FDA earns plaudits for flexible approach to approval of drugs for rare diseases

Drugmakers should not fear investing in new medicines for rare diseases because federal regulators have shown considerable flexibility when approving them, says a new report from the National Organization for Rare Disorders.

The report was released Tuesday in conjunction with the start of a three-day U.S. conference on rare diseases and orphan drugs. Federal law offers financial incentives for drug companies to develop so-called "orphan" drugs for diseases that affect 200,000 or fewer U.S. patients, but the standard for federal approval is the same as for more mainstream medicines — even though the pool of test patients is much smaller.

"This review of [Food and Drug Administration] actions concludes that two of every three orphan drugs approved show FDA's historic flexibility in its review of effectiveness data on orphan drug therapies," study author Frank Sasinowski said in a statement. "Therefore, FDA has demonstrated in its actions on orphan products that it recognizes the importance of therapies for persons with rare disorders. It would be helpful for such flexibility and importance to be recognized in a formal FDA policy, and for FDA officials to incorporate and recognize that flexibility in a systematic way in their evaluations of each new therapy in development and under FDA review for Americans with any rare disease."