FDA Home Page | Search FDA Site | FDA A-Z Index | Contact FDA

Problem | Desired Outcomes | Key Strategies | FY 2004 Goal Highlights | Current Status | FY 2002 Performance Goals | FY 2001-2002 Highlights

Problem

FDA oversees the public health standards of an industry that produces almost $1.5 trillion worth of regulated products. The products include the entire food supply, except for meat and poultry; over-the-counter and prescription medications; blood products; vaccines; tissues for transplantation; medical equipment and implantable devices; devices that emit radiation; animal drugs and feeds; and cosmetics.

FDA faces two challenges in assuring that U.S. citizens fully reap the health benefits of modern medical technology, and enjoy a safe and nutritious food supply:

1. FDA must assure the safety and effectiveness of drugs, biologics, food additives, medicated animal feeds, and medical devices before they are allowed on the market.This challenge means keeping pace with the increasing scientific complexity of products that emerge from a $50 billion a year research and development effort. FDA scientists must accurately judge the readiness of these products to be marketed, and help these innovative products reach the market as quickly as possible.

There must also be a sufficient number of reviewers to handle the product review workload to provide timely feedback to application sponsors to identify and address deficiencies. Any delays might:

• Postpone critically needed disease prevention and treatment, especially for a growing population of elderly and immune-compromised patients;
• Increase the cost of bringing a new product to market by lengthening the time a product is in development; and,
• Result in fewer low-cost alternatives for patients (including children and the elderly).

4. FDA must also monitor the safety of products once they are on the market. U.S. consumers spend an estimated $326 billion annually on medical products that are produced world-wide, and make their way to the market throughout a wide variety of distribution channels.

To ensure that these products are safe, the Agency must oversee their entire life cycle - from production through distribution, and use/ consumption.

Desired Outcome

FDA's long-term goal is to contribute to improving the health status of U.S. citizens by minimizing the risks associated with use of medical products and the consumption of food in the U.S. This becomes more likely when informed consumers and health professionals make wise choices concerning the dissemination and use of these products. FDA is examining specific outcome measures that will reflect progress towards this goal, and will be working to identify data sources to accurately measure this progress.

Key Strategies

FDA recognizes that improvements in U.S. public health will only be possible through a coordinated effort on the part of many partners in the public and private sectors, as well as the consumer. In this joint effort, FDA makes a significant contribution in several ways:

• Thorough, science based scrutiny prior to market entry assures that only safe and effective products are used to treat and prevent disease and promote health.
• Efficient product review processes make life saving new technologies available to the market on a timely basis so that they begin to make a difference earlier.
• Inspection of facilities ensures that products are being manufactured according to Good Manufacturing Practices (GMPs), and that these standards are based on rigorous science and sound quality control principles. This reduces the probability that defective or hazardous products will ever enter the distribution system.
• Effective collaborations with health and regulatory partners will leverage the use of FDA's standards through a wider domain at the international, state and local levels of governance.
• Rigorous risk analysis will help the Agency focus on the highest risks at all points in the product life cycle; and it will lead to a cost-effective deployment of FDA resources.
• FDA's educational efforts are directed at consumers, health professionals and all those who can ensure safe use of products - e.g., correct use of medications, healthy eating habits, etc This, in turn, contributes to better treatment outcomes and improved health status for U.S. citizens. Collaborative educational efforts with outside organizations further strengthen this influence.

These multiple roles will contribute greatly to the availability and appropriate use/ consumption of FDA-regulated products. Although product availability and safe use are still only part of the equation they are necessary ingredients that must be present to improve public health outcomes.

The discussion that follows outlines specific strategies that FDA is embarking on in the areas of premarket review, postmarket inspection and enforcement; and risk analysis that underpin the entire effort:

1. Premarket Strategies

FDA has adopted a number of strategies to improve its product review processes, including:

