Orphan Drug Act of the 1980′s

From the US Food and Drug Administration:

The original definition of “rare disease or condition” in the Orphan Drug Act was amended in October 1984 by P.L. 98-551 to add a numeric prevalence threshold to the definition:

“…the term rare disease or condition means 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.”

The Orphan Drug Act (P.L. 97-414, as amended) includes various incentives that have stimulated a considerable amount of interest in the development of orphan drug and biological products. These incentives include tax credits for clinical research undertaken by a sponsor to generate required data for marketing approval, and seven years of marketing exclusivity for a designated drug or biological product approved by the FDA.

The FDA, through OOPD, funds the development of orphan products through its grants program for clinical studies.

Gaucher in the News

These are articles from my google alerts and other interesting articles I find about Gaucher’s Disease and Cerezyme in the news. There is so much that I need to learn, and I just don’t have the time yet, as we are trying to get Hannah’s medical treatment and insurance situation figured out. I’m posting these here mostly for me to keep track of and if anyone else is interested in learning what is new on the research front.

• First Patient Enrolled In Open-Label, Switchover Trial Of PrGCD For The Treatment Of Gaucher Disease
• Group Charged With ‘Playing God’ Over Genetic Testing
• Amicus Therapeutics Announces Positive Progress of Three Lead Clinical Programs
• New treatment has Plymouth boy with rare genetic disorder beating the odds
• Protalix BioTherapeutics – Gaucher disease case study” will be the keynote presentation
• Genetic Diseases and Dysmorphic Syndromes Gaucher’s Disease

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