An Overview of FDA s Regulatory Compliance Agenda

Transcription

1 An Overview of FDA s Regulatory Compliance Agenda The FDA Regulatory and Compliance Symposium Managing Risks-From Pipeline to Patient Annenberg Hall at Harvard University Boston, MA August 24, 2005 John M. Taylor, III Divisional Vice President, Federal Government Affairs, Abbott Laboratories Former Associate Commissioner for Regulatory Affairs U.S. Food & Drug Administration

2 Introduction Thank you for having me here today. I am honored to speak you this afternoon. I have been asked to provide you with an Overview of the FDA s Regulatory/Compliance Agenda. I will not touch upon drug safety and prescription drug advertising today because others will cover those issues in more detail, however, I will give you my thoughts on other aspects of the Agency s present and future regulatory and compliance agenda. I am not going to focus on specific cases or regulatory actions that may be on the horizon. My crystal ball is not that clear but I will discuss the framework that I believe constitutes the Agency s compliance agenda and give you examples of past, current, and future initiatives and projects that illustrate the goals and framework of that agenda. As you know, for almost one hundred years, the Food and Drug Administration has fulfilled its mandate to protect the public health by enforcing the nation s Food and Drug laws. FDA s mission, however, has become much more complicated. The medical products that the Agency regulates are greater in number and more sophisticated than ever and that does not include the investigational products that are under development. In addition, there is a broader diversity of dietary supplements on the market and a broader range of food choices from the United States and overseas. These are obviously positive developments. 2

3 At the same time though, FDA is unfortunately facing some increasingly difficult threats to the public health that are more complex than those of the last century. These threats include: large-scale criminal activity that is responsible for diverting and counterfeiting FDA regulated products; potentially unsafe medications and foods that may result from increasingly complex production processes; new infectious disease threats; an increasing number of imported pharmaceuticals from unknown sources; terrorism; and the intentional contamination of FDA regulated products. While FDA has received some new resources in recent years to carry out its mission to protect the public health, the growing size and complexity of the Agency s job clearly outpaces its resources and this has real implications for FDA. Accordingly, the Agency has committed resources to craft strategies that focus on reviewing its old compliance methods and where appropriate utilizing new methods that get the best bang for the buck in terms of improving the public health. Background I m going to talk in more detail about what FDA is doing to confront and overcome these challenges, however, first let me provide some background that will help set the stage for where the Agency is headed regarding its regulatory and compliance agenda. For many years, the Agency has used a variety of traditional law enforcement techniques and tools, for example investigations, inspections, import examinations, seizures, injunctions, prosecutions, recalls, and enforcement correspondence, to ensure that FDA 3

4 regulated products are in compliance with the applicable laws and regulations, that noncompliance is identified and corrected, and that any unsafe products are removed from the marketplace. In the mid 1990s, however, the number of inspections, import examinations, and enforcement actions conducted by FDA decreased due to resource constraints and other factors. In response to this trend, FDA began utilizing strategies that were focused more heavily on the use of nontraditional enforcement activities. This allowed the Agency to make progress in using its limited resources more efficiently and effectively. These nontraditional activities included outreach programs that encouraged communication between the Agency and industry regarding FDA s new compliance initiatives. In addition to embracing outreach opportunities, the Agency developed other innovative non-traditional regulatory approaches that helped the Agency leverage its resources devoted to ensuring that FDA s regulated industry was in compliance with the Federal Food, Drug, and Cosmetic (the Act). As a part of the Agency s leveraging efforts, FDA developed training programs that complemented the outreach activities noted above and other intervention strategies aimed at helping industry achieve voluntary compliance with the Act. The medical device grassroots initiative, the implementation of the medical device quality systems regulation, the implementation of the BSE feed rule, and 4

5 the Seafood HACCP training initiatives are examples of Agency initiatives in the late 1990s where these training programs were used. Through these activities, the Agency learned the value of outreach, leveraging, education, and innovation. FDA became more open and more willing to listen and the Food and Drug Modernization Act (FDAMA) codified or added to the steps that the Agency was taking to improve communication with its stakeholders. When I first arrived at the Agency, FDA was not terribly transparent nor was it willing to communicate openly regarding certain compliance issues. I think that this approach hampered FDA s ability to communicate the Agency s expectations to industry and sometimes had diminishing returns in terms of encouraging voluntary compliance. So I think that many of the steps that the agency took to improve communications in the late 90s were indeed positive ones. Despite all of these positive efforts, the Agency realized that non traditional means of ensuring compliance could not be used to the exclusion of traditional tools and that regulatory actions still must form the cornerstone of its compliance strategy. Consequently, for those situations where voluntary compliance was not being achieved, the Agency realized that it still needed to perform its traditional enforcement functions well and be prepared to act in a timely and efficient manner. Therefore, in 1999, at the direction of then Commissioner Henney and Associate Commissioner Baker, I led a working group that undertook an evaluation of the Office of Regulatory Affairs (ORA) 5

