Natural history studies for rare diseases

Natural history studies are epidemiological studies to describe and understand the features and evolution of a disease by collecting real-world data from patients.

They are particularly key to the development of new therapies for rare diseases because the data can be interrogated to provide insights into the disease and patient populations, identify endpoints for measuring therapeutic efficacy and identify patients eligible for trials. In addition, natural history data can even be used as a historical comparator in single-arm trials.

What will you learn in this webinar?

In this 90-minute webinar, Beacon and Healx were joined by speakers from industry, clinical research and patient groups to discuss:

• The importance of natural history studies to the rare disease community
• Best practices and how to overcome challenges when designing meaningful natural history studies
• The implementation of novel technologies such as machine learning to enhance data collection in natural history studies
• Examples of how the data from such studies can be applied to improve clinical trials and the pathway to therapeutic development