Diseases: A Review of the Literature
