Orphan Drug Act of 1983

The Orphan Drug Act of 1983 is a law passed in the United States to facilitate development of

Orphan drug designation does not indicate that the therapeutic is either safe and effective or legal to manufacture and market in the United States. That process is handled through other offices in the US Food and Drug Administration. Instead, the designation means only that the sponsor qualifies for certain benefits from the federal government, such as market exclusivity and reduced taxes.

In 1982 an informal coalition of supporters and families of patients with rare diseases who formed National Organization for Rare Disorders (NORD) and others, called for change to legislation to support development of orphan drugs, or drugs for treating rare diseases.^[2] They succeeded in getting the United States Congress to pass the Orphan Drug Act (ODA) in early 1983.^[2][3][4] Only thirty-eight orphan drugs had been approved prior to the 1983 Act; by 2014 "468 indication designations covering 373 drugs have been approved."^[5] Partly as a result of the 1983 US Orphan Drug Act, Japan adopted it in 1993 as did the European Union in 2000.^[5]

Background

Emergence of orphan diseases

In response to incidents such as difficulties with

• Tax incentives.
• Enhanced patent protection and marketing rights.
• Clinical research subsidies.
• Creating a government-run enterprise to engage in research and development.

Legislation

The plight of patients with rare diseases became an important political issue in the late 1970s and early 1980s. The US government was subject to pressure from activist groups such as NORD^[2] and many others.^[6]

The chief sponsor of the bill (H.R. 5238) was

Market exclusivity is particularly appealing to pharmaceutical firms as an incentive to pursue orphan drug development. The seven-year market exclusivity period differs from traditional patent law in that it does not begin until the drug is granted FDA approval and is independent of the drug's current patent status. Furthermore, if a market competitor wishes to introduce a drug for the same indication, the onus is on the competitor to prove that their drug is therapeutically superior (e.g. increased efficacy, less toxicity, etc.) when compared to the present drug indicated for the rare disease of interest. This incentive creates an attractive monopolistic market for companies interested in developing a product for any given rare disease.[6]

Television historian and

Drug companies nearly universally believe the ODA to be a success.[13] Before Congress enacted the ODA in 1983 only 38 drugs were approved in the USA specifically to treat orphan diseases.^[14] In the US, from January 1983 to June 2004, a total of 1,129 different orphan drug designations have been granted by the Office of Orphan Products Development (OOPD) and 249 orphan drugs have received marketing authorization. In contrast, the decade prior to 1983 saw fewer than ten such products come to market. From the passage of the ODA in 1983 until May 2010, the FDA approved 353 orphan drugs and granted orphan designations to 2,116 compounds. As of 2010, 200 of the roughly 7,000 officially designated orphan diseases have become treatable.^[13] In 2010, drugmaker Pfizer established a division to focus specifically on the development of orphan drugs^[15] as other large pharmaceutical companies focused greater efforts on the orphan drug research.^[16]

Some critics have questioned whether orphan drug legislation was the real cause of this increase (claiming that many of the new drugs were for disorders that were already being researched anyway, and would have had drugs developed regardless of the legislation), and whether the ODA has really stimulated the production of truly non-profitable drugs; the act also received some criticism for allowing some pharmaceutical companies to make a large profit off of drugs that have a small market but still sell for a high price. While orphan drug status is given to drugs with "no reasonable expectation" of profitability, some orphan drugs have gone on to net large profits and/or receive widespread use.

The [pharmaceutical] industry has taken advantage of the incentives to charge excessive profits and to reap windfalls far in excess of their investments in the drug.

— Henry Waxman, primary sponsor of the ODA

In an effort to reduce the burden on manufacturers applying for orphan drug status, the FDA and the European Medicines Agency (EMA) agreed in late 2007 to utilize a common application process for both agencies. However, the two agencies will continue to maintain separate approval processes.^[19]

See also

• Rare Diseases Act of 2002

References