Article | September 10, 2019

The FDA's New Guidance On Natural History Studies In Rare Diseases: What You Need To Know

rare disease, patient voice

In March 2019, the FDA released draft guidance on the design and implementation of natural history studies to support the development of safe and effective treatments for rare diseases. The document, Rare Diseases: Natural History Studies for Drug Development, addresses one of the major challenges sponsors encounter when developing therapies for rare disease: the lack of natural history data to guide the design of successful clinical trials.

The natural history of a disease is the course it takes in the absence of investigational intervention. To gain insight into this, investigators conduct observational studies designed to follow the course of the disease. The objective of these studies is to identify demographic, genetic, environmental, and other factors that correlate with the development and outcomes of the disease.

Read more to learn about the important role natural history studies play in drug development and their benefits.

VIEW THE ARTICLE!
Signing up provides unlimited access to:
Signing up provides unlimited access to:
  • Trend and Leadership Articles
  • Case Studies
  • Extensive Product Database
  • Premium Content
HELLO. PLEASE LOG IN. X

Not yet a member of Clinical Leader? Register today.

ACCOUNT SIGN UP X
Please fill in your account details
Login Information
I'm interested in newsletter subscriptions.
ACCOUNT SIGN UP

Subscriptions

Sign up for the newsletter that brings you the industry's latest news, technologies, trends and products.

You might also want to: