FDA Updates Information on Rare Neurodegenerative Disease Program

The FDA has updated information on its Rare Neurodegenerative Disease (RNDD) program.

The program was launched when the “Accelerating Access to Critical Therapies for ALS (also referred to as ACT for ALS) law was enacted in December 2021,” and it “supports research in amyotrophic lateral sclerosis (ALS) and other rare neurodegenerative diseases in adults and children.”

Under this program, “FDA awards grants and contracts to public and private entities to cover costs of research and development of medical products intended to prevent, diagnose, mitigate, treat or cure ALS and other rare neurodegenerative diseases in adults and children. This program supports research in ALS as well as other rare neurodegenerative diseases.”

Neurodegenerative diseases are diseases that occur when “nerve cells in the brain or peripheral nervous system lose function over time and ultimately die. In general, they are incurable and debilitating conditions and are progressive.” Rare diseases or conditions are those with “a prevalence of fewer than 200,000 people in the United States or in the case of an acute disease (e.g., less than one year duration), an annual incidence of fewer than 200,000 per year.”