Orphan Drugs

Prosperity and Misery

by Waldemar Janiec


People have always need drugs. Drug manufacturing costs are high. Pharmaceutical companies profit from manufacturing drugs to treat diseases, which occur commonly, in a large number of patients. They do not profit from manufacturing drugs for a small group of people, suffering from diseases which are difficult to treat and occur rarely. Drug which were so very needed to treat rare diseases were like abandoned orphans. That is why drugs to treat rare diseases, started to be called orphan drugs. The general term for referring to drugs needed to treat rare diseases as orphan drugs was quickly accepted throughout the world. The main reason was the passing of The Orphan Drug Act, in the United States, in 1983. The aim of this act was to create the financial fundaments for the search and manufacture of orphan drugs needed to treat rare diseases. The passing of this act was one of the breakthrough events in medicine. The act provided hope to find a way to treat diseases, for which previously there had been no cure.

The discovery of each orphan drug to treat rare diseases led through many paths. Some paths ended with success. others with failure. There were cases that great misfortunes contributed to the discovery of orphan drugs.

The showing of the difficulties and ways of searching for orphan drugs will allow for a better understanding of the fate and misery of orphan drugs, which are so very needed by those suffering from rare diseases.

The descriptions of facts and drugs in the book, are based on the quoted literature. The literature provided should enable the reader to reach the source of the information and enable him to formulate his own judgements of the facts described, which may be different than the author’s interpretations. The aim of the book is to show the aspects of orphan drugs from the side of social reception, that is why the chosen facts have been described in a belletristic version, where the description of the compound processes of biochemical or molecular changes, is either missing or has been significantly shortened.

On the cover photo: Michał Janiec,

Photography and cover design: Sławomir Janiec

Translated from the Polish edition by Szczepan Witaszek

Graphics: Arek Garcia, Cologne ©2014

© 2015 Copyright Waldemar Janiec and FBC-SOFT Sp. z o.o. Publishing House


About the author:

Prof. dr hab. of medical sciences Waldemar Janiec is retired academic of the Silesian Medical Academy (currently Silesian Medical University). He is a lecturer of Pharmacology and Pharmacodynamics and co- author of the VII edition of Polish Pharmacopeia. In his book “Orphan drugs. Prosperity and Misery”, he displays problems of treating rare disorders and describes many interesting events which accompanied the discovery of drugs, which have been given the name of orphan.

Publisher, Jakub Janiec

What are orphan drugs? Can drug even be orphan? Who needs such drugs? Does anybody produce orphan drugs and for what purpose?