Rare disease developers applaud clarity in FDA guidance
Several new guidance documents issued by the FDA are poised to significantly impact the rare disease development landscape.
Regulation
From real to regulatory: why RWD needs trial-grade standards
FDA appoints Tracy Beth Høeg as fifth CDER head this year
EuropaBio calls for “bold approach to investment” amid EU Biotech Act
FDA’s revolving door reveals Pazdur as new CDER head
OS endpoint focus will realign industry to “ultimate goal”
FDA creates new pathway for rare disease drugs, analysts unconvinced
Pharma’s AI prospects get nudged into the future with EU’s AI act
