FDA Releases Action Plan for Rare Neurodegenerative Diseases

FDA unveiled its Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis (ALS) on June 23, 2022. The plan is a five-year strategy for improving and extending the lives of people living with rare neurodegenerative diseases through advancing the development of, and improving patient access to, safer and more effective medical treatments and products.

The action plan outlines how FDA will move forward in addressing challenges in drug development for rare neurodegenerative diseases. FDA will take actions such as regulatory science initiatives, enhancements to existing programs, and new policy initiatives. The plan was developed in accordance with the Accelerating Access to Critical Therapies for ALS Act, which was signed into law on December 23, 2021.

“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, MD, in a press release. “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.”

Source: FDA