National Organization for Rare Disorders
Remarks Presented Before The
FDA Pulmonary-Allergy Drugs Advisory Committee
Silver Spring, Maryland
March 9, 2010

Good afternoon. My name is Diane Edquist Dorman. I am Vice President for Public Policy for the National Organization for Rare Disorders (NORD). I have no personal financial relationship with InterMune, Inc. From 2003 to 2005, however, NORD did administer an expanded access program on behalf of InterMune for perfenidone.

I am here today, not on behalf InterMune or their therapy under consideration by this Advisory Committee for the treatment of idiopathic pulmonary fibrosis. Rather I am here on behalf of the millions of men, women and children in the United States affected by one of the 7,000 known rare diseases that, in the aggregate affect approximately 30 million people.

Rare disease research and the development of orphan therapies to treat them are unique in many respects. Patient populations are generally very small and geographically dispersed across the United States, Europe and Asia, and few researchers and biopharmaceutical companies are willing to take on the financial risk associated with this vital work

For those reasons and many more, NORD has been dedicated to helping people with rare or "orphan" diseases, and assisting the organizations that serve them. We are the primary non-governmental clearinghouse for information on rare disorders, and we are committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and service.

Today there are nearly 350 orphan drugs and biologics that treat only about 200 rare diseases. Given that there are thousands more rare diseases without any specific treatment, it is easy to understand that there are millions of people who can only hope that one day someone will take on the significant financial risk to develop a therapy for their condition.


As you deliberate today, I ask only that you keep in mind that patients affected by rare diseases are willing to take on a far great degree of risk than those affected by more widely understood diseases affecting larger populations.

Thank you.

Contact Information: Diane Edquist Dorman
Vice President, Public Policy

National Organization for Rare Disorders
1779 Massachusetts Avenue, NW, Suite 500

Washington, DC 20036
Office/(202) 588-5700
ddorman@rarediseases.org

www.rarediseases.org
www.rarediseaseday.us