Debating a new pathway for ultrarare
Woodcock, patient advocates, biotech leaders call for reforms
Science has repeatedly thrown patients with devastating ultrarare diseases a lifeline, but all too often it has been snatched away before they can seize it. Scores of ultrarare therapies have been abandoned or stalled over the past four years, and many others have been envisioned but never developed, despite evidence that they could be effective treatments or cures.
The bottleneck is especially frustrating for patients and their families because they’ve seen how public policy can stimulate drug development. Despair over market failures has, on several occasions, led patients and their families to demand economic incentives for orphan drug development. Congress listened, enacting laws over the past 40 years that provided market exclusivity, R&D tax credits, and rare pediatric disease priority review vouchers. Hundreds of drugs for orphan diseases have been brought to patients as a result. Yet drug development for the rarest of these conditions remains a nearly impassable road. ...