Strategic Priorities: Cross-Cutting Strategic Priorities
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2.0 Cross-Cutting Strategic Priorities
The Commissioner has selected five cross-cutting areas to serve as strategic priorities over the next five years. These include efforts to: 1) Advance Regulatory Science and Innovation; 2) Strengthen the Safety and Integrity of the Global Supply Chain; 3) Strengthen Compliance and Enforcement Activities to Support Public Health; 4) Address the Unmet Public Health Needs of Special Populations; and 5) Advance Medical Countermeasures and Emergency Preparedness.
2.1 Advance Regulatory Science2 and Innovation
Recent innovative breakthroughs in science and technology — ranging from sequencing of the human genome to advances in the application of nanotechnology to new medical products — have the potential to transform our ability to prevent, diagnose and treat disease. These developments will result in moving treatment strategies towards approaches that are tailored or personalized to individual patients, thus maximizing the benefit of treatments while decreasing their safety risks. Similarly, advances in research and information technologies are enabling us to more efficiently identify microbial pathogens, track and trace food contamination outbreaks, develop new approaches to safety assessment, and determine where foods and other FDA-regulated products are produced or manufactured, stored, how they are transported, where they go and who uses them. These tools also can play an important role in preventive health by enabling more comprehensive immunization strategies, especially in the face of emerging pandemics.
For these advances to reach their full potential, FDA must play an increasingly integral role as an agency that both protects the public health by ensuring safe and effective products, and promotes the public health by fostering innovative approaches and solutions on behalf of some of the most compelling health and medical challenges. FDA must also participate more actively in the scientific research enterprise directed towards new treatments and interventions. We must also modernize our evaluation and approval processes to ensure that innovative products reach the consumers and patients who need them, when they need them. These new scientific tools, technologies, and approaches form the bridge to critical 21st century advances in public health. They form what we call regulatory science: the science of developing new tools, standards and approaches to assess the safety, effectiveness, quality and performance of FDA-regulated products.
A robust regulatory science program will strengthen biomedical advances, food safety, and many other aspects of FDA’s work. Advances in regulatory science are critical to effectively translate cutting-edge developments in science and technology into promising products and therapies for the Americans who need them. Americans cannot take full advantage of the breakneck speed of biomedical research unless we also achieve innovations in regulatory science. Just as biomedical research has evolved over the past few decades, regulatory science must also evolve in important and powerful ways. As an example, nanomaterials have recently received considerable national and international attention with the development of new and unique hybrid materials to treat disease and as new analytical tools for biotechnology and in the life sciences. Research on nanotechnology-based materials will provide a better understanding of current safety tests and the toxicity of nanomaterials in products for human use, such as over the counter drugs, cosmetics, food ingredients and packaging or dietary supplements.
Stronger regulatory science is necessary to bridge the gap between basic research discoveries and approved drugs, devices, food ingredients and packaging, and biologics. Modernizing toxicology safety assessment is a critical gap in regulatory science because advanced toxicology studies are essential to advance safe product development. Modernizing approaches to toxicology studies will develop new and better ways to predict how novel compounds will behave in humans. These studies will reduce drug and other product development costs, the time to market for new technologies, and help prevent adverse health events.
The National Center for Toxicological Research (NCTR) plays an important role in conducting FDA mission-critical, peer-reviewed, translational research to develop a scientifically sound basis for regulatory decisions and reduce risks associated with FDA-regulated products. This NCTR research evaluates the biological effects of potentially toxic chemicals or microorganisms; defines the complex mechanisms that govern their toxicity; aids in understanding critical biological events in the expression of toxicity; and develops new scientific tools and methods to improve assessment of human exposure, susceptibility, and risk. NCTR works in partnership with each product center within the FDA to expand agency research efforts within the centers to develop new tools and standards for evaluating safety of novel drugs, biologics, or devices.