• Increase the number of inspections and target high-risk clinical trials - e.g., vulnerable populations such as mentally impaired, pediatric, etc.; and increase training for investigators; improve the inspection process for Institutional Review Boards (IRBs); and enhance follow-up compliance activities.
• Develop disease indication-specific guidance on clinical evidence to support premarket applications.
• Continue the Human Drug Review Program supported by the reauthorization of the Prescription Drug User Fee Act (PDUFA) to keep pace with the review of increasingly complex new drugs and vaccines.
• Continue the Medical Device Review Program supported by the Medical Device User Fee and Modernization Act (MDUFMA) to keep pace with the increasingly complex medical device review process.
• Increase efforts to promote safe and effective over-the-counter (OTC) drug products in the United States.
• Develop standards for new products of emerging technologies, including new drugs, biologics, medical devices and bioengineered foods, to facilitate safe product development guided by current science.
• Implement a comprehensive quality control systems approach to medical product review, and ensure the Agency's premarket review processes are consistent, high-quality and efficient. FDA is working with stakeholders to implement this in the least burdensome way.
• Pediatrics
  
  • Increase the number of drugs that are adequately labeled for children.
  • Provide incentives for the effective development and dissemination of information on how to properly use therapies in children.
• Premarket Generic
  
  • Improve the generic drug review program to reduce review backlogs and expedite the review of new applications; and implement improved regulations governing generic drug competition.
• Dietary Supplements
  
  • Continue prompt review and evaluation of premarket notifications for dietary ingredients.

2. Inspection and Enforcement Strategies

• Import Strategies
  
  • Enhance the automated import monitoring system (OASIS) to improve cost effectiveness in screening imports and expand coverage at ports.
  • Increase criminal investigations of fraudulent medical product imports.
  • Implement the European Mutual Recognition Agreement; and participate in international standard setting forums such as the International Conference on Harmonization (ICH) to continue to advocate for rigorous standards.
    
• Domestic Strategies
  
  • Leverage inspection efforts through contracts and grants with the States, other third parties and outreach to small firms.
  • Review Human Drug, Biologics and Animal Drug GMPs to emphasize science-based risk management and quality control.
  • Ensure that inspectors have the scientific and technological support necessary to make quick and valid judgments about medical product and food compliance.
  • Focus resources on the highest risk firms for food and medical product areas, which will bring the greatest health benefit.

3. Risk Analysis Strategies

• Identify statistical analysis and modeling methods, such as computer simulation and monitor best practices that could potentially be applied or extended in Agency risk management.
• Armed with appropriate information and analytical tools, apply principle of "reasonable certainty of no harm" in making risk management decisions.
• Assess opportunities for more standardized methods and approaches to risk analysis in support of risk management decisions.
• Introduce the knowledge of new genetic systems and computer-assisted toxicology (toxicoinformatics) into the risk management process.

Performance Goal Highlights for FY 2004

Premarket

• Meet PDUFA III commitments for the review of original New Drug Application (NDA), Product License Application (PLA), and Biologic Licensing Application (BLA) submissions.
• Complete Review and Action upon 90 percent of fileable original generic drug applications within 6 months after submission date.
• Complete Review and Action on 90 percent of prescription-to over-the-counter switch applications within 10 months of receipt.
• Increase the number of drugs that are adequately labeled for children.
• Complete the safety evaluation of 75 percent of food and color additive petitions within 360 days of receipt.
• Complete Review and Action on 90 percent of New Animal Drug Applications and reactivations of such applications within 295 days; 90 percent of investigational animal drug study submissions within 320 days; and review 90 percent of investigational animal drug submissions consisting of protocols, without substantial data within 125 days.
• Complete Review and Action on 90 percent of Device Premarket Approval Application (PMA) first actions within 180 days.
• Protect human research subjects who participate in drug studies and assess the quality of data from these studies by conducting onsite inspections and data audits under FDA's Bioresearch Monitoring Program.
• Respond to at least 95 percent of premarket notifications for new dietary ingredients within the statutory time frame (75 days);

Postmarket

• Inspect 55 percent of an estimated 630 registered high-risk human drug manufacturers.
• Meet the biennial inspection statutory requirement by inspecting 50 percent of the approximately 2,700 registered blood banks, source plasma operations, and biologics manufacturing establishments to reduce the risk of product contamination.
• Utilize risk management to target inspection coverage for Class II and Class III domestic medical device manufacturers at 20 percent. [Class II Devices require standards to be developed prior to marketing. Class III Devices are considered to be higher risk devices, such as heart valves].
• Utilize risk management to target inspection coverage for Class II and Class III foreign medical device manufacturers at 20 percent.

