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On December 23, 2021, President Biden signed into law (Public Law 117-79), the “Accelerating Access to Critical Therapies for ALS Act” (ACT for ALS). 

The ACT for ALS, among other things, requires the Department of Health and Human Services (HHS), through the Food and Drug Administration (FDA) and the National Institutes of Health (NIH), to implement a Public-Private Partnership for Rare Neurodegenerative Diseases through cooperative agreements or contracts to advance the understanding of neurodegenerative diseases and foster development of treatments for ALS and other rare neurodegenerative diseases. It requires the FDA to publish and implement a 5-year action plan to foster drug development and facilitate access to investigational drugs for ALS and other rare neurodegenerative diseases. Finally, it requires that the FDA award grants or contracts to public and private entities to cover costs of research on, and development of interventions intended to prevent, diagnose, mitigate, treat, or cure ALS and other rare neurodegenerative diseases in adults and children.

FDA Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis

As part of its requirements under the ACT for ALS, FDA has developed a five-year plan describing actions the FDA intends to take to:

• Foster the development of safe and effective drugs that improve or extend, or both, the lives of people living with ALS and other rare neurodegenerative diseases; and
• Facilitate access to investigational drugs for ALS and other rare neurodegenerative diseases.

The intent of this action plan is to advance innovation that promotes and accelerates drug development for the treatment of rare neurodegenerative diseases including ALS. The action plan will be periodically reviewed, as appropriate, and may evolve over time.  

• The action plan is available here: FDA’s Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis 
• The public docket is available here: Public Docket no: FDA-2022-N-1279. We welcome stakeholder comments on the Action Plan. Comments should be submitted at www.regulations.gov under Docket no: FDA-2022-N-1279.

FDA Rare Neurodegenerative Disease Grant Program

Through the establishment of the FDA’s Rare Neurodegenerative Disease Grant Program, the ACT for ALS requires that the FDA award grants and contracts to public and private entities to cover costs of research on, and development of interventions intended to prevent, diagnose, mitigate, treat, or cure ALS and other rare neurodegenerative diseases in adults and children.  

These awards will be administered by the Office of Orphan Products Development which administers programs that provide incentives for sponsors to develop products for rare diseases.  Grants and contracts will be used for the development of tools, methods and processes to characterize the natural history of the neurodegenerative diseases, to identify molecular targets for these diseases, and to increase efficiency and productivity of clinical development of therapies.  

Additional Background Information

• Press Release
• Rare Disease Therapy Development and Access Remain Top FDA Priorities During COVID-19
• FDA Rare Disease Day 2022
• CDER's ARC Program
• Developing Products for Rare Diseases & Conditions
• Orphan Products Grants Program

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