Pharm-Olam Blog Importance of Natural History Study to Rare Disease Drug Development

Rare disease drug development varies from the pathways seen in more established indications. The sample sizes are often extremely small, and establishing a good base of clinical data can be difficult. Natural history studies help bridge the gap. Per the FDA, the natural history of a disease can be defined as “the natural course of a disease from the time immediately prior to its inception, progressing through its presymptomatic phase and different clinical stages to the point where it has ended, and the patient is either cured, chronically disabled or dead without external intervention.” A natural history study effectively allows medical professionals to collect information about an indication.