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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:

  • 1 Dosing Investigator
  • 3 Clinical Sites
  • 1 CRO
  • 2 FDA INSPECTIONS
  • 0 FDA SIGNIFICANT CONCERNS

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