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Introduction

Mission Statement of the U.S. Food and Drug Administration


The FDA is responsible for protecting the public health by assuring the safety, efficacy, and security of human and veterinary drugs, biological products, medical devices, our nation’s food supply, cosmetics, and products that emit radiation. The FDA is also responsible for advancing the public health by helping to speed innovations that make medicines and foods more effective, safer, and more affordable; and helping the public get the accurate, science-based information they need to use medicines and foods to improve their health.


SOURCE: FDA, 2007a.

The FDA regulates products representing roughly 25 percent of all consumer spending in the United States (Coalition for a Stronger FDA, 2007). Since 1992, when the Prescription Drug User Fee Act (PDUFA) was enacted, federal funding for the agency has diminished. Currently the FDA’s Center for Drug Evaluation and Research (CDER) relies on the fees that it receives from the industry it regulates to fund its essential programs. At the same time, the issue of prescription drug safety has received widespread public and congressional scrutiny as a result of the highly publicized recall of the arthritis drug Vioxx because of its link to serious cardiovascular events, and more recently the increased risk of suicidal ideation among children being treated for depression with selective serotonin reuptake inhibitors (SSRIs). There is growing public concern about the ability of the current drug safety system to prevent future Vioxx-like events.

In light of this increased scrutiny, the FDA commissioned the Institute of Medicine (IOM) to convene an ad hoc committee of experts to conduct an independent assessment of the current U.S. drug safety system. A con-



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Challenges for the FDA: The Future of Drug Safety - Workshop Summary 1 Introduction Mission Statement of the U.S. Food and Drug Administration The FDA is responsible for protecting the public health by assuring the safety, efficacy, and security of human and veterinary drugs, biological products, medical devices, our nation’s food supply, cosmetics, and products that emit radiation. The FDA is also responsible for advancing the public health by helping to speed innovations that make medicines and foods more effective, safer, and more affordable; and helping the public get the accurate, science-based information they need to use medicines and foods to improve their health. SOURCE: FDA, 2007a. The FDA regulates products representing roughly 25 percent of all consumer spending in the United States (Coalition for a Stronger FDA, 2007). Since 1992, when the Prescription Drug User Fee Act (PDUFA) was enacted, federal funding for the agency has diminished. Currently the FDA’s Center for Drug Evaluation and Research (CDER) relies on the fees that it receives from the industry it regulates to fund its essential programs. At the same time, the issue of prescription drug safety has received widespread public and congressional scrutiny as a result of the highly publicized recall of the arthritis drug Vioxx because of its link to serious cardiovascular events, and more recently the increased risk of suicidal ideation among children being treated for depression with selective serotonin reuptake inhibitors (SSRIs). There is growing public concern about the ability of the current drug safety system to prevent future Vioxx-like events. In light of this increased scrutiny, the FDA commissioned the Institute of Medicine (IOM) to convene an ad hoc committee of experts to conduct an independent assessment of the current U.S. drug safety system. A con-

