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.
Making Clinical Trial Recruitment More Efficient
Strategies for Accelerating Rare Disease Clinical Development
Rare disease trials differ from those in most other therapeutic areas because the indications are more complex and involve smaller patient…
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.