C-Path secures FDA Grant for new public-private partnership to benefit people with rare neurodegenerative diseases

Reviewed by Emily Henderson, B.Sc.Sep 14 2022

Critical Path Institute (C-Path) has announced it will serve as the convener of the Critical Path for Rare Neurodegenerative Diseases (CP-RND), a new public-private partnership (PPP) to benefit people across multiple rare neurodegenerative diseases, supported by a grant from the U.S. Food and Drug Administration (FDA). The Agency announced the PPP today in a press release.

These innovative and collaborative approaches are needed to accelerate medical product development for the benefit of individuals living with these rare diseases, and their families. I am thrilled about this partnership that will strengthen collaborations between C-Path, FDA and NIH, and bring together a diverse group of stakeholders."

Wainwright Fishburn, C-Path Board Chair

C-Path's proven proficiencies will be leveraged to ensure the success of this effort, specifically the organization's track record in generating tangible solutions that have accelerated drug development in numerous diseases. This, together with the capabilities of its Rare Disease Cures Accelerator-Data and Analytics Platform(RDCA-DAP ®) to integrate multiple patient-level data sources across rare diseases, will provide the foundation for leveraging advances in basic and clinical sciences, supported by NIH, and innovative regulatory science, supported by FDA.

In June, the FDA unveiled its Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis (ALS) -; a five-year strategy for improving and extending the lives of people with rare neurodegenerative diseases, including ALS, by advancing the development of safe and effective medical products and facilitating patient access to novel treatments. The plan was developed in accordance with the provisions of the Accelerating Access to Critical Therapies for ALS Act, including the requirement to establish a public-private partnership this fiscal year.

"It is an honor for C-Path to be part of this transformative partnership with FDA and NIH. CP-RND will leverage all five of C-Path's core competencies -; data management and standards, quantitative analytics and modeling, biomarkers, clinical outcome assessments and regulatory science -; as well as C-Path's concentration areas from its neuroscience/neurology, pediatrics, and rare disease programs," explained Klaus Romero, M.D., M.S., F.C.P., C-Path's Chief Science Officer and Executive Director of Clinical Pharmacology. "This partnership with FDA and NIH will positively transform medical product development and make CP-RND a template for comprehensive and meaningful collaborative science."

As with previous PPPs at C-Path, CP-RND will provide a neutral environment for pre-competitive collaboration including, but not limited to industry, patients, advocacy groups, academia, non-profit organizations, and others, to come together and actively contribute to advance promising solutions for the benefit of these communities. To learn more about C-Path's CP-RND, visit https://c-path.org/programs/cp-rnd and contact the team at [email protected].