Over the next five years, FDA will create and implement a strategic plan for expanding and modernizing the field of regulatory science within the agency. This plan will complement each of FDA’s program specific strategic plans, which focus on advancing scientific approaches and tools critical for FDA to translate science into regulation and public health. In addition, FDA will develop an innovation strategy that focuses on its role in facilitating the development of new biomedical products and emerging technologies. At the same time, we will work to strengthen science as a whole within FDA. The agency charged with judging the safety and effectiveness of drugs and other medical products — and monitoring the safety of those products as long as they are on the market — must possess a scientific capability equal to that task and become engaged in mission-critical fields of applied research, including systems biology, wireless healthcare devices, nanotechnology, medical imaging, robotics, cell- and tissue-based products, regenerative medicine, and combination products. In that spirit, FDA has already begun identifying strategies to recruit and retain outstanding scientists and will work collaboratively with other agencies, industry, and academia to define and advance science in high-priority areas critical to the health of individuals, the national healthcare system, and global public health.
The strategic plan for advancing regulatory science will pave the way for a range of regulatory activities, including setting standards for products that address unmet public health needs, identifying and mitigating the spread of disease using informatics, modernizing toxicology and hazard assessments, protecting the food supply, and regulating tobacco. The plan will enable FDA to leverage the latest in science and technology, along with FDA know-how, to bring a new generation of medical products — personalized therapies, stem-cell therapies, and genetic diagnostics, among others — to the American people.
SCIENTIFIC COMPUTING INITIATIVE
FDA houses the largest known repository of clinical data — unique, high-quality data on the safety, effectiveness and performance of drugs, biologics and devices, both before and after approval. Despite the availability of these data, questions about subpopulation responses and underlying placebo effects remain unanswered. FDA data could be used to address fundamental questions about patient subsets who respond in varying ways to new therapies, or for whom a drug is more or less safe. In October 2010, FDA competitively awarded funds to a Johns Hopkins University-led research team to advance Patient Centered Outcomes Research (PCOR) by leveraging FDA’s immense stores of data. The Partnerships in Comparative Effectiveness Science (PACES) initiative will facilitate the development of PCOR studies, which will allow FDA to better understand which interventions are most effective for patients under specific circumstances — a key part of the FDA's public health mission. The standardization of FDA data, coupled with a better understanding of clinical interventions, has the potential to ultimately transform the regulatory review and decision-making process.
The electronic receipt of study data is vital to the efficiency of the medical product review and approval process, as is developing an environment conducive to analyses of large data sets. The ultimate goal of having the ability to review multiple studies that can be compared and ana¬lyzed requires data harmonization and standardization such that comparisons between data can be made effectively. The data must then be organized in a com¬mon database so that it can be queried by topic and analyzed to address key questions. These goals require investments in informatics hardware and software and the development of standardized data models for relational databases and scientific computing. FDA has sponsored the development of a clinical trials repository that will be capable of handling this task.
2.2 Strengthen the Safety and Integrity of the Global Supply Chain
Challenges Presented by Globalization
- Increasing volume of imported products
- Greater complexity in imported products
- More foreign facilities supplying the United States
- Incomplete regulatory information about supply chains
- Patch work of foreign, federal, and state oversight of product safety
- Greater opportunities for economic adulteration and intentional fraud
- National security threats
- Enforcement tools that do not reflect today’s commercial practices
- Corporations lacking accountability
- Current import regulatory system an honor system
Today, maintaining the safety of America’s food and medical products is a serious challenge. Complex global supply chains, international trade, the foreign sourcing and manufacture of regulated products, and the increase in the volume and complexity of imported products have forced FDA to reevaluate its approach to supply-chain safety. We have seen the warning signs: contaminated heparin, melamine-tainted pet food, counterfeit glucose test strips, to name just a few. Globalization presents a host of internal and external challenges to the design, development, manufacturing, and distribution of regulated food and medical products — challenges that make critical the prevention, detection, intervention, and response to product safety.
