by Scot Pittman
A recently released draft, five-year plan from the FDA includes procedures aimed at increasing the Agency's involvement in the area of orphan diseases. Rare, or orphan, diseases are those that affect less than 200,000 people in the United States; but with more than 7,000 known rare diseases, they are anything but rare and affect a significant number of people in the U.S. (30 million) and worldwide (250 million). Many of these diseases, however, lack meaningful treatment options for those afflicted.
FDA's draft, five-year plan includes meaningful steps to address the lack of treatment options for orphan diseases. Starting next year, FDA will complete a staffing and implementation plan for the Rare Disease Program of the Center for Drug Evaluation and Research ("CDER"). This will include adding five staff members and a liaison in the Rare Disease Program for the Center for Biologics Evaluation and Research ("CBER").
The draft plan outlines a continual program of guidance and policy dissemination that will aid FDA reviewers in staying informed about the latest in development of drug and biologic treatments for orphan diseases. The Rare Disease Program staff will also increase its outreach to the industry through public meetings seeking input on complex issues involved in the approval of drugs for these rare diseases.