DEPARTMENT OF HEALTH AND HUMAN SERVICES
NATIONAL INSTITUTES OF HEALTH
Fiscal Year 2017 Budget Request
Statement for the Record
Senate Appropriations Subcommittee on Labor, Health and Human Services, Education and Related Agencies
Catherine Y. Spong, M.D., Acting Director
Eunice Kennedy Shriver National Institute of Child Health and Human Development
Mr. Chairman and Members of the Committee: I am pleased to present the President's Fiscal Year (FY) 2017 budget request for the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) of the National Institutes of Health (NIH).
Understanding human development, both normative and atypical, is at the core of NICHD's mission. Unlike many of the other NIH Institutes and Centers (ICs), NICHD takes a lifespan approach to its work, supporting research that ranges from efforts to increase our understanding of basic biological mechanisms to testing health interventions aimed at improving the lives and health of children, women, and families, including those with disabilities.
Minimizing Duplication, Maximizing Expertise
In this increasingly complex world, highly specific expertise may be required to ensure that any preventive measures or development and application of therapies will meet the needs of each affected population. Because of the breadth of its mission, NICHD can provide a lifecourse perspective and a depth of expertise to research efforts related to pregnancy, infants, and the typical or atypical development of children through young adulthood. This breadth also means that NICHD works across scientific disciplines and often seeks or is tapped to lead trans-NIH, collaborative efforts that bring together other ICs' and non-NIH scientists' knowledge and skills so that the overall efforts are additive, not duplicative.
Human Placenta Project – NICHD is leading an initiative known as the Human Placenta Project that will develop technologies to assess, in real time, the structure and function of the human placenta. The least understood human organ, the placenta has substantial implications for the lifelong health of both mother and child, since so many health issues can be traced back to the earliest stages of development. The Project's goals include understanding normal and abnormal placental development, developing biomarkers to predict adverse pregnancy outcomes, examining the effects of environmental factors, and developing interventions to prevent abnormal placental and fetal development. Each year, NICHD brings together a group of broad thinkers that includes subject matter experts in various technologies, placental biologists, and clinicians to generate creative approaches to developing and applying technologies in new, noninvasive ways to understand placental function. The impact of the Human Placenta Project extends beyond enhancing pregnancy to providing additional insights into health and disease. New knowledge will increase our understanding of tumor biology and transplant medicine as the placenta mimics tumor growth and invasion early in gestation and mediates and promotes the health of two genetically distinct entities.
PregSource™ – About 6.5 million pregnancies occur each year in the United States, and yet we have relatively little scientific information about what constitutes a normal pregnancy. In the coming year, NICHD will launch "PregSource™," a longitudinal, crowd-sourced, citizen science approach that will gather information about pregnancy from the experts – pregnant women. Through web-based questionnaires or mobile apps, PregSource™ will expand our knowledge about what women experience physically and emotionally during pregnancy and after giving birth, the effects of pregnancy on women's lives, the special challenges some pregnant women face (such as health issues related to disability or a chronic health condition), and what features are common to many pregnancies. Ultimately, this information will help to fill the large gap in knowledge about medications used by pregnant women and other issues commonly experienced during pregnancy. In return, PregSource™ will provide participants with links to evidence-based information about pregnancy from 20 trusted national partners, including several NIH ICs and professional societies. Approved researchers can view de-identified, amalgamated information that may suggest fruitful research avenues about pregnancy. This innovative approach to gathering information was based on the highly successful DS-Connect®: The Down Syndrome Registry, which was launched by NICHD and its Down Syndrome Consortium partners in 2013. With more than 3,200 registrants across the United States, DS-Connect® has facilitated recruitment into clinical trials on several aspects of Down syndrome.
Birth Defects – With other NIH ICs and the Common Fund of the NIH Office of the Director, in FY 2017 NICHD will continue to provide leadership for extended basic and translational research efforts related to birth defects. Structural or functional anomalies affect about three percent of births and are the leading cause of infant death in the United States. Through intramural and extramural programs, NICHD supports collaborative teams of basic and clinical scientists studying the developmental biology, epidemiology, and genetics of structural birth defects. New FY 2017 initiatives in structural birth defects research will catalyze the development of new research teams and support data analysis that uses and complements the genomic sequencing data developed under the Gabriella Miller Kids First Pediatric Research program, funded by Congress since FY 2014.
Best Pharmaceuticals for Children – Although progress has been made in recent years, approximately three-quarters of all medicines marketed still do not carry Food and Drug Administration (FDA) approved labeling for use in neonates, infants, children, and adolescents. Although it is widely recognized that children are not small adults, only five of the 80 drugs most frequently used in newborns and infants are labeled for pediatric use. For many drugs, developmental, metabolic, and pharmacokinetics-pharmacodynamic data are needed for their safe and effective use in children, yet sometimes private pharmaceutical companies are reluctant to incur the expense of gathering these data. In the Best Pharmaceuticals for Children Act of 2002, which was reauthorized in 2007 and 2012, the Congress charged NIH with setting priorities and funding research on pediatric therapeutic needs, tapping NICHD to lead this effort. Through an active trans-NIH Working Group, NICHD has collaborated with other ICs to identify therapeutic gaps in pediatric diseases, disorders, or conditions for which more complete knowledge of drugs and biologics would benefit specific pediatric populations, and develop future clinical trials. Using this approach, and also consulting with pediatric experts from FDA, academia, and industry, NICHD regularly publishes a prioritized list of drugs or indications that require further investigations. Four additional drugs commonly prescribed for children now have pediatric labeling, with others pending approval at FDA. Most recently, NICHD staff met with FDA pediatric leadership in December 2015 to discuss how to further improve the process for getting drugs labeled for pediatric use.
