Accelerating Access to Critical Therapies for ALS Act – ACT for ALS
![Brain graphic](https://public4.pagefreezer.com:443/content/FDA/14-02-2023T13:44/https://www.fda.gov/files/styles/main_image_medium/public/Brain%20Graphic_1200x675-01.png?itok=k0nPYH8X)
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.
- 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. Stakeholders may provide comments through November 28, 2022 on needs for future funding opportunities.
FDA posted a request for information (RFI) related to a brain computer-interface (BCI) focused systematic literature review and qualitative study in patients living with ALS, their caregivers, and their healthcare providers. This RFI will help the FDA plan for a potential request for proposals for contract services in 2023. The deadline for responses to the RFI is December 5, 2022 at 2 p.m. (EST).
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.
Additional Background Information
- FDA and NIH Launch Public-Private Partnership for Rare Neurodegenerative Diseases
- FDA Releases Action Plan for Rare Neurodegenerative Diseases, Including ALS
- 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
Contact Us
For meeting requests or inquiries, please email us at ACTforALS@fda.hhs.gov.
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