More than 20 million import lines of food, devices, drugs, and cosmetics arrived at U.S. ports of entry in Fiscal Year (FY) 2010, more than three times the number of imports 10 years ago. Today, there are more than 130,000 importers of record and about 300 ports of entry in the United States. The array of regulated products comes from 300,000-plus facilities in more than 150 different countries.
History of Import Volume vs. Import Staffing Levels
Staffing levels expressed as Full-Time Equivalents (FTE), including Foreign Inspections
The growing challenges of globalization have far outstripped the FDA’s resources for inspection and quality monitoring, and the inability to maintain adequate oversight means potential risk to consumers grows every year. Addressing these challenges will require a paradigm shift — to a focus on prevention, on stopping threats before they ever become reality. The border must be viewed as a final checkpoint for preventive controls, rather than the primary line of defense against unsafe imports.
FDA must require more — and better — information about product supply chains and monitor this information throughout the product life cycle, and regulatory standards must foster corporate responsibility to identify, protect, and control risks. Such an effort will entail more strategic and consistent coordination among foreign, federal, and state counterparts. FDA needs novel and updated compliance and enforcement tools, a greater global safety net that includes a global alliance of regulators, coordination of activities with international capacity-building and standards-setting organzations, new adequate funding to allow for inspections, examinations and sample collections and analysis, and updated systems, including IT support, to assist with the increased workload. These changes are essential if we are to protect product safety in a way that Americans expect and deserve.
ANALYTICAL TOOLS INITIATIVE
FDA continues to implement the Analytical Tools Initiative (ATI), established to explore new or previously unused technologies for analysis and directed screening in the field and for rapid or high throughput analysis in the laboratory. The ATI was established to bridge the gap between industry/academia and FDA scientists on new technologies and applications to FDA-regulated products. The ATI’s main objective is to provide rapid analytical tools for field investigators and/or FDA scientists.
In support of this initiative, FDA is assessing tools for the field investigator and analyst. Field-deployable kits and instruments are being evaluated to incorporate into the “investigator’s toolbox” to be used at the site of sample collection. Instruments for the laboratory, such as hand-held devices, are also being evaluated to enhance laboratory capacity and capability.
FDA is training field staff on the use of Counterfeit Detector 3 (CD3), a hand-held device for import personnel to detect suspected counterfeit drugs and/or packaging. FDA is also training field staff on the use of a portable X-ray Fluorescence (XRF) device capable of detecting toxic elements in imported products such as foods and dietary supplements. The focus of interest for toxic elements is primarily for the five elements: Lead (Pb), Cadmium (Cd), Mercury (Hg), Arsenic (As), and Selenium (Se); although additional media and analyte targets may be identified in the future.
Once training is completed, these devices will be deployed to investigators located at ports of entry to assist with efforts to ascertain the safety of products offered for import.
2.3 Strengthen Compliance and Enforcement Activities to Support Public Health
To protect the public health, FDA must act swiftly and aggressively to guarantee effective enforcement of and compliance with FDA laws and regulations. FDA enforcement actions affect not only the manufacturing and distribution of foods, drugs, biologics, medical devices, tobacco, and cosmetics, but also their development and marketing. Enhancing FDA’s compliance and enforcement programs will strengthen the agency’s focus on preventing problems and responding rapidly when violations occur.
FDA is implementing a number of new programs designed to sharpen the effectiveness and timeliness of its regulatory, compliance, and enforcement systems. The agency established deadlines for industry to respond to significant inspection findings, which enable FDA to take enforcement action more rapidly if a manufacturer has not corrected violations documented after an inspection. FDA has implemented processes to prioritize follow-up inspections after the agency has issued a Warning Letter, classified a major recall, or taken other significant enforcement and compliance actions. These processes will ensure that violative products are no longer available to the American public and that necessary corrective actions are taken to prevent harmful products from being manufactured and sold.
