FDA to form task force, public-private partnership on rare neurodegenerative diseases
The US Food and Drug Administration (FDA) released a five-year action plan for accelerating drug development for rare neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS), as well as improving access to novel therapies.“The effects of rare neurodegenerative diseases are devasting, with very few effective therapeutic options available to patients,” Robert M. Califf, MD, FDA Commissioner, said in a statement. “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 of those suffering by facilitating access to new therapies.”
The action plan, which implements provisions of the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS), includes establishing the FDA Rare Neurodegenerative Diseases Task Force and a public-private partnership for rare neurodegenerative diseases. Both of these activities would begin in Fiscal Year 2022. The action plan also calls for developing disease-specific science strategies throughout the next five years.
The Rare Neurodegenerative Diseases Task Force will build on work FDA has already started around rare diseases, including the Accelerating Rare disease Cures (ARC) Program, which was launched in May 2022 to accelerating the availability of treatments. The agency plans to leverage the ARC Program’s inter-center, interdisciplinary infrastructure in launching the new task force.
The task force will be co-led by senior representatives from the Center for Biologics Evaluation and Research (CBER) and the Center for Drug Evaluation and Research (CDER), in coordination with a senior official from the FDA Office of the Commissioner. The task force will also be charged with external outreach to the rare neurodegenerative disease communities.
The public-private partnership will include the FDA, the National Institutes of Health (NIH), and one or more groups, with the charge of supporting research and innovation.
As part of the goal of developing disease-specific science strategies, FDA will assess unmet medical needs, evaluate the landscape of clinical development, and pinpoint challenges to drug development for each disease. The agency has already developed an ALS-specific science strategy that includes identifying and validating biomarkers and potential surrogate endpoints to be used in clinical development, enhancing clinical trial design and infrastructure, exploring the use of digital health technologies to improve disease understanding, and supporting the development of study data standards for ALS.
FDA has already published final guidance on drug development and trial designs for ALS and draft guidance on human gene therapy for neurodegenerative diseases. (RELATED: FDA Finalizes ALS Drug Development Guidance, Regulatory Focus 23 September 2019)
In the action plan, FDA officials note that the ACT for ALS legislation authorizes $100 million each year from FY 2022 to 2026 and that the agency’s ability to carry out the plan is contingent on Congress appropriating and distributing those funds.
FDA Action Plan
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