FDA and NIH Launch Public-Private Partnership for Rare Neurodegenerative Diseases

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Today, 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 today 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.

To learn more about the Critical Path for Rare Neurodegenerative Diseases, contact the C-Path team at
CP-RND@cpath.org.

Additional Resources:

FDA Releases Action Plan for Rare Neurodegenerative Diseases, Including ALS

FDA Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis (ALS)

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NINDS is the nation’s leading funder of research on the brain and nervous system. The mission of NINDS is to seek fundamental knowledge about the brain and nervous system and to use that knowledge to reduce the burden of neurological disease.

About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit https://www.nih.gov.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.