DEPARTMENT OF HEALTH AND HUMAN SERVICES
NATIONAL INSTITUTES OF HEALTH>
Fiscal Year 2005 Budget Request
Witness appearing before the
Senate Subcommittee on Labor-HHS-Education Appropriations
Duane Alexander, M.D., Director
National Institute of Child Health and Human Development
April 1, 2004
William Beldon, Acting Deputy Assistant Secretary, Budget
Mr. Chairman and Members of the Committee:
I am pleased to present the fiscal year (FY) 2005 President's budget request for the National Institute of Child Health and Human Development (NICHD). The FY 2005 budget includes $1,280.9 million, an increase of $39.1 million over the comparable FY 2004 appropriation of $1,241.8 million.
The NIH Roadmap provides the schema to guide the NICHD in achieving its programmatic and research goals.
Today I would like to share with you how the research supported by this committee is improving the lives of children, mothers, adults and families, and helping to reduce health disparities. The NICHD is participating in the trans-NIH obesity initiative identifying how primary care physicians can help children maintain a healthy weight.
ENCOURAGING HEALTHY BIRTH OUTCOMES
Preeclampsia is a condition that affects five out of every hundred women who become pregnant. Preeclampsia can occur suddenly, and without warning, causing women to develop dangerously high blood pressure. In some cases, the condition may progress to eclampsia in which women experience potentially fatal seizures. Infants born to mothers with preeclampsia may be extremely small for their age or may be born prematurely, putting them at risk for a variety of other birth complications. Although a woman's high blood pressure and seizures can be treated, the only cure for preeclampsia is delivery of the baby. In a significant step toward treating preeclampsia, researchers have identified substances in the blood that have the potential to predict who will develop preeclampsia. This knowledge may help us treat women before preeclampsia becomes a serious problem, for them and their infant.
We have also intensified our research in the area of stillbirth, a devastating occurrence that affects far too many families. Health care providers use the term stillbirth to describe the loss of a fetus after the 20th week of pregnancy. Stillbirth can occur before delivery or as a result of complications during labor and delivery. In at least half of all cases, researchers can find no cause for the pregnancy loss. We hope to change that. The NICHD has established the Stillbirth Collaborative Network, which consists of research centers in Texas, Utah, Rhode Island, and Georgia. In each center, a team of specialists, including obstetricians, nurses, statisticians, and even grief counselors will seek to understand the causes of stillbirth and eventually find ways to prevent these deaths.
One way to increase the chances of a healthy pregnancy and healthy birth outcome is to avoid alcohol during pregnancy. Infants born to mothers who drink heavily during pregnancy are known to be at risk for mental retardation and birth defects. They are also at increased risk for Sudden Infant Death Syndrome (SIDS). NICHD researchers have now identified another reason that women should not consume alcohol during pregnancy: exposure to alcohol before birth affects the developing nervous system in the arms and legs.
Recently, scientists in NICHD's Maternal-Fetal Medicine Units Network reported a breakthrough in reducing a major cause of infant mortality and the subsequent long term health problems associated with prematurity. The scientists, working collaboratively in 14 academic health centers across the U.S., demonstrated that progesterone administered to women at risk for premature birth could significantly reduce the likelihood of early delivery. This was a very significant discovery and we were delighted that others recognized its importance. A few weeks ago, Parade magazine identified this discovery as one of the ten most significant health advances of the past year.
NEW FRAGILE X CENTERS TO DEVELOP TREATMENT OPTIONS
In 2003, the NICHD funded three new Fragile X research centers. Teams of researchers at each of the centers-located in North Carolina, Texas, and Washington state-are developing new ways to diagnose both the mild and severe forms of the condition, as well as new treatments. Fragile X syndrome is the most common genetically-inherited form of mental retardation currently known. It occurs in 1 out of every 2,000 males and in 1 in 4,000 females. The syndrome is caused by a mutation in a specific gene, known as FMR1, on the X chromosome. In its fully-mutated form, the FMR1 gene interferes with normal development, resulting in mental retardation. In a partially mutated form, the FMR1 gene can cause fragile X syndrome in the children of a parent who is a carrier. Until recently, it was thought that carriers did not have any symptoms. Researchers have learned that some people with a form of fragile X have mild cognitive and emotional problems. In addition, some female carriers are likely to undergo premature menopause. In older male carriers, the fragile X is associated with a neurological degenerative syndrome. Identifying a means to predict which carriers will develop the symptoms could be a first step toward developing new treatments for these often overlooked symptoms. The Fragile X Research Centers are focusing their research on how the fragile X affects the developing brain and nervous system, how the disorder progresses throughout an individual's life span, and treatments that can improve the behavior and mental functioning of people with fragile X syndrome.
