The field of endeavors known as “regulatory science” has grown out of the need to link and integrate knowledge within and among basic science research, clinical research, clinical medicine, and other specific scientific disciplines whose focus, aggregation, and ultimate implementation could inform biomedical product development and regulatory decision making. The U.S. Food and Drug Administration (FDA) defines regulatory science as “the science of developing new tools, standards, and approaches to assess the safety, effectiveness, quality, toxicity, public health impact, or performance of FDA-regulated products.”2 Substantial efforts have been devoted to defining regulatory science and communicating its value and role across the scientific and regulatory ecosystems. Investments are also being made in technology infrastructure, regulatory systems, and workforce development to support and advance this burgeoning discipline.
Since its inception, the Forum on Drug Discovery, Development, and Translation (the Forum) of the National Academies of Sciences, Engineering, and Medicine (the Academies) has focused on the need for strengthening the scientific basis of drug regulation. In February 2010, the Forum
1 The planning committee’s role was limited to planning the workshop, and the workshop summary has been prepared by the workshop rapporteurs as a factual summary of what occurred at the workshop. Statements, recommendations, and opinions expressed are those of individual presenters and participants, and are not necessarily endorsed or verified by the Forum or the Academies, and they should not be construed as reflecting any group consensus.
2 See http://www.fda.gov/ScienceResearch/SpecialTopics/RegulatoryScience/ucm268095.htm (accessed April 11, 2016).
held a workshop that was summarized in the Institute of Medicine (IOM) report Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary (IOM, 2011), which examined the state of the science of drug regulation and considered approaches for enhancing regulatory science. In September 2011, the Forum convened another workshop that was summarized in the report Strengthening a Workforce for Innovative Regulatory Science in Therapeutics Development: Workshop Summary (IOM, 2012), which considered opportunities and needs for advancing innovative regulatory science through workforce and career development.
Over the past several years, models to support the discipline of regulatory science have advanced. FDA’s Centers of Excellence in Regulatory Science and Innovation (CERSIs) enhance training and educational opportunities for regulatory scientists. Private funders have also established programs; for example, in 2011, the Burroughs Wellcome Fund launched its Innovations in Regulatory Science Awards (IRSA) initiative, which aims to strengthen regulatory systems capacity by funding regulatory science–based research and collaborations.
On October 20–21, 2015, the Forum held a public workshop to facilitate dialogue among stakeholders about the current state and scope of regulatory science, opportunities to address barriers to the discipline’s success, and avenues for fostering collaboration across sectors. The workshop, co-sponsored by the Burroughs Wellcome Fund, held discussion panels that explored key needs for strengthening the discipline of regulatory science, including considering what are the core components of regulatory science infrastructure to foster innovation in medical product development.
The field of regulatory science is broad and touches many aspects of research. This workshop did not attempt to comprehensively discuss all the challenges and opportunities facing the field. To focus the discussions, the planning committee adopted the theme of innovation in regulatory science through integration of information. Presenters and participants were invited to examine and discuss how large-scale generation of information, particularly in light of the recent advent of “big data,” presents new opportunities to strengthen the connections among the regulatory science disciplinary components and advance the field (see Box 1-1 for the full Statement of Task).
This report is a summary of the workshop. Statements, recommendations, and opinions expressed are those of individual presenters and participants and are not necessarily endorsed or verified by the Forum or the Academies, and they should not be construed as reflecting any group con-
sensus. The workshop was webcast live, and online participants were able to contribute to the discussions through the hashtag #RegulatoryScience. The presentations, videos, and tweets are archived on the Forum website.3
3 For more information, see http://nationalacademies.org/hmd/Activities/Research/DrugForum/2015-OCT-20.aspx (accessed April 5, 2016).
The summary is organized as follows:
- Chapter 2 characterizes the current landscape of information integration in regulatory science presented at the meeting.
Chapter 3 summarizes discussion of four case studies of regulatory science applications that served as focal lenses to illuminate how enhanced approaches to obtaining, accessing, and integrating information could advance the science throughout and across medical product development. The four case studies were as follows:
- Identification and Development of Meaningful Biomarkers
- Integrating Clinical Trial Data
- Next-Generation Surveillance
- Innovative Modeling for Integrating Data
- Chapter 4 describes needs for regulatory science infrastructure and workforce.
- Chapter 5 summarizes challenges, opportunities, and key focus areas for advancing regulatory science offered by individual workshop participants.