The Rare Disease Act of 2002 is a law passed in the

United States The United States of America (U.S.A. or USA), commonly known as the United States (U.S. or US) or America, is a country Continental United States, primarily located in North America. It consists of 50 U.S. state, states, a Washington, D.C., ...

that establishes the statutory authorization for the

Office of Rare Diseases The Office of Rare Diseases Research is a division of the National Center for Advancing Translational Sciences (NCATS) that oversees the Rare Diseases Clinical Research Network The Rare Diseases Clinical Research Network (RDCRN) is an initiative ...

as a federal entity able to recommend a national research agenda, coordinate research, and provide educational activities for researchers.

Background

rare disease A rare disease is any disease that affects a small percentage of the population. In some parts of the world, an orphan disease is a rare disease whose rarity means there is a lack of a market large enough to gain support and resources for discov ...

or disorder is defined in the U.S. as one affecting fewer than 200,000 Americans. There are more than 6,000 known rare diseases, and it is estimated that about 25 million Americans are affected by them (as of 2002). Prior to the RDA was the Orphan Drug Act of 1983 which was designed to facilitate the development and commercialization of drugs to treat rare diseases, termed ''

orphan drugs An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases. The assignment of ...

''. This act however did not provide for the creation of a centralized structure able to coordinate research or recommend agendas that would better facilitate research and education.

Legislative history

S. 1379 (Rare Diseases Act of 2001) was introduced in 2001 by Sen.

Edward Kennedy Edward Moore Kennedy (February 22, 1932 – August 25, 2009) was an American lawyer and politician who served as a United States senator from Massachusetts for almost 47 years, from 1962 until his death in 2009. A member of the Democratic ...

(D-MA) but died in committee. H.R 4013 was introduced by Rep. John Shimkus (R-IL) on May 20, 2002 and had 54 co-sponsors. It was signed into law on November 6, 2002.

Provisions

It establishes the Office of Rare Diseases under the

National Institutes of Health The National Institutes of Health, commonly referred to as NIH (with each letter pronounced individually), is the primary agency of the United States government responsible for biomedical and public health research. It was founded in the lat ...

. The law provided for a total of $24,000,000 in annual funding between 2003–2006.

Impact

The NIH established the

Rare Diseases Clinical Research Network The Rare Diseases Clinical Research Network (RDCRN) is an initiative of the Office of Rare Diseases Research (ORDR). RDCRN is funded by the ORDR, the National Center for Advancing Translational Sciences and collaborating institute centers. The RDCR ...

in 2003 with a $51 million grant over five years in response to the law. The network consists of seven Rare Diseases Clinical Research Centers (RDCRCs) and a Data and Technology Coordinating Center (DTCC). According to Stephen Groft, Pharm.D., director of NIH's Office of Rare Diseases "The network will facilitate increased collaboration and

data sharing Data sharing is the practice of making data used for scholarly research available to other investigators. Many funding agencies, institutions, and publication venues have policies regarding data sharing because transparency and openness are consid ...

between investigators and patient support groups working to improve the lives of those affected by these diseases and potentially prevent or eliminate these diseases in the future." The RDCRC's will be able to utilize the resources available at the 82 General Clinical Research Centers distributed across the United States. Since the program’s launch, nearly 29,000 participants have been enrolled in RDCRC clinical studies. As of October 2014, the network is composed of around 2,600 researchers which includes NIH staff, academic investigators and members of 98 patient advocacy groups. There are 91 studies underway. For fiscal year 2014, the NIH awarded a total of $29 million to expand the Rare Diseases Clinical Research Network and study 200 rare diseases.

References

{{reflist

External links

Text of the Rare Diseases Act of 2002

NIH Office of Rare Diseases Research
National Institutes of Health Rare diseases Acts of the 107th United States Congress United States federal health legislation