Commenters concerned about scope of FDA’s BsUFA regulatory science pilot

The US Food and Drug Administration’s (FDA) research roadmap for its Biosimilar User Fee Amendments (BsUFA) III regulatory science pilot program garnered comments from stakeholders broadly supportive of the initiative, but who also shared concerns that the agency may be trying to achieve too much with its pilot program.

The goals of the BsUFA III regulatory research pilot program are “advancing the development of interchangeable products” and “improving the efficiency of biosimilar product development” through the end of BsUFA III in September 2027. Research priorities for the agency include improving the accuracy and capability of chemistry, manufacturing, and controls (CMC) characterizations; and to develop alternatives to human studies for biosimilars. (RELATED: FDA announces FY 2023-2027 BsUFA science and research priorities, Regulatory Focus 26 January 2023)

“The BsUFA III regulatory research pilot program aims to leverage FDA’s purview, at the intersection of scientific advancement, public health, and regulatory policy, to identify knowledge gaps and direct research to advance biosimilar development,” FDA wrote in a document requesting comment on the research roadmap.

Stakeholders who responded to FDA’s request for comment were supportive of the initiative, but some commenters were concerned with the pilot’s research priorities relative to the main goals of the program.

“We are concerned that some of the research priorities that have been selected by FDA are neither feasible nor would they lead to improvements in the efficiency of biosimilar product development,” Samsung Bioepis wrote in their comment. “It is a key priority that every project funded is regulatorily relevant and specifically applicable to biosimilars. These must be identified in each application ahead of their being seriously considered for funding, such that were the project to be funded the development of biosimilars can be materially more efficient.”

Looking forward to BsUFA IV, Samsung Bioepis said that it is important for the pilot program to have relevance for biosimilar developers today to lay the groundwork for future development. “A decision on the sustainability of this research program itself, given that it is supported by the biosimilar industries own user fees, should be a consideration sooner rather than later,” they wrote.

The Biosimilars Council (BC), a division of the Association for Accessible Medicines (AAM), described the research priorities in the roadmap as a “good starting point,” but expressed concern that “the scope of research and work needed to make progress on ten priority areas is too much to attempt concurrently, especially when the program is being established.”

“BC recommends FDA work with stakeholders to further prioritize the research agenda and then to allocate the research funding to a broad variety of high-priority projects,” they wrote. “BC recommends FDA, along with stakeholder input, develop an approach, and consider variables, to outline priorities into tiers such as short-term, midterm, and long-term.”

The Pharmaceutical Research and Manufacturers of America (PhRM) also said the pilot program should stay consistent with the goals outlined in FDA’s commitment letter.

“PhRMA recognizes that the biosimilar and interchangeable biosimilar landscape will continuously evolve, and believes the lessons learned and the scientific knowledge acquired through the pilot program could help enhance FDA’s regulatory decision-making and facilitate science-based recommendations,” they wrote.

Near-term changes

Sandoz, a division of Novartis focused on developing generics and biosimilars, noted in their comment that they “strongly support” the pilot program. With the latest analytical assays, "there are existing opportunities to consider alternatives to and reduce the size of studies involving human subjects,” they explained, but the proposed research projects should align with both goals of the program.

“It would be inappropriate to study advanced analytics under the BsUFA III regulatory science program simply to develop additional or more complex assays for analysis of biological drugs without these efforts being aimed at achieving the second objective of the Roadmap,” they wrote.

Members of the Biosimilars Forum said they believe FDA can streamline components of biosimilar development today without waiting for the completion of BsUFA III research programs.

“Other highly credible and experienced regulatory agencies are already embracing streamlined development by implementing waivers for the confirmatory comparative clinical efficacy studies,” they wrote. “It is important to acknowledge these advancements and successful experiences so that developers are not producing data that is no longer scientifically relevant, nor indeed pertinent, to regulatory decision-making.”

BsUFA III Research Roadmap

Commenters concerned about scope of FDA’s BsUFA regulatory science pilot

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