Food and Drug Administration

Mission

To protect and promote public health through food, drug, medical device, and cosmetic regulation.

Evaluation Program

The Food and Drug Administration’s FY2001 Evaluation Program ultimately reflected some of the goals established and promulgated by the Department of Health and Human Services (HHS) for which FDA has responsibility.  HHS’s goals are products of its strategic performance planning process, and FDA uses its own strategic framework to accomplish these goals.  This process also satisfies the implementation requirements of the Government Performance and Results Act (GPRA) and the Food and Drug Administration Modernization Act of 1997 (FDAMA).   The strategic and performance process is an evolving set of program directions for FDA as changes occur in FDA’s dynamic environment.  FDA’s challenges, now and in the future, will rest on its ability to leverage its efforts in that environment, which grows increasingly complex and more institutionally networked.  The Agency will strive to maintain the scientific knowledge base necessary to achieve greater effectiveness in assuring the quality and availability of the products it regulates.

One goal area within FDA’s strategic framework is Pre-Market Review, where the objective is to make timely and cost-effective pre-market review decisions, while assuring product safety and efficacy.  The results achieved and reported in the Prescription Drug User Fee Act (PDUFA) FY2000 Performance Report, are one indication of how well FDA is meeting its goal of making timely pre-market review decisions. The focus of performance goals under PDUFA is to expedite the entire drug development and review process, from research to approval, without compromising the safety or the quality expected from the Agency’s application review process. 

Another goal area within FDA’s strategic framework is Post-Market Assurance, a goal that strives to strengthen the assurance that products on the market are safe.  One strategy of accomplishing this goal is by targeting high-risk products.  The Tobacco Program illustrated one of the initiatives in this area.

Completed Evaluations

An Assessment of Thimerosal Use in Childhood Vaccines

This report (published in PEDIATRICS, Vol. 107, No. 5, May 2001) provides an overview and summary assessment of the use of thimerosal, a mercury derivative, as a preservative in vaccines.   The findings, made available in draft form to vaccine advisory committees, led to strong recommendations to vaccine manufacturers to remove thimerosal from childhood vaccines, recommendations that have now been implemented.  This risk assessment consisted of hazard identification, dose-response assessment, exposure assessment, and risk characterization. The risk assessment focused on infants and young children because of their small body size, developing brain, and exposure to vaccines containing thimerosal.  A literature review was conducted in order to identify the known toxicity of thimerosal and related organic mercury compounds, and to determine the doses at which toxicity occurs.  Maximum potential exposure to mercury from vaccines was calculated for children 6 months and 2 years of age, under the U.S. childhood immunization schedule, and compared with mercury exposure limit standards.  This assessment showed no evidence of harm caused by doses of thimerosal in vaccines, except for local hypersensitivity reactions.  However, depending on the immunization schedule, vaccine formulation, and infant weight, cumulative exposure of infants to mercury from thimerosal during the first 6 months of life may exceed EPA guidelines.  This article concludes that mercury in vaccines can be reduced or eliminated by using products formulated without thimerosal as a preservative.
PIC ID: 7736; CONTACT: Leslie K. Ball, 301-827-3070; PERFORMER: Food and Drug Administration, Washington, DC

U.S. Regulation of Pharmaceutical Outcomes Research

Outcome research often produces information that is particularly attractive for use in pharmaceutical product promotion.  FDA is then faced with making a determination as to whether the information used in pharmaceutical product promotion is adequate to support labeling and advertising claims.  A lack of consistency in the terminology used, and a lack of understanding and agreement on the evidentiary standards that should apply to the use of these data may hinder outcome evidence.  This article presents categories of outcomes information (such as economic, productivity, compliance, and satisfaction) and discusses the unique set of scientific and regulatory issues associated with each.  The article concludes that the future of outcomes research in drug development will be shaped by the consumers of outcome information and the perceived added value of these outcome measures.  Furthermore, it is envisioned that development of this field of research will benefit by integration of outcomes researchers with the rest of the clinical development team to facilitate in the production of adequate evidence to meet developing regulatory hurdles.  The final report appears in published form in: “Value in Health”, vol. 4, No. 2, 2001.  Author:  Laurie B. Burke, Chief, Evidence Review Branch, Food and Drug Administration (FDA).
PIC ID: 7733; CONTACT: Laurie Burke, 301-827-3910; PERFORMER: Food and Drug Administration, Consumer Research Staff, Washington, DC

