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...