Standards for clinical trials in sexual dysfunction in women: research designs and outcomes assessment.

INTRODUCTION Clinical trial design in female sexual dysfunction (FSD) is an evolving science, with some areas of controversy. AIM To develop an evidence-based, expert consensus-report on design of FSD clinical research. METHODS Literature review including the Food and Drug Administration (FDA) clinical trial guidelines with critique by six experts from three countries, modified after public presentation and debate. MAIN OUTCOME MEASURE Expert opinion and recommendations were based on grading of evidence based literature, internal committee dialogue, open presentation, and debate. RESULTS Design of clinical research for regulatory approval is driven by FDA guidelines. Diagnostic and Statistical Manual-IV definitions and consideration of comorbidity of sexual disorders may complicate patient selection and outcomes. Measures for study end points include satisfying sexual events utilizing a daily diary, sexual distress, and patient-reported outcomes measures of the construct under study. Currently, trial duration is recommended to be 6 months for efficacy trials to allow for modification of behavioral adaptations to changes in desire. Important issues include safety assessments, generalizability, having a representative study population, stratification by reproductive status, partner assessment, contextual and interpersonal factors, symptom duration and severity, management of placebo response, and drug dosing. Statistical analysis should include assessment of change from baseline to end point between study drug and placebo, determination of statistically significant change vs. clinically meaningful effects, linear mapping of all measures of the same construct, and determination of responders and remitters. CONCLUSIONS Future trials should include clear population definitions, direct and indirect measures of the specific FSD construct, and procedures to allow generalizability of diagnosis and treatment to the target population.