Orphan Drug Amendment

Passage of the first Orphan Drug Amendment to encourage development of drugs for small markets. 

Orphan Drug Amendments of 1983 Provided incentives for development of drugs that treat diseases with less than 200,000 patients in USA. 

The Orphan Drug Act of 1983, which amended the 1938 Federal Food, Drug, and Cosmetic Act, provides incentives for the development of drugs for treatment of diseases affecting fewer than 200,000 patients in the USA. The FDA maintains an office of Orphan Product Development to provide special assistance and grants to scientists with an interest in these products. Information on orphan drugs is also available from The National Organization for Rare Disorders. 

The estimated 130h pertains to Sec. Sec. 316.20,316.21, and 316.26. These apply primarily to initial applications/requests seeking orphan-drug designation. Many applications/requests received in the Office of Orphan Products Development contain multiple volumes include an exact duplicate copy of the original and may include 50 or more documented references. Additional information is requested when an application/request is denied. The sponsor usually supplies the requested information in the form of an amendment. 

In the Food, Drug, and Cosmetic Act of 1938, as amended, the location of what we affectionately refer to as orphan drugs is Subchapter B—Drugs for Rare Diseases or Conditions. The original parts of the subchapter were added in 1983, with amendments made in 1984 and 1985. 

In 1984, an amendment to the Act changed the standard for orphan drug designation from profitability to prevalence, which was set at less than 200 000 patients in the United States. The requirement of unprofitability was dropped from the Act. 

In 1985, another amendment to the Act made it possible for patented and patentable medicines to receive orphan drug designation (a pre-marketing classification) and orphan drug status (a post-NDA approval classification). 

In 1988, a further amendment established the time period for filing for orphan drug designation. This clarified that the designation must be prior to filing the NDA. 

In 1990, the US Congress passed a fourth amendment that would have allowed shared exclusivity for companies that developed an orphan drug virtually simultaneously and to lose exclusivity under certain conditions. However, this amendment was vetoed by President G. Bush, in whose judgment it was anticompetitive. 

An October 1993 amendment of the Pharmaceutical Affairs Law (Article 14-4) made provision for priority review for orphan drugs, orphan medical devices and innovative drugs or medical devices that have been authorised to be highly necessary from a medical standpoint . The new ordinance sets out standards to define both orphan drugs and the criteria by which innovative drugs will be decided for priority review. 

Congress passed the Orphan Drug Act (Public Law 97-414) in 1983, providing strong incentives for private firms to discover and develop treatments for rare diseases and conditions. Amended... 

The Orphan Drug Act authorized grants for clinical research on potential orphan products, and one of its more recent amendments (Public Law 100-290) extended this authority to preclini-cal studies. These grants represent a direct subsidy for orphan drug R D.31 The Office of Orphan Products Development administers the program in a manner parallel to other Public Health Service grants. 

Legislation to amend the Orphan Drug Act has been slow. Early in 1994 a compromise proposal was introduced, which would reduce the period of marketing exclusivity from 7 to 4 years, allow more than one version of an orphan drug if they were developed simultaneously, and allow withdrawal of exclusivity for orphan products when the patient population exceeds 200 000. Orphan drugs of limited commercial potential could be granted an additional 3 years exclusivity if sales information and related data justified it. ... 

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