FDA Promotes Its Scientific Mission

In testifying before Congress last week on FDA regulation of long-awaited biosimilars, Janet Woodcock, director of the Center for Drug Evaluation and Research (CDER), emphasized the importance of ensuring that the evaluation of new therapies is based on sound science. “We have to get the science right,” Woodcock told the Senate subcommittee of the Health, Education, Labor and Pensions Committee, in explaining FDA’s lengthy and deliberative process for issuing important policy advisories on biosimilar naming, labeling, and interchangeability. Woodcock noted that a “sound scientific framework” is key to assuring prescribers and patients that biosimilars are safe and of high quality and should come to market.

Biosimilars are one of many FDA programs that benefit from strong scientific expertise at the agency, as outlined in a report from FDA’s advisory Science Board. The analysis, “Mission Possible: How FDA Can Move at the Speed of Science,” examines a number of important programs and responsibilities that rely heavily on FDA’s scientific capabilities.

The report highlights regulatory science efforts important for developing more novel drugs and biologics, including qualification of biomarkers and encouraging more efficient and lower-cost clinical trials through the use of trial networks and “master protocols.” Science can enhance medical product quality, purity, and safety by facilitating use of new DNA technology to characterize medical products, assessing products made using nanotechnology, evaluating biosimilar production, screening for evidence of product contamination or counterfeiting, and assessing evidence of antibiotic resistance. FDA’s scientific expertise also is important for ensuring food safety and proper use of tobacco products.

FDA has achieved important improvements in its scientific and technical capabilities since issuing an important report in 2007 on “FDA Science and Mission at Risk,” but notes that limited resources can set back the agency’s ability to bring new medical products to patients and to protect public health. Continued progress requires more collaboration with external organizations, such as the Reagan-Udall Foundation; changes in policies to facilitate the hiring of outstanding scientists; and continued growth in resources to support these initiatives and ever-growing areas of responsibility.

FDA acting commissioner Stephen Ostroff echoed these findings in unveiling the report at a symposium last week for science writers. Ostroff noted how FDA scientific expertise has been crucial in supporting the nation’s response to the Ebola virus outbreak, tackling antimicrobial resistance, identifying counterfeit products, accelerating the approval of new medical products, and mapping the pathway to precision medicine. The “timing is terrific” for this report, he said, emphasizing that scientific capability is crucial to ensure that FDA makes correct decisions about product safety and efficacy.

At the symposium, FDA scientists described a number of research initiatives that illustrate the value of scientific expertise in advancing biomedical development and regulatory understanding. Innovative projects, for example, are investigating new technology for early detection of traumatic brain injury, enhancing agency understanding of new 3D printing applications, and using computational modeling and simulation to better predict toxicity. Investigators in the Center for Biologics Evaluation and Research (CBER) explained how they are examining a surprising reemergence of mumps and pertussis (whooping cough) in the United States, with an eye to developing new assays, animal models, and bioinformatics techniques to better understand the limitations in protection from current vaccine regimens.

The Science Board report applauds increased funding for FDA from Congress and from user fees in recent years to support its expanding responsibilities, including oversight of biosimilars, accelerated review of generic drugs, and increased oversight of imported medicines. Legislation before Congress, however, would impose additional costs on the agency of approximately $1 billion over five years. Such an expansion in responsibilities could set back progress in medical product development, the report warns, by “forcing FDA to divert its scientific staff away from its currently successful review programs.”