WHEN A RANDOMIZED CONTROLLED TRIAL IS NEEDED TO ASSESS DRUG SAFETY - THE CASE OF PEDIATRIC IBUPROFEN

Drugs are frequently made available for use before risks of rare but s erious reactions have been identified and quantified. While this situa tion may be acceptable for drugs used to treat serious conditions, gre ater information on safety is needed for drugs used to treat less seri ous conditions, and particularly those medications available without p rescription. Spontaneous reports and observational studies can provide useful data in most instances, but nonrandomised studies are inadequa te in the presence of confounding by indication (i.e. when patients tr eated with a drug differ in their underlying risk of adverse outcome f rom patients given alternate treatments, independent of the effect of the drug), Such is the case in the US with regard to the use of paedia tric ibuprofen as an antipyretic. In this setting, a rigorous and larg e randomised controlled trial is needed to provide valid and statistic ally stable risk estimates. A trial of this kind is a feasible way to develop clinically meaningful data on safety with respect to rare but serious adverse reactions.