Rare Disease R&D: FDA To Use NIH Registry For Natural History Studies
NIH plans to select two dozen groups to participate in a Global Rare Disease Patient Registry pilot project, which FDA hopes will lead to a better understanding of the conditions and speed drug development.
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Agency will make $2m available in 2017.
Straightforward draft document seems aimed at entities that have limited experience working with the agency.
Rare disease advocate says the focus should be on ensuring the accelerated approval expansion and surrogate endpoint guidances become FDA priorities as the rare disease drug program is set to grow under PDUFA V.