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Defining Regulatory Science
The concept of regulatory science is not a new one. The FDA Science Board’s 2007 Science and Mission at Risk report (FDA Science Board, 2007) describes regulatory science as a science-based decision-making process needed to fulfill the responsibilities of a public health agency: “FDA must have the scientific staff and resources to undertake the regulatory research that will provide a basis to: (1) improve capacity for safety and efficacy evaluations and monitoring of candidate and licensed products, (2) modernize current regulatory pathways, and (3) develop new regulatory pathways where there are currently none.” According to the report, this capacity is important because “decisions made in regulation development, pre-market approvals, legal actions and related public health emergencies must be based on understanding of contemporary and emerging science within the context of the risk analysis paradigm” (FDA Science Board, 2007, p. 14).
While a number of descriptions of regulatory science have been put forth, no formal definition exists. Some alternative definitions of regulatory science are presented in Box 2-1. The following general definition was submitted to the IOM Drug Forum for discussion at this workshop by Carl Peck, Professor of Bioengineering and Therapeutic Sciences at the University of California, San Francisco, Center for Drug Development Science, and former Director of FDA’s CDER (1988–1993) (Peck, 2010, p. 1):
Regulatory science is a broad term concerning drug and other product regulations, regulatory standards, law and procedures across many disciplines. It is a systemized body of knowledge (practiced by FDA and
BOX 2-1 Some Definitions of Regulatory Science While no official definition of regulatory science has been promulgated by any U.S. regulatory agency or standards-setting body, the following definitions highlight a common theme: drawing science and policy together for the benefit of public health and safety:
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similar regulatory agencies world-wide) comprising public protection-oriented medical product regulations, policy and decisions using scientific methods employing empirical and causal evidence utilized in the evaluation and approval of all the products that FDA regulates.
Thus the role of regulatory science is inherent in FDA’s functioning as a scientific agency. It plays a critical role in all aspects of the agency’s mission, including:
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review and assessment of laboratory data;
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review and assessment of animal and human clinical data;
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methods development;
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facilities inspection; and
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development of technical and scientific standards for preclinical
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assessment, product development, postmarket surveillance, manufacturing, packaging standards, food safety standards, and food processing technologies.
Garret FitzGerald, Professor of Translational Medicine and Therapeutics, University of Pennsylvania School of Medicine, offered the following guidelines for determining what the term does not denote:
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a new set of regulations;
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an approach to speeding up the approval process; or
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an attempt to establish cutting-edge biomedical science at FDA.
Nevertheless, regulatory science can aid in the above areas by boosting scientific capacity. It is not a cure-all for the shortfalls in drug regulation, yet is critical to FDA’s accomplishment of its complex goals. Box 2-2 summarizes FDA Commissioner, Margaret Hamburg’s talk on regulatory science, which encapsulates these intricacies. Workshop participants suggested that, because it lies beyond the traditional domain of biomedical science, the practice of making regulatory decisions on the basis of the best science possible is an emerging area of meta-science.
Jeffrey Drazen, Editor-in-Chief of the New England Journal of Medicine and Co-Chair of the Drug Forum, described regulatory science as “a science that has been evolving and is continuing to evolve, but it’s not as hard a science as we would like.” Thus, enhancement of the regulatory science discipline today represents a concerted attempt to systemize and standardize daily regulatory processes and bolster regulatory decision-making capabilities with a sound scientific base. The following chapter explores why such efforts are necessary.
BOX 2-2 Excerpts from the Commissioner’s Speech FDA Commissioner, Margaret Hamburg, provided an overview of regulatory science priorities at the agency. Commissioner Hamburg referred to regulatory science as “vital to enhance the quality and integrity of FDA’s regulatory decisions.” The Commissioner’s speech defined the need for and application of regulatory science; discussed collaborative efforts to advance regulatory science; and described models and initiatives that, if adequately funded, could significantly advance the field of regulatory science. The Commissioner emphasized that “[o]utreach and collaboration are central to regulatory science efforts. When successful, these collaborative efforts will help predict which discoveries will succeed or fail as actual products, thereby reducing product development costs and getting better products to patients faster.” She remarked that FDA should actively participate in research and development by partnering with academia, industry, and other government agencies. The following excerpts represent topical highlights from the Commissioner’s presentation at the workshop:
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latory science and try to bridge the gap between research, discovery, and innovation and the evaluation and development of new safe and effective products. “…[S]cience is a global enterprise. There are enormous opportunities to collaborate with scientific colleagues around the world on matters of mutual concern, but also to engage with sister regulatory agencies to address the sharing of important information and strategies and to harmonize standards and approaches.” In looking forward, the Commissioner said, achieving an enhanced regulatory science at FDA and beyond will require not only a concrete, coordinated plan, but also resources. She reported that, for the first time in history, the President’s fiscal year 2011 budget included a targeted initiative to advance regulatory science for public health. She reported that FDA “will support scientific excellence by recruiting, training, and retaining FDA scientists through meaningful career ladders, fellowship programs, scientific collaborations, exchanges, and other professional development activities.” In addition to the collaboration with NIH, the Commissioner announced, other interagency partnerships will be explored, such as with the National Institute of Standards and Technology (NIST) regarding areas of standards development and qualification, and with the Defense Advanced Research Products Agency (DARPA) to help provide regulatory input on novel products at the early development stage in its programs. She also noted that FDA will continue to engage outside advisory groups, such as the FDA Science Board, to gather information to help define regulatory science priority areas and foster and support the work necessary to address them. In closing, the Commissioner stated, “I think we really have the opportunity to lay out an important set of ideas and actions and also to help … shape this emerging field of regulatory science as a discipline. We are living in a century where the advances in biology are astounding and ripe for action. With our collective effort, these advances can be transformed into therapies that will alleviate suffering and products that will enhance our quality of life.” |