CBER Acting Head Highlights Regulatory Flexibility, AI and Rare Disease Priorities

CBER acting director Katherine Szarama outlined an expansive vision for the biologics center during remarks at the Food and Drug Law Institute’s annual conference in Washington, DC 5/6, emphasizing regulatory flexibility, scientific rigor and increased engagement with industry as the agency confronts a rapidly expanding pipeline of complex biologic therapies. Speaking during a wide-ranging discussion moderated by Hogan Lovells partner Elizabeth Jungman, Szarama described CBER as a center operating at “the intersection of science and policy,” while balancing pressure to accelerate innovation in areas including cell and gene therapies, rare disease treatments, vaccines and xenotransplantation.

Szarama, who recently became acting director earlier this month after previous director Vinay Prasad left the agency, said CBER now oversees 439 products with approximately 1,100 staff and is managing growing numbers of investigational applications and increasingly complex scientific questions. “We must continue moving forward based on the available science to make our most informed decisions and put forth a path to progress,” she said.

Szarama said the biologics pipeline has shifted dramatically toward rare diseases and advanced therapies, with orphan-designated products accounting for as much as 70% of CBER approvals in recent years. She noted that CBER approved 21 biologic products in 2025 across its offices that oversee therapeutic products, vaccines, and blood products.

The center is also seeing a sharp rise in investigational new drug applications involving gene therapies and genetically modified cell therapies. By 2023, CBER received more than 200 such IND submissions annually, with roughly 61% targeting rare diseases, she said. The growing pipeline is driving FDA efforts to modernize regulatory frameworks and expand early engagement programs with sponsors. Szarama highlighted several initiatives, including the Collaboration on Gene Therapies pilot, the Support for Clinical Trials Advancing Rare Disease Therapeutics program, and the Chemistry, Manufacturing and Controls Development and Readiness Pilot. “These are development programs. They’re not approved products, but they represent a maturing pipeline,” she said.

Much of the discussion focused on how FDA is approaching evidentiary standards for biologics, particularly amid increased agency discussion around single pivotal trial approvals and use of external controls in rare diseases. Szarama said FDA’s interpretation of what constitutes an “adequate and well-controlled” trial has evolved over time, especially in settings where randomized studies may be impractical. “I think over time, adequate and well-controlled has started to evolve into broader populations of patients that have well-understood natural histories that then could be relied on as external controls,” she said.

Still, she cautioned that single-trial approvals require robust confirmatory evidence and emphasized that FDA continues to weigh disease severity, duration, lack of existing therapies and patient risk-benefit considerations when evaluating programs.

She also acknowledged industry concerns about perceived late-stage shifts in FDA expectations during development programs. Szarama suggested some disagreements emerge as broader scientific and clinical issues surface later in development, including questions raised through advisory committee discussions, patient input and evolving understanding of diseases.

Szarama also addressed growing interest in artificial intelligence across drug development and FDA operations, describing AI tools as useful but still heavily dependent on human oversight. “I think AI is like a great graduate student,” she said. “It’s very eager to take feedback and adjust its thinking, but it hasn’t necessarily gotten to the point where it’s going to work independently.”

She advised sponsors not to rely on AI-generated summaries without careful review but said FDA is increasingly using AI-supported systems internally to improve document management, data organization and review workflows.

Szarama highlighted FDA’s “Hive” supercomputing platform and said standardization efforts tied to electronic common technical document submissions are helping the agency automate portions of file assignment and data summarization. She also pointed to growing FDA interest in AI-supported bioinformatics and personalized cancer vaccine development.

During the discussion, Szarama defended FDA’s increasingly public-facing communications strategy, arguing that speeches, interviews and public workshops provide important insight into the agency’s evolving thinking even before formal guidance documents are finalized. “The quieter we get, the lower the noise floor, the harder it is to respond, and then everything becomes a signal,” she said.

She encouraged sponsors to view guidance development as an iterative process and to engage FDA early and frequently during product development. “Bring us your questions,” Szarama said in closing. “The depth of thought, their willingness to research a question, their collegial partnership with other centers, other offices, other programs across FDA is unrivaled.”