Uncontrolled Extensions of Clinical Trials and the Use of External Controls - scoping opportunities and methods.
Sign Up to like & getrecommendations! Published in 2021 at "Clinical pharmacology and therapeutics"
DOI: 10.1002/cpt.2346
Abstract: Increased interest in real-world evidence (RWE) for clinical and regulatory decision making and the need to evaluate long-term benefits and risks of pharmaceutical products raise the importance of understanding the use of external controls (ECs)… read more here.
Keywords: extensions clinical; trials use; clinical trials; uncontrolled extensions ... See more keywords
Integrating external controls in case–control studies improves power for rare‐variant tests
Sign Up to like & getrecommendations! Published in 2022 at "Genetic Epidemiology"
DOI: 10.1002/gepi.22444
Abstract: Large‐scale sequencing and genotyping data provide an opportunity to integrate external samples as controls to improve power of association tests. However, due to the systematic differences between genotyped samples from different studies, naively aggregating the… read more here.
Keywords: rare variant; power; external controls; variant ... See more keywords
Use of quantitative bias analysis to evaluate single‐arm trials with real‐world data external controls
Sign Up to like & getrecommendations! Published in 2024 at "Pharmacoepidemiology and Drug Safety"
DOI: 10.1002/pds.5796
Abstract: Use of real‐world data (RWD) for external controls added to single‐arm trials (SAT) is increasingly prevalent in regulatory submissions. Due to inherent differences in the data‐generating mechanisms, biases can arise. This paper aims to illustrate… read more here.
Keywords: external controls; use quantitative; single arm; real world ... See more keywords
Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls
Sign Up to like & getrecommendations! Published in 2022 at "Neuromuscular Disorders"
DOI: 10.1016/j.nmd.2022.02.009
Abstract: Using external controls based on real-world or natural history data (RWD/NHD) for drug evaluations in Duchenne muscular dystrophy (DMD) is appealing given the challenges of enrolling placebo-controlled trials, especially for multi-year trials. Comparisons to external… read more here.
Keywords: placebo; drug; world natural; rwd nhd ... See more keywords
rdborrow: an R package for causal inference incorporating external controls in randomized controlled trials with longitudinal outcomes
Sign Up to like & getrecommendations! Published in 2025 at "Journal of Biopharmaceutical Statistics"
DOI: 10.1080/10543406.2025.2489283
Abstract: ABSTRACT Randomized controlled trials (RCTs) are considered the gold standard for treatment effect evaluation in clinical development. However, designing and analyzing RCTs poses many challenges such as how to ensure the validity and improve the… read more here.
Keywords: external controls; controlled trials; causal inference; randomized controlled ... See more keywords
ProxECAT: Proxy External Controls Association Test. A new case-control gene region association test using allele frequencies from public controls
Sign Up to like & getrecommendations! Published in 2018 at "PLoS Genetics"
DOI: 10.1371/journal.pgen.1007591
Abstract: A primary goal of the recent investment in sequencing is to detect novel genetic associations in health and disease improving the development of treatments and playing a critical role in precision medicine. While this investment… read more here.
Keywords: external controls; association test; case control; proxecat proxy ... See more keywords