Sound drug development requires a comprehensive understanding of the disease being treated. To design reliable clinical studies and achieve meaningful outcome measures, researchers must apply known etiology and thorough knowledge of the disease’s progression. For rare diseases, this information is often minimal at best: patient numbers are small and historical data is spread across treating physicians who operate around the world.
It’s essential that pharma companies, patient advocates, and other stakeholders use data on the natural history of these diseases to drive discussion and formulate drug development strategy. There are two prevalent data collection approaches: registry studies, which may include broad collection of defined data, and natural history studies, used for controlled, detailed collection of data that is subject to regulatory scrutiny.
Key elements of this webinar will include:
- The importance of natural history information in rare disease research and the regulatory importance of comprehensive disease data
- The difference between registry and natural history studies, and how to decide which will better meet your needs
- How to design and conduct a registry or natural history study, and what resources are available to help you proceed
Angi Robinson, Executive Director, Pediatrics & Rare Disease, Premier Research
Juliet Moritz, Executive Director, Rare Disease & General Medicine, Premier Research