With the enactment of the FDA Food Safety Modernization Act on January 4, 2011, FDA is now authorized to mandate a recall of unsafe food if a food company fails to do it voluntarily. The law also provides a more flexible standard for administrative detention (the procedure FDA uses to keep suspect food from being moved); allows FDA to suspend the registration of a food facility associated with unsafe food, thereby preventing it from distributing food; and directs the agency to improve its ability to track both domestic and imported foods.
Another important component of FDA’s enforcement program is protecting the public health through criminal prosecution. FDA’s criminal enforcement program uses stringent sanctions, including prison sentences, fines, restitution, and forfeiture to deter conduct that violates FDA-enforced laws. These sanctions are made public, thereby informing consumers and helping to ensure greater compliance to protect the public health. The success of FDA’s criminal enforcement efforts sends a clear message to would-be violators: crimes involving FDA regulated products will not be tolerated.
Ensuring that products are effective and that they are safely manufactured and delivered to the American consumer requires the cooperation of a broad network of FDA field offices, local, state, and territorial regulatory authorities, and foreign government officials. In the next five years, FDA will work more closely with these regulatory and enforcement partners to share laboratory and enforcement data. This increased collaborative approach will enable faster identification of threats to the public health and quicker response times, and will strengthen our ability to ensure appropriate corrective actions are taken to reduce the likelihood of harmful products being manufactured and distributed.
2.4 Expand Efforts to Meet the Needs of Special Populations
Therapeutic breakthroughs and clear and timely public health information should be available to all Americans. There are numerous challenges to achieving this goal. For example, historically women and minorities have been underrepresented in clinical trials, making it difficult to assess whether a medical product will be safe and effective for them. Underrepresentation is also true for the pediatric population because many medical products are tested only in adults. Questions regarding effectiveness, pediatric dosing and side effects for drugs often go unanswered. Similarly, medical devices are often not tested in or sized for a relevant pediatric population. Assuring that products are safe and effective for people with rare diseases is particularly challenging, because the patient populations are too small to support standard clinical trials. For these groups, there may be fewer — or riskier — available therapies. FDA is committed to reducing this risk through targeted scientific programs and culturally sensitive outreach efforts.
As we entered the 21st century most therapies being used in children still had not been adequately studied. As a result of legislative research incentives and mandates, there have been more than 385 labeling changes with new pediatric information. Over 15% of the studied products were shown not effective in children and 20% had new safety information specific for children identified.
Despite this progress, much work remains to be done. Over the next five years we need to develop validated endpoints for neonates if we are to more effectively treat this vulnerable population. We also need to explore why so many products that work in adults appear to not work in children—is it dosing, differences in pathophysiology, or for medical devices the need to incorporate new design concepts? Additional efforts will focus on understanding the long-term safety of products, including devices, used chronically in children. The limited number of children with certain conditions means pediatric trials require many sites and are global in nature. In efforts to ensure children are enrolled in scientifically and ethically sound trials, FDA will expand its collaboration with the European Union, Japan, Australia, and Canada, and will establish liaisons for collaboration with other countries.
FDA will be supporting scientific advances in women’s health through grants for FDA and collaborative regulatory science research. Building on ongoing activities, we will also plan to host new internal and external scientific dialogues and workshops to identify gaps and opportunities in women’s health research.
The challenges are not only scientific. FDA must make available targeted public health information that is appropriate for the target population. Language barriers create additional challenges. FDA is planning an array of targeted activities to meet these challenges.
FDA created a new Office of Minority Health in 2010 to address an array of challenges to reduce health disparities in the United States. FDA will have a focal point for the ongoing and new activities to meet the public health needs of minority populations. FDA is already undertaking obesity prevention programs targeting minority populations. We are part of a coalition of Latino consumers and providers called "Latino Initiatives Committee Por Tu Familia”, which plans educational workshops to promote healthy behaviors. FDA manages partnership agreements with national and community-based organizations to increase access to FDA health information for Hispanic Americans, Asian-Americans and Pacific Islanders, and Native Hawaiians. We are exploring additional partnerships to improve health literacy for underserved populations and to diversify options for FDA’s distribution of health information.
