Rare Daily Staff
The U.S. Food and Drug Administration issued draft guidance to inform the design and implementation of natural history studies that can be used to support the development of therapies for rare diseases.
Information obtained from a natural history study can play an important role at every stage of drug development, from drug discovery to the design of clinical studies intended to support a drug’s marketing approval.
The draft guidance describes the potential uses of a natural history study in all phases of drug development for rare diseases. It 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.
“One of the challenges we know innovators encounter developing therapies for rare diseases is the lack of natural history data to guide the design of successful clinical trials,” said FDA Commissioner Scott Gottlieb. “Such data comes from observational studies that track how rare diseases develop and progress over time. Sometimes rigorous natural history models can help inform development programs, and even serve as comparator arms for studies where it may be impractical to randomize patients to placebo.”
In April, the FDA is holding a public meeting to obtain patients’ and caregivers’ perspectives on the impacts of rare diseases on daily life and to assess their common experiences.