Clinical Trials of Drugs Used Off-Label in Neonates: Ethical Issues and Alternative Study Designs

TitleClinical Trials of Drugs Used Off-Label in Neonates: Ethical Issues and Alternative Study Designs
Publication TypeJournal Article
Year of Publication2008
AuthorsAmin, SB, McDermott, MP, Shamoo, AE
JournalAccountability in Research: Policies & Quality Assurance
Volume15
Issue3
Pagination168-187
Type of ArticleArticle
Publication Languageeng
ISBN Number08989621
Accession Number32964889
KeywordsACTIVE , ALTERNATIVE , Clinical , Drugs , ethics , National , neonatal , neonates , NEWBORN , off-label , Placebo , Testing , United , unlicensed
AbstractThe use of drugs for indications unapproved by the Food and Drug Administration, often called “off-label use,” is widespread in children and neonates, and presents ethical and safety challenges. Since the passage of the Best Pharmaceuticals for Children Act (BPCA) in 2002, both the FDA and National Institutes of Health (NIH) have taken initiatives to facilitate and encourage research to achieve the necessary labeling for drugs routinely used in infants and children. However, there are ethical issues that a physician may face when designing clinical trials of drugs in neonates that are routinely used off-label and widely believed to be beneficial. The authors describe these challenges and provide recommendations,including alternative study designs, to resolve them in an ethical framework that takes into account the Belmont Report, the statement of the World Medical Association (WMA), and federal regulations.
NotesAmin, Sanjiv B. 1; Email Address: sanjiv_amin@urmc.rochester.edu; McDermott, Michael P. 2; Shamoo, Adil E. 3; Affiliations: 1: Department of Pediatrics, University of Rochester School of Medicine, Rochester, New York, USA; 2: Departments of Biostatistics and Computational Biology and Neurology, University of Rochester School of Medicine, Rochester, New York, USA; 3: Departments of Biochemistry and Molecular Biology and Epidemiology and Preventive Medicine, University of Maryland, School of Medicine, Baltimore, Maryland, USA; Issue Info: Jul-Sep2008, Vol. 15 Issue 3, p168; Thesaurus Term: TESTING; Subject Term: CLINICAL trials; Subject Term: DRUGS; Subject Term: NEWBORN infants; Subject Term: OFF-label use (Drugs); Subject: UNITED States; Author-Supplied Keyword: active control; Author-Supplied Keyword: alternative study designs; Author-Supplied Keyword: clinical trials; Author-Supplied Keyword: ethics; Author-Supplied Keyword: neonatal emergency research; Author-Supplied Keyword: neonates; Author-Supplied Keyword: off-label; Author-Supplied Keyword: placebo; Author-Supplied Keyword: unlicensed ; Company/Entity: UNITED States. Food & Drug Administration ; Company/Entity: NATIONAL Institutes of Health (U.S.); Number of Pages: 20p; Illustrations: 2 Charts; Document Type: Article
DOI10.1080/08989620802194392
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