Analysis of Hazard Analysis Critical Control Point (HACCP) Survey Data

The Food and Drug Administration (FDA) is placing increasing emphasis on the use of Hazard Analysis and Critical Control Point (HACCP) systems to ensure the safety of foods.  Over a number of years, Research Triangle Institute under contract with FDA, conducted studies to estimate the costs of implementing HACCP in food plants under FDA jurisdiction.  The project was conducted in three phases. The task of this report is to provide a final report on the outcome of the three phases of the overall project, and to provide a Microsoft Access tool that combines the data from the three phases of the overall project and that can be used to analyze the effects of various regulatory options related to HACCP.  Section II of the report describes the results of the survey on HACCP practices in plants under FDA jurisdiction (Phase III) and includes a brief description of the sampling frame used to conduct the survey (from Phase I).  Section 3 describes the results of the HACCP cost data collection process (from Phase II).  Finally, Section 4 describes the interactive Microsoft Access tool that combines the data from the three phases of the project.
PIC ID: 6866; CONTACT: Clark Nardinelli, 202-205-8702; PERFORMER: Food and Drug Administration, Rockville MD

Food Testing Laboratory Industry Database:  Final Report

In September 2000, FDA contracted with the Research Triangle Institute to collect information on the characteristics and capabilities of the 546 private laboratories that submit test packages to the FDA and to prepare a dataset containing this information.  The primary objective of this study was to provide FDA with information that could be used to assess the quality and uniformity of results reported by private laboratories.  Private laboratories performing analyses on regulated food products that may submit analytical data to FDA in order to demonstrate compliance with the Food, Drug, and Cosmetic Act were included (FDA’s 1997 definition of private laboratories.)   Private laboratories were defined as independent providers of services that were not owned by the firms utilizing those services.  Data on private laboratories were collected from FDA sources (such as OASIS) and publicly available sources on the range of companies offering food-testing services nationally and a survey of food testing laboratories in other countries.  This information was entered into an Access database containing information on laboratory location, contact information, economic variables, test capabilities, and quality assurance programs.
PIC ID: 7731; CONTACT: Clark Nardinelli, 202-205-8702; PERFORMER: Research Triangle Institute, Research Triangle Park, NC

Maternity Care Practices: Implications for Breastfeeding

The purpose of this study was to assess the impact of the type and number of Baby-Friendly practices experienced on breastfeeding.  The Infant Feeding Practices Survey, a longitudinal study conducted by the FDA, was administered to women prenatally through 12 months postpartum.   The study focused on 1,085 women with prenatal intentions to breastfeed for more than 2 months who initiated breastfeeding, using data from the prenatal and neonatal periods.  Five predictor variables included indicators of the absence of specific Baby-Friendly practices (late breastfeeding initiation, introduction of supplements, no rooming-in, not breastfeeding on demand, use of pacifiers) and number of Baby-Friendly practices experienced.  The main outcome measure was breastfeeding termination before 6 weeks.  The findings:   Only 7% of mothers experienced all 5 Baby-Friendly practices.  The strongest risk factors for early breastfeeding termination was late breastfeeding initiation and supplementing the infant.  Compared with mothers experiencing all 5 Baby-Friendly practices, mothers experiencing none were approximately eight times more likely to stop breastfeeding early.  Additional practices decreased the risk for early termination. Based on the analysis presented in this study, it can be concluded that increased Baby-Friendly Hospital Initiative practices improve the chances of breastfeeding beyond 6 weeks.  The need to work with hospitals to increase adoption of these practices is illustrated by the small proportion of mothers who experienced all 5 practices measured in this study.  See final report in Birth, Vol. 28, #2, June 2001. 
PIC ID: 7734; CONTACT: Sara Fein, 202-205-5349; PERFORMER: Food and Drug Administration, Washington, DC

Women’s Participation in Clinical Trials and Gender-Related Labeling

Through a retrospective review of clinical trial protocols and labeling for 185 new molecular entities approved by the FDA Center for Drug Evaluation and Research, this study assessed the extent to which women participated in clinical trials over a five-year period (1995-1999).  In addition to looking at clinical trials for all products examined, the study’s scope included the percentage of women participating in the trials by year, phase (although 72 percent of the protocols did not specify phase), and product type (i.e., the division within the agency responsible for the product review).  As well, the study looked at the frequency with which FDA medical officers’ evaluations of the products commented about whether the sponsors’ studies revealed gender differences.  Differences in this frequency over time, by phase, and by product type were not examined.  The study also examined the frequency with which package inserts contained some type of statement related to gender (e.g., whether or not there were similar effects of the drug in men and women) and whether this frequency differed by product type.  The findings indicated that, overall, women appeared to participate in the clinical trials at nearly the same rate as men, and that labeling of two-thirds of the products contained some statement about gender (although only 22 percent described actual gender effects).  Of the effects discussed, 90 percent were pharmacokinetic, 12 percent were safety, and 5 percent were efficacy.
PIC ID: 7737; CONTACT: Teresa Toigo, 301-827-4460; PERFORMER: FDA, Center for Drug Evaluation & Research, Rockville, MD

