The Children's Hospital of Philadelphia (CHOP), in collaboration with the Center for Clinical Epidemiology and Biostatistics (CCEB) at the University of Pennsylvania, proposes to develop an innovative research training program in pediatric pharmacoepidemiology, specifically designed to train pediatricians to be rigorous, independent academic investigators able to use the range of approaches available in epidemiology to study the use and effects of medications in pediatric patients.
The specific aims of the training program are to: 1) provide in-depth knowledge of the research techniques appropriate to clinical pharmacoepidemiology research;2) apply these research techniques to pediatric populations;3) provide an understanding of the basic principles of clinical pharmacology and how these principles apply to pediatric patients;4) provide opportunities for the study of topics related to pharmacoepidemiology in a pediatric setting;5) provide research experience with mentors in pediatric pharmacoepidemiology;and 6) bring together faculty and fellows through seminar series at CHOP and at the CCEB. Trainees will earn a Master of Science in Clinical Epidemiology (MSCE) degree from the CCEB. This multidisciplinary, two- to three-year training program consists of core courses in epidemiology, clinical research methodology, and biostatistics;required courses in pharmacoepidemiology and pharmacokinetics;elective courses in advanced methods for epidemiology and biostatistics, and/or pharmacology;a required clinical experience in pediatric pharmacoepidemiology;a teaching seminar series;instruction in the responsible conduct of research and regulatory affairs;extensive independent readings and attendance at research seminars at CHOP and the CCEB;and applied research in pediatric pharmacoepidemiology under the close supervision of a senior mentor. Strengths of the proposed program are: 1) the long history of successful research training programs in the CCEB and CHOP;2) the collaborative links that have been forged among the faculty between these academic entities;3) the comprehensive course offerings and research programs that are available to trainees;4) the internationally renowned pharmacoepidemiology program housed in the CCEB;5) the wealth of databases available at CHOP for pharmacoepidemiologic analyses;and 6) the extensive successful training records of the program director and faculty.

Public Health Relevance

There is a major national shortage of qualified pediatric clinicians able to conduct rigorous clinical research in pedantic pharmacoepidemiology. This training program addresses these shortages through the collaborative efforts of pediatricians and pharmacoepidemiologists. Specific research areas of focus of mentors include: pediatric cancer drug development;effectiveness of pharmaceuticals for common problems in general pediatrics, and the relationship between antibiotic use and antibiotic resistance.

National Institute of Health (NIH)
Eunice Kennedy Shriver National Institute of Child Health & Human Development (NICHD)
Institutional National Research Service Award (T32)
Project #
Application #
Study Section
Pediatrics Subcommittee (CHHD)
Program Officer
Giacoia, George
Project Start
Project End
Budget Start
Budget End
Support Year
Fiscal Year
Total Cost
Indirect Cost
Children's Hospital of Philadelphia
United States
Zip Code
DiNofia, Amanda M; Seif, Alix E; Devidas, Meenakshi et al. (2018) Cost comparison by treatment arm and center-level variations in cost and inpatient days on the phase III high-risk B acute lymphoblastic leukemia trial AALL0232. Cancer Med 7:3-12
Miller, Tamara P; Li, Yimei; Kavcic, Marko et al. (2017) Center-level variation in accuracy of adverse event reporting in a clinical trial for pediatric acute myeloid leukemia: a report from the Children's Oncology Group. Haematologica 102:e340-e343
James, Karen E; Xiao, Rui; Merkel, Peter A et al. (2017) Variation in the Treatment of Children Hospitalized With Antineutrophil Cytoplasmic Antibody-Associated Vasculitis in the US. Arthritis Care Res (Hoboken) 69:1377-1383
DiNofia, Amanda M; Salazar, Elizabeth; Seif, Alix E et al. (2016) Bortezomib Inpatient Prescribing Practices in Free-Standing Children's Hospitals in the United States. PLoS One 11:e0151362
Miller, Tamara P; Li, Yimei; Kavcic, Marko et al. (2016) Accuracy of Adverse Event Ascertainment in Clinical Trials for Pediatric Acute Myeloid Leukemia. J Clin Oncol 34:1537-43
James, Karen E; Xiao, Rui; Merkel, Peter A et al. (2016) Clinical course and outcomes of childhood-onset granulomatosis with polyangiitis. Clin Exp Rheumatol :
Miller, Tamara P; Getz, Kelly D; Kavcic, Marko et al. (2016) A comparison of discharge strategies after chemotherapy completion in pediatric patients with acute myeloid leukemia: a report from the Children's Oncology Group. Leuk Lymphoma 57:1567-74
Ipe, Tina S; Wilkes, Jennifer J; Hartung, Helge D et al. (2015) Severe hemolytic transfusion reaction due to anti-D in a D+ patient with sickle cell disease. J Pediatr Hematol Oncol 37:e135-7
Taxter, Alysha J; Wileyto, E Paul; Behrens, Edward M et al. (2015) Patient-reported Outcomes across Categories of Juvenile Idiopathic Arthritis. J Rheumatol 42:1914-21
Miller, Tamara P; Troxel, Andrea B; Li, Yimei et al. (2015) Comparison of administrative/billing data to expected protocol-mandated chemotherapy exposure in children with acute myeloid leukemia: A report from the Children's Oncology Group. Pediatr Blood Cancer 62:1184-9

Showing the most recent 10 out of 17 publications