TY - JOUR
T1 - Institution-based prospective inception cohort studies in neonatal rare disease research
AU - Armstrong-Wells, Jennifer
AU - Goldenberg, Neil A.
N1 - Funding Information:
Dr Armstrong-Wells is funded in part by a Career Development Award from the National Institute of Health, Building Interdisciplinary Research Careers in Womens’ Health (BIRCWH) K12 ( KD HDO57022 ). Dr Goldenberg is funded in part by a Career Development Award from the National Institutes of Health, National Heart Lung and Blood Institute ( 5K23HL084055 ).
PY - 2011/12
Y1 - 2011/12
N2 - In rare diseases, wherein the conduct of randomized controlled clinical trials (RCTs) is challenging, cohort studies can offer important, and, in certain instances, high quality (e.g. the prospective inception cohort study) evidence on relationships among risk factors, treatments, and outcomes. The objective of this perspective article is to provide an overview of salient issues in the design and application of institution-based prospective inception cohort studies in neonatal rare disease research, with emphasis on quality assurance measures. Rigorous implementation of the prospective inception cohort study is challenging, and application to neonates renders it even more difficult. However, when performed collaboratively among institutions employing uniform methods and quality assurance mechanisms, institution-based prospective inception cohort studies can provide optimal observational evidence to inform the design and execution of RCTs in this special pediatric population.
AB - In rare diseases, wherein the conduct of randomized controlled clinical trials (RCTs) is challenging, cohort studies can offer important, and, in certain instances, high quality (e.g. the prospective inception cohort study) evidence on relationships among risk factors, treatments, and outcomes. The objective of this perspective article is to provide an overview of salient issues in the design and application of institution-based prospective inception cohort studies in neonatal rare disease research, with emphasis on quality assurance measures. Rigorous implementation of the prospective inception cohort study is challenging, and application to neonates renders it even more difficult. However, when performed collaboratively among institutions employing uniform methods and quality assurance mechanisms, institution-based prospective inception cohort studies can provide optimal observational evidence to inform the design and execution of RCTs in this special pediatric population.
KW - Children
KW - Cohort
KW - Neonates
KW - Quality assurance
KW - Rare disease
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U2 - 10.1016/j.siny.2011.07.004
DO - 10.1016/j.siny.2011.07.004
M3 - Review article
C2 - 21820983
AN - SCOPUS:80053617561
SN - 1744-165X
VL - 16
SP - 355
EP - 358
JO - Seminars in Fetal and Neonatal Medicine
JF - Seminars in Fetal and Neonatal Medicine
IS - 6
ER -