Rare Diseases Need Ongoing Natural History Studies To Speed Trials, FDA Says
CDER Director Janet Woodcock says rare disease advocates should consider organizing patients to study a rare disease history and progression.
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US FDA officials say they need knowledge of the disease to have the confidence to use accelerated approval.
Orphan Disease Natural History Grants Latest FDA Incentive For Drug Development
Agency will make $2m available in 2017.
As Orphan Drugs Gain More Attention, FDA Releases Development Guidance
Straightforward draft document seems aimed at entities that have limited experience working with the agency.