Randomized trials often utilize a select group of study participants. This group does not typically represent the general population. Furthermore, sample sizes are often small to reduce cost. To improve power and generalizability, external control groups may be added to the randomized study. It is possible to incorporate a suitably selected external control group into a randomized clinical...
Randomised controlled trials (RCTs) are the gold standard of evidence to support causal conclusions on the benefits and risks of medicines in regulatory decision making along the lifecycle [1]. However, single-arm trials (SATs) are also frequently used for various reasons during drug development. While RCTs allow adjustment for confounding via design, the contextualization of SATs requires...
Bayesian dynamic borrowing (BDB) methods are popular for incorporating historical data in rare disease or paediatric clinical trials, in particular with regard to control groups. They can be used to leverage the historical information while mitigating the consequences of potential prior-data conflicts to some degree. However, these methods do not consider baseline covariate information that...
Drug development in the era of precision medicine increasingly uses basket trials and other multi-subgroup designs, where targeted therapies are evaluated across biomarker-defined patient subtrials. For many targeted agents and immunotherapies, the objective in early development is no longer the maximum tolerated dose (MTD), but the optimal biological dose (OBD) that achieves the best...
