Rare Disease Day 2013

RDD_whiteToday, February 28th is Rare Disease Day.  Rare Disease Day is an international advocacy day to bring widespread recognition of rare diseases as a global health challenge. The day is celebrated on the last day of February every year.  In the United States, any disease affecting fewer than 200,000 people is considered rare.  Mesothelioma is estimated to be diagnosed in 3,500 American each year, making it very rare.

This year is a very important year for rare diseases, as 2013 marks the 30 year anniversary of the Orphan Drug Act (ODA).  The ODA changed the way that pharmaceutical companies viewed rare diseases, like mesothelioma, and the ways they sought to treat them.  The ODA created incentives for the development of treatments for Orphan diseases.  The ODA grants a special status called an “orphan designation” to a product to treat a rare disease or condition upon request.

The ODA was approved by the U.S. Congress in December 1982 and signed into law by President Ronald Reagan on January 4, 1983.  The ODA is considered very successful and has resulted in more than 2,700 potential therapies in the research pipeline and more than 400 approved products.[i]  Cancer was the main group of diseases that was targeted for orphan approvals.[ii]  Twelve of the drugs that have received an orphan designation for the treatment of mesothelioma, including the one FDA approved treatment that is currently available to mesothelioma patients, Alimta.

The Meso Foundation is a member of the National Organization for Rare Diseases, and is proud to represent the mesothelioma community in this regard.  We have joined other member organizations in meetings at the NIH and the FDA, and routinely work together in our advocacy efforts.

To learn more about Rare Disease Day, visit:

http://rarediseaseday.us/

To learn more about Orphan Drug Designations, visit the FDA website:

http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/HowtoapplyforOrphanProductDesignation/default.htm


[i] www.fda.gov Accessed February 20, 2013

[ii] Orphanet J Rare Dis. 2008 Dec 16;3:33. doi: 10.1186/1750-1172-3-33.  Incentives for orphan drug research and development in the United States. Seoane-Vazquez E, Rodriguez-Monguio R, Szeinbach SL, Visaria J.

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