FDA and NIH Launch Partnership for Rare Neurodegenerative Diseases

Earlier this week, the FDA and NIH announced their launch of a public–private partnership for research on rare neurodegenerative diseases. The partnership, called the Critical Path for Rare Neurodegenerative Diseases (CP-RND), will be facilitated by the Critical Path Institute (C-Path).

The goal is for the organization to assemble a group of individuals with a vested interest in rare neurodegenerative diseases. Researchers, patient communities, advocacy groups, and private organizations will all be stakeholders in this new 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, MD, MPH, in the FDA Press Release. “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.”

The launch of this partnership will provide a platform for drug development for rare neurodegenerative diseases. Additionally, the organization hopes to encourage the use of the Rare Disease Cures Accelerator-Data Analytics Platform (RDCA-DAP), an FDA-funded program.

Additional goals of this partnership include the following:

• furthering the understanding of rare neurodegenerative diseases and their history
• identifying molecular targets for the treatment and diagnosis of these diseases
• developing more efficient, predictable, and productive methods to treat these disorders

According to the FDA press release, “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.”

As this partnership expands and develops, it may provide a collaborative place for neurodegenerative disease research. Partnerships across multiple federal organizations may also garner additional resources that are allocated to these disorders. Physicians focused on neurodegenerative diseases are encouraged to follow up with this organization regularly to stay up to date on current technologies, therapies, and interventions.