IMPROVING TREATMENT FOR CRITICALLY ILL CHILDREN
Critical care medicine for children is an emerging field where, in general, physicians continue to rely upon adult treatments that have not yet been tested for effectiveness in a young population. To change this situation, the NICHD will help establish a national pediatric critical care research network to develop and evaluate treatments for children with disabling conditions. The initiative will foster collaborations among scientists in many different fields and will support research such as the best approach to care for children with brain injury, the most effective way to transition a critically ill child from an acute care to a rehabilitation setting, and the care of critically ill children in the event of a bioterrorism attack.
CUTTING OBESITY THROUGH RESEARCH AND PROGRAMS
The increase of overweight and obesity among adults and children is a major public health concern. In fact, in a recent analysis of international data, NICHD researchers documented that U.S. teenagers had more overweight than youth in 14 other developed countries. Like many other health conditions that affect adults, the antecedents of adults having obesity can be found in childhood. Young children who have overweight are likely to have overweight as adults. There is no single explanation for the increase in childhood overweight and there is no single solution. However, we know we must devise successful interventions that help children maintain a healthy weight. As part of the trans-NIH initiative, the NICHD will lead a major effort to determine whether a weight control program for children and youth led by primary care physicians as part of a comprehensive community-based effort can be successful. Currently, most weight management programs are administered through specialty clinics. However, there is strong evidence that an appropriate intervention by a physician can have a significant impact on personal behaviors such as tobacco use. Effective weight management programs in a primary care setting would be accessible to large numbers of children and would minimize the geographic, social, and economic barriers that commercial weight management programs can impose.
We are also developing an exciting research-based program that helps to teach young children the fundamentals of good nutrition and physical activity as well as how to make sense of the messages that appear in the media. Three years ago, this committee provided funds to the NICHD and other health agencies to develop programs that encourage young people to engage in healthy behaviors. In response to this directive, the NICHD has developed "Media Smart Youth," an after school program for children between nine and 13 years of age. The program focuses on good nutrition and physical activity. But it also provides skills to young children to interpret the messages about food and snacks they see on television, in magazines, and on the Internet. As part of their activity, the children who take part in Media Smart Youth develop messages about the importance of good nutrition and physical activity for their peers. The program has been tested with youth groups around the country. In fact, the children at P.S. 127 in the Bronx who took part in this program developed a message about physical activity for young people that appeared for 30 minutes on the Panasonic "jumbotron" screen in Times Square.
HELPING YOUNG CHILDREN PREPARE FOR SCHOOL
The preschool years are crucial for learning language, social skills, and developing the intellectual capabilities that set the stage for later success in school. Yet, comparatively little is known about how to help young children obtain the greatest benefit possible from the preschool experience. In December 2003, NICHD joined with two other HHS agencies and the Department of Education, and launched a five year research initiative to find the best ways to help preschoolers at risk for failure in school acquire the skills they need for school success. The initiative provided $7.4 million in funding for the first year. Eight projects were funded to test research-based approaches to preschool curricula, Internet based approaches to training preschool teachers, and the importance of parental involvement for preparing children to enter school. Funds requested for FY 2005 will allow us to expand this effort by funding academic researchers and small businesses to develop and produce more effective measurements of outcomes from preschool interventions.