Current Status

Premarket Review

Human Drugs and Biologics:   For new drugs and biologics, the story is one of great success. FDA has moved from criticisms of a "drug lag" with other countries a decade ago to the current situation in which new drugs are approved in the U.S. as fast as or faster than anywhere in the world, with the same high standards Americans expect. This was accomplished largely by the assurance of sufficient scientific staff funded by industry fees that supplement appropriated funds.

Generic Drugs:   In the area of generic drugs, recent increases have enabled the program to raise performance targets.

OTC Drugs:   For over-the-counter (OTC) drugs, the FDA has finalized 52 monographs or "recipes" for marketing these drug products without the need for FDA preclearance. The monographs save manufacturers costs and reduce barriers to competition, as they allow both large and small companies to enter the market place with OTC drug products that have to meet the same, uniform criteria. By the end of FY 2004, the Agency expects to have initiated the review process (e.g. submit as advance notice of proposed rulemaking (ANPR)) for as many as 110 major drug category monographs. In the next 10 years, FDA expects to review and complete approximately 25 monographs, a number of which are major product categories (e.g. internal and external analgesic, and health care antiseptics). The road from ANPR to when a monograph is finalized is a complex and time-consuming rule-making process and can take several years.

Pediatric Medicine:   The original pediatric exclusivity provision of FDAMA has done more to generate clinical studies and useful prescribing information for the pediatric population than any other regulatory or legislative process to date. As a result of this provision, FDA has worked with NIH to develop off-patent drugs and to elicit study proposals from industry. FDA has issued over one hundred written requests for studies that could affect thousands of pediatric patients. The Agency has also issued a number of grants of pediatric exclusivity. The Best Pharmaceuticals for Children Act (BPCA), enacted on January 4, 2002, will continue to provide incentives for the effective development and dissemination of information on how to properly use therapies in children.

Postmarket

Global Vigilance - Imports of all FDA regulated products have been increasing over the last several years - growing at an annual rate of 10 to 12 percent. In FY 2001, FDA physically examined less than one percent of all entries offered for import into the United States. Plans for a highly integrated web-enabled import monitoring system are being developed to allow the Agency to prevent unsafe import entries on a cost-effective basis. Alternatively, FDA would need restructured regulatory authority that would place more responsibility on exporters to assure that products entering the U.S. are safe.

Domestic Industry Monitoring - The law requires that FDA inspect certain biologics, human and animal drugs and feeds, and medical device manufacturers at least once every two years. Although at least 50 percent of statutory establishments should be inspected annually, only 19 percent of human drug, and 20 percent of medical device statutory establishments were inspected in FY 2001. However, the Agency did exceed its goal in inspecting over 50 percent of registered blood banks, source plasma operations, and biologics manufacturing establishments in FY 2001. Beginning in FY 2003, FDA has developed a new high risk performance goal to inspect the highest risk drug manufacturers.

FY 2002 Performance Goals

Highlights of FY 2001-2002 Accomplishments

Premarket

Medical Product Research Oversight

• While new products continue to be approved in record times, the Agency also took steps to safeguard the interests of human subjects in clinical trials prior to products reaching the commercial review stage. In the medical device area alone, FDA conducted 238 domestic and foreign inspections under the bioresearch monitoring program. FDA relies heavily on the integrity of data generated from clinical trials in making many of its review decisions.
• FDA issued an interim rule to provide additional safeguards for children participating in clinical studies. The new rule provides specific criteria, such as an assurance of informed consent by the children and their parents that have to be maintained by the Institutional Review Boards that supervise the trials.

