KGI to Celebrate Rare Disease Day with Orphan Drug Workshop

Keck Graduate Institute (KGI) will celebrate Rare Disease Day on Feb. 28 by hosting its second annual workshop with the U.S. Food and Drug Administration (FDA) for pharmaceutical and biotech companies and academics to learn how to write and file an Orphan Drug designation application.

Hosted by KGI's Center for Rare Disease Therapies, the goal of the workshop is to simplify and demystify the application process so more companies and individuals will pursue development of drug therapies and medical devices for people who are suffering from rare diseases. Eighteen applications for rare disease therapies were filed after last year's workshop.

Rare diseases affect nearly 25 million Americans. No treatment is available for most of the 7,000 identified rare diseases, and for those that can be treated the cost to the patient is often prohibitive.

Dr.Tim Coté, director of the FDA's Office of Orphan Products Development, will conduct the workshop in which participants will be guided by FDA officers through the application process. Meetings will be conducted in private rooms to maintain confidentiality. Participants will be able to submit their proposals in person to the FDA at the end of the two-day workshop.

"This is a highly efficient way to get an application done correctly and into the hands of the FDA," said Ian Phillips, PhD, KGI's Norris Professor and director of the Center for Rare Disease Therapies.   

The workshop coincides with Rare Disease Day, an international effort to raise awareness about the difficulties of combating rare diseases since, for most patients, drugs and treatments are not available.

The Office of Orphan Products Development offers special incentives to companies to develop these products because there often is little profit in developing a drug for a disease that may afflict a small number of people.

To receive orphan designation, the proposed therapy must target a rare disease (one with a patient population of 200,000 or less), and it must show promise for treatment.

Companies or institutions that have a product for treatment of a rare disease and sufficient data to show it may be promising should send the following information to James Bona, FDA workshop coordinator, at james.bona@fda.hhs.gov or fax to 301-847-8621: names of proposed attendees; contact information; proposed orphan product, and the rare disease targeted.

Upon notification of acceptance to the workshop by the FDA, attendees must complete registration for the workshop at: www.kgi.edu/fda_workshop.xml