Rehabilitation Research – Although NIH was already supporting multiple lines of research focused on medical rehabilitation for physical, sensory, and cognitive disability across the lifespan, the establishment of the National Center for Medical Rehabilitation Research (NCMRR) by the Congress in 1990 provided a new focus on the science of medical rehabilitation and how results could be used to meet the challenges facing individuals with physical disabilities. A component of NICHD, NCMRR leadership emphasizes the need for medical rehabilitation across the lifespan, working across the various NIH ICs that have relevant and complementary missions. NCMRR serves as the focal point for the coordination and development of policies, plans, initiatives, and objectives that further rehabilitation research, convening the NIH Medical Rehabilitation Coordination Committee (MRCC) on a monthly basis to coordinate ongoing research and training activities. In addition, MRCC works with other agencies within the Department of Health and Human Services, the Department of Veterans Affairs, the Department of Defense, and the National Science Foundation to develop collaborative projects that serve to enhance and encourage rehabilitation science. A major conference on rehabilitation research in May 2016 will bring together scientists from across the rehabilitation research spectrum, which will serve to inform and catalyze the research community through a new rehabilitation research plan, expected in early FY 2017.
Training Opportunities in Endocrinology – NICHD's intramural researchers also engage in regular collaborations with scientists from other ICs. Together with the National Institute of Diabetes and Digestive and Kidney Diseases, and the National Institute of Dental and Craniofacial Research, NICHD collaborates on the Inter-Institute Endocrinology Training Program, an accredited program that provides a comprehensive training experience for physicians who seek a broad education in both research and clinical endocrinology. Led by world-renowned experts in the field, trainees can learn how to manage a wide variety of common and rare endocrine disorders. For example, the program provides training in specialized diabetes clinics to teach how to provide intensive insulin therapy in pediatric patients with severe diabetes.
IDeA States Pediatric Clinical Trials Network – Following the closing of the National Children's Study in late 2014, passage of the National Pediatric Research Network Act, and after extensive input from the research and other stakeholder communities about pediatric research priorities, NIH decided to augment its pediatric research efforts through the creation of a new national pediatric network, the IDeA States Pediatric Clinical Trials Network (ISPCTN). The new ISPCTN will utilize the Institutional Development Awards (IDeA) Program, which was authorized and funded by the Congress to broaden the geographic distribution of NIH funding across the United States by enhancing the ability to compete for funding of institutions located in States in which the success rate for NIH grant applications historically has been low. The goal of ISPCTN is to develop a pediatric research infrastructure and build capacity at academic institutions in IDeA States for pediatric clinical trials; increase access to clinical trials for children (some of whom may have rare conditions) in rural and medically underserved areas who are unable to travel long distances; and allow researchers across the country who are conducting pediatric clinical trials to recruit a greater diversity of pediatric participants in a cost-effective manner. By creating teams of pediatric clinical trial specialists in IDeA States throughout the country, children who have never before had realistic access to cutting edge research will have the opportunity to participate. In addition, investigators conducting any IRB-approved, funded pediatric clinical trial (not only those in the IDeA States) may work with the Data Coordinating and Operations Center to recruit additional pediatric participants from IDeA State sites for their trials; thus, trials on rare conditions may be able to complete recruitment within a shorter period of time, even though there might be a limited number of individuals in any one geographic area. ISPCTN will be overseen by the Program Director for the new Environmental influences on Child Health Outcomes (ECHO) program in the NIH Office of the Director, and managed by NICHD.
Zika Virus – The rapid spread of the Zika virus across the Americas has emerged as a potentially critical public health issue. Although the virus was initially discovered in 1947, recent reports from Brazil suggested a possible connection with an increase in severe birth defects, including microcephaly, which sounded the alarm to the public health community. NICHD is coordinating with and contributing to the overall effort by providing expertise in developmental biology, pregnancy, and preventive measures such as contraception. Efforts include work to understand how the virus is transmitted and how best to prevent it; the effects of the virus on the body, especially during pregnancy and timing of the infection; the impact on the developing fetus; new assays to test therapeutic candidates, and improved diagnostics to rapidly identify the virus and distinguish it from related viruses. Joined by six other NIH ICs, NICHD issued a rapid funding opportunity announcement to stimulate research on Zika, particularly on the seminal issue of whether the virus causes fetal abnormalities and pregnancy complications (fetal loss, stillbirth, microcephaly, ventriculomegaly, brain anomalies, and ocular abnormalities). In addition, NICHD is coordinating with the National Institute of Allergy and Infectious Diseases and sites in several countries, including Brazil, to conduct a prospective longitudinal study on Zika-related adverse pregnancy and infant outcomes. Since evidence of a link between the Zika virus and microcephaly and other abnormalities is now sufficiently strong, NICHD will again be called upon to utilize its knowledge base on the long-term consequences of physical and intellectual disabilities to help children and families affected by the consequences of the disease.