NORD calls for FDA 'statement of policy' on orphan drug development

Noting that only about 200 of the nearly 7,000 diseases classified as rare currently have treatments, the National Organization for Rare Disorders (NORD) has called for a US Food and Drug Administration 'statement of policy' on regulation of therapies for rare disorders.

The NORD also urged the FDA to work with the research community to reduce regulatory uncertainty in the development of medicines for rare disorders.

"There are still about 5,800 rare disorders for which there are no FDA-approved therapies," said Frank Sasinowski, chairman of the NORD board of directors, in the opening address at a two-day public hearing sponsored by the FDA, adding: "Perhaps most discouraging is that many affected with these rare disorders do not even see any research being conducted on their conditions.