Criteria for BTD - hoping we can sell it on the last bullet Requirements for breakthrough therapy designation
To qualify for this designation, a therapy must meet two primary criteria:
- The therapy must target a disease or condition with significant morbidity or mortality rates. YES
- Early clinical data should indicate that the therapy offers substantial improvement over available therapies on one or more clinically significant endpoints. SORT OF
Substantial improvement in treatments is characters by three key factors:
- Efficacy, where the new treatment demonstrates better effectiveness than existing options.SORT OF
- Enhanced safety, with fewer or less severe side effects. YES
- A novel mechanism of action, particularly in addressing unmet medical needs. YES. This is the big one to me. One or two and done is an unmet need for rural and travelling patients. Mechanism is very novel.