DEPARTMENT OF HEALTH AND HUMAN SERVICES
NATIONAL INSTITUTES OF HEALTH
Fiscal Year 2004 Budget
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 8, 2003
Kerry N. Weems, Acting Assistant Secretary for Budget, Technology and Finance
William R. Beldon, Acting Deputy Assistant Secretary for Budget
Mr. Chairman and Members of the Committee:
I am pleased to present the FY 2004 President's budget request for the National Institute of Child Health and Human Development (NICHD). The fiscal year (FY) 2004 budget includes $1,245 million, an increase of $41 million over the FY 2003 enacted level of $1,205 million comparable for transfers proposed in the President's request.
The NIH budget request includes the performance information required by the Government Performance and Results Act (GPRA) of 1993. Prominent in the performance data is NIH's second annual performance report which compares our FY 2002 results to the goals in our FY 2002 performance plan.
Forty years ago, the U.S. Congress charged the NICHD with a broad mandate. The Institute was asked to develop a research program to ensure that people are able to have children when they want them; that every child is born healthy; that women suffer no adverse consequences from the reproductive processes; and that children experience healthy physical, cognitive, behavioral, and social development, reaching adulthood free of disease and disability, and able to lead productive lives.
We have made exceptional progress toward those goals during the last 40 years. Infant mortality has been cut by more than 70 percent, largely due to NICHD research that has lead to new ways to treat and prevent respiratory distress syndrome, to manage premature infants, and to reduce Sudden Infant Death Syndrome. Mental retardation in the United States has been significantly reduced because we have conquered and controlled some of its leading causes: Hemophilus influenza type b (Hib) meningitis, phenylketonuria (PKU), measles encephalitis, and jaundice. Infertility that deprived millions of couples from conceiving children can now be diagnosed and in many cases treated. Transmission of HIV infection from mother to baby has been reduced from 27 percent to less than 2 percent in the U.S. as a result of research showing the effectiveness of administering antiretroviral drugs to the mother during pregnancy and to the infant just after birth.
We look forward to building on 40 years of scientific achievements and we would like to share with you recent achievements that are improving the health of the American people
PREMATURE BIRTH: NEW RESEARCH MAY REVERSE A TREND
The number of infants who are born prematurely is increasing. While infant mortality rates have decreased significantly in recent years, the number of premature low birth weight babies born has increased by 11 percent over the last two decades. The number of premature very low birth weight infants, weighing less than 1500 grams, has increased by 24 percent. Research supported by the NICHD has helped many premature infants to survive. But these infants can develop neurological, respiratory, or other conditions causing life-long disabilities. Recently, NICHD scientists discovered that weekly injections of progesterone, a readily available hormone, can lower premature birth by more than one-third among women who are at risk of premature delivery. In this study, like many clinical studies, some of the women received the progesterone and some received a placebo injection. The results were so dramatic that the scientists halted the study and administered progesterone to all women enrolled in the study.
ORAL CONTRACEPTIVES AND BREAST CANCER: NO ASSOCIATION
The NICHD research has also provided reassuring evidence to women and their physicians who may be concerned about a possible relationship between oral contraceptive use and breast cancer. About 80 percent of U.S. women born since 1945 have used oral contraceptives. Conflicting studies had caused concern about the possible effect of oral contraceptive use on breast cancer risk. The NICHD's Women's Contraceptive and Reproductive Experiences Study found that women between the ages of 35 and 64 who took oral contraceptives at some point in their lives were no more likely to develop breast cancer than other women the same age who never took oral contraceptives. Many women who took oral contraceptives during their reproductive years are now reaching the ages of greatest breast cancer risk. This study should resolve the long-standing concern that oral contraceptive use might be associated with an increased risk of breast cancer in later life.
