Critical Path for Rare Neurodegenerative Diseases Initiative for Drug Development

Effects of Neurodegenerative diseases are profound and devastating to patients and families with very few therapeutic options available. Moreover, many of these diseases also fall under rare disease category with few patients available to study and learn their natural history, or to enroll in clinical trials and evaluate drug effects. As such neurodegenerative disease such as ALS need particular attention from multiple stake holders to help develop therapies/cures for such diseases.

FDA and NIH jointly selected Critical path institute (C-Path) to convene the new public-private partnership: Critical path for neurodegenerative diseases (CP-RND) in 2022 whose mission it is to bring together experts in rare neurodegenerative diseases, private entities, patient communities and others with the goal of facilitating interactions and collaboration with FDA, NIH and external biomedical community to help accelerate and advance our understanding of disease pathology, develop treatment options, diagnostics, biomarkers etc.

This talk will be presented by the C-Path representative of the CP-RND project. It will cover the status of the project, specific work streams that include strategies to include and aggregate patient level data (clinical trial, natural history and real-world data), strategies to generate drug development diagnostic tools and biomarkers (including fluid, imaging and digital modalities), reasonably likely to predict surrogate endpoint candidates, and finally how industry and regulatory partners can work together under the framework that CP-RND has assembled.