Case Study
Clinical Operations
How we helped a rising biotech with a novel gene therapy product engage with regulators, investigators, and clinical sites as they dosed patients
By The Numbers:
- 100% Manual Processing
- 5 sprints in 1 year
- 7 countries globally
- 97.5% compliance KPI
- Trending towards
- 90% automated AE form intake
Challenge:
- Small gene therapy organization and a novel drug with potentially serious adverse events and a complex dosing procedure
- Limited global experience dosing the target patient population and therapy
- Currently dosing at a single site with challenging investigator
- Close monitoring by regulatory agencies and ongoing inspections
Approach:
- Supported the existing principal investigator to improve the relationship
- Prepared and guided the clinical site through FDA inspections
- Revised the protocol to reduce adverse events and improve patient safety
- Engaged additional clinical sites and investigators to diversify patient dosing
Results:
- Successful FDA inspection with no significant follow-up required
- Improved relationship with the principal investigator
- Additional dosing sites prepared, patients screened, and investigators engaged
- Closer monitoring of CRO leading to reduced expense