Considerations For Natural History Studies

One of the cornerstones of developing therapies for rare diseases is the use of natural history (or noninterventional) data to help inform the clinical development process. Though you may think natural history studies aren’t relevant or valuable to your clinical program, they may be worth keeping under consideration. Natural history data can play a surprisingly helpful role in the clinical development process, with many potential uses and benefits for drug developers.

WHAT ARE NATURAL HISTORY STUDIES?

Defining statements excerpted from the FDA Draft Guidance, Rare Diseases: Natural History Studies for Drug Development, March 2019:

• The natural history of a disease is traditionally defined as the course a disease takes in the absence of intervention in individuals with the disease.
• A natural history study is a preplanned observational study intended to track the course of the disease.
  • Its purpose is to identify demographic, genetic, environmental, and other variables that correlate with the disease’s development and outcomes.
• Natural history studies are likely to include patients receiving the current standard of care and/or emergent care, which may alter some manifestations of the disease.
• Disease registries are a frequent platform to acquire the data for natural history studies.