In this BioCentury op-ed, Dr. Joseph Muenzer joins other advocates in calling for a fit-for-purpose regulatory pathway for ultrarare diseases. Standard placebo-controlled clinical trials are often not feasible for these conditions, especially those affecting the brain where symptom onset and progression vary greatly between patients. These challenges can add years to the approval process, leaving patients without treatment while irreversible damage occurs.
Read the full article: FDA delays mean more deaths: Ultrarare diseases need a fit-for-purpose pathway.