The results are cause for celebration. The US Food and Drug Administration's OD program is a "tremendous triumph," commented Janet Woodcock, director of the Center for Drug Evaluation and Research (CDER), at a May conference commemorating ODA's 25th anniversary sponsored by the Drug Information Association (DIA). FDA has awarded OD status to 1850 products over the past 25 years. The awards have resulted in 326 approved medications that are used by about 12 million patients worldwide. Yet roughly 6000 to 8000 rare diseases still exist, Woodcock noted, so "we still have a very long way to go."
Woodcock expressed optimism that the emergence of molecular medicine will provide knowledge that can help identify more orphan conditions. Recent genomic discoveries can expand scientists' understanding of many serious conditions. In addition, personalized medicine promises more effective treatment and fewer side effects for many medicines. At the same time, high prices for many ODs, which reflect their development and production costs, create challenges for insurance coverage and patient access.Success story
Before the ODA, pharmaceutical companies did not invest resources in developing drugs that were unlikely to make a profit, noted Abbey Meyers, prime mover behind ODA and founder of the National Organization for Rare Disorders (NORD). Patient groups thus looked to academia and the National Institutes of Health (NIH) for new treatments. But mention of the rare-disease problem on the popular TV show Quincy and new approaches to spur industry involvement prompted policymakers to examine the problem seriously.
But the incentives for industry also generated controversy. Some orphan therapies such as human growth hormone became immensely profitable, raising questions about exclusivity and manufacturer "salami slicing," a strategy by which sponsors would seek OD status for a narrow indication and then promote the drug to much broader populations. "Many of the controversies over drugs that became profitable were spurred by competitors who lost the race to approval," Meyers commented. "NORD's greatest service has been to protect ODA from changes."