Advancing Regulatory Science and Innovation
On the forefront of regulatory science and innovation, CVM was involved in essential methods innovation research aimed at addressing new veterinary drug safety and effectiveness for nanotechnologies, proteomics, and stem cell therapies. The challenge was to establish science-based and valid regulatory reviews and decisions involving data obtained from novel and emerging technologies. FDA’s regulatory role creates unique scientific and technological needs that can be met only by a robust regulatory science program.
Several projects were underway during the year to ensure CVM is ready to evaluate data obtained from new and emerging technologies that raise safety and effectiveness questions, including:
Stem cell research. CVM conducted its first stem cell study designed to investigate stem cell markers in canine cell lines, with three cell markers already identified. Study results would contribute to CVM’s pre-market review process and allow for safe and effective use of these innovative products in animals.
Nanotechnology. The application of nanotechnology in veterinary medicine has shown great promise, especially in the growing area of nanopharmaceuticals. During the year, our scientists completed their first nanotechnology study, and a protocol was approved for a second study.
Proteomic and genomic clinical biomarkers. Several projects to identify and investigate the use of biomarkers also made considerable advancement this year, including a Medical Countermeasures Initiative (MCMi) funded project with FDA’s Center for Drug Evaluation and Research (CDER) to utilize microarrays to rapidly characterize antibiotic resistance genes of bacterial bio-threat agents. Other research is also identifying host candidate protein biomarkers indicative of diagnosing bovine pneumonia, and new drug efficacy markers for Non-Steroidal Anti-Inflammatory Drug (NSAID) treatment of bovine mastitis. Details of these and other projects can be found elsewhere in this report, especially in the sections Animal Biotechnology and Science at CVM.
We are developing the appropriate risk-based questions to facilitate approval of innovative technologies. Establishing a scientific environment that cultivates and facilitates creativity and innovation allows CVM scientific experts to tailor the evaluation of safety and effectiveness for innovative products through a proactive, risk-based approach, rather than relying on more traditional scientific methods that may not be sufficient for evaluating the new drug products we are seeing today.
To achieve this goal, the Office of New Animal Drug Evaluation (ONADE) created a process for forming technology teams and focus groups to utilize knowledge within and outside ONADE to develop predictable processes for evaluating innovative products. These processes allow our review scientists to become familiar with innovative products and study design options much earlier in drug development than in years past and to tailor the development plan to the specific risks associated with that product. Proactive efforts at this stage in drug development can increase the predictability of the regulatory pathway for sponsors, maintain a low number of review cycles by reducing missteps, and reduce the overall time to approval for innovative new animal drugs.
One example of this new review process is the formation of a tech team to address the use of stem cells in veterinary regenerative medicine. Under ONADE’s leadership, the stem cell tech team includes scientific experts from ONADE, the Animal Biotechnology Interdisciplinary Group, the Office of Surveillance and Compliance, and the Office of Research. The stem cell tech team addresses policy development and scientific review associated with this emerging technology.
Building on previous efforts to establish procedures to identify key scientific questions and issues related to stem cell products, the stem cell tech team made significant in-roads toward addressing policy development and scientific review associated with this emerging technology during FY 2012. The stem cell tech team executed (and continues to execute) a coordinated, multi-disciplinary, Center-wide risk-based approach to the regulation of stem-cell based products. This is an excellent example of a cross-cutting effort bringing a diversity of knowledge, expertise, and experience to address an emerging technology that does not lend itself to the more traditional approaches for new animal drug review. Along with addressing emerging issues associated with the review of veterinary stem cell products, the stem cell tech team is making significant progress toward developing guidance on cell-based products.
The biomarker focus group is another example of how CVM scientists are proactively exploring innovative tools to aid drug development. This effort is part of FDA’s strategic goal to modernize and advance regulatory science by developing new tools and approaches to assess the safety, effectiveness, quality, and performance of FDA-regulated products.