There has been a rapid expansion in the use of non-randomized evidence in the regulatory
approval of treatments globally. An emerging set of methodologies have been utilized to …

Highlights•Considerations for the use of external controls in oncology clinical trials are
multifaceted and depend on the intended use.•Review of recent research and some …

Platform trials are a type of randomized clinical trial that allow simultaneous comparison of
multiple intervention groups against a single control group that serves as a common control …

Purpose: Combinations of immune-checkpoint inhibitors (ICI) with other cancer therapies
have been approved for advanced cancers in multiple indications, and numerous trials are …

The pharmaceutical industry and its global regulators have routinely used frequentist
statistical methods, such as null hypothesis significance testing and p values, for evaluation …

Pulmonary fibrosis is a pathologic process associated with scarring of the lung interstitium.
Interstitial lung diseases (ILDs) encompass a large and heterogenous group of disorders, a …

For the past decade, it has become commonplace to provide rapid answers and early
patient access to innovative treatments in the absence of randomized clinical trials (RCT) …

Background One of the greatest challenges in the current IBD clinical trial landscape is,
perhaps, the recruitment and retention of eligible participants. Seamless testing of promising …

Randomized controlled trials (RCTs) are the gold standard design to establish the efficacy of
new drugs and to support regulatory decision making. However, a marked increase in the …

Objectives External control arms (ECAs) provide useful comparisons in clinical trials when
randomised control arms are limited or not feasible. We conducted a systematic review to …