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Accelerating Access to Critical Therapies for ALS Act – ACT for ALS

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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.

Public-Private Partnership for Rare Neurodegenerative Diseases

On September 14, 2022, FDA and NIH announced the launch of the Critical Path for Rare Neurodegenerative Diseases (CP-RND) – a public-private partnership aimed at advancing the understanding of neurodegenerative diseases and fostering the development of treatments for amyotrophic lateral sclerosis (ALS) and other rare neurodegenerative diseases. The FDA and NIH have selected the Critical Path Institute (C-Path) as the convener of this partnership.

To learn more about the Critical Path for Rare Neurodegenerative Diseases, visit the CP-RND webpage here. To learn more about the Critical Path for Rare Neurodegenerative Diseases, contact the C-Path team at cp-rnd@c-path.org.

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.  

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. 

NEW: Request for Applications for FDA Rare Neurodegenerative Disease Grant Program, Deadline to submit applications: May 6, 2024

The U.S. Food and Drug Administration (FDA) announced a new funding opportunity for the FDA Rare Neurodegenerative Disease Grant Program to support efficient natural history studies alone or in conjunction with the development and validation of clinical outcome assessments (COAs) and/or biomarker studies to address the unmet needs in rare neurodegenerative diseases for children and adults. 

Through the support of studies with high quality and interpretable data elements, FDA expects to address critical knowledge gaps, remove major barriers to progress in the field, exert a significant and broad impact on a specific rare neurodegenerative disease or multiple rare neurodegenerative diseases with similar pathophysiology and facilitate rare disease product development.

The FDA Rare Neurodegenerative Disease Grant Program was established by the Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act. This program is administered by FDA’s Office of Orphan Products Development to promote medical product development for rare neurodegenerative diseases such as ALS. To apply, visit RFA-FD-24-024: Natural History, Clinical Outcome Assessment, and Biomarker Studies of Rare Neurodegenerative Diseases (U01) Clinical Trials Optional (nih.gov)

FDA, NIH, Critical Path Host Public Meeting on 2024 Priorities for CP-RND

On March 8, 2024, FDA and NIH partnered with the Critical Path Institute (C-Path) to host a hybrid in-person and virtual public meeting to share the 2024 priorities for Critical Path for Rare Neurodegenerative Diseases (CP-RND) public private partnership. Participants from C-Path, FDA and NIH provided updates on efforts focused on rare neurodegenerative diseases. You may access a recording of this meeting here: https://www.youtube.com/watch?v=ugzCROY3-jA

Critical Path to Rare Neurodegenerative Diseases (CP-RND): An Introduction to the Patient Community Webinar

On March 15, 2023, C-Path held a webinar to share an overview of CP-RND to the patient stakeholder group and provide the opportunity for feedback and questions from participants. You may access a recording of this webinar here: CP-RND: An Introduction to the Patient Community - YouTube

FDA/NIH ACT for ALS Stakeholder Engagement Meeting

On Aug. 24, 2022, FDA and NIH hosted a Stakeholder Engagement Meeting to provide updates on the ACT for ALS. You may access the recording of this meeting below or at https://youtu.be/mqEWQvK7Uyg.

Click Here to Watch

Announcements

  • On September 29, 2023, the FDA announced it has awarded more than $16.9 million in funding over the next four years to support important research through its Rare Neurodegenerative Disease Grant Program, including studies for amyotrophic lateral disease (ALS) as well as other rare neurodegenerative diseases such as Niemann-Pick type C and myotonic dystrophy type 1. This grants program was established under the Accelerating Access to Critical Therapies for ALS Act. See Rare Neurodegenerative Disease Grant Program for the full list of awardees. 
  • FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies. Selected participants of the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program will be able to obtain frequent advice and regular ad-hoc communication with FDA staff to address product-specific development issues, including, but not limited to, clinical study design, choice of control group and fine-tuning the choice of patient population. Under CDER’s eligibility criteria, the product must be intended to treat rare neurodegenerative conditions, including those of rare genetic metabolic type. The FDA will be accepting applications to the START program between Jan. 2, 2024, and March 1, 2024. More information on the program’s eligibility requirements can be found in the Federal Register Notice.   

Additional Information


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