Biogen's Experience Highlights Challenges With Expanded Access Programs
Executive Summary
Expanded access program for rare pediatric disease treatment did not enroll many patients due to clinician community overwhelmed by patient demand and questions over who would pay ancillary drug costs.
You may also be interested in...
Biogen Spinraza Approval: The Perfect Antidote To Sarepta Headlines?
Approval of Biogen/Ionis’ Spinraza gives the drug development community a successful, collaborative model for a rapid pathway to full approval for a rare disease therapy; it also provides a timely, compelling rebuttal to claims that US FDA’s drug review operations are in dire need of reform.
Eisai/Biogen’s Leqembi Sees Clear Path To Regular Approval For Alzheimer’s Disease
US FDA will ask its advisory committee to weigh anti-amyloid drug’s benefit-risk in populations at higher risk for adverse events, but agency says lecanemab’s risks ‘do not appear to preclude traditional approval’ and the CLARITY AD trial demonstrated clinical efficacy and a reduction in brain amyloid.
FDA Staffing: Retirements Account For More Than One-Third Of FY 2023 Departures
Combination of an aging workforce and departures that were delayed by the pandemic are taking a toll on the agency’s current staffing levels, Chief of Staff Julie Tierney says.