US ‘eroding’ lead in rare diseases a matter of national security: Report - BioSpace
• A Congressional advisory panel has warned that the U.S. is losing its lead in rare disease drug development, labeling the trend a matter of national security. • To combat this, the panel recommends implementing supportive policies, including a finalized FDA platform designation and the adoption of novel trial designs for small patient populations. • The FDA is already taking action via a new pilot program to shorten the timeline from drug identification to first-in-human studies and improve submission quality.
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