The US Food and Drug Administration (FDA) has released its proposed guidance to industry, aiming to assist companies in the preparation of natural history studies. These are often integral to rare disease market access, as the nature of rare diseases is that relatively few patients have been followed through the disease course.
The guidance includes sections on:
- Uses of a natural history study
- Types of natural history studies
- Study protocol, data elements, and research plans
- Data collection, storage, and dissemination
- Human subject protection
- Interacting with FDA