FDA, NIH launch partnership for rare neurodegenerative diseases

The FDA and NIH announced the launch of the Critical Path for Rare Neurodegenerative Diseases, a public-private partnership aimed to advance the understanding of neurodegenerative diseases and the development of treatments.

The institutions have selected the Critical Path Institute, a nonprofit organization, as the convener of the 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,” FDA Chief Medical Officer Hilary Marston, MD, MPH, said in a press release from the association. “Collaboration across public and private sectors can accelerate the progress to address this urgent need. The partnership will leverage the shared expertise of all participants to create a path towards new breakthroughs in treating these diseases.”

According to the release, Critical Path Institute will bring together experts in rare neurodegenerative diseases, including patient communities, advocacy organizations and private entities.

Areas of focus will include patient-focused drug development, and the institution will use the FDA-funded Rare Disease Cure Accelerator-Data and Analytics Platform to bring together data to facilitate the characterization of diseases and their natural history; identification of molecular targets; and increased efficiency, predictability and productivity of clinical development of therapies.

“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,” Walter Koloshetz, MD, director of the National Institute of Neurological Disorders and Stroke, said in the release.