Completing a natural history study for ultra-rare diseases can be critical to understanding the disease and its progression. The data from the natural history can be essential to informing the design and outcome selection of an interventional study. We explore the challenges of designing and running an extraordinary complex natural history study - with both retrospective and prospective data – which then had to pivot to virtual patient visits mid-way through the data collection process.
Our experts will share their experience, successes, and lessons learned on:
- The need for and challenges of a Natural History study of Canavan Disease
- Case Study: Strategies for addressing challenges of a unique study design
- Pivoting to a virtual & in-person hybrid model during COVID-19