FDA’s Accelerated Approval Program creates an opportunity to bring promising treatments for serious conditions to patients sooner. Yet the pathway introduces a consequential planning challenge: the evidence that supports initial approval and the evidence required to verify clinical benefit must...
Paulla Dennis
The rapid advancement of cell and gene therapies (CGT) continues to reshape the clinical development landscape. As these innovative modalities move beyond ultra-rare diseases and academic institutions, clinical trial stakeholders must contend with a new set of operational realities—chief among...
In recent years, the pharmaceutical landscape for rare diseases has witnessed a remarkable evolution. Rare diseases, often referred to as orphan diseases, affect small patient populations, yet collectively they impact more than 300 million people worldwidei and account for a...