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 firstname.lastname@example.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.
NEW: The U.S. Food and Drug Administration (FDA) has a funding opportunity available for Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01).
The FDA’s Orphan Products Grants Program awards grants to clinical investigators to support the development of safe and effective medical products for patients with rare diseases. Clinical trial grants for orphan products are a proven method of successfully fostering and encouraging the development of new safe and effective medical products for rare diseases and conditions. The program has supported clinical research since 1983 and has funded clinical trials that have facilitated the approval of more than 80 products.
This program is administered by the FDA’s Office of Orphan Products Development. The deadline to submit applications is October 24, 2023. To learn more and apply, visit https://grants.nih.gov/grants/guide/rfa-files/RFA-FD-23-001.html.
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.
- 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.
- FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene
- 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
For meeting requests or inquiries, please email us at ACTforALS@fda.hhs.gov.
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