FDA takes new steps to advance natural history studies for accelerating novel treatments for rare diseases

"Despite a recent wave of medical progress, most rare diseases still have no approved therapies. This presents a significant unmet public health need. It remains a top priority for the FDA to provide tools and clear advice to drug developers so that they can advance innovations that can help address these significant unmet medical needs," said FDA Commissioner Scott Gottlieb, M.D.

"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. 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.

One key way we’ve addressed these opportunities is by providing funding for rare disease natural history studies through our Orphan Products Grants Program. Another is to provide critical guidance, like the one we are issuing today that specifically addresses how to approach natural history studies. 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. 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The U.S. Food and Drug Administration today issued the draft guidance, Rare Diseases: Natural History Studies for Drug Development. This draft guidance is intended to help inform the design and implementation of natural history studies that can be used to support the development of safe and effective drugs and biological products for rare diseases.

Specifically, this guidance describes the broad 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.

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. Hearing directly from patients and caregivers may help the agency and medical product developers further understand and advance the development of treatments for rare diseases, potentially through the creation of novel endpoints or trial designs that focus on commonalities across a variety of rare diseases.

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