Rare diseases and orphan drug Act
Back Story Of The Orphan Drug Act
Ronald Reagan is remembered for many things, but few would associate his presidency with the subject of medicinals. Yet it was Reagan who signed into law the two acts that have, during the past twenty-five years, had the greatest impact on the American supply of medicines. In 1984, he signed the Drug Price Competition and Patent Restoration Act, sponsored by Henry Waxman, Democratic Representative from California, and Orrin Hatch, Republican Senator from Utah. Popularly known as the Hatch-Waxman Act, this law opened the gates for American consumers to enjoy the price advantage of generic drugs and antibiotics. It allowed the generic drug industry to develop into the major enterprise it is today, at the same time giving the Food and Drug Administration (FDA) the tools to prod that industry into improved manufacturing and quality control procedures so that American generic drug products are of equal quality to their brand name counterparts. The other major piece of drug legislation presided over by Reagan was the Orphan Drug Act, which he signed on January 4, 1983. The term “orphan drug” reflects the existence of diseases that are “orphaned” (i.e., little research or pharmaceutical study is conducted) because of their rarity. Research-intensive pharmaceutical firms tend to devote their resources to widespread conditions such as asthma, diabetes, and hypertension. Aside from these and other common ailments, however, it is estimated that there are more than 6, 000 rare disorders, which collectively affect some 25 million people in this country . Prior to 1983, these disorders were largely neglected by the drug industry.
It is just as difficult to discover and develop a remedy for a rare disease as it is for a common affliction and just as time-consuming and costly to satisfy all the necessary requirements to gain FDA approval. If the potential market comprises only a few thousand individuals, what return on investment can the drug firm expect? It is said that “big pharma” these days is not interested in a drug with potential sales of less than 100 million dollars.
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Rare Diseases Orphan Product Development Act of 2002 : report (to accompany H.R. 4014) (including cost estimate of the Congressional Budget Office) (SuDoc Y 1.1/8:107-702)
Book (U.S. G.P.O.)
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