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

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

In August 2024, FDA announced a new funding opportunity for the FDA Rare Neurodegenerative Disease Grant Program to fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare neurodegenerative diseases for children and adults. Applications are due October 22, 2024.

Through the establishment of the FDA 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 are 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.

For information about awards, see Rare Neurodegenerative Disease Grants Awarded. For current rare disease grant opportunities, including grants under this program, see Funding Opportunities for Rare Disease Research.

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 at https://youtu.be/mqEWQvK7Uyg.

Announcements

In February 2024, FDA published a peer-reviewed article, Trends in drug development for amyotrophic lateral sclerosis (Nature Reviews Drug Discovery), which discusses trends in orphan designation for treatments related to ALS since 1983.
The FDA Rare Neurodegenerative Disease Grant Program supports research in ALS as well as other rare neurodegenerative diseases in adults and children. In June 2024, researchers funded under this program published a peer-reviewed scoping review titled: Remote-Use Applications of the ALSFRS-R Clinical Outcome Assessment Tool: A Scoping Review (Value In Health), which discusses the findings of prior publications on the remote collection of data from assessments with this commonly used clinical outcomes assessment tool. Currently, for investigational studies, the ALSFRS-R requires an in-person assessment. The researchers analyze barriers and facilitators for the adaptation of a remote-use ALSFRS-R which will decrease travel burden on people with ALS and their care partners.
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.

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