 |
 |
 |
FDA
Home Page | Search FDA Site |
FDA A-Z Index | Contact FDA
The FDA Plan for Statutory Compliance addresses requirements set forth in Section 406 of the Food and Drug Administration Modernization Act of 1997 (FDAMA) (see Appendix A). The Plan identifies those actions necessary to bridge the gap between what FDA is required to do by statute* and expected to do by the public--and what the Agency currently is able to accomplish with existing resources. A high-performing FDA working in concert with its stakeholders is absolutely crucial to promote and to protect the health and well-being of the American public. Given the myriad escalating technological, economic, and health risk challenges, this will not be an easy task for FDA. The passage of FDAMA presents FDA with an opportunity to demonstrate innovative and bold approaches in meeting these challenges for the 21st century. This Plan is one positive step toward moving FDA into conformance with the views of Congress and the Agency's stakeholders.
This document demonstrates that FDA already is making great progress in managing health risks--a job that is becoming more complex and often fraught with uncertainty and unpredictability. The Plan also highlights the fact that the Agency clearly is unable to meet all of the challenges it is expected to address with its current level of resources. Innovation and creative collaboration with external stakeholders will certainly enhance the Agency's abilities to reduce health risks in the long run; but additional resources are essential to help FDA fulfill its statutory mandates.
[* Statutory requirements encompass all provisions of the Federal Food, Drug, and Cosmetic Act (FD&C Act) and its amendments, including FDAMA.]
The Plan specifically addresses the six objectives stipulated by Congress in FDAMA Section 406(b):
To achieve these objectives, the Plan identifies Agency-wide strategic directions for the next 5 years, and specific performance goals for Fiscal Year (FY) 1999. Thus, the total plan presents a picture of the Agency's long- and short-term future that will be reviewed and modified as part of ongoing discussions with FDA's stakeholders, with future Department of Health and Human Services (DHHS) leadership and other parts of the Administration, and with Congress.
This Plan is one element of a total strategic framework mandated by FDAMA that enables FDA to address increasingly complex public health challenges. This framework, outlined in Section 903 of the Federal Food, Drug, and Cosmetic Act as amended by FDAMA (see Appendix A), contains the following key elements:
These elements reflect certain broad themes. First, the Agency should devise and implement strategies in a more open, multi-organizational environment. Congress emphasized throughout FDAMA that consultation, collaboration, and synergy-building with external organizations are paramount to FDA achieving its mission of protecting and promoting public health. Simply put, FDA cannot do the job alone.
Second, Section 903 provides FDA with a more systematic approach to strategic management. The essential elements are clearly articulated: a clear mission, consultation with stakeholders, a plan based on stakeholder input to carry out the intent of the mission, and provision for ongoing feedback, accountability, and adjustment to the plan. The Agency recognizes the importance of this plan for action accountability, as outlined in Section 406(b) of FDAMA, and in establishing an ongoing dialogue with stakeholders to continually improve strategies.
Third, Congress has recognized that an array of capabilities including public education and research {Section 903(d)(2)} are essential elements required to carry out its responsibilities under the Act. The six objectives outlined in FDAMA 406(b) also explicitly stipulate education and scientific expertise as being central to the Agency's modernization plan. Successful public health promotion and protection decisions depend upon a well-developed science infrastructure and an informed public. Without these two elements, desired health outcomes are not possible.
Figure 1 illustrates how FDA is integrating the mandates in Section 903 to form the components of an effective strategic management process. As the figure illustrates, effective implementation of the FDAMA plan depends upon several elements:
Part One of the Plan provides the broad Agency-wide context--steps 1 through 5 above. Part Two of the Plan maps the specific plan for achieving objectives. In Part Two, the specific performance targets for FY 1999 are established based on the Agency's existing resources, reinventions, and collaborative arrangements. FY 2000 performance targets currently are being developed as part of the FY 2000 Budget process and are not included in the Plan. Many factors influence FDA's choice of performance levels, including: extrapolations of past performance, anticipated workload, creative re-engineering to improve internal efficiencies, successful collaboration with FDA's outside stakeholders, and strategic priorities.
