title: Assessment of congenital anomalies in infants born to pregnant women enrolled in clinical trials.
abstract: In 2011 and 2012, the Division of Microbiology and Infectious Diseases at the National Institute of Allergy and Infectious Diseases, National Institutes of Health, held a series of meetings to provide guidance to investigators regarding study design of clinical trials of vaccines and antimicrobial medications that enroll pregnant women. Assessment of congenital anomalies among infants born to women enrolled in these trials was recognized as a challenging issue, and a workgroup with expertise in epidemiology, pediatrics, genetics, dysmorphology, clinical trials, and infectious diseases was formed to address this issue. The workgroup considered 3 approaches for congenital anomalies assessment that have been developed for use in other studies: (1) maternal report combined with medical records review, (2) standardized photographic assessment and physical examination by a health professional who has received specific training in congenital anomalies, and (3) standardized physical examination by a trained dysmorphologist (combined with maternal interview and medical records review). The strengths and limitations of these approaches were discussed with regard to their use in clinical trials. None of the approaches was deemed appropriate for use in all clinical trials. Instead, the workgroup acknowledged that decisions regarding the optimal method of assessment of congenital anomalies will likely vary depending on the clinical trial, its setting, and the agent under study; in some cases, a combination of approaches may be appropriate. The workgroup recognized the need for more research on approaches to the assessment of congenital anomalies to better guide investigators in optimal design of clinical trials that enroll pregnant women.
authors.affiliation: Centers for Disease Control and Prevention, Atlanta, Georgia.
authors.forename: Sonja A
authors.initials: SA
authors.lastname: Rasmussen
authors.affiliation: Harvard School of Public Health, Boston, Massachusetts.
authors.forename: Sonia
authors.initials: S
authors.lastname: Hernandez-Diaz
authors.affiliation: University of Mississippi Medical Center, Jackson.
authors.forename: Omar A
authors.initials: OA
authors.lastname: Abdul-Rahman
authors.affiliation: Food and Drug Administration, Silver Spring.
authors.forename: Leyla
authors.initials: L
authors.lastname: Sahin
authors.affiliation: The EMMES Corporation, Rockville.
authors.forename: Carey R
authors.initials: CR
authors.lastname: Petrie
authors.affiliation: National Institutes of Health, Bethesda, Maryland.
authors.forename: Kim M
authors.initials: KM
authors.lastname: Keppler-Noreuil
authors.affiliation: Saint Louis University School of Medicine, Missouri.
authors.forename: Sharon E
authors.initials: SE
authors.lastname: Frey
authors.affiliation: National Institutes of Health, Bethesda, Maryland.
authors.forename: Robin M
authors.initials: RM
authors.lastname: Mason
authors.affiliation: National Institutes of Health, Bethesda, Maryland.
authors.forename: Mirjana
authors.initials: M
authors.lastname: Nesin
authors.affiliation: University of Utah School of Medicine, Salt Lake City.
authors.forename: John C
authors.initials: JC
authors.lastname: Carey
mesh_terms: D000890:Anti-Infective Agents / Q000008:administration & dosage; D002986:Clinical Trials as Topic; D000013:Congenital Abnormalities / Q000175:diagnosis / Q000453:epidemiology; D005260:Female; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D011247:Pregnancy; D037841:Pregnant People; D014481:United States; D014612:Vaccines / Q000008:administration & dosage
chemical_list: D000890:Anti-Infective Agents; D014612:Vaccines
keywords: birth defects; clinical trials; congenital anomalies; pregnant women.