Survey of Current Guidance for Child Health Clinical Trials. The StaR Child Health Project: Standards for Research with Children
(2009; 37 pages)


At present, between 50 and 90% of daily prescriptions for sick children use "off label" drugs. Recently, legislations were introduced to stimulate the pharmaceutical industry to investigate the pharmacological effect and safety of both new and existing medicines in children. The quality of pediatric randomized controlled trials is often suboptimal, in part because guidance for their design and execution is lacking. Also, evidence indicates that the quality of reporting of randomized controlled trials is less than optimal. The aim of this survey is to identify, classify, and appraise existing guidance on the design, conduct and reporting of pediatric clinical trials.

We systematically reviewed all relevant methodological and regulatory literature on standards or guidelines for clinical drug trials in children, over the period 1999-2009. The descriptives and contents of these guidelines were extracted and their quality was appraised by a modified version of the Appraisal of Guidelines Research and Evaluation (AGREE) instrument.

Of 60 documents found on the internet and 3779 articles found in bibliographic databases, 22 internet guideline documents and 18 scientific publications which addressed recommendations for pediatric clinical trials were selected. The appraisal of these guidelines showed that the methods of research guideline development were poorly described. Areas of pediatric research that were addressed varied greatly and empirical evidence for recommendations was scarce. Most research guidelines are limited to "what one should aim to do" instead of "how to do it".

There is a need for readily accessible, clear guidelines on how to design, conduct and report clinical drug trials in children in a scientifically valid and ethical way. To enhance their acceptance, these guidelines should be developed using transparent methods with input from investigators, regulators, WHO, and the pharmaceutical industry. Parallel to their development attention should be paid to their active promotion, implementation, and evaluation.

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