Clinical trials (CT) are the ultimate test of the development hypothesis that conditions can be identified under which a novel medicinal product can be shown to be a safe and efficacious treatment option.
Therefore, CTs and the documentation of their orderly planning, conduct, analysis, and reporting are essential in the regulatory profiling of new drug products and substances.
CT expose trial participants to risks and inconvenience with little (while time-limited) or no (while placebo-treated) individual medication-related benefit; this constraint is particularly obvious in early clinical development when investigating healthy subjects or investigating patients with out-of-scope doses.
Therefore, a favourable benefit:risk-relation requires minimising individual risk and maximising group-ethical benefit (for the future treatment of other patients).
The need to optimise and survey the benefit:risk-relation of CT is reflected by mandatory risk-based quality standards of orderly trial conduct (WMA Declaration of Helsinki & GCP) and the public sanctioning of the compliance with these standards by competent authorities and ethics committees.