FDA and NIH combining forces to further combat Rare Neurodegenerative Diseases

Sept. 15, 2022

The U.S. Food and Drug Administration and the National Institutes of Health (NIH) announced the launch of the Critical Path for Rare Neurodegenerative Diseases (CP-RND) – a public-private partnership aimed at advancing the understanding of neurodegenerative diseases and fostering the development of treatments for amyotrophic lateral sclerosis (ALS) and other rare neurodegenerative diseases. The FDA and NIH have selected the Critical Path Institute (C-Path) as the convener of this partnership.

“There is a crucial need to develop new treatments that can improve and extend the lives of people diagnosed with rare neurodegenerative diseases, including ALS. Collaboration across public and private sectors can accelerate the progress to address this urgent need,” said FDA Chief Medical Officer, Hilary Marston, M.D., M.P.H. “The partnership we are announcing will leverage the shared expertise of all participants to create a path towards new breakthroughs in treating these diseases. We look forward to working with NIH, C-Path, and other public and private partners to carry out this important effort.”

“This public-private partnership will convene the entire ALS community to develop novel strategies and approaches to therapy development and clinical testing with the goal to finally produce a treatment that stops the tragic progression of ALS,” said Walter Koroshetz, M.D., director of the National Institute of Neurological Disorders and Stroke (NINDS), part of the NIH.

C-Path will convene the partnership, bringing together experts in rare neurodegenerative diseases, including, but not limited to, patient communities, advocacy organizations, and private entities. The direction and priorities for the effort will be determined with input from the partners. Areas of focus will include patient-focused drug development, and utilization of the FDA-funded Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) to bring together scientific data on rare neurodegenerative diseases to facilitate the characterization of neurodegenerative diseases and their natural history, the identification of molecular targets for neurodegenerative disease, and increased efficiency, predictability, and productivity of clinical development of therapies.

Building on and leveraging the shared expertise of the participants, the goal of this partnership is to generate actionable solutions that can tangibly accelerate drug development for rare neurodegenerative diseases.

The Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act (Act for ALS) was signed into law on December 23, 2021 by President Biden and requires HHS, through FDA and NIH, to establish and implement a public-private partnership not later than one year after enactment. This partnership is a key component of the FDA’s Action Plan announced in June.

FDA release

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