Orphan Drugs & Treatment of
Rare Diseases
Drugs
for rare diseases-so-called orphan drugs-can be difficult to research, develop,
and market. Proof of drug safety and efficacy in small populations must be
established, but doing so is a complex process. Furthermore, because basic
research in the pathophysiology and mechanisms of rare diseases receives
relatively little attention or funding in both academic and industrial
settings, recognized rational targets for drug action may be few. In addition,
the cost of developing a drug can greatly influence priorities when the target
population is relatively small. Funding for development of drugs for rare
diseases or ignored diseases that do not receive priority attention from the
traditional industry has received increasing sup-port via philanthropy or
similar funding from not-for-profit foundations such as the Cystic Fibrosis
Foundation, the Huntington’s Disease Society of America, and the Gates
Foundation.
The
Orphan Drug Amendments of 1983 provides incentives for the development of drugs
for treatment of a rare disease or condition defined as “any disease or
condition which (a) affects less than 200,000 persons in the U.S. or (b)
affects more than 200,000 persons in the U.S. but for which there is no
reasonable expectation that the cost of developing and making available in the
U.S. a drug for such disease or condition will be recovered from sales in the
U.S. of such drug.” Since 1983, the FDA has approved for marketing more than
300 orphan drugs to treat more than 82 rare diseases.
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