FDA Releases Action Plan for Rare Neurodegenerative Diseases, Including ALS

Five-Year Plan Fulfills a Requirement of the Accelerating Access to Critical Therapies for ALS Act

Today, the U.S. Food and Drug Administration unveiled its Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis (ALS) – a five-year strategy for improving and extending the lives of people living with rare neurodegenerative diseases by advancing the development of safe and effective medical products and facilitating patient access to novel treatments.

“The effects of rare neurodegenerative diseases are devastating, with very few effective therapeutic options available to patients. We recognize the urgent need for new treatments that can both improve and extend the lives of people diagnosed with these diseases,” said FDA Commissioner Robert M. Califf, M.D. “To face that challenge and to accelerate drug development, we need innovative approaches to better understand these diseases while also building on current scientific and research capabilities. 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.”

The action plan is a blueprint for how the agency will move forward in aggressively tackling challenges in drug development for rare neurodegenerative diseases, including ALS, in order to improve patients’ health. Specific actions include regulatory science initiatives, enhancements to existing programs and new policy initiatives. The plan has been developed in accordance with the provisions of the Accelerating Access to Critical Therapies for ALS Act, or ACT for ALS, that President Biden signed into law on December 23, 2021.

Within a five-year span, the plan will focus on bolstering scientific advancement and promoting innovation for rare neurodegenerative diseases by engaging in targeted activities including:

Establishing the FDA Rare Neurodegenerative Diseases Task Force (FY 22).
Establishing the public-private partnership for rare neurodegenerative diseases (FY 22).
Developing disease-specific science strategies (FY 22 – FY 26).
Leveraging ongoing FDA regulatory science efforts.

The ALS Science Strategy is an element of the plan focused specifically on ALS. It provides a forward leaning framework for FDA activities to assess key regulatory science priorities. The focus areas of the ALS Science Strategy are to:

Improve characterization of disease pathogenesis and natural history —including quantifying disease progression, predictive and prognostic biomarkers, and efficient translation and implementation of basic science discovery is needed.
Facilitate patient access to new drugs whenever possible and promote greater participation in clinical trials by reducing barriers and burdens faced by diverse populations; utilizing digital health technologies and decentralized trial designs; and ensuring mechanisms for expanded access (generally outside of a clinical trial) are appropriately integrated into development programs.
Enhance clinical trial infrastructure and agility to enable early selection of promising therapeutic candidates for further development, optimize clinical trial design, improve access to the trials, streamline clinical trial operations, and reduce the time and cost of drug development.

Key to the success of the FDA’s implementation of the ALS Science Strategy will be patient engagement, public workshops, research projects, coordination across FDA centers and offices and collaboration with the National Institutes of Health (NIH).

ACT for ALS also requires the Department of Health and Human Services (HHS) to implement a Public-Private Partnership for Neurodegenerative Diseases between NIH, FDA and one or more outside entities through cooperative agreements, contracts or other appropriate mechanisms to advance the understanding of neurodegenerative diseases and foster development of treatments for ALS and other rare neurodegenerative diseases. It also directs the agency to award grants and contracts to public and private entities to cover the costs of research and development of interventions that are intended to prevent, diagnose, mitigate, treat or cure ALS and other rare neurodegenerative diseases.