Clinical operations in rare disease trials have unique challenges due to small patient populations, limited understanding of the disease, and lack of established clinical endpoints. In this case study, we will explore:
- Adaptive trials designs to adjust clinical protocols based on interim results
- Specialized approaches to managing sponsor and site relationship
- Decentralized trials and leveraging technology to perform remote assessments
- Collaboration with patient communities and others
We may also discuss approaches that we previously employed but concluded as not suitable or too premature for rare disease trials.