FDA Shares Plan for ALS, Other Neurodegenerative Diseases

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by Margarida Maia, PhD |

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The U.S. Food and Drug Administration (FDA) has published on its website a five-year action plan meant to help advance the development of — and access to — treatments that may make life better and longer for people with amyotrophic lateral sclerosis (ALS) or other rare neurodegenerative diseases.

The development and publication of this strategy was a requirement of the Accelerating Access to Critical Therapies for ALS Act, which President Joe Biden signed into law in December. It will make available $100 million annually up to fiscal year 2026 to improve the prevention, diagnosis, and treatment of rare neurodegenerative diseases such as ALS.

“We recognize the urgent need for new treatments that can both improve and extend the lives of people diagnosed with these diseases,” Robert M. Califf, MD, the FDA’s commissioner, said 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,” Califf added. “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 plan is a roadmap for how the agency intends to address the unmet medical needs of people with rare neurodegenerative diseases such as ALS, with respect to program enhancements, policy development, regulatory science initiatives, and other appropriate initiatives.

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It also involves efforts and collaborations between the agency, the Department of Health and Human Services, and the National Institutes of Health.

Specific actions for this fiscal year include setting up a task force and a public-private partnership for rare neurodegenerative diseases. The multidisciplinary task force, comprised of members from several of the agency’s centers, will work toward establishing a consistent, cross-cutting approach to the development of treatments for rare neurodegenerative diseases, and the partnership will be used to advance these plans.

Over the next five years, the agency also will leverage some of its ongoing programs, such as the Patient-Focused Drug Development Program, which draws on patients’ experiences and views to inform how to best develop and test potential new treatments.

Disease-specific science strategies also will be developed to overcome the unmet medical needs and challenges of each disease. The ALS Science Strategy will focus solely on ALS.

“Executing the ALS Science Strategy within this action plan will enhance FDA’s ability to address substantive issues in ALS drug development, deepen its scientific expertise to enhance regulatory decision-making, and promote its collaboration with external stakeholders,” the agency stated in the plan.

“The ALS Science Strategy will serve as an example for how FDA can systematically and strategically foster and advance drug development for rare neurodegenerative diseases,” the plan continued.

The implementation of this strategy will be ensured by an ALS working group that is expected to be established this year within the agency’s rare neurodegenerative diseases task force.

Other near- and medium-term activities of the ALS Science Strategy are framed around obtaining deeper knowledge about how ALS begins and evolves over time. This includes the promotion of data sharing, the development of better preclinical models, and the identification of biomarkers of the disease and the response to treatments.

Other activities will focus on improving ALS patients’ access to investigational treatments by reducing barriers to entry into clinical trials, using digital health technologies and decentralized trial designs, and encouraging the incorporation of expanded access mechanisms into clinical development programs.

Expanded access offers people with ALS who did not make it into clinical trials a possible route for gaining access to an experimental therapy.

Longer-term activities (expected in 2025 and 2026) “will incorporate the insights and knowledge gained from earlier activities in the ALS Science Strategy and focus on enhancing the design and conduct of clinical trials,” the agency stated.

These will work to speed up the selection of potential therapeutic candidates for further development, improve trial access, streamline operations, and reduce the time and cost of therapy development.

To carry through its activities, the agency is counting on patient engagement, public workshops, research projects, and the collaborative efforts brought through the task force and the public-private partnership.

From time to time, the action plan will be reviewed and updated if necessary.