FDA recognizes that communications must be adapted to meet the needs of many groups who differ with respect to literacy, language, culture, race/ethnicity, disability, and other factors. As part of FDA’s Strategic Plan for Risk Communication, FDA committed to specific actions designed to improve our capacity to effectively communicate with different populations. These include: training FDA staff on health literacy and basic risk communication principles, considerations, and applications; partnering with consumer and patient organizations to increase availability of FDA communications in a variety of languages and for literacy-challenged audiences; and regularly measuring plain language and appropriate reading level for audiences targeted by communications.
Social media tools can help meet some of FDA’s communication challenge. We are planning to use social networks to create a virtual community of organizations and individuals to disseminate FDA science-based information on women’s health. We will also be collaborating with other government partners, to integrate FDA information on women’s health into their programs.
SCIENTIFIC INNOVATION FOR RARE DISEASE THERAPIES
An estimated 6,000 rare diseases, most of which are serious or life-threatening, affect more than 25 million Americans. Because each disease, by definition, affects fewer than 200,000 Americans, market incentives may be insufficient to drive the investment needed to develop medical products to prevent, diagnose, and treat these conditions. Because the patient populations are small, product testing presents significant scientific challenges calling for innovative approaches. FDA has identified this issue as a priority and is taking steps to meet the needs of these patients.
Perhaps our best known work to advance therapeutics for rare diseases is our Orphan Product Program. This program fosters clinical studies of promising therapies, designates eligible orphan products, and confers marketing exclusivity for orphan drugs and other benefits designed to spur product development. This important program anchors FDA’s efforts to meet the public health needs of people with rare diseases.
We are working to build on this foundation. For example, FDA established two new expert review groups, the Rare Disease Review Group and the Neglected Disease Review Group, consisting of FDA staff scientists from an array of pre-clinical and clinical disciplines. These groups are currently evaluating FDA activities and will be recommending options to the FDA Commissioner for further supporting and facilitating the development and evaluation of medical products for these conditions. As part of this review, the groups will take into account the recommendations of the 2010 Institute of Medicine (IOM) study of national policy for rare disease research and related medical product regulation.
In February 2010, FDA created a position of Associate Director for Rare Diseases in the Center for Drug Evaluation and Research (CDER), to help develop scientific and regulatory innovations for development and evaluation of new treatments for patients with rare diseases. Building on this new capacity, FDA is planning a series of scientific workshops to address important and difficult rare disease research issues and is developing a “rare disease database” to establish the natural history of rare diseases to assist with planning trials to test rare disease therapies. We are enhancing collaborations to increase transparency, share advice, and establish new programs with an array of public and private research and patient advocacy groups.
2.5 Advance Medical Countermeasures and Emergency Preparedness
The events of 9/11 and the subsequent bioterrorist attack involving the mailing of anthrax-containing envelopes forever changed the way Americans view public health and their personal security. Despite considerable financial and human resource investments since 2001, the United States does not yet have the range of medical countermeasures (MCMs) it requires to rapidly and effectively respond to a deliberate chemical, biological (such as anthrax or smallpox), radiological or nuclear event or naturally-occurring infectious disease outbreaks. There are few FDA-approved MCMs (drugs, vaccines, diagnostic tests, personal protective equipment, and supplies) to respond to these types of public health emergencies. Moreover, there is limited capability to rapidly develop a new MCM in response to a new or emerging threat, and only limited capacity to ramp up production of existing MCMs once an event is detected. The complex regulatory pathway required for approval of these types of medical products is one major contributing factor to the limited availability of MCMs.
