Rare Disease Challenges At US FDA: CDER Director Says More Staff, Not Reorganization, Will Fix Issues
Executive Summary
Rather than changing the agency's structure to create more focus on rare diseases, Patrizia Cavazzoni wants funding for people to do the necessary work.
You may also be interested in...
Ultra-Rare Orphan Drugs: Advocates Begin Laying Groundwork For New Incentive, Special Pathway
Rare disease advocates push for National Academies of Sciences, Engineering and Medicine study on how or whether ultra-rare should be defined, which could create legislative pressure. In an interview, Ultragenyx CEO Emil Kakkis suggests patient thresholds, accelerated approval triggers.
Does The Orphan Drug Act Need A Tune-Up?
A better description of the US FDA’s regulatory flexibility may be necessary, but tinkering with the foundational law for rare disease drug development could open the door for more substantial and unwanted changes.
Rare Disease Patients: Placebo Controls, Sham Surgeries Not Appropriate For Gene Therapy Trials
Advocates also worry that long follow-up times could prevent participation in future clinical trials.