SIDS RESEARCH SUPPORTS PROGRAM OUTREACH
We have known for more than 10 years that placing infants on their backs to sleep reduces their risk of Sudden Infant Death Syndrome (SIDS). In fact, since the NICHD launched the Back to Sleep SIDS risk reduction campaign in 1994, the rate of SIDS in the U.S. has declined by more than 50 percent. The NICHD continues a vigorous research program to learn more about the causes and prevention of SIDS. For instance, a team of NICHD-funded researchers in Ohio recently discovered that infants who were placed to sleep on their backs were less likely to develop fevers, get stuffy noses or develop ear infection. Ear infections alone cost the health care system an estimated $5 billion a year. So this simple behavior of placing infants on their backs to sleep not only saves lives, it can save the health care system large sums money by reducing the use of antibiotics to treat ear infections. We also learned that infants who are normally placed to sleep on their backs are at greatly increased risk of SIDS when they are occasionally placed to sleep on their stomachs. New research on SIDS continues to shape our SIDS risk reduction outreach campaign. More recently, a major focus of the campaign has been reducing the risks of SIDS in African American communities.
SIDS rates for African American babies have declined significantly since the NICHD initiated its Back to Sleep campaign ten years ago. Yet, the SIDS rate for African American infants is more than twice that of white infants. To address this health disparity, the NICHD joined forces with three national African American organizations in a unique collaboration to reduce the risks of SIDS in African American communities. The Alpha Kappa Alpha Sorority, the National Coalition of 100 Black Women, and the Women in the NAACP, sponsored three regional summit meetings to raise SIDS awareness and train community leaders to be resources and spokespersons for SIDS risk reduction in their communities. The summit meetings were held in Tuskegee Alabama, Detroit Michigan, and Los Angeles California, and they helped build an infrastructure to involve faith-based, community, and service organizations in reducing the risks of SIDS and in promoting the health of infants. In Detroit, for instance, the summit ended with a "SIDS Sunday," which was held at Hartford Memorial Baptist Church on the Sunday following that summit. Afterwards, other churches across the region held a "SIDS Sunday," where pastors shared SIDS information from their pulpits, in their church bulletins, and with nurses and care givers in their childcare centers and nurseries. The successful collaboration of researchers, government officials, and the community will create a strong foundation for launching other interventions to eliminate health disparities.
MOTHERS LEAVING WELFARE HAD NO EFFECT ON PRESCHOOLERS
A study that received much of its funding from the NICHD demonstrated that when a mother leaves welfare to enter the labor force, it does not seem to have any negative effects on preschoolers or young adolescents. The study was undertaken in response to the Personal Responsibility and Work Opportunity Reconciliation Act of 1996, which mandated stricter welfare requirements for all welfare recipients. The researchers theorize that the positive and negative effects of going off welfare and getting a job may cancel each other out. For example, the increase in family income that comes with leaving welfare-thought to relieve the stress on a family-may make up for the decreased amount of time that mothers spend with their young children. In addition, mother's transition to work had a slightly positive effect on teens, reducing the teens' levels of anxiety. Conversely, teens whose mothers left the job market and went on welfare developed increased anxiety levels.
MICROBICIDES THAT CAN PREVENT SEXUALLY TRANSMITTED INFECTIONS
The NICHD is funding a number of projects to develop microbicidal compounds to prevent the spread of sexually transmitted infections and HIV. These compounds not only have the potential to prevent the spread of disease-causing bacteria and viruses, but may also be effective in preventing pregnancy. One project is a large scale test of the contraceptive effectiveness of Buffergel, a compound that kills the microorganisms that cause sexually transmitted diseases, and shows promise as a contraceptive. Another project is studying a microbicidal spermicide, C31G. The compound's effectiveness will be compared to that of a conventional spermicide preparation. Working with the National Institute of Allergy and Infectious Diseases, the NICHD has funded a new system to test the quality of potential microbicides to determine if they warrant further testing in human beings.
SAFER DRUGS FOR USE WITH CHILDREN
In January, 2002, President Bush signed into law the Best Pharmaceuticals for Children Act (BPCA). The law recognizes that drugs may have different effects in children than they do in adults, and seeks testing for drugs given to children. For roughly 75 percent of the drugs approved by the U.S. Food and Drug Administration (FDA) for adults, there is inadequate information available to ensure the safety and effectiveness of the drugs in children. Moreover, there is little or no data to guide physicians in prescribing dosages of these drugs for children. Working in close collaboration, the NICHD and the FDA, as directed by the BPCA, identified several high priority drugs to be tested. The NICHD is currently establishing partnerships with pediatric drug study networks in other NIH Institutes to expedite the study of other clinically important drugs.
