FDA Releases Action Plan for Rare Neurodegenerative Diseases, Including ALS

The U.S. Food and Drug Administration unveiled its Action Plan for Rare Neurodegenerative Diseases, a five-year strategy for improving and extending the lives of people living with rare neurodegenerative diseases by advancing the development of therapies and facilitating patient access to treatments.

“The effects of rare neurodegenerative diseases are devastating, with very few effective therapeutic options available to patients,” said FDA Commissioner Robert Califf.  “We recognize the urgent need for new treatments that can both improve and extend the lives of people diagnosed with these diseases.”

The action plan is a blueprint for how the agency will tackle challenges in drug development for rare neurodegenerative diseases, including ALS, 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, establishing the public-private partnership for rare neurodegenerative diseases, developing disease-specific science strategies, and 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.

ACT for ALS also requires the Department of Health and Human Services 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.

Author: Rare Daily Staff