Natural History Studies For Rare Diseases: Development Strategies For External Comparator Arms Leveraging Real World Insights

By Emily Bratton, PHD, MSPH,Cassie Holt, PHD, MPH, CPH, Sarah Kromrey, PHD, Jeffrey Keefer, MD, PHD

This paper explores essential principles for defining the right strategy for natural history studies and how to design such studies in a way that the data can be used as an external comparator to augment future regulatory filings and payer submissions. For more information, download the available white paper.