Drugs prescribed to pregnant women are also a concern. Although nearly two-thirds of all pregnant women take at least four to five drugs during pregnancy and labor, the effects of these drugs on a pregnant woman and her fetus remain largely unstudied. In addition, little is known about how pregnancy-related changes in cardiac output, blood volume, intestinal absorption, and kidney function may influence drug absorption, distribution, utilization, and elimination. Therefore, the NICHD will establish a new network of Obstetric-Fetal Pharmacology Research Units that will allow investigators to conduct key pharmacologic studies of drug disposition and effect during normal and abnormal pregnancies.
NATIONAL CHILDREN'S STUDY
In a few short years, The National Children's Study has evolved from a concept to an exciting research collaboration poised to answer critical questions about child development. The FY 2005 budget request continues planning dollars for this important project, but does not reflect funding to launch the study itself, since it is still being developed. The National Children's Study plans to examine the effects of environmental influences on the health and development of more than 100,000 children across the United States, following them from before birth until age 21. The NICHD serves as the lead agency on this ambitious project, working closely with the National Institute of Environmental Health Sciences, the Centers for Disease Control and Prevention, and the U.S. Environmental Protection Agency. The collaboration involves government agencies, the research community, industry, and community groups.
NIH ROADMAP AND CLINICAL RESEARCH
To ensure that the necessary clinical research workforce is available to translate laboratory findings to improved treatments for patients, the NIH Roadmap is strengthening several stages in the career path for these researchers. One new program will provide clinical research experience and didactic training during medical and dental school. Another will train doctorate-level professionals in multi disciplinary collaborative clinical research settings that reflect the diversity of today's clinical research team. To attract community practitioners to clinical research, the NIH plans to create a cadre of National Clinical Research Associates, community practitioners trained in clinical research who will refer patients to large clinical trials to enhance patient recruitment and more rapidly test potential therapies. The NIH is also identifying ways to improve peer review of clinical research grant applications and to enhance promotion and tenure policies in academia for clinical researchers.
Duane Frederick Alexander, M.D.
August ll, l940, Baltimore, Maryland
Married Marianne Ellis, Washington D.C.,
Pennsylvania State University, University Park,
Johns Hopkins University School of Medicine, Baltimore,
Intern and Fellow, Department of Pediatrics,
Assistant Resident and Fellow, Department of
1968-1970: Surgeon (LCDR), U.S. Public Health Service, and Clinical Associate, Children's Diagnostic and Study Branch, National Institute of Child Health and Human Development (NICHD), National Institutes of Health (NIH), Bethesda, Maryland
1971-2000: From Surgeon to Assistant Surgeon General, U.S. Public Health Service
Fellow in Pediatrics (Developmental Disabilities), John F. Kennedy Institute for Habilitation of the Mentally and Physically Handicapped Child, Johns Hopkins Hospital, l970-71
Maryland Board of Medical Examiners
Diplomate, American Board of Pediatrics, 1973
Assistant to the Scientific Director, NICHD, NIH
Medical Officer (Staff), National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research, DHEW
Assistant to the Director, NICHD, NIH
Deputy Director, NICHD, NIH
Director, NICHD, NIH
Department of Health and Human Services, Office of Budget
William R. Beldon
Mr. Beldon is currently serving as Acting Deputy Assistant Secretary for Budget, HHS. He has been a Division Director in the Budget Office for 16 years, most recently as Director of the Division of Discretionary Programs. Mr. Beldon started in federal service as an auditor in the Health, Education and Welfare Financial Management Intern program. Over the course of 30 years in the Budget Office, Mr. Beldon has held Program Analyst, Branch Chief and Division Director positions. Mr. Beldon received a Bachelor's Degree in History and Political Science from Marshall University and attended the University of Pittsburgh where he studied Public Administration. He resides in Fort Washington, Maryland.