Sample size derivation is a crucial element of planning any confirmatory trial. The required
sample size is typically derived based on constraints on the maximal acceptable Type I error …

In clinical trials, it is common to have multiple clinical outcomes (eg, coprimary endpoints or
a primary and multiple secondary endpoints). It is often desirable to establish efficacy in at …

There are several steps to confirming the safety and efficacy of a new medicine. A sequence
of trials, each with its own objectives, is usually required. Quantitative risk metrics can be …

In clinical trials with time‐to‐event data, the evaluation of treatment efficacy can be a long
and complex process, especially when considering long‐term primary endpoints. Using …

The identification of surrogate markers is motivated by their potential to make decisions
sooner about a treatment effect. However, few methods have been developed to actually …

Clinical trials often collect data on multiple outcomes, such as overall survival (OS),
progression-free survival (PFS), and response to treatment (RT). In most cases, however …

Background Go/no-go decisions after phase II and sample size chosen for phase III are
usually based on phase II results (eg, the treatment effect estimate of phase II). Due to the …

Receptor occupancy in targeted tissues measures the proportion of receptors occupied by a
drug at equilibrium and is sometimes used as a surrogate of drug efficacy to inform dose …

Quantitative decision-making (QDM) principles address the issues related to the mapping of
results to decisions, the synthesis of information and the quantification of uncertainty. Since …

To design a phase III study with a final endpoint and calculate the required sample size for
the desired probability of success, we need a good estimate of the treatment effect on the …