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Scott Gottlieb Stepping Down as Head of the FDA

The announcement on March 5th (in a staff letter which was shared on Twitter) that Scott Gottlieb, Commissioner of the FDA, would resign at the end of the month has created considerable speculation in the press. Along with questions about why (Dr. Gottlieb gave family...

Today is Rare Disease Day

The last day of February is designated Rare Disease Day, a campaign to raise awareness of rare diseases and their impact on patients, caregivers, and society. On this day local organizations host events to improve public knowledge of rare diseases and advocate for...

Draft Guidance on Rare Disease Drug Development

The CDER/CBER divisions of the FDA released a draft guidance in February 2019 describing various approaches to addressing challenges common in rare disease drug development. This latest guidance focuses on issues faced by sponsors in clinical trials; issues in early...