VASECTOMY AND PROSTATE CANCER: NO ASSOCIATION
Another study supported by the NICHD answered an important question for men who have had vasectomies. About one out of six American men over the age of 35 has had a vasectomy. Some studies conducted in the United States in the early 1990s reported a moderately increased risk of prostate cancer among men who underwent vasectomy. Other studies found no such risk. Because of this conflicting evidence, many urologists have increased prostate cancer screening of men who had vasectomies and have discouraged vasectomies in men with a family history of prostate cancer. The NICHD study found that men who had a vasectomy were no more likely to develop prostate cancer than those who had not had a vasectomy. The study also found that men who had vasectomies as long as 25 years ago did not have an increased risk of prostate cancer. These results should reassure men who have had or who are considering a vasectomy.
STROKE PATIENTS IMPROVE FUNCTION OF IMPAIRED LIMB
The results of other NICHD-supported research provide encouraging news to some stroke victims. Until recently, therapy for stroke victims often involved teaching patients to strengthen their less impaired limb for several weeks after a stroke. The prevailing view among rehabilitation professionals was that patients' motor ability reached a plateau at about six months after a stroke. They believed that additional therapy would provide little if any additional benefit. But new research has shown that the use of the impaired limb can improve significantly a year or more after a stroke. Using "Constraint Induced Therapy," researchers showed that constraining the good or less affected limb for 10 days can help restore a great deal of mobility to the impaired limb.
TRAUMATIC BRAIN INJURY NETWORK FOR BETTER TREATMENTS
Traumatic brain injury is one of the leading causes of death and disability in children and adults. An estimated two million head injuries occur in the United States each year. As a result of advances in emergency medicine at the accident scene and the hospital, many TBI victims are living longer. However, many will live with persistent physical, cognitive, behavioral and social deficits that compromise their quality of life. Research over the last two decades has demonstrated that not all neurologic damage occurs at the moment of injury, but evolves over the minutes, hours, and days after an accident. Research also has dramatically improved the immediate care, follow-on care, and rehabilitative process for TBI patients. Yet there are many unanswered questions about the underlying damage and the reasons for reduced functioning associated with TBI. In addition, to determine the most appropriate therapies for children and young adults with TBI, multiple sites are needed to evaluate various interventions with many patients. To address this need, the NICHD recently established the Traumatic Brain Injury Clinical Trials Network. The Network will evaluate medical, rehabilitative, and educational interventions to identify which ones most effectively improve the long-term outcomes of TBI patients.
NEW FRAGILE X CENTERS WILL DEVELOP TREATMENT OPTIONS
Fragile X syndrome is the most common genetically-inherited form of mental retardation currently known. The condition occurs in every 1 out of 2,000 males and in 1 in 4,000 females. The syndrome is caused by a mutation in a specific gene (FMR1) on the X chromosome. In its fully-mutated form, the FMR1 gene interferes with normal development. In a partially mutated (premutation) form, the FMR1 gene can cause fragile X syndrome in the children of a carrier (a person who has the premutation gene). Until recently, however, the premutation form was not thought to cause symptoms in carriers. Scientists have now identified a subgroup of premutation FMR1 carriers with symptoms that appear to be associated with the gene. Symptoms included mild cognitive and emotional problems and, in female carriers, premature menopause. In older male carriers, the premutation gene is associated with a neurological syndrome. Identifying a genetic basis could be a first step toward accurate diagnosis and, possibly, development of new treatments for these often overlooked symptoms. In addition, to develop improved diagnostic techniques and treatment options, the NICHD will begin funding three new Fragile X research centers in FY 2003. Each center will call upon the combined expertise of several researchers working in diverse fields to investigate different aspects of the disorder. The new Fragile X Research Centers will study issues such as how the fragile X affects the developing brain and nervous system, how the disorder progresses throughout an individual's life span, and effective treatments that can improve the behavior and mental functioning of people with fragile X syndrome.
