Rare Disease

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Rare Diseases: Natural History Studies for Drug Development Guidance for Industry

Published by FDA

Natural HistoryRare Disease

This 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. Although the focus of this guidance is rare diseases; the recommendations in the guidance may be applicable to drug development for nonrare diseases.

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New-born screening programs

Published by IRDiRC

Genetic ConditionNewborn Sreening ProgrammePatient InformationRare DiseaseResearch and Drug Development

The purpose of NBS is to detect potentially fatal or disabling conditions in newborns as early as possible and possibly before onset of symptoms. Such detection allows the early treatment which may significantly modify the natural history of the disease and potentially prevent developmental delays, physical disabilities and eventually death.

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