Pediatric registries at the Food and Drug Administration: design aspects that increase their likelihood of success

• Published in: Pharmacoepidemiology and drug safety Vol. 25; no. 5; pp. 602 - 605
• Main Authors: Winiecki, Scott K., Tejero-Taldo, M. Isabel, Avant, Debbie, Murphy, Dianne, McMahon, Ann W.
• Format: Journal Article
• Language: English
• Published: England Blackwell Publishing Ltd 01.05.2016; Wiley Subscription Services, Inc
• Subjects: Biological Products - adverse effects; Child; Drug Approval; Drug-Related Side Effects and Adverse Reactions; FDA approval; Humans; Pediatrics; Pharmaceutical industry; pharmacoepidemiology; postmarketing; product surveillance; Product Surveillance, Postmarketing - methods; Registries; United States; United States Food and Drug Administration; US Food and Drug Administration; postmarketing; US Food and Drug Administration; product surveillance; registries; pediatrics; pharmacoepidemiology
• ISSN: 1053-8569; 1099-1557
• DOI: 10.1002/pds.3939

Purpose To determine aspects of the design of pediatric registries that contribute to the success of registries conducted as a postmarketing study following approval of drugs or biological products by the US Food and Drug Administration. Methods Pediatric registries for drugs and biological products were identified by searching the US Food and Drug Administration Postmarketing Requirements and Commitments database. Based on the recruitment of patients, the meeting of predetermined deadlines, and the submission of data, we classified studies as successful, unsuccessful, or unevaluable. Design aspects of successful and unsuccessful registries were examined for commonalities. Results Thirty‐eight studies were identified, and ten registries met the criteria for successful. Four (40%) successful registries utilized a registry established prior to product approval, and six (60%) were disease‐based. Among unsuccessful registries, none were disease‐based or utilized a pre‐existing registry. Conclusions Characteristics identified as more common to successful registries included utilizing a disease‐based registry and a registry established prior to product approval. Future studies might examine a larger sample of registries to see if these aspects consistently result in successful studies. Published 2015. This article is a U.S. Government work and is in the public domain in the USA.