On August 19, 2010, Department of Health and Human Services (HHS) Secretary Kathleen Sebelius released a report of an extensive review of the federal government’s processes and infrastructure required to develop, approve, and stockpile MCMs. The review identified FDA as one of the most critical components of the Nation’s Public Health Emergency Medical Countermeasures Enterprise. FDA is responsible for evaluating medical product safety and effectiveness; as a result, FDA has significant understanding of the challenges associated with product development. The development and regulatory review of these products requires specialized knowledge and scientific expertise. Harnessing FDA knowledge and expertise in the form of a comprehensive MCM initiative will help to establish regulatory pathways based on the most advanced scientific foundations available, accelerate MCM development towards approval, and realize the promise of new technologies for faster development and flexible, rapidly scalable manufacturing of vaccines and other MCMs.
To help achieve these goals, FDA must work closely with its federal government Enterprise partners, as well as industry and academia. To this end, FDA has developed a MCM Action Plan with 3 pillars:
1. Enhancing the Regulatory Review Processes for MCMs
To advance high priority Enterprise MCMs and related technologies, FDA will establish multidisciplinary Public Health and Security Action Teams that will tackle the range of regulatory, scientific and policy issues facing MCM development and approval. These Action Teams will ensure consistent regulatory approaches and efficient implementation of best regulatory review practices while fostering proactive communication with sponsors and federal government partners. These Action Teams will also allow FDA to anticipate challenges that inevitably emerge during product development and, by engaging with Enterprise partners, address issues that contribute toward delays in MCM development.
2. Advancing Regulatory Science for MCM Development and Evaluation
FDA’s MCM regulatory science program will be implemented through internal and collaborative research, as well as through partnerships with academia, federal government agencies and industry to explore solutions to complex scientific regulatory problems and to identify situations in which the application of new science could simplify or speed product development and/or the regulatory process.
3. Optimizing the Legal, Regulatory and Policy Framework for Effective Public Health Response
To ensure that laws and policies adequately support preparedness and response, FDA will conduct a review of the strengths and weaknesses of existing legal and policy approaches to MCM development, distribution, dispensation, post-use surveillance and data collection and, where needed, develop new approaches.
MCMs are essential for saving lives and maintaining public confidence in our government in the aftermath of a public health emergency involving chemical, biological, radiological or nuclear threats or naturally-occurring emerging infectious diseases. FDA will establish a comprehensive program to support the federal government’s efforts in achieving the vision of the Nation’s MCM Enterprise: “Our Nation must have the nimble, flexible capacity to produce MCMs rapidly in the face of any attack or threat, known or unknown, including a novel, previously unrecognized, naturally occurring emerging infectious disease.”3
In the area of clinical trial design for studying new antibacterial drugs, FDA is updating its guidance documents and also is involved in work assessing new endpoints for clinical trials of antibacterial drugs. In addition, FDA is working with the Office of the Assistant Secretary for Planning and Evaluation to develop innovative strategies that promote the development of antibiotics to treat resistant organisms.
FDA also remains committed to advancing its emergency preparedness and response capabilities. Integrating outputs of the efforts above with efforts to maintain and improve FDA’s capabilities for responding to emergencies are critical to the agency fulfilling its public health protection responsibilities. Whether a natural disaster, foodborne illness outbreak, contaminated drug or biologic product, faulty medical device or harmful pet food, FDA must be prepared and have the needed resources and tools available to provide a coordinated response across field and headquarters organizational components. In order to accomplish this, FDA will continue to provide the needed training and information management tools needed for emergency response planning and critical decision making. Additionally, FDA will continue to expand its emergency exercises and after-action review programs to strengthen preparedness and make improvements for future responses.
In addition to the five priorities discussed above, “Manage for Operational Excellence and Accountability” is another essential cross-cutting area, which is one of the four strategic goals that will be addressed in the next section.
Figure 1 below depicts the relationship between the cross-cutting strategic priorities and the strategic goal areas discussed in the next section.
2Regulatory Science encompasses the development and use of new tools, standards, and approaches to more efficiently develop products and to facilitate the evaluation of product safety, effectiveness, quality, and performance. 3United States. Dept. of Health and Human Services. Assistant Secretary for Preparedness and Response. The Public Health Emergency Medical Countermeasures Enterprise Review. Washington: GPO, 2010. Print.
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