FDA Orphan Disease Clinical Trial Grants Aim To Reduce Financial Risk
Executive Summary
Agency awards $22m to support 15 clinical trials and approximately $10m for six natural history studies in rare diseases. FDA Commissioner Gottlieb highlights the program as a way for the agency to help de-risk clinical development.
You may also be interested in...
Rare Diseases: Growing Understanding Will Unlock Accelerated Approval, FDA Says
US FDA officials say they need knowledge of the disease to have the confidence to use accelerated approval.
Keytruda's New Tissue-Agnostic Indication Could Prompt Orphan Policy Changes
US FDA contemplating how to match orphan designation process with drug development moving to molecularly-targeted therapies.
Companies Reveal Hurdles In Providing Drugs Via Expanded Access Programs
GSK, Stealth BioTherapeutics and Blueprint Medicines discuss the difficulties getting participation of physicians, the excessive cost of expanded access, and whether physicians should report research data.