CDER Identifies Key Regulatory Science Needs and Strategies

FDA released this week a report from its Center for Drug Evaluation and Research (CDER) that identifies regulatory science needs that will guide CDER’s strategic planning in the years ahead and allow it to fulfill its regulatory mission. The report, Identifying CDER’s Science and Research Needs, discusses internal research initiatives and goals for contributing to the agency’s overall regulatory science efforts.

Behind the report is the agency’s Critical Path program, which was launched in 2004 to transform the way FDA-regulated medical products are developed, evaluated, and manufactured. More than 200 representatives from various offices within FDA were interviewed to help identify the areas of greatest need. The report notes that computational infrastructure needs and data standards are not addressed because efforts are already underway to in these areas. “Communicating our science and research needs represents an important step in stimulating research and fostering collaborations with our external partners,” said CDER director Janet Woodcock, MD, in a release about the report.

The 24-page report, put together by the CDER Science Prioritization
and Review Committee, goes into detail about how to address key science and research needs, which are broken down into seven areas:

• To improve access to postmarket data sources and explore feasibility of their use in different types of analyses;

• To improve risk assessment and management strategies to reinforce the safe use of drugs;

• To evaluate the effectiveness and impact of different types of regulatory communications to the public and other stakeholders;

• To evaluate the link among product quality attributes, manufacturing processes, and product performance;

• To develop and improve predictive models of safety and efficacy in humans;

• To improve clinical trial design, analysis and conduct; and

• To enhance individualization of patient treatment (including biomarkets and drug product behavior in specific populations).

The first four bulletpoints may be of most importance to pharmaceutical manufacturers. For example, the first point about postmarket data calls for determining the accuracy of available postmarket data sources and to use that data, along with premarket data, in safety analyses. The second point, improving risk assessment and management strategies, notes that regulators need to better characterize the risks associated with drugs that have not gone through the FDA approval process and to enhance risk-mitigation strategies for those drugs that have. For example, the report notes that, “to ensure the safe use of drugs, we need to evaluate whether our regulatory activities are resulting in improved outcomes for patients…. This will necessitate identification of additional appropriate outcome measures, methodologies, and accompanying metrics.” A more in-depth look at counterfeit and subquality products is also called for in the report.

The third regulatory-science need regarding regulatory communication suggests that CDER “determine whether current approaches (content, media, and format) are effective in conveying important medical information, and whether they are having an impact on patient and health care provider behavior and patient outcomes.” And the fourth point regarding product process and performance suggests that CDER officials need to “better understand the impact of variability in the drug components and manufacturing process parameters on product quality and performance.” Combination products and biologics are specifically called out in this section of the report. Achieving this goal, according to the report, may require implementation of advanced analytical technology as well as quality risk-management principles and quality-by-design approaches.

Looking ahead, CDER intends to explore the ideas and opportunities noted within the report with other groups such as industry, academia, and nonprofit organizations. “The intent is to enable priorities to be established among the categories and identify gaps that could be addressed through targeted research projects,” states the report. Public comments on the report are due within 60 days of the report’s publication in the Federal Register, or Sept. 25, 2011.