New FDA designation
The definition “Orphan drug” is commonly applied to a medicinal product intended to treat a disease so rare that sponsors are reluctant to develop them under usual marketing conditions.
Reluctancy is due to the fact that
The process from the discovery of a new molecule to its marketing is long (10 years in average);
drugs for rare diseases are almost never cost effective (development cost several tens of millions of euros );
success is not guaranteed (among ten molecules tested, only one may have a therapeutic effect).;
Although all these uncertainties about the benefits of therapy relative to the costs incurred in research and development to produce it, Pharmaceutical Industry continues to invest on orphan drugs R&D, not because of economic reasons but to respond to public health needs.
The Food and Drug Administration (FDA) is the American agency responsible for protecting and promoting public health through the regulation and supervision of human and veterinary drugs, biological products, medical devices, our nation’s food supply, cosmetics, and products that emit radiation.
FDA is also responsible for advancing the public health by helping to speed innovations that make medicines more effective, safer, and more affordable and by helping the public get the accurate, science-based information they need to use medicines and foods to maintain and improve their health. FDA also has responsibility for regulating the manufacturing, marketing and distribution of tobacco products to protect the public health and to reduce tobacco use by minors. Please visit FDA website http://www.fda.gov/ for more info.