6 compliance functions and its use of traditional enforcement tools. My working group focused mostly on ORA but we looked at the Agency s compliance functions as a whole. Based on our review, we recommended that the Agency and ORA needed to improve its compliance functions so that FDA could use its traditional enforcement tools in a more timely and efficient manner when an individual or a firm had not voluntarily complied with the Act. Accordingly, we drafted a report that set forth our ideas and proposed approaches to bringing greater quality and balance to FDA s use of its traditional and nontraditional tools. Recent Efforts to Strengthen the Agency s Compliance Agenda As a result of this review and similar efforts throughout FDA, the Agency at the beginning of this decade began reemphasizing the use of efficient risk management in those compliance programs where it was already being utilized. At the same time the Agency emphasized the use of efficient risk management in those compliance programs where it was not being utilized. By efficient risk management, I mean the use of the best: biomedical science, food science, risk analysis and risk management science so that the Agency can accomplish the most with the limited resources and authorities it has to protect the health of the public-without imposing costs that unnecessarily make food and medicines less affordable or available. The utilization of scientific risk-based principles is not new to FDA but the greater utilization of these principles in the context of FDA s compliance efforts 6

7 required strengthening and became one of the Agency s top priorities. To further this goal, elements of both Commissioner McClellan s and Commissioner Crawford s strategic plans included revisiting the currency and applicability of long standing regulations in light of scientific and technological advances since the inception of those rules; strengthening the involvement of scientific experts from throughout FDA in the Agency s compliance functions so that they can help quantify risk; identifying and targeting violations of the Act and its regulations that have the greatest public health significance; strengthening and consolidating FDA s IT capability so that the Agency can evaluate and use its inspectional and enforcement data better to identify compliance trends; identifying the best enforcement tools to maximize the Agency s enforcement impact; focusing inspection resources in those areas where the Agency finds violations with the greatest public health significance; and focusing resources based on the element of risk associated with the violations noted above. Now, this is by no means an easy undertaking. As you know, properly assessing risk poses many challenges. It is a challenge to assess risk because FDA s knowledge of science and risk is constantly evolving, and an assessment of those risks and their priorities will invariably change. In addition, as a part of crafting a meaningful strategic approach to compliance, FDA needs to ensure that inspectional, compliance, regulatory and enforcement strategies, compliance programs, compliance policy guides, and resource allocations reflect the change in risk. Moreover, risk is not measured merely by counting the 7

8 number of bodies in the street. Risk can also be defined by the risk to the integrity of the Act and its implementing regulations; so, as you can see, many elements of risk have to be evaluated, reconciled, and applied consistently across products and program areas. As I have noted on other occasions, there are undoubtedly disparities between FDA s inspectional efforts, compliance efforts, resource allocations and the risks associated with certain FDA regulated products. FDA, however, has taken and continues take meaningful steps to improve its ability to assess and prioritize risks. Obviously it is different to quantify risks associated with particular violations versus particular products, but if the Agency can continue to make inroads into this arena it will undoubtedly enhance its surveillance and analytical capabilities and its regulatory and compliance strategies. The contemplation and implementation of a meaningful compliance agenda that utilizes risk management principals to assist in the planning of FDA s inspectional and enforcement activities is indeed a daunting task. However, the benefits of using a risk based approach include: a better articulation of why the Agency cites certain inspectional observations or brings enforcement actions in one context but not another; a better regulatory and compliance infrastructure that is less subject to swings in the enforcement pendulum; a greater emphasis on mission relevance when the Agency performs its compliance functions; and a better understanding and a subsequent decrease in actions based on the disparity between perceived and actual risks. Towards this end and across all 8

9 program areas- FDA has initiated a critical, comprehensive review of its practices relative to the application of risk management principals in the planning and prioritizing of its regulatory and compliance efforts. These efforts reflect the Agency s commitment to the consistent adoption of risk management principles throughout all of the Agency s components and functions. Current and Future Efforts to Strengthen the Agency s Compliance Agenda There are some key principles driving the Agency s adoption of a broader scientific risk based compliance agenda. Some of these principles are based on the Agency s historical experiences while others are based on the need to confront current and future scientific, technological, and regulatory challenges. These principles include: using clear and consistent guidance and communications with regulated firms to promote voluntary compliance with the law; using leveraging and working with partners, including other Federal and state agencies as well as private oversight organizations, to bring more resources and a more coordinated, powerful approach to enforcement; using the latest science and risk management principles and making sure that the Agency s practices reflect and allow for, the latest innovations in production, inspection, and enforcement techniques; and using deterrents because the other steps don t always work and the Agency still needs to be able to seek out bad actors aggressively, and punish them using the most effective tools available, including civil enforcement actions and criminal prosecutions that will 9

