Recent breakthroughs in science and technology — ranging from sequencing of the human genome to advances in the application of nanotechnology to new medical products — have the potential to transform our ability to prevent, diagnose and treat disease. These developments will result in moving treatment strategies towards approaches that are tailored or personalized to individual patients, thus maximizing the benefit of treatments while decreasing their safety risks. Similarly, advances in research and information technologies are enabling us to more efficiently identify microbial pathogens, track food contamination outbreaks and determine where foods and other FDA-regulated products are produced or manufactured, how they are transported, where they go and who uses them. These tools also can play an important role in preventive health by enabling more comprehensive immunization strategies, especially in the face of emerging pandemics.
For these advances to reach their full potential, the Food and Drug Administration (FDA) must play an increasingly integral role as an agency not just dedicated to ensuring safe and effective products, but also to promote public health and participate more actively in the scientific research enterprise directed towards new treatments and interventions. We must also modernize our evaluation and approval processes to ensure that innovative products reach the patients who need them, when they need them. These new scientific tools, technologies, and approaches form the bridge to critical 21st century advances in public health. They form what we call regulatory science: the science of developing new tools, standards and approaches to assess the safety, efficacy, quality and performance of FDA-regulated products.
This document outlines a broad vision for advancing regulatory science and unleashing its potential to improve public health. It discusses the role of the FDA, working with partners, to strengthen the field, both within the agency and throughout the Nation.
The document is organized into two sections: (1) The Promise of Regulatory Science and (2) A Collaborative Implementation Framework. The first section provides background on the emerging and promising field of regulatory science as well as examples of current activities. It then goes on to explore seven different public health areas in which advancements in the field can help deliver better, safer, more innovative products to Americans. The second section lays out a strategic framework that will guide FDA as we lead this nationwide effort to advance regulatory science and leverage its potential to fulfill the agency’s fundamental mission — to promote and protect the public health.
FDA proposes to build on the success of the Critical Path Initiative and other projects by leading an effort to advance regulatory science through its new Regulatory Science Initiative. This initiative will be supported by a four-part framework, outlined in greater detail in the final section of this document.
- Leadership, coordination, strategic planning and transparency to support science and innovation
- Support for mission-critical applied research, both at FDA and collaboratively
- Support for scientific excellence, professional development and a learning organization
- Recruitment and retention of outstanding scientists
With the President’s $25 million budget request for fiscal year (FY) 2011, FDA plans to expand ongoing efforts within the agency and build additional partnerships with academia, industry and government around the country. A new office dedicated to regulatory science will lead strategic development and coordination within FDA, and early efforts will focus on recruiting key personnel and building senior leadership.
In FY2011, the bulk of the budget will be used to mobilize external collaborations and partnerships and support studies in four major regulatory science research areas:
- Transforming Product Development for Patients: Bringing Progress to Patients
(eg: Methods for Modernizing Toxicology, Biomarkers for Personalized Medicine, the Stem Cell Initiative and Updating Drug Review Standards)
- Science to Address Emerging Technologies in FDA-regulated Products
(eg: Nanotechnology and Expertise to Regulate New Animal Biotech Products)
- Information Sciences for Health Outcomes
(eg: Medical Device Registry and Scientific Computing for Data Analyses)
- Addressing Unmet Public Health Needs
(eg: Nutrition and Public Health)
Activities in FY2011 will also set the stage for a network of Centers of Excellence in Regulatory Science to be integrated with expanded intramural FDA research and with other clinical research networks. Funding for external programs would be competitive and focus on pilot and feasibility studies to form Centers of Excellence.
This document discusses in detail how advances in regulatory science can speed progress in FDA’s high-priority public health areas.
There is no single discovery — no magic bullet — to address our unique set of modern scientific regulatory challenges. But one thing is clear: if we are to solve the most pressing public health problems we face today, we need new approaches, new collaborations and new ways to take advantage of 21st century technologies. And we need them now.