[Orphan drugs].
BACKGROUND
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.


CONCLUSIONS
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.
Univerzitet u Novom Sadu, Medicinski fakultet, Zavod za farmaciju. magistrakon@orcon.net.nz
Svetlana
S
Golocorbin Kon
Aleksandra
A
Vojinović
Mladena
M
Lalić-Popović
Nebojsa
N
Pavlović
Momir
M
Mikov
D055808:Drug Discovery; D006801:Humans; D009965:Orphan Drug Production / Q000191:economics* / Q000331:legislation & jurisprudence / Q000706:statistics & numerical data; D035583:Rare Diseases / Q000188:drug therapy*; D055771:Serbia