In-Progress Evaluations

Impact of the Mammography Quality Standards Act of 1992

The final report for this study has been drafted but not yet finalized or cleared.  The study estimated the economic impact of regulations developed under the MQSA.  It evaluated conditions of facilities after implementation of the regulations in order to evaluate whether patient access to quality screening services has been adversely affected.  The Contractor examined agency data sets as well as other public sources to estimate the effect of regulations on facility closures and patient access.  Field contacts were used to discuss impacts with industry experts.  Preliminary findings are that the regulations have not adversely affected patient access to high quality mammography screens.
PIC ID: 6080.4; EXPECTED COMPLETION: FY 2003; CONTACT: Steve Tucker, 301-827-5339; PERFORMER: Eastern Research Group, Lexington, MA

Nationwide Evaluation of X-Ray Trends (NEXT)

The Nationwide Evaluation of X-Ray Trends (NEXT) survey program is a collaboration jointly administered by the Food and Drug Administration (FDA) and State radiation control program agencies.  Each year a selected diagnostic x-ray is selected for survey, and nationally representative data are collected from US facilities on patient exposure levels and associated indicators of diagnostic quality.  Surveys are repeated periodically to observe trends in the state of practice, and the results are published by the Conference of Radiation Control Program Directors (CRCPD), Frankfort, KY.  Please refer to web site address.
PIC ID: 4984; EXPECTED COMPLETION: FY 2002; CONTACT: Orhan Suleiman, 301-594-3533; PERFORMER: Food and Drug Administration, Rockville MD

Drug Metabolism:  Drug-Drug Interactions

Recent examples of studies that have provided information to specific review decisions include: (1) interactions and metabolism studies of anti-cancer drugs such as paclitaxel, (2) anti-abuse drugs such as LAAM, (3) anti-HIV drugs such as the protease inhibitors, (4) anti-epileptic drugs, (5) cardiovascular drugs, and (6) anti-malarial drugs. In addition to the general technical expertise brought to bear on these problems, results from the program have been incorporated into final product labeling for some drugs, including paclitaxel and saquinavir. Labeling changes for other drugs are under discussion. Also, cutting-edge issues regarding cases of intermediate potency for cytochrome P450 pathways are under investigation.
PIC ID: 7103; EXPECTED COMPLETION: FY 2004; CONTACT: Jerry Collins, 301-827-5471; PERFORMER: Food and Drug Administration, Rockville MD

Food Labeling and Package Survey (FLAPS)

This survey was implemented by the Food and Drug Administration (FDA) in 1977 with the purpose of producing a database containing label and package information obtained from a sample of processed food products.  Food Labeling and Packaging (FLAPS) data enable FDA personnel to keep abreast of market responses to food labeling rules via changes in package labels.   Data collection for the most recent survey (2000-2001) commenced on 5-1-2000 and should be completed by the end of FY 01.  Data analysis will commence on 11-01-02 and end by 9-28-02.  The end of FY02 schedules two reports for completion.
PIC ID: 5711.3; EXPECTED COMPLETION: FY 2002; CONTACT: Lori A. LeGault, 202-205-5269; PERFORMER: Information Resources Inc., Chicago, IL

Prescription Drug User Fee Act Reports

The Prescription Drug User Fee Act (PDUFA) of 1992 provided FDA with greater resources for the review of human drug and biologic applications.  Fees that FDA collected from drug and biologics firms were used to reduce the time required to evaluate human drug applications without compromising review quality.  FDA has submitted annual Performance and Financial Reports to Congress on progress in streamlining the drug review process and use of PDUFA fees.  The original act expired September 30, 1997, but the FDA Modernization Act of 1997 amended and extended PDUFA through September 30, 2000.  The amended act is now referred to as PDUFA II and its predecessor as PDUFA I.
PIC ID: 6079.6; EXPECTED COMPLETION: FY 2002; CONTACT: Dennis Hill, 301-827-5255; PERFORMER: Office of Policy, Planning, and Legislation, FDA, Rockville MD