Clinical and regulatory considerations for rare disease therapy

MEDI 324

Marlene E. Haffner, marlene.haffner@fda.hhs.gov, FDA, Director, Office of Orphan Products Development, 5600 Fishers Lane, HF-35, Rockville, MD 20857 and Janet Whitley, janet.whitley@fda.hhs.gov, Food & Drug Administration, 1431 Harbor Bay Parkway, Alameda, CA 94502.
The US Orphan Drug Act (Act), signed in 1983, opened entire new horizons for the development of drug and biological products to treat patients with rare diseases. In the 10 years prior to the passage of the Act approximately 10 products were developed by pharmaceutical companies for the treatment of rare diseases. Since the passage of the Act, which includes incentives to both pharmaceutical firms and academic researchers, more that 285 products have been developed to treat these frequently life-threatening diseases. The development of products to treat rare diseases is still not easy. Patients may not be available for clinical trial enrollment or because the number of affected patients is very small, statistical significance may be difficult to demonstrate. The authors will discuss the issues surrounding the development of rare disease therapies as well as the incentives available to the developers.
 

Medicinal Chemistry of Rare Diseases
1:30 PM-5:00 PM, Wednesday, 13 September 2006 Moscone Center -- Room 103, Oral

Division of Medicinal Chemistry

The 232nd ACS National Meeting, San Francisco, CA, September 10-14, 2006