Over the years, Congress has dramatically expanded the responsibilities of the FDA. The Federal Food and Drugs Act of 1906, the first national statute enacted by Congress to regulate the American food and drug supply, gave FDA's predecessor agency the authority to remove adulterated or misbranded foods and drugs. In ensuing years, Congress enacted a series of statutes that expanded FDA's responsibilities in a number of directions, including: new product areas (cosmetics, biologicals, and medical devices); additional product characteristics (e.g., efficacy as well as safety); and additional perspectives from which to monitor products (e.g., monitoring prior to market introduction as well as postmarket monitoring).
Beginning in 1996 with the passage of the Animal Drug Availability Act (ADAA) and continuing in 1997 with the passage of FDAMA, Congress enhanced FDA's mission in ways that recognized the Agency would be operating in a 21st century characterized by increasing technological, trade, and public health complexities. To meet these challenges, Congress added explicit phrasing to the Agency's mission statement to ensure that FDA would coordinate its own efforts with regulatory counterparts worldwide. In addition, Congress recognized that external scientists, medical experts, and public health experts must play an increasing role in Agency responsibilities. It defined a new emphasis to be placed on regulatory processes and required more interaction with stakeholders. Through FDAMA, Congress intends to ensure timely availability of safe and effective new products that benefit the public, and to ensure that our nation continues to lead the world in new product innovation and development.
FDAMA defines FDA's new mission as follows:
The Administration shall--
FDA must address a wide range of challenges that serve as potential obstacles to successfully carrying out its health protection mission in the 21st century. To the extent that these challenges remain unaddressed, a gap between expectation and performance will persist. This Plan represents a blueprint for addressing these challenges, thereby narrowing the gap.
Key challenges that FDA faces now and in the near future include:
Each of these challenges is discussed briefly below.
Each year, FDA-regulated firms add more than $2 billion to domestic research and development efforts. For pharmaceuticals alone, this effort currently exceeds $20 billion total, which is triple the effort of only 10 years ago. The growth in research budgets at public agencies such as NIH surely will result in a greater number and wider variety of products that FDA must, by statute, regulate. More importantly, the speed of product development also is accelerating. By streamlining the commercial review process, FDA has helped to reduce the time between discovery and Agency evaluation. But this streamlining also gives the Agency very little time to develop a regulatory framework to handle new technologies. Thus, it is imperative for FDA to continue to engage in close interaction with industry in the early stages of product research and development.
The volume, variety, and speed of new product development presents FDA with the twofold goals of 1) ensuring that consumers enjoy timely public health benefits from these products; and 2) minimizing the health risks associated with consumers' use of these products. FDA resources devoted to premarket review of these products must be carefully allocated so that both goals are addressed. The Agency's current level of resources, however, cannot adequately address both goals in all of the product areas for which the Agency has responsibility.
Product complexity continues to increase. FDA-regulated products will be characterized by unprecedented technological sophistication, while also providing unparalleled health benefits for the U.S. public. The continued benefits of genetic engineering warrant particular attention. New products generated by the biotechnology revolution cover a broad spectrum, including: genetic probes that serve as powerful diagnostics; genetically engineered drug and gene therapies; and biotechnology-based food modifications such as protein-enhanced vegetables. Increased understanding of the human genome, as well as of the genetic make-up of other organisms (genomes of other animals and plants), will yield many new and different products and applications.
The number of sources that produce these new genetically engineered products continues to escalate. The number of biotechnology firms grew dramatically from the early 1980s through 1993, so that by 1993 there were 1,272 firms, more than a threefold increase over the pre-1981 number. By April 1997, nearly 300 biotechnology drugs were in development, tripling the number that were in development in 1989. FDA must have access to the necessary scientific expertise to be able to address the complexity of these new products, and to provide sound regulatory decisions.
Microprocessor and miniaturization technologies are rapidly expanding and enabling significant improvements in implantable medical devices such as pacemakers, cochlear implants, and closed-loop medicine delivery systems that monitor conditions within the body and administer treatments as required. Progress in artificial intelligence has increased companies' ability to apply pattern recognition techniques in such products as Pap smear readers and neural net classifiers.
