Conducting clinical trials in rare diseases and advancing research pipelines requires a collaborative, multi-stakeholder approach. This process requires cooperation from industry, academic researchers, advocacy groups, and patients as well as their caregivers and families. In this white paper, we look at the barriers drug developers face when positioning a new trial in the rare disease space and how to minimize these barriers by applying a multi-stakeholder collaborative approach to drug development.
Achieving Challenging Recruitment Goals and Timelines in a Pediatric Type 2 Diabetes Study
Recruiting pediatric patients for type 2 diabetes mellitus (T2DM) studies is extremely challenging. Patients are difficult to identify and often face…
Successful Adaptation to the Evolution of Complex Dose Escalation Within a Large Master Protocol Basket Design Across IO Co-Therapies
A case study describing a Phase I dose escalation and cohort expansion study of an immune checkpoint inhibitor in patients with advanced solid…
Successes in Phase II Autologous Cellular Therapy Oncology Program
A case study describing two Phase II, multicenter studies assessing the efficacy and safety of an autologous cellular therapy treatment.