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FDA Rolls Out 5-Year Plan for Addressing Rare Neurodegenerative Diseases
The US Food and Drug Administration (FDA) recently announced its 5-year Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis (ALS). The plan was developed in accordance with the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS), which President Biden signed into law in December 2021.
“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,” said FDA Commissioner Robert M. Califf, MD.
Related: ALS Assessments Should Include Comprehensive Sensory Testing, Researchers Advise
A blueprint for how the agency will proceed through fiscal year 2026, the plan includes establishing a rare neurodegenerative diseases task force and creating a public-private partnership for rare neurodegenerative diseases between the FDA, National Institutes of Health, and outside entities. The plan also calls for developing disease-specific strategies, leveraging ongoing FDA regulatory efforts, and awarding grants and contracts to public and private entities to cover the costs of research and intervention development.
The ALS Science Strategy part of the plan focuses on a trio of priorities:
- improving the characterization of disease pathogenesis and natural history, including quantifying disease progression, predictive and prognostic biomarkers, and efficient translation and implementation of research findings;
- facilitating patient access to new drugs and promoting greater participation in clinical trials; and
- enhancing clinical trial infrastructure and agility to enable early selection of promising therapeutic candidates and reduce the time and cost of drug development.
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