New combination products, such as food-drug and drug-device combinations, will continue to be generated through the application of biotechnology techniques. Such developments foster improved versions of products already developed and approved, as well as entirely new products. New biological-based products will require the development of new data profiles, because the data used to determine the safety of chemical-based products of the past are neither sufficient nor appropriate for predicting the safety of these new products.
Biotechnology also is being used to develop new assessment tools. More emphasis is being placed on new approaches to assess the product safety of food, dietary supplements, and health care products. These tools include bioassays to improve safety assessments of carcinogenicity and to address emerging concerns of neurotoxicity, immunotoxicity, and developmental toxicity.
New technologies have provided an explosion of innovative diagnostic and therapeutic health products. The consequences of this explosion, however, include a parallel expansion of adverse effects associated with product use. Although the benefits realized from these products still greatly outweigh the problems associated with consumption, these problems must be addressed. To illustrate, FDA received more than one-quarter million reports of suspected drug-related adverse effects in 1997, and this number of adverse reports continues to increase annually. FDA estimates that nearly one million patient injuries and deaths each year are associated with the improper use of FDA-regulated products. Additional injuries and deaths occur under conditions of proper use and accidental injury. For example, of the more than 70,000 injury reports related to medical devices received annually, approximately 25 to 40 percent of the injury or death reports may be attributed to device misuse or operator error. Injury reports received by FDA only represent between 1 to 10 percent of all injuries associated with the use of medical devices. Using these figures, as many as 400,000 incidents per year resulting in patient injury or death may, at least in some way, be attributed to the user-device interaction.
Currently, the FDA Center for Food Safety and Applied Nutrition (CFSAN) receives reporting on food additives, cosmetics, and special nutritionals from the field offices and other sources. To achieve efficiency in monitoring and responding to adverse events, the Center is proposing the establishment of an integrated adverse event reporting system for food and cosmetic products. As the Agency develops more comprehensive adverse event reporting systems, particularly in collaboration with other institutions, the number of reported adverse events likely will increase. If surveillance capability does not expand, the magnitude and severity of product use problems will, to a large extent, remain unknown, and the health risks will be unaddressed.
FDA continues to face a range of threats to public health that appear in a random and discontinuous pattern. For example, crippling infectious diseases such as tuberculosis are re-emerging, bovine spongiform encephalopathy (BSE) became epidemic in the United Kingdom and was unexpectedly linked to the human disease, Creutzfeld-Jakob disease (nvCJD), and more virulent and antibiotic-resistant bacteria have been discovered in food products around the world. These unpredictable threats, coupled with the growing incidence of disease-causing organisms' resistance to existing drug therapies, challenge both industry and FDA to bring innovative, safe, and effective treatments to the market rapidly. The Agency also must address crises that require emergency responses, whether they are the discovery of pesticides in selected imported products, Escherichia coli outbreaks, or intentional product tampering. These events are byproducts of several factors, including continually expanding global trade; new entrants into domestic industries--particularly where emerging technologies are present; and economic pressures on regulated firms to reduce costs in order to ensure short-term survival.
The unpredictable nature of a significant portion of FDA's compliance activity also acts as a severe limitation to fulfilling statutory mandates of inspectional coverage. FDA is attempting to augment its inspection capability with strategies that call for collaboration with states, use of third parties to verify industry compliance, and augmenting industry quality control mechanisms. But even these augmentation strategies require front-end investments to develop systemic capabilities such as data validation, data sharing, and auditing to determine whether protocols are in place. In addition, some stakeholders oppose other third-party involvement. Consequently, in the short run FDA--even in conjunction with collaborators--will not be able simultaneously to satisfy statutory inspection requirements and address all current health and safety threats.
A more knowledgeable and diverse consumer population is escalating expectations for more information, as well as information that is more tailored to their particular needs, concerning the safety of FDA-regulated products. American consumers have become more health-conscious during the 1990s and are seeking more information on the impact of medical products and food on their health. FDA must distinguish between the risks perceived by consumers and their actual risks, and respond accordingly. Based on the additional information that FDA provides, consumers are playing a larger role in protecting their ownhealth.
