Ocata Therapeutics Inc fka OCAT

[rocky301]

3 decent reads on Orphan Status/approval and off-label drug use

Below is a decent read of "highlights" and the report is about one year old. No, I am not paying $3800 to get full report..lol

1)Opportunities in Orphan DrugsStrategies for developing maximum
returns from niche indications
http://www.globalbusinessinsights.com/content/rbhc0251m.pdf


2)Orphan Status Designations(Some FDA Stats)
The 1983 US Orphan Drug Act (ODA) established fiscal incentives for companies that produced promising drugs to treat rare diseases, defined as a disease that occurs in <200,000 people in the US. The OOPD recently published two articles assessing the characteristics and distribution of orphan designations and approvals by the US FDA. The first, by Braun et al, determined that the FDA granted 1,892 orphan designations from 1983 to August 2008, of which 326 received marketing approval. More than one drug can be designated for each orphan disease, and more than 200 diseases are represented among the approvals. Cancer was the most commonly designated category of disease and also had the greatest number of marketing approvals. The largest number of approvals for any condition was growth hormone deficiency (nine) and the second largest was AIDS (eight), which also had the largest number of orphan designations (57). Orphan drugs can be designated for more than one disease, with 1,243 drugs representing the 1,892 designations. In addition, there were 247 different drugs among the 326 orphan designated drugs that received marketing approval, 36 of which received two or more marketing approvals. Approximately half of the approvals occurred by four years after being granted designation, and the most common patient population size was fewer than 10,000 patients. In summary, there were only ten drugs that received FDA approval for the treatment of a rare disease in the 8-10 years before the ODA, thus the ODA may have contributed to a large number of drugs being developed for a variety of rare diseases, which has implications on future development and marketing of rare disease therapeutics.
http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/ucm217869.htm

3)Subject: Off-Label Drug and Approved Orphan Drug Use
Current Effective Date: 04/21/2010
Clinical UM Guideline
http://www.anthem.com/medicalpolicies/guidelines/gl_pw_a048553.htm