FDA Issues New Guidance for Rare Diseases

Rare Diseases: Natural History Studies for Drug Development Guidance

On March 22nd, the FDA issued the draft guidance, Rare Diseases: Natural History Studies for Drug Development Guidance for Industry. A natural history study describes the course of a rare disease over time, identifying demographic, genetic, environmental, and other variables that correlate with its development and outcomes. Natural history studies can be prospective or retrospective.

Information obtained from a natural history study can be used to help to develop programs at every stage of drug development, from drug discovery to the design of clinical studies intended to support marketing approval of a drug and beyond into the postmarketing period, and even serve as comparator arms for single arm studies where placebo is impracticable.

This draft guidance is intended to help inform the design and implementation of natural history studies. Specifically, the guidance covers the strengths and weaknesses of various types of natural history study designs, common data elements and research plans, and a practical framework for the conduct of a natural history study.

In addition to this guidance, the FDA also provided funding support for rare disease natural history studies through the Orphan Products Grants Program which was initiated in 2016 and granted 6 Natural History Studies in 2017. Basically, Orphan products refer to drugs, biologics, medical devices, and medical foods that are indicated for a rare disease or condition.

“Because the natural history of many rare diseases remains relatively unknown, investing in rigorous models can help advance the development of treatments for these conditions.” – Scott Gottlieb, FDA Commissioner

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