10 stand up in court. These principles form the cornerstone for the agency s compliance agenda and I will now go into more detail about some of these elements. With regards to communication, by building upon the steps taken in the late 1990s, and working to help businesses understand the relevant statutes and regulations -especially the large number of small businesses that the Agency regulates - FDA can improve compliance with the law. FDA does not and will not have the luxury of resources or time to play gotcha. IF you do not believe me just look at the Agency s budget trends. Instead an important component of efficient and effective enforcement is clarity and effective communication that can be used to get more companies into compliance with the law at a lower cost. At the end of the day companies have the primary responsibility for assuring their own compliance and the best tools available to achieve this goal so the Agency must continue to step up its efforts to issue clear compliance guidances to industry, to provide opportunities for communication, and to sponsor more educational programs so that industry has a clear understanding of the Agency s expectations. A good example of this effort is the outreach and educational work that the Agency has done in connection with its rollout of the regulations implementing the Bioterrorism Act. This rollout will undoubtedly serve as a model for important regulations that the Agency will implement in the future. I believe that FDA wants to make sure everyone knows how to comply 10

11 with rules designed to protect consumers and patients and that the Agency will remain committed to this goal. In regard to leveraging, the Agency is helping to address the ever increasing demands placed upon it by exploring the utilization of first party and third party inspectional approaches and by looking for better ways to build upon its relationships with its regulatory partners. The following are just a few examples of the partnerships that the Agency has formed. FDA has worked closely with the Federal Trade Commission (FTC) under a liaison agreement that coordinates FDA s authority over claims on product labeling and FTC s authority over advertising claims. In addition, the Agency has an ongoing collaboration with the FTC that is focused on their dual law enforcement and consumer education efforts in connection with the fraudulent marketing of supplements and other health products on the Internet. The FDA also works with the Department of Homeland Security s Bureau of Customs and Border Protection (CBP) and Immigration Customs and Enforcement (ICE) on numerous counterfeit drug cases and on safety and enforcement issues related to the growing number of pharmaceutical imports. In addition, the Agency entered into an MOU with CBP that led to the training and cross designation of thousands of CBP agents who are working with the Agency to implement and enforce the FDA bioterrorism regulations that are meant to protect the safety of the nation s food supply. 11

12 FDA is also working closely with the states on such issues as inspections and testing, infectious diseases, illegal pharmacies, counterfeit drugs, counterterrorism and the enforcement of the Agency s BSE feed rule. In addition, FDA works with the Health and Human Services (HHS) Inspector General to address kickbacks and other fraudulent and abusive practices that increase health care costs, including improper drug promotion activities; with the Drug Enforcement Administration to address the growing problem of prescription drug abuse and controlled substances sold illegally over the Internet; and with the Securities and Exchange Commission (SEC) to address misleading statements made by publicly held medical product firms. To cement the last of these relationships, FDA finalized a formal relationship regarding referrals to the SEC when FDA sees companies misrepresenting its discussions with them to the public markets. The Agency appreciates these valuable partnerships. They enable FDA to get maximum impact and reach from its limited resources and I believe that the Agency will continue to build on these existing relationships and establish new relationships with other partners in the future. From the standpoint of science, FDA is working to make sure that all of its decisions are based on the most up to date science available and are risk based. I wholeheartedly believe that the Agency s continued implementation of key initiatives that improve upon, and advance, the public health is dependent upon an appreciation of the roles that cutting edge science, 12

13 coupled with the adoption of risk-based principles, play in their success. One initiative that is illustrative of this approach is the Agency s recent efforts to enhance its pharmaceutical quality program. In 2002, began to strengthen the incorporation of risk - based practices into its pharmaceutical program s core regulatory and compliance functions as part of the Agency s Pharmaceutical Quality Initiative entitled, Pharmaceutical cgmps for the 21 st Century: A Risk-Based Approach. The title of the initiative is a bit of a misnomer because the initiative focuses on issues beyond FDA s current good manufacturing practice regulations (cgmps). Specifically, this initiative builds upon and enhances the Agency s existing efforts to ensure pharmaceutical quality in the areas of human and veterinary drugs and biologics and it demonstrates the Agency s eagerness to look at its CGMP regulations in a different light due to changes in science and technology. As the Agency approached the 25 th anniversary of the last major revisions to the drug cgmp regulations, FDA recognized that it was time to step back and evaluate the currency of both the cgmp program and the pre-market review of chemistry and manufacturing issues. The Agency s Pharmaceutical Quality Initiative builds upon the many successes of these two programs and helps them continue to be successful in the future by keeping pace with advances in pharmaceutical science and manufacturing technologies. Moreover, the initiative was designed to improve public health promotion and protection by focusing on the integration of quality systems and risk management approaches 13

