FDA releases 5-year action plan to combat neurodegenerative diseases

The FDA on June 23 announced a 5-year strategy for improving and extending the lives of people with rare neurodegenerative diseases by advancing development of safe and effective medical products and facilitating access to novel treatments.

The plan, which will serve as a blueprint for how the agency will move forward in tackling challenges in drug development, was developed in accordance with the Accelerating Access to Critical Therapies for ALS Act that President Joe Biden signed into law in December 2021, the FDA said in a press release.

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

According to the release, during the 5-year span, the plan will focus on bolstering scientific advancement and promoting innovation by:

• establishing the FDA Rare Neurogenerative Disease Task Force;
• establishing the public-private partnership for rare neurodegenerative diseases;
• developing disease-specific science strategies; and
• leveraging ongoing FDA regulatory science efforts.

A key focus of the plan is the ALS Science Strategy, which provides a forward-learning framework for FDA activities to assess key regulatory science priorities. Highlights of the strategy include:

• improving characterization of disease pathogenesis and natural history;
• facilitating patient access to new drugs whenever possible and promoting greater participation in clinical trials by reducing barriers and burdens faced by minority populations; and
• enhancing clinical trial infrastructure and agility to enable early selection of promising therapeutic candidates for further development, optimizing clinical trial design, improving access to the trials, streamlining trail operations and reducing the time and cost of drug development.

Per the release, success of the FDA’s implementation of the ALS Science Strategy will rely on patient engagement, public workshop, research projects and collaboration with the NIH.

“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,” Califf said. “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.”