BioCentury
ARTICLE | Regulation

Signal crossing

Caregivers want more real-world, caregiver-reported outcomes in inborn errors

June 16, 2014 7:00 AM UTC

FDA's workshop on neurological symptoms in inborn errors of metabolism revealed conflicts between how caregivers would like symptoms assessed and what drug companies need to test to get therapies approved.

The June 10 workshop was the eighth in FDA's patient-focused drug development initiative commitment outlined in PDUFA V...