FDA’s  2011 strategic plan identified eight priority areas for regulatory science, of  which seven related to medical products where the agency determined  that new or enhanced engagement is essential to the continued success  of the agency’s public health and regulatory mission. FDA Lacks Measurable Goals to Assess Progress Meeting Its Regulatory Science Priorities

FDA’s 2011 and 2013 strategic planning documents do not identify  measurable goals, such as targets and time frames, for assessing  progress in regulatory science. FDA Lacks Information Necessary to Track Funding and Strategically Plan for Its Regulatory Science Priorities across the Agency

FDA does not have the information necessary to track funding and  conduct strategic planning agency-wide for its regulatory science priorities  because most of the centers and offices did not collect information on the  FDA priority areas that were addressed by the projects they funded. Six centers and offices—CDRH, NCTR, OCET, OIP, OMH, and  OWH—fund regulatory science projects that address multiple FDA  priority areas, but generally did not collect information about how  funds are distributed across those priority areas. All six had to  retrospectively identify associated FDA priority areas for our review. Standards for internal control in the federal government state that  complete and accurate data are needed to make operating decisions and  to allocate resources. In addition, to ensure program goals are met, our  work encourages agencies to manage efforts that cut across the  agency. 2.) FDA Reported That the Selected Regulatory Science Projects Led to Changes in Agency and External Stakeholder Practices

FDA reported that the 17 regulatory science projects that we selected for  review helped to advance regulatory science. Dissemination of project findings. These projects resulted in the development of tools or  proposed standards for use by industry or the implementation or use of  new tools or standards by industry or outside organizations. For  example, a CDRH study of radio interference with automated external  defibrillators led FDA to recommend to an international commission  that standards for these types of defibrillators be modified to account  for radio interference. FDA projects targeted at  advancing regulatory science have led to internal and external impacts in  understanding new science associated with medical products. Such goals are a best practice for strategic  planning and could enable FDA to assess and report its progress in  addressing its identified priority areas and strategically plan and allocate  resources for its broader regulatory science initiative. Systematic tracking by each center or office is  needed for the agency to examine obligations across, or progress within,  specific priority areas and would help the agency to strategically plan for  its regulatory science initiative as a whole. Develop and document measurable goals, such as targets and time  frames, for its regulatory science efforts so it can consistently assess  and report on the agency’s progress in regulatory science efforts. Nevertheless, FDA should develop measureable goals  that are related to the impacts that are discussed in HHS’s comments,  including the effectiveness and efficiency of FDA’s regulatory review, new  pathways for medical product development, enhancements in the  agency’s ability to provide useful guidance to sponsors, and new  technologies to monitor manufacturing and real world use of approved  medical products. Appendix I: Description of Projects Included in Our Review of Achievements Resulting from FDA’s Regulatory Science Projects

We examined the achievements related to regulatory science for 17  projects funded by the Food and Drug Administration (FDA). Center or office funding project: ORSI  Priority area(s) covered: Clinical evaluations and personalized  medicine  Funding obligations and time period of funding: $1.425 million  (fiscal years 2010 through 2012)
Description of project: This project had three main objectives: 1)  create protocols, processes, and standards by which a consortium for  tuberculosis biomarkers—composed of three organizations central to  tuberculosis clinical drug development—would operate; 2) create a  repository for receiving, storing, and shipping samples to designated  investigators; and 3) establish a peer review panel to review  proposals related to the discovery and qualification of tuberculosis  biomarkers, especially some surrogate markers.
Why GAO Did This Study

FDA has faced challenges regulating medical products, owing in part to rapid changes in science and technology. In 2010, FDA established a regulatory science initiative that identified eight priority areas for medical products where new research was needed to advance its mission. Legislation enacted in 2012 required FDA to establish a plan for measuring its progress on its regulatory science efforts.
GAO was asked to examine FDA's progress on its regulatory science efforts related to medical products. In this report, GAO (1) evaluates FDA's strategic planning efforts to address its regulatory science priorities, (2) describes FDA's funding targeted at regulatory science projects, and (3) describes the achievements of selected FDA regulatory science projects. GAO compared related FDA strategic planning documents to federal internal control standards and leading practices for strategic planning. GAO reviewed FDA data on obligations targeted at regulatory science projects for fiscal years 2010 through 2014 and reviewed the achievements FDA reported from a sample of 17 projects, chosen to ensure nine FDA centers and offices and priority areas are represented.

What GAO Found

The Food and Drug Administration (FDA) lacks measurable goals to assess its progress in advancing regulatory science—the science supporting its effort to assess the products it regulates. The agency issued strategic planning documents in 2011 and 2013 to guide its regulatory science efforts and identify priority areas for conducting work, but these documents do not specify the targets and time frames necessary for the agency to measure progress overall or within each of the eight priority areas related to medical products. According to leading practices for strategic planning, identifying and using consistent measurable goals in planning and progress documents is important to assessing effectiveness. While FDA cited examples of its achievements in regulatory science in a 2015 report, FDA cannot assess how those achievements constitute progress towards its goals. In addition, FDA lacks information about how funding targeted at regulatory science is distributed across the priority areas. Decisions to award these funds are made by individual FDA centers and offices, which generally did not collect information on the associated priority areas of funded projects. Rather, FDA retrospectively identified these areas for the purpose of GAO's review. The lack of consistent information limits FDA's ability to examine obligations across, or progress within, specific priority areas. Standards for internal control in the federal government state that complete and accurate data are needed to make operating decisions and allocate resources. Furthermore, multiple centers or offices fund projects toward a given priority area and leading practices for strategic planning encourage agencies to manage efforts that cut across the agency.
For the 17 regulatory science projects GAO reviewed, FDA identified achievements ranging from the dissemination of project findings to changes in both agency and external stakeholder practices. For example, FDA reported that all projects resulted in some type of change within FDA. About half of the projects resulted in the agency developing standards, methods, tools, or training that it could use internally, and about one-third of the projects affected guidance or regulations. FDA also reported that about half of the projects resulted in the development of new tools or standards for use by industry or other stakeholders, in areas such as setting new standards for defibrillators to account for radio interference.

What GAO Recommends

To improve strategic planning for regulatory science efforts, FDA should (1) develop and document measurable goals, including targets and time frames, and (2) systematically track funding across its regulatory science priority areas. The Department of Health and Human Services agreed with GAO's recommendations.