Natural History, Clinical Outcome Assessment, and Biomarker Studies of Rare Neurodegenerative Diseases (U01) Clinical Trials Optional

This grant, titled "Natural History, Clinical Outcome Assessment, and Biomarker Studies of Rare Neurodegenerative Diseases (U01)", is directed towards research involving neurodegenerative diseases that are not commonly encountered. The aim of this funding opportunity is to carry out studies concerning the natural development of these uncommon diseases. This could be done as a standalone study or in combination with processes to develop and verify the effectiveness of clinical outcome assessments (COAs) and/or biomarker studies. These are all aimed at tackling certain challenges faced in handling these rare neurodegenerative diseases in both children and adults.

Through this grant, the Food and Drug Administration (FDA) anticipates filling vital knowledge gaps in this research field and reducing the obstacles that are hampering progress. By focusing on a specific rare neurodegenerative disease or a group of these rare diseases with similar traits, this grant aims to effect a wide-ranging, significant impact. Additionally, it also hopes to expedite the development of products intended for treating such rare neurodegenerative diseases. Note that clinical trials are optional for this grant.