Orphan Drug Reviews: Industry Wants FDA To Move Out Of Its Comfort Zone
FDA may have to exit its drug review comfort zone and accept more trials outside the classic double-blind, placebo-controlled realm to promote more orphan drug development, industry and advocacy organizations told the agency
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Reviewer expertise may be better leveraged in more focused roles, but basic tenets of rare disease program should not fundamentally change as part of proposed Office of New Drugs reorganization.
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CDER Director Janet Woodcock says rare disease advocates should consider organizing patients to study a rare disease history and progression.