14 into existing programs and encouraging the adoption of modern and innovative manufacturing technologies. It also sought to enhance the integration of the pre-approval review and cgmp programs and achieve a more consistent interpretation and application of FDA regulations and policies across Agency organizational components including programs outside the pharmaceutical arena. Therefore, to the extent that the initiative impacted the device or food programs, representatives from these programs were involved in the initiative. In addition, the initiative focused on strengthening the Agency s use of existing and emerging science and analysis to ensure that limited resources are targeted to address important quality issues, especially those associated with predictable or identifiable health risks. As a part of the Agency s assessment of its product quality programs, FDA assessed current practices as well as available new tools of manufacturing science. FDA s assessment helped it create a new framework for the regulatory oversight of manufacturing quality that has put the Agency on a path to restructure its oversight of pharmaceutical quality regulation, thereby developing the product quality regulatory system of the future. All of these steps enhance the agency s ability to protect the public health. Following the second year anniversary of its launch, FDA issued a final report that highlights specific steps that the agency has taken and will take to develop and implement a quality system management and risk-based product quality regulatory system for pharmaceuticals. The report, which is on FDA s 14

15 website, describes the accomplishments and plans for the future resulting from FDA s completed assessment of its GMP regulations, and current practices and the new tools in manufacturing science that will enable a progression to controls based on quality systems and risk management. You will see in the final report that the Agency has accomplished many projects and identified future projects that further the goals noted above and should lead to a strengthening of the agency s regulatory and compliance framework by using the latest science and technology. Despite the fact that some of the projects that are associated with the initiative are ongoing and may take some time to come to fruition, the following remain FDA s guiding principles: risk based orientation; science based policies and standards; integrated quality systems orientation; international cooperation; and strong public health protection and they will continue to drive the Agency s work in the future. The Agency s primary focus though will remain the same; to strengthen the agency s oversight of pharmaceutical quality and to minimize the risks to the public health associated with pharmaceutical product manufacturing. These activities constitute concrete steps toward achieving FDA s goals for a 21 st century, state-of-the-art regulatory system for pharmaceutical quality and manufacturing. Moreover, the lessons learned in this initiative are being applied to FDA programs outside the pharmaceutical arena. For example, FDA s evaluation of the Agency s recall and enforcement correspondence processes and the Agency s review of the food CGMP program are being 15

16 conducted in a manner that utilizes some of the lessons leaned from the evaluation of the pharmaceutical program. These accomplishments and projects not only contribute to the overall protection of the public health, they simultaneously ensure that safe and effective drugs and medical devices and safe foods are available to the American public. One point of emphasis though, FDA remains committed to strong enforcement of the existing cgmp requirements, even as it is examining and revising its approach to these regulatory programs. That is why the Agency continues to take enforcement actions, as it deems appropriate, for failure to comply with cgmp regulations. This leads me to the importance of providing meaningful deterrents in cases where FDA can t achieve full compliance through better guidance, better science, and better partnerships. The Agency continues to bring significant civil and criminal enforcement actions. By pursuing significant actions and publicizing them, the Agency sets a clear deterrent for illegal behavior, particularly in areas related to the most serious threats to public health. Whether it is injunctions, seizures, civil penalties or prosecutions, the Agency s willingness to pursue court actions is intended to and does - send a clear message that if you threaten the public health in order to make a profit the Agency will take steps to stop you. To underscore this message and enhance its ability to recognize potential actions that will have the most impact in terms of protecting the pubic health, ORA is holding conferences at each of its Districts to discuss 16

17 how to identify and bring those cases that have the greatest public health impact. This continues to be an important message because as I stated earlier, the other steps that the Agency takes to encourage voluntary compliance do not always work and the Agency still needs to ensure compliance with the Act using the most effective tools available. Conclusion Today, I have tried to highlight what I believe is the Agency s regulatory and compliance agenda and touch upon areas where this agenda is being applied. I have also discussed some of the steps that FDA is taking to strengthen its regulatory and compliance infrastructure and processes. Despite the challenges that confront FDA, its first priority is still to protect the public health and there are many areas where the agency is pursing this goal. For example: preventing prescription drug diversion, especially involving drugs of abuse; continuing to work hard to prevent the importation of unsafe foods or potentially unsafe drugs that are improperly labeled, poorly stored or poorly manufactured; fighting counterfeiters of FDA regulated products; acting against FDA regulated products sold illegally over the Internet; enhancing the nation s food security; ensuring that pharmaceuticals, devices, and foods are properly manufactured or processed; and preventing emerging infectious disease threats, just to name a few. These are real challenges that underscore the agency s need to continue to evolve and use the best approaches available as part of its 17

18 regulatory and compliance agenda in order to ensure that its resources are being used as efficiently as possible to protect the public health. 18