STRATEGIC ALLIANCES WITH MINORITY GROUPS TO REDUCE SIDS
Less than ten years ago, the NICHD initiated a campaign urging parents and care takers to place infants on their backs to sleep to reduce the risk of Sudden Infant Death Syndrome (SIDS). Since that time, the SIDS rate in the U.S. has declined by more than 50 percent. This dramatic decline represents a significant public health achievement because the SIDS rates had remained tenaciously steady prior to the NICHD campaign. Although the SIDS rates have declined in all populations since the campaign began, the SIDS rate among African American infants remains double that of white infants. Among Alaska Natives and many American Indian tribes, the rates are higher still. To begin closing this gap, the NICHD has formed strategic alliances with the Alpha Kappa Alpha sorority, The National Coalition of 100 Black Women, and The Women in the NAACP. In collaboration with these organizations, the NICHD has planned and will support a series of "summit" meetings in three U.S. cities with high rates of African American SIDS deaths. These summits will enlist the resources of faith-based and community organizations, public health officials, and service organizations to help establish an infrastructure that will provide information, material, and support for reducing SIDS among African American infants. Each organization will take the lead in organizing one of the summit meetings and will continue to serve as the catalyst for SIDS risk reduction activity in that city and its surrounding region.
The NICHD has also initiated a project with American Indian and Alaska Native groups to reduce SIDS and infant mortality in these populations. At NICHD-sponsored meetings in Minneapolis, MN and Rapid City, SD, representatives of Tribal Chairman's Health Boards and Alaska Native health organizations provided the NICHD with a blueprint to support the activities of community health workers involved in SIDS risk reduction education. The NICHD will develop and disseminate the materials for this effort during the current year.
TESTING DRUGS TO IMPROVE HEALTH OF CHILDREN AND PREGNANT WOMEN
In FY 2004, the NICHD will continue to invest in research and programs that benefit the American people. One such investment is the fulfillment of the Best Pharmaceuticals for Children Act (BPCA). The immature physiology of children means that drugs approved to prevent or treat illness in adults may have different effects in younger patients, requiring children's physicians to prescribe different doses and make other adjustments in drug therapies. However, for approximately seventy-five percent of the pharmaceuticals approved by the Food and Drug Administration (FDA) for adults, there are inadequate safety and efficacy data to allow approval for pediatric uses, or to guide physicians in prescribing these drugs for children. The BPCA, signed into law in January 2002, directs the NIH to issue contracts to test in children off-patent prescription drugs already approved for adults. Working with the FDA and other experts, the NICHD identified a priority list of drugs to be tested through the Institute's Pediatric Pharmacology Research Units (PPRUs) and at other sites. The FY 2004 budget request includes an increase of $25 million, across all of the NIH Institutes and Centers (ICs), for these studies.
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 prescribed 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.
EXPANSION OF NEWBORN SCREENING THROUGH MICROARRAY TECHNOLOGY
At present, all states routinely screen all newborns for only two disorders: phenylketonuria (PKU) and congenital hypothyroidism. These are conditions for which effective treatments are available. In addition, most states screen for a mix of 1 to 15 other disorders, but some commercially available tests can screen for up to 50 conditions. A Secretarial-level panel and the American Academy of Pediatrics have recommended that an expanded and standardized approach to newborn screening be developed. To address this need, the NICHD proposes to apply the knowledge and techniques garnered from the Human Genome Project. Using cord blood and microarray technology, there is the potential to identify disease genes at birth for more than 200 single gene defects associated with mental retardation, nearly 100 associated with immunodeficiency disorders, approximately 10 causes of muscular dystrophy, and cystic fibrosis. Although treatments are available for many of these conditions, effective study of potential new treatments for others requires a population who has not yet developed symptons of the condition. Screening of newborn infants can provide this population. This testing could be done in one procedure so that economies of scale and simplicity may overcome one of the major obstacles to widespread acceptance of expanded newborn screening: cost.
The NICHD will collaborate with several other ICs, research institutions, and industry to develop the appropriate microarray chip and associated technology for mass screening and pilot test the new screening technology. This approach would maximize the use of newborn screening for preventive purposes. Moreover, by developing this translational research, NICHD will fulfill one of the objectives of the NIH road map activities.
Mr. Chairman, I will be happy to provide answers to any questions you have.