FDA Publishes Strategy for Advancing Regulatory Science

FDA released a strategic plan to advance regulatory science on Aug. 17, 2011, in an effort to “keep pace with and utilize…new scientific advances” and to promote public health. The plan is based on the agency’s five-year strategic goals (see “FDA Releases Strategic Priorities for 2011–2015”) and its October 2010 report on advancing regulatory science (see “FDA Reports on Advancing Regulatory Science”).

“As new discoveries yield increasingly complex products, this strategic plan ensures that our experts are equipped to make science-based decisions resulting in sound regulatory policy. It positions us to foster innovation through better science without compromising our high safety standard,” stated FDA Commissioner Margaret A. Hamburg in a press release about the report.

The plan outlines eight priority areas that describe how FDA intends to gather and communicate information and advance tools and product quality. Of the eight priorities, six focus on pharmaceutical development and manufacturing—the other two address food safety and communication. Each priority area has a public-health component. Below are some highlights.

Priority 1 is to modernize toxicology to enhance product safety. Because nonclinical safety assessment tends to be exposure-based and conducted in healthy test systems, says the plan, certain responses are not addressed, such as those tied to genetic background or progression of a disease. They agency would like to close these gaps by investing in and implementing better models of human adverse responses, biomarkers, and computational methods as well as in silico modeling.

Priority 3 is to support new approaches to improve product manufacturing and quality. The agency notes in the report that it needs to foster innovations by doing more research with industry and academia. It would like to develop and evaluate novel and improved manufacturing methods by looking into the effects of continuous manufacturing on product quality, how innovative approaches affect product failure rates, and the role of excipient ingredients and complex dosage forms on final products. The role of excipients in drug performance has been a large area of discussion in recent months, especially with regard to atypical actives, which are addressed in PharmTech’s September 2011 special issue on ingredients.

Under this same priority area, the need for new analytical methods and statistical approaches, including testing their feasibility and value, is noted, as is the risk reduction of microbial contamination. Quality by design and process analytical technology are highlighted in this regard. (FDA is publishing a review of its QbD program in PharmTech’s September issue).

Priority 4 is to ensure FDA readiness to evaluate innovative emerging technologies such as nanotechnology. To accomplish this goal, the agency intends to develop additional expertise and infrastructure to establish assessment tools for novel therapies, stimulate and ensure safe innovative development, and enhance information-technology applications.

Priority 5 is aimed at harnessing diverse data using information sciences. The idea is to integrate and analyze data from various sources, including by “unlocking” data from product applications reviewed by FDA. Simulation models for product life cycles and risk assessment are also envisioned. Because the agency has so much information at its fingertips, including toxicology data, FDA would like to incorporate that information into a single database to facilitate the use of predictive toxicology models and model validation, for example.

Priority 7 focuses on facilitating the development of medical countermeasures. These countermeasures are meant to respond to potential chemical or biological threats, infectious disease, and so forth. The agency launched an initiative in 2010 to facilitate the development and approval of medical countermeasures. The use of animal models, modernized tools, and biomarkers are part of the plan.

Priority 8 is to strengthen social and behavioral science to help consumers and professionals make informed decisions about regulated products. Over the past few years, FDA has been more transparent (see “Transparency and Safety,” and “FDA Aims to Communicate Better”) about its work and is trying to better educate the public and collaborate more with industry and academia. This strategic goal builds on those past efforts and aims to improve understanding of consumer decision-making and evaluate the effectiveness of FDA’s communications.

Of note, Priority 2 addresses ways to improve innovation in personalized medicine and clinical evaluations, and Priority 6 addresses food safety.

The final portion of the FDA strategic plan discusses its partnerships with government agencies and the private sector, staff training and professional development, and direct-funding mechanisms. Each area is directly tied to achieving the plan and advancing regulatory science. Overall, notes the report’s concluding section, FDA intends to use the plan to enable the best available resources and tools to keep pace with current science and technology and to promote public health.

FDA has an online podcast featuring Dr. Vicki L. Seyfert-Margolis, the senior advisor for Science Innovation and Policy in the FDA Commissioner’s office.