FDA Offers Plan for Developing Drugs for Neurodegenerative Disease, Including ALS

The FDA on Thursday released its action plan for developing therapies to treat neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS), following through on its duties under a law signed late last year.

The agency released the action plan in a statement.

The Accelerating Access to Critical Therapies for ALS Act (ACT for ALS), which was signed into law by President Joe Biden, requires the HHS to implement a public-private partnership for Rare Neurodegenerative Diseases through cooperative agreements or contracts. These agreements, established through the FDA and the National Institutes of Health (NIH), would help foster development of treatments for ALS and other rare neurodegenerative diseases.

The act required FDA to publish and implement a 5-year action plan in order to foster drug development and facilitate access to investigational drugs for ALS and other rare neurogenerative diseases. It also requires the FDA to award grants or contracts to public and private entities that would cover costs of research on and development of interventions that are designed to prevent, diagnose, mitigate, treat, or cure ALS and rare neurodegenerative diseases in adults and children.

Also included in the action plan were elements to facilitate

• interactions and collaboration between FDA and stakeholders,
• consideration of cross-cutting clinical and regulatory policy issues,
• identification of key regulatory science and policy issues critical to advancing development of safe and effective drugs, and
• enhancement of collaboration and engagement across FDA and with other operating divisions within HHS, the public-private partnership for rare neurodegenerative diseases, and the broader neurodegenerative disease community.

The ACT for ALS allocates $100 million for each of the fiscal years 2022 to 2026 to carry out these goals. The action plan is contingent on the FDA receiving these funds each year. The action plan is open for public comment from stakeholders.

“We recognize the urgent need for new treatments that can both improve and extend the lives of people diagnosed with [rare neurodegenerative diseases],” FDA Commissioner Robert M Califf, MD said in a statement. “This action plan, especially including the use of public-private partnerships and direct involvement of patients, will ensure the FDA is working toward meeting the task set forth by Congress to enhance the quality of life for those suffering by facilitating access to new therapies.