Pediatric Research in the Era of Clinical and Translational Science Awards
Article Outline
Abbreviations: CTSA, Clinical and Translational Science Award, NIH, National Institutes of Health
The National Institutes of Health (NIH) Reform Act of 2006 includes a section providing for dedicated funding for pediatric clinical research in the context of Clinical and Translational Science Awards (CTSAs). The timing of this action is in many ways opportune, in that the technical resources available for pediatric research need to encompass the range of powerful new tools that have emerged to advance clinical research, such as genome-wide association studies, ultrasound/magnetic resonance fusion imaging, and engineered nanostructures and devices. Pediatricians have been quick to exploit new therapeutic interventions in their approaches to severe or lethal diseases, and they have an enviable tradition for working together. With this background, the emphasis that CTSAs place on translational research should create an environment that will support new approaches to many of the chronic or disabling disorders of childhood. Pediatric research will benefit most from CTSAs if research teams emerge that are experienced in applying the sophisticated new approaches and technologies to clinical research in children.
Pediatric research tends to benefit from the fact that the parental decision makers are highly motivated to make decisions that will give their children the best chance of clinical benefit. The fact of parental choice requires that pediatricians in both primary and specialist care settings be familiar with the questions being asked by researchers and with the decision making process that parents use to weigh the risks and benefits of research participation.
Overall, about 1/3 of graduating medical students anticipate a career in medical or clinical research.1 Research usually follows subspecialty training, and about 1/3 of third-year pediatric residents expect to go into subspecialty training programs.2 The estimate that only 10% of the graduates of pediatric residency programs actually develop careers in pediatric research is worrying and prompts the question of why so few young pediatricians are attracted to careers in, or close to, research. Are 90% of graduating pediatricians perfectly satisfied with the treatments that are currently available, or are careers in research seen as risky and insufficiently rewarding?
For those families with children afflicted with a high-morbidity disease (eg, cystic fibrosis, ataxia-telangiectasia, type 1 diabetes mellitus), the small proportion of pediatricians embarking on research careers must be disheartening. But even focusing on these desperate circumstances is perhaps missing the opportunities that are becoming a focus of excitement in clinical medicine. “First do no harm” is useful and even important guidance for physicians, but as a philosophy, it is handicapped by rather pessimistic expectations. An exhortation that sufficed in antiquity should be updated to urge physicians to provide care that is predictive, personalized, preemptive, and participatory.3 When medical students in earlier generations were expected to present preventive strategies at attending rounds, sadly, there were few such strategies available to offer. Much has changed; for example, avoiding salt retention (by diet or angiotensin-converting enzyme inhibitor therapy) will reduce hypertension and arteriosclerosis, and wearing crash helmets reduces the severity of brain injury in road traffic accidents. In terms of lives saved, these approaches are scarcely “rocket science,” and they need to be considered only a beginning. The potential of the twenty-first century derives from interrupting the pathogenesis of common and expensive diseases. The future that pediatrics deserves lies in the prevention of such common disorders as asthma, obesity, and schizophrenia. Asthma affects an estimated 3.5 million adults in the US, at an annual cost exceeding $17 billion.4 Some 66% of US adults are obese, with combined direct and indirect costs exceeding $78 billion in 1998 dollars.5 Schizophrenia cost the US some $65 billion in 2002, with annual costs of around $15,000 per patient for medical care, lost wages, social services, and the consequences of incarceration and suicide.6
The annual costs of these conditions are cited for adults to underscore the point that if they are to be reduced, preventive strategies will have to be initiated in childhood. The survival of patients with traditionally pediatric conditions into adulthood has already blurred the traditional borders between adult and pediatric clinical practice. Adding a role for pediatricians in preventing disorders traditionally considered adult disorders will further diminish the adult–pediatric divide.
The advances that bring adult and pediatric health care together will come, of course, from a better understanding of behavioral traits and disease pathophysiology and from the identification of an ever-increasing number of loci linked to the susceptibility for multiple chronic diseases. Many of these loci are thought to affect the regulatory controls of genes and may not be expressed at a protein level. If intervention directed toward a single susceptibility component could reduce the risk of disease, then the number of loci linked to chronic (and expensive) disorders could be a source of hope rather than despair. Advances are occurring on the pharmaceutical front as well, with the identification of a couple of new “druggable” targets in an average year.7 But the most cost-effective preventive strategies for pediatricians could be environmental or behavioral, rather than depending on chronic medication.
The most cost effective preventive strategies for pediatricians could be environmental or behavioral, rather than depending on chronic medication.
Achieving the full potential of the NIH’s new CTSAs will require the cost-efficient use of a sophisticated infrastructure that encompasses genotyping, imaging, and statistical cores among other specialized resources. Both a sense of direction and a career structure also will be essential for the clinical researchers who will benefit from CTSA infrastructure. To be eligible for one of these awards, a participating institution must be able to award higher degrees in clinical research (MS or PhD), and the application must include a mentored scholars K program. This emphasis acknowledges that clinical advances come from systematic and academic study, and that research skills deserve recognition. One gauge for the future of pediatric research will be the number of graduating fellows in pediatric subspecialties who enter these programs and ultimately advance to achieve independent NIH funding.
The students, trainees, and scholars who benefit from the many research career development opportunities provided through CTSAs will have the greatest impact on the health of the nation if they are able to look beyond the traditional disorders of childhood to the many conditions that present in adolescence and adulthood that they will have unique opportunities to prevent.
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- The economic burden of schizophrenia in the United States in 2002. J Clin Psychiatry. 2005;66:1122–1129
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PII: S0022-3476(07)00344-7
doi:10.1016/j.jpeds.2007.04.007
© 2007 Mosby, Inc. All rights reserved.
