FDA Orphan Designations At Record Levels; Resources Need To Keep Up
Executive Summary
The development of drugs and biologics for rare diseases has reached a regulatory milestone that underscores industry’s interest in the orphan drug market. FDA’s Office of Orphan Product Development is now granting one new orphan drug designation, on average, every day of the year. That activity underscores the success of the Orphan Drug Act, but creates a new problem: Finding the funding to support it.
You may also be interested in...
Political Appointees Shouldn't Influence Approval Decisions, Califf Says
FDA commissioner offers new details about why he left final decision on Sarepta's controversial Duchenne muscular dystrophy drug to FDA's expert bureaucrats.
Accelerated Approvals Convert Much Faster If Confirmatory Trial Already Underway, FDA Cancer Data Shows
Oncology Center of Excellence’s ‘Project Confirm’ finds that if a confirmatory trial is ongoing at the time of accelerated approval, conversion to regular approval takes an average of three years versus 4.9 years if the trial starts after AA.
CBER Staffing Remains Challenging, But Permanent Virtual Work May Help Offset Expected Retirements
Peter Marks predicts that staffing challenges within CBER can be alleviated by offering employees the flexibility to work remotely – permanently. With important posts in the vaccines office still to be hired, it could be an important incentive.