The elderly population provides a good illustration of why FDA must target its information and regulatory policies to fit the needs of particular market segments. Although the elderly are by no means the only segment with special needs, their numbers have become much more prominent in the general population. By the year 2000, Americans aged 75 and older will be the fastest growing group. The elderly (those over 65) have disproportionately high health care demands. Challenges associated with this patient subpopulation, such as multiple drug interactions, different physiological characterizations and reactions to drug regimens, and the need for better medical device design for home self-diagnostics and therapies, will become more acute. These challenges will require greater inclusion of the elderly in clinical testing for drugs, medical devices, and other FDA-regulated products. Further, the increasing educational needs of the elderly will require more focused education programs, including specific dietary information and foods targeted to their nutritional requirements. The elderly population and food service workers who prepare food for the elderly also will require special education initiatives concerning proper food handling, because as the population ages it becomes more susceptible to foodborne diseases.
FDA participates in the world community of developed, underdeveloped, and developing economies and regulatory authorities. Radical changes in the dynamics of the world structure are underway, driven by several forces: 1) an increasing number of global and multinational firms that produce FDA-regulated products; 2) increasing sophistication of unified economic, political, and regional entities (e.g., the European Union [EU] and Pacific Rim countries); and 3) the response to these conditions on the part of regulatory/standard-setting entities.
The larger drug, biological, device and food firms now operate as multinational companies. New products will be developed, produced, and marketed through a highly networked and global commercial system. The system will have great power to satisfy consumer needs, but will be much more complex to monitor for potential risk than has been the case in the past. This situation will require sophisticated international regulatory responses. Further, the regulatory response by U.S. interests must preserve the delicate balance at the international level between preventing unnecessarily high-risk products from entry into the country, while providing access to novel, important therapies or foods to the American public.
The multinational and global firms are sharing center stage with an increasingly organized set of regional economic and political entities such as the EU, Pacific Rim organizations, North America Free Trade Act (NAFTA) participants, etc. These entities are amassing the economic and political power to attract world trade. The pace of their development is often uneven, but the longer term direction is clear. Raw materials and joint ventures that stretch across national borders are all becoming international elements for FDA to regulate where previously these were purely domestic phenomena. The Agency must now make new decisions on how (or if) to manage each of these new elements. Increasingly FDA must take into account the global trade implications of its decisions.
Organizations such as the International Committee on Harmonization (ICH), the International Standards Organization (ISO), the Global Harmonization Task Force, the International Cooperation on Harmonization of Technical Requirements of Registration for Veterinary Medicinal Products (VICH), and Codex are becoming increasingly important in the determination of the level of acceptable product safety, quality, and efficacy for products trading in the international arena. FDA must maintain a viable voice as standards are prepared and speak with a voice that represents the interests of all of its stakeholders, whether they are consumers, patients, health practitioners, or the regulated industry.
Imported products regulated by FDA represent a significant component of total U.S. consumption. In some sectors, such as seafood, the percentage of total consumption represented by imports is approximately 50 percent. FDA's responsibilities in the import arena continue to expand, without a corresponding increase in resources to do the job. To illustrate: The volume of imports has grown steadily over the past few decades. By 1998 an estimated 4 million FDA-regulated import line items arrived in the U.S. The number of food items, representing the majority of those imports, increased by 21 percent over the last year alone! During that same period, FDA resources to address imports remained essentially level.
And the complexity is increasing--the reality of a truly global economy is adding significant regulatory challenges for FDA. These products are originating in countries that often have less developed health/safety regulatory structures. The increase in volume, variety, and sources of imports may be accompanied by increases in novel pathogens, microbial contamination, and other public health concerns and regulatory challenges for FDA. Developing countries, which once provided raw materials for U.S. manufacturers, and assemblers are increasingly providing finished products to the U.S. market. This conversion could increase the risks associated with such products.
Recent budget proposals and appropriations acts have addressed emerging public health issues (such as AIDS) and long-standing public health problems that received insufficient attention in the past (including reducing youth tobacco use, improving food safety, and accelerating prescription drug approvals). While those problems continue to need attention, inflation has reduced real resources available for FDA's other public health responsibilities, which are necessary to meet the obligations delineated in FDAMA. These include inspections to ensure product safety; review of devices, food additives, blood products, animal drugs, and generic drugs; and adverse event reporting and followup.