Rare Disease Drug Development Increases but Challenges Remain

In a recent study published by Tufts University, researchers found that rare disease drug development is now one of the fastest growing areas in drug R&D, with nearly one-third of all products in the global R&D pipeline targeting treatments for rare disease indications.

Yet while innovative biopharmaceutical companies have increased R&D investment in rare disease drug development in recent years, challenges remain. For example, the report indicates that:

Clinical through approval phase durations for rare disease drug development take on average four years longer than those for non-rare diseases; and clinical trials for rare disease drugs overall engage more investigative site to recruit fewer patients, reflecting the difficulties of patient identification and enrollment.

The truth is, 95 percent of rare diseases currently have no treatment options, thus maintaining the incentives of the Orphan Drug Act (ODA) remains as critical as ever for companies looking for essential investment for development in this space. Since the law’s passage in 1983, more than 500 new therapies have been approved by the FDA to treat orphan diseases — which impact 200,000 or fewer in the United States — compared to just 34 approvals before enactment.

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