Studies Will Determine Pediatric Outcome Measures to Facilitate Future Research
Tuesday, November 17, 2009
Studying drugs in pediatric populations is challenging because drugs often affect children differently than they do adults. The scarcity of pediatric studies limits the ability of doctors and scientists to predict drug dosing, safety and efficacy in children. To address this gap, the National Institutes of Health announced today 18 grants to help determine outcome measures and increase the likelihood of success of future trials of treatments for children.
The grants were awarded to 17 Clinical and Translational Science Award (CTSA) institutions to support 18 studies of pharmaceutical treatments for children. The CTSA consortium is a national network of 46 medical research institutions working together to improve the way biomedical research is conducted across the country. The $8.5 million in funding will support studies which focus on three areas critical to health: pediatric cardiology, neonatology and pediatric neurology.
The awards will be administered by the National Center for Research Resources (NCRR) while the funding was provided by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). The NCRR and NICHD are part of the NIH.
“These awards exemplify what the CTSA program supports: innovative, transformative and collaborative research,” said Barbara Alving, M.D., director of NCRR. “These CTSA institutions are uniquely positioned to deliver outcomes in these studies that may translate into better treatments for children in the future."
The funding is part of NIH’s continuing efforts in studying drugs for use in pediatric populations. The Best Pharmaceuticals for Children Act of 2002 established a process to study on-patent and off-patent drugs for children and to improve pediatric therapeutics through collaboration on scientific investigation, clinical study design, weight of evidence, and ethical and labeling issues.
“People of different ages require research outcome measures that are tailored to their size and condition,” said Steven Hirschfeld, M.D., Ph.D., NICHD associate director for clinical research. “These studies will fill a critical gap by providing the precision needed to understand outcomes for younger patients who may respond differently than adults, and thereby enhance the quality of pediatric research.”
As an example, premature infants often suffer from a lung condition called bronchopulmonary dysplasia (BPD), which requires the use of oxygen and other supportive measures, and places them at high risk for developing chronic respiratory obstruction later in life. However, researchers have found that the success of therapies used to treat BPD in newborns did not necessarily correlate with success in staving off the chronic conditions, suggesting the currently used outcome measures are not reliable. To address this deficiency, two separate projects have been funded and will be conducted by researchers at the University of North Carolina at Chapel Hill and at Tufts University. The goals of the of these projects are to develop new tools to define the extent of functional impairment due to BPD in neonates and to establish a registry to track and quantify the onset and extent of chronic lung problems in these at-risk infants as they grow older, respectively.
In another example, the tools such as blood pressure cuffs that doctors use to diagnose and treat hypertension were developed for adults and have never been validated in children. As a result, researchers have had a hard time detailing the effectiveness of treatments for high blood pressure in children. To address this problem, researchers at Case Western Reserve University received funding to adapt the blood pressure cuffs to children. To better understand blood pressure patterns in children, a consortium of five CTSAs led by investigators at the Albert Einstein College of Medicine will enroll children between the ages of 10 and18 years into a six-month study of the effect of home-based diet and lifestyle modification on blood pressure.
The National Center for Research Resources (NCRR), a part of NIH, provides laboratory scientists and clinical researchers with the resources and training they need to understand, detect, treat and prevent a wide range of diseases. NCRR supports all aspects of translational and clinical research, connecting researchers, patients and communities across the nation.
About the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD): The NICHD sponsors research on development before and after birth; maternal, child and family health; reproductive biology and population issues; and medical rehabilitation. For more information, visit the Institute’s Web site at www.nichd.nih.gov/.
About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov.