RESUMO
BACKGROUND: Assessment of symptoms or disease improvement by study participants is an important aspect of assessing new dermatological therapies in clinical trials, especially for chronic skin diseases that lack objective severity markers. OBJECTIVES: We sought to determine the frequency and prominence of reporting of participants' subjective efficacy outcomes in dermatological clinical trials. Our secondary objective was to determine whether participant and physician outcomes agree in terms of direction and magnitude. METHODS: Systematic review of 125 randomized controlled trials identified from the Archives of Dermatology, British Journal of Dermatology, Clinical & Experimental Dermatology, Journal of Dermatological Treatment and Journal of the American Academy of Dermatology published between 1994 and 2001 (25 from each). Studies were retrieved in hard copy from the Cochrane Skin Group specialized register of trials and data were abstracted and summarized. RESULTS: Participant efficacy outcomes were mentioned in some form in only 32 of 125 trials (25.6%, 95% exact confidence interval 18.2-34.2%). Of these 32 studies, participant outcomes were mentioned only in the methods section in two studies, in the methods and results section without further data in nine studies and with further data in 21. Data were presented in figure format only in 12 of these studies and in tables and figures in nine. Participant efficacy outcomes were mentioned in the abstract section in just over half (53%) of the 32 trials that included participant efficacy outcomes. There was not enough information to assess agreement in direction and magnitude of participant vs. assessor outcomes. Overall, only 17 papers (13.6%) clearly declared their main outcome measures beforehand in the introduction or methods section. CONCLUSIONS: Asking study participants for their views of treatment efficacy seems like a good idea in dermatological clinical trials, yet only about a quarter of the trials examined in this review did so. Even when such information was recorded, it was often poorly and incompletely reported and given low prominence within the trial report. Our study findings call for a more comprehensive uptake for including participant efficacy outcomes alongside other assessor outcomes in clinical trials and, when included, to report those outcomes in full.