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Challenges for the FDA: The Future of Drug Safety - Workshop Summary sensus report with recommendations for the system’s improvement—The Future of Drug Safety: Promoting and Protecting the Health of the Public (hereafter referred to as the IOM report)—was released on September 22, 2006. The IOM committee that produced the report identified four major vulnerabilities in the U.S. drug safety system: (1) chronic underfunding; (2) organization problems, particularly inadequate integration of pre-and postmarket data review; (3) a range of technical problems related to the insufficient quantity and quality of postmarket data and inadequate capability to systematically monitor the risks and benefits of drugs after marketing (an underlying issue being the use of modern information technology and informatics, and the human expertise and systems that enable their use); and (4) unclear regulatory authority and insufficiently flexible regulatory tools (IOM, 2007a). Since the IOM report was issued, the FDA has taken a number of steps toward implementing the improvements recommended by the report (see Box 1-1). Like many government agencies, however, the FDA is financially BOX 1-1 Highlights of the FDA’s Response to the IOM Report Strengthening the Science Improving how the agency assesses risk is a central component of the FDA’s efforts to improve pharmaceutical drug safety. The agency is operating in this area with the belief that new scientific discoveries and the expanded availability of new data sources for pharmacoepidemiological research are creating an emerging science of safety that will support a life-cycle approach to drug safety (e.g., by helping to build safety into products prior to approval and by targeting patients who are more likely to benefit from a given product). The FDA has already started or is taking steps toward initiating a pilot program to review systematically new safety data for new molecular entities (NMEs) approximately 18 months after approval; upgrading the electronic Adverse Event Reporting System* (AERS) and expanding safety database resources in an effort to strengthen epidemiological surveillance methods and tools; and employing several Critical Path Initiative** activities designed to improve safety evaluation and establish best practices for protocol reviews. Improving Communication and Information Flow Improving the FDA’s communication and information flow is another key component of the agency’s current efforts to strengthen the effectiveness of the U.S. drug safety system. The FDA believes that open and transparent communication among the agency, health care providers, and patients is paramount to the rapid and

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Challenges for the FDA: The Future of Drug Safety - Workshop Summary strained by its existing responsibilities, and fully implementing the recommended improvements to the drug safety system would require significant financial commitments from the agency. Without providing a full analysis of the costs associated with its recommendations, the IOM report left open a number of questions about the ability of the agency to achieve the envisioned improvements. Attempting to implement the report’s 25 recommendations without a substantial funding increase could further strain the FDA’s resources, thereby making it more difficult to identify safety problems associated with new drugs, to monitor routinely submitted safety data, and to relay the resulting information to the public. effective dissemination of new drug safety information. Accordingly, the agency has already started or is taking steps toward strengthening the role of advisory committees, designing two pilot projects to evaluate different ways of involving Office of Surveillance and Epidemiology (OSE) staff in reviews and regulatory decision making, implementing new procedures for postmarket decision making, implementing a postmarket electronic tracking system, establishing a new advisory committee on communication, publishing an Internet newsletter on postmarket findings, and issuing new guidance on risk communication. Improving Operations and Management In its effort to improve its culture of safety throughout the life cycle of the products it regulates, the FDA is reinvigorating the senior management team of the Center for Drug Evaluation and Research and charging it with leading the organization in an integrated manner, enlisting external organizational consultants to address concerns about tensions between preapproval and postapproval staff and clarify their respective roles and responsibilities, and improving the way in which scientific disagreements are handled.    *The Adverse Event Reporting System is a computerized information database that supports the FDA’s postmarket safety surveillance program. It stores safety information for all approved drugs and therapeutic biologic products.    **The Critical Path Initiative is a national effort led by the FDA to prompt the development of new technologies and leverage those technologies to expedite the product development process for new drugs, biologic products, and medical devices. SOURCE: Galson, 2007. To address this gap, the IOM’s Forum on Drug Discovery, Development, and Translation convened a 1-day symposium in March 2007 to consider the types and magnitude of resources needed to implement some of the most resource-intensive recommendations of the IOM report. The symposium’s presentations and discussions were in most cases framed

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Challenges for the FDA: The Future of Drug Safety - Workshop Summary by selected recommendations from the report, and are summarized here in seven key areas: addressing the FDA’s resource challenges; strengthening the scientific base of the agency; integrating pre- and postmarket review; enhancing postmarket safety monitoring; conducting confirmatory drug safety and efficacy studies; enhancing the value of clinical trial registration; and enhancing the FDA’s postmarket regulation and enforcement. The presentations and discussions included the types and magnitude of resources required in these areas. A session at the close of the symposium looked to the future, exploring prerequisites for revitalizing the U.S. drug safety system and the future of drug safety regulation. It should be noted that, while the IOM report suggested some organizational and cultural changes at the FDA, those recommendations were not a focus of the discussions during the symposium. Additionally, participants did not deliberate upon whether the FDA in its current form is properly configured to lead the efforts that were discussed.