Abstract
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.
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.
| Original language | English |
|---|---|
| Number of pages | 37 |
| Publication status | Published - Apr 2009 |
| Externally published | Yes |