FDA October 2025 Highlights: Streamlining Biosimilar Development, Strengthening Submission Quality, and Advancing Public Health Protection

October 2025 marked a decisive month for the U.S. Food and Drug Administration (FDA) as it advanced four major regulatory initiatives to modernize drug review, improve transparency, and enhance public health protection.

From accelerating biosimilar development to restricting unapproved pediatric products, the agency demonstrated its continued commitment to efficient, evidence-based regulation that balances innovation with safety.

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1. Accelerating Biosimilar Development and Reducing Drug Costs

On October 29, the FDA released a new draft guidance to simplify biosimilarity studies and reduce the cost and time associated with developing biosimilars and interchangeable biologics.

The guidance, “Scientific Considerations in Demonstrating Biosimilarity to a Reference Product: Updated Recommendations for Assessing the Need for Comparative Efficacy Studies,” proposes eliminating unnecessary clinical trials in favor of advanced analytical testing.

Key updates include:

• Allowing developers to rely primarily on analytical comparability data instead of large human efficacy studies.

• Discouraging switching studies for interchangeable biosimilars, reducing duplication and confusion.

• Enhancing early engagement between sponsors and FDA review divisions.

FDA Commissioner Dr. Marty Makary emphasized that reforming biosimilar development will “achieve massive cost reductions for advanced treatments” while maintaining rigorous scientific standards.

With biologic drugs accounting for more than half of U.S. drug spending, this reform is expected to increase competition, lower costs, and expand access for patients with chronic diseases such as cancer, autoimmune disorders, and diabetes.

For Regulatory Affairs professionals, the shift underscores the need to focus on analytical data integrity, manufacturing quality, and early dialogue with regulators to navigate a faster, data-driven review process.

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2. Increasing Transparency with FDA Filing Checklists

On October 23, the FDA made public the internal filing checklists used by its Center for Drug Evaluation and Research (CDER) reviewers to assess whether a submitted New Drug Application (NDA) or Biologics License Application (BLA) is complete and ready for review.

The initiative aims to prevent Refuse-to-File (RTF) outcomes caused by procedural oversights, which can delay promising therapies by more than a year.

Over the past decade, more than 200 applications have received RTF notifications, often due to missing documentation or incomplete data sets.

FDA Commissioner Dr. Makary called the publication of these checklists a “common-sense step to make the FDA process easier to understand and more transparent.”

CDER Director Dr. George Tidmarsh added that the change will promote clearer communication between the agency and sponsors and help “eliminate preventable RTF actions.”

The checklists—now part of CDER’s MAPP 6025.4, “Good Review Practices: Refuse to File”—offer a valuable resource for Regulatory Affairs teams.

They provide insight into discipline-specific expectations, helping sponsors enhance submission quality, conduct internal readiness checks, and avoid costly resubmissions.

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3. Restricting Unapproved Fluoride Drug Products for Children

On October 31, the FDA announced enforcement actions to restrict unapproved ingestible fluoride drug products marketed for use in children.

The agency found that these products, in circulation since the 1940s, had never undergone formal FDA review for safety, efficacy, or manufacturing quality.

Recent scientific evaluations revealed that while topical fluoride effectively prevents dental caries, ingestible forms may alter the gut microbiome and pose potential developmental risks.

The agency concluded that such products should not be used in children under age 3 or in older children not at high risk of tooth decay.

FDA Commissioner Dr. Makary stressed that “there are better ways to protect children’s teeth than taking unapproved ingestible fluoride,” highlighting growing evidence linking high fluoride intake to possible neurodevelopmental effects.

The FDA also issued letters to healthcare professionals, warning against the use of these unapproved products and announcing plans to develop a national fluoride research agenda in collaboration with the NIH and HHS.

This decision reflects a broader trend toward reevaluating legacy drugs using modern scientific standards—an approach increasingly central to the FDA’s post-market oversight strategy.

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4. Launching the National Priority Voucher Program

On October 16, the FDA announced the first recipients of its Commissioner’s National Priority Voucher (CNPV) pilot program, a new initiative designed to fast-track applications addressing critical national health priorities.

Nine sponsors received vouchers for products that target major unmet needs—ranging from infertility and diabetes to rare cancers, blindness, and domestic drug manufacturing resilience.

Voucher recipients will benefit from accelerated review timelines of 1–2 months, enhanced communication with FDA review teams, and multidisciplinary evaluations using a “tumor board–style” model.

According to Dr. Makary, the program “modernizes the review process and meets the needs of the American people” by focusing regulatory resources on treatments with the greatest potential impact.

For Regulatory Affairs professionals, this marks the emergence of a new priority review mechanism—one that rewards innovation aligned with public health priorities such as affordability, domestic manufacturing, and unmet medical needs.

It complements existing expedited programs like Fast Track, Breakthrough Therapy, and Priority Review Vouchers, providing sponsors with another pathway to accelerate access to transformative therapies.

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A Modernized Regulatory Vision

Taken together, these four October initiatives reflect the FDA’s evolving approach to science-based, transparent, and patient-centered regulation.

By streamlining biosimilar requirements, clarifying submission expectations, addressing unapproved pediatric products, and incentivizing innovation, the agency continues to redefine how drugs are developed, evaluated, and approved in the United States.

For Regulatory Affairs professionals, these updates highlight a clear message: the future of regulatory work will depend on strategic communication, data integrity, and readiness for accelerated review models.

As the FDA advances its modernization agenda, aligning regulatory strategy with this evolving landscape will be key to achieving both compliance and innovation success.