Human Drugs and Biologics

• FDA successfully negotiated the reauthorization of the Prescription Drug User Fee Act, which will give the Agency access to resources necessary to effectively support human drug development and expedite premarket application review.
• FDA continues to review and approve medical products on a timely basis. For new drug and biologics applications received during FY 2000, the Agency met or exceeded all 15 performance review goals committed to. New drugs are approved in the U.S. as fast as or faster than anywhere in the world, with the same high standards Americans expect. During the past year, several new therapies were approved including:
  • Gleevec, a new oral treatment for patients with chronic myeloid leukemia was reviewed and approved in two and half months; and,
  • Xigris, the first biologic treatment for the most serious life threatening forms of sepsis, which claims 225,000 lives in the U.S. each year.
• FDA has also made progress in increasing the number of drugs that are adequately labeled for children. Before FDA can approve such labels, Agency-requested studies must be conducted. As of July 2002, FDA had requested 588 studies that address dosing, safety and use of drugs for children. Over 34,000 patients are projected as participants in these studies. As of May 2002 over 30 pediatric labels have been approved. Accurate dosing and safety information is now available for children who are being treated for allergies, diabetes, heartburn, high blood pressure and many other conditions. In January 2002, the President signed the "Best Pharmaceuticals for Children Act" which reauthorized the pediatric exclusivity provisions of the 1997 "FDA Modernization Act" and provided new approaches for assuring the appropriate study of drugs that were not being studied under the exclusivity provisions. FDA, in conjunction with NIH, has initiated the various steps needed to implement this legislation.

Medical Devices

FDA successfully negotiated the authorization of the Medical Device User Fee and Modernization Act (MDUFMA), which will give the Agency access to resources necessary to effectively support medical device development and expedite premarket application review.

In 2002, several new products were approved including:

• The Given Diagnostic Imaging System, a new swallowable capsule containing a tiny camera that can facilitate early detection of colon cancer;
• Expanded use of defibrillators to treat heart patients; new drugs to treat rare pediatric liver disease (nitisinone); and broader use of a brain implant to treat Parkinson's disease.

Foods

• FDA has completed 100 percent of its reviews for "new dietary ingredients" notifications within the 75-day deadline, which exceeds its 95 percent target. The Agency also completed review of all 80 notifications received in 2001, which also exceeds its 95 percent target.

Postmarket

Human Drugs and Biologics

• FDA met its statutory requirements by inspecting 50 percent of registered blood banks, source plasma operations, and biologics manufacturing establishments to reduce the risk of product contamination.
• The Agency ensured that recipients of blood products will be better protected thanks to licensing of the first nucleic acid test systems intended for screening of plasma donors. These systems are expected to further ensure the safety of plasma-derived products by permitting earlier detection of HIV and HCV infections in donors.
• In FY 2001, FDA became part of a coordinated HHS-wide effort to establish common reporting portal that will simplify the task for those who are submitting adverse events information for medical products, and for the Federal agencies that are collecting it.
• FDA made progress in automating the receipt and processing of drug safety reports that will allow the Agency to be more responsive to public health issues, reduce resources associated with data management, and apply better data and better science to the drug regulatory process.

Medical Devices

• FDA continued to improve reporting of adverse events associated with medical device use by recruiting several additional hospitals into the Medical Product Surveillance Network. This reporting network is intended to reduce the estimated 300,000 injuries and deaths associated with medical device use and misuse.

Foods

FDA took further steps to understand and manage the risk associated with dietary supplements, including the following:

• Implemented the Cooperative Agreement with the National Center for Natural Products Research at the University of Mississippi. Work done by the National Center to identify and analyze specific components in dietary supplement ingredients, including botanical ingredients, is an essential component to FDA's research and regulatory programs directed at ensuring the safety and effectiveness of dietary supplements.
• Developed "Tips for the Savvy Supplement User," to assist consumers in making informed decisions and evaluate information about dietary supplements.
• Reviewed 43 of 44 notifications for new dietary ingredients within the 75-day statutory timeframe.

BSE Risk Management

FDA participated in the USDA sponsored training of risk analysts to use and continue developing the Harvard Center for Risk Analysis (HCRA) BSE simulator. The simulator has been installed in FDA's Center for Veterinary Medicine (CVM) to run computationally-intense risk assessment and risk management models. FDA's Risk Analysis Team is running the simulator to test the impact on risk of BSE infectivity in cattle under alternative compliance strategies. The risk management approach to BSE risks in the U.S. enables FDA to make difficult resource allocation decisions under the scientific uncertainty attendant health risk issues.

Top of page

Previous Page | Table Contents | Strong FDA

Office of Planning Home Page
FDA Home Page | Search FDA Site | FDA A-Z Index | Contact FDA | Privacy | Accessibility

FDA Office of Planning