Gaining Regulatory Approval Following A Single-Arm Phase I/II Study

A small biotech company engaged Veristat early in clinical development for help with a novel biologic being tested for treatment of an ultra-rare and aggressive hematologic malignancy with no available effective therapies. Using the results of a single-arm Phase I/II study, Veristat and the sponsor company collaborated to present a thorough, well-planned strategy for demonstrating the benefits and risks of the targeted therapy in this patient population to regulatory authorities. Based on Veristat’s significant experience, the sponsor engaged our team for strategic consulting, statistical and programmatic support, and medical writing to plan and execute the marketing application process, eventually leading to both FDA and EMA approval.