Critical Path for Rare Neurodegenerative Diseases

Overview

The Problem

Neurodegenerative diseases occur when nerve cells in the brain or peripheral nervous system progressively lose structure and/or function and ultimately die. Significant challenges remain in research and drug development for rare neurodegenerative diseases and advances are needed to improve the lives of those affected. 

The Solution

Critical Path for Rare Neurodegenerative Diseases (CP-RND), brings together multiple experts in rare neurodegenerative diseases, including ALS, as well as biopharmaceutical companies, regulators, patient communities and advocacy organizations to accelerate and advance our understanding of disease pathology, treatment options, diagnostics and drug development.  

C-Path has built a robust infrastructure within a pre-competitive framework that provides a safe harbor for commercial drug developers to bring data and shared learnings. This, combined with independent efforts from the FDA and NIH, provides a prime opportunity and unique global venue for accelerating drug and therapeutic development in rare neurodegenerative diseases. CP-RND also facilitates interactions and collaboration with the FDA and NIH, as well as the external biomedical research community to discuss cross-cutting clinical and regulatory policy issues, and foster consistent decision making. 

CP-RND is focused on increasing our understanding of disease pathogenesis and natural history by quantifying disease progression. These efforts include evaluating potential biomarkers from patient-level data and optimizing clinical trial design, including exploration of innovative trial designs, to improve the efficiency and success of drug development.

This, together with the capabilities of C-Path’s Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) integrates multiple patient-level data sources across rare diseases, providing the foundation for leveraging advances in basic and clinical sciences. 

The Impact

CP-RND aims to enhance the medical advances for rare neurodegenerative diseases, including ALS, by:  

• Bringing the experiences of those living with rare neurodegenerative diseases, including ALS, to the forefront of the discussion to inform researchers of the diseases impacts on daily life, what priorities patients have for research and treatment, key outcome measures they would find most valuable and best ways to improve their clinical trial experience.
• Leveraging expertise from industry partners, physicians, academicians and other key opinion leaders across disease areas to advance qualifications and regulatory tools for the broader CP-RND ecosystem.  
• CP-RND held its first conference for Rare and Orphan Diseases in September 2023 in Washington, D.C. The conference shared the program’s draft proposal for advancing research, harmonizing data, and working collaboratively across multiple research efforts to reduce burden on people with living with the disease and increase speed and efficiency of research.

Interested in participating in this collaboration? Please inquire and include your contact information at CP-RND@c-path.org. 

Patient Engagement

ALS is a relentless disorder characterized by the degeneration of both upper and lower motor neurons, leading to progressive weakness and paralysis.  In approximately 30% of cases, non-motor neurons in the frontal and temporal regions of the brain are also affected, causing impairments in various cognitive domains. The disease is uniformly fatal, with an average survival of only about three years after diagnosis, although some individuals succumb even faster and others survive for more than a decade. About 10% of ALS cases are inherited—typically as a dominant trait linked to a growing number of ALS-causing genes (“familial” ALS), while the remaining cases have no apparent family history of ALS (“sporadic” ALS).  The cumulative lifetime risk for ALS is estimated at 1:400, which predicts that more than 800,000 individuals now alive in the US will develop ALS.

CP-RND actively engages the ALS community through conference and participation, hosting Discussion Forums regularly throughout the year, and participation in collaborative efforts convened by other ALS community stakeholders. CP-RND will focus its efforts on landscaping activities to inform the design of new research projects supported through this initiative, generation of standards, and creation of drug development tools (biomarkers, digital health technologies, trial simulation tools, biological disease classification, novel COAs aimed at accelerating clinical trials in all potential persons impacted with ALS.

Collaborators

• Foundations for the National Institute of Health
• National Institute of Health
• U.S. Food and Drug Administration

Team