Purpose: Few data on the efficacy and safety of drugs in children are available as in the past, these children were not included in randomized controlled trials (RCTs). Data on the efficacy and safety of drugs in children are extrapolated from adults. The EMA recognizes the need for long-term safety studies on various drugs, a need that can best be answered by pharmacoepidemiological studies. In this article, we provide currently available information on study designs within the field of paediatric drug research. Methods: A PubMed search was conducted on all pharmacoepidemiological studies in children. In addition, data from handbooks on pharmacoepidemiology were consulted. Data were reviewed and the relevant literature on study designs in paediatric pharmacoepidemiology is described. Results: The various study designs in pharmacoepidemiology have their specific indications, all with their specific limitations. Case reports and case series are mainly used for signal detection of safety issues whereas case control and cohort studies are used for safety hypothesis testing. Observational studies can be conducted using data from automated databases that guarantee large sample size and long-term follow-up, which is ideal for safety studies, especially in case of rare events. Conclusion: Pharmacoepidemiological studies are crucial in research on the safety of drugs in children. Knowledge of the different pharmacoepidemiological methods is important to guarantee optimal use and correct interpretation of the data.

Case-control, Children, Cohort, Effectiveness, Pharmacoepidemiology, Safety
dx.doi.org/10.1007/s00228-010-0930-2, hdl.handle.net/1765/21771
European Journal of Clinical Pharmacology
Article in press - dd December 2010
Erasmus MC: University Medical Center Rotterdam

Verhamme, K.M.C, & Sturkenboom, M.C.J.M. (2011). Study designs in paediatric pharmacoepidemiology. European Journal of Clinical Pharmacology (Vol. 67). doi:10.1007/s00228-010-0930-2