Concepedia

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

Francesco Muntoni, James Signorovitch, Gautam Sajeev, Nathalie Goemans, Brenda Wong, Cuixia Tian, Eugenio Mercuri, Nicolae Done, Hallee E. Wong, Jackson Moss,

Neuromuscular Disorders · 2022 · 36 citations · 32 references

Open access

References

32

An Introduction to Propensity Score Methods for Reducing the Effects of Confounding in Observational Studies

Peter C. Austin · Multivariate Behavioral Research · 2011 · 11.2K citations · Full text

Treatment Effect, Quasi-experiment, Causal Inference +21

Models for Longitudinal Data: A Generalized Estimating Equation Approach

Scott L. Zeger, Kung‐Yee Liang, Paul S. Albert · Biometrics · 1988 · 4.5K citations

Tobacco Control, Generalized Linear Models, Longitudinal Data Analysis +12

Constructing a Control Group Using Multivariate Matched Sampling Methods That Incorporate the Propensity Score

Paul R. Rosenbaum, Donald B. Rubin · The American Statistician · 1985 · 4.2K citations

Treatment Effect, Causal Inference, Developmental Psychology +19

Optimal caliper widths for propensity‐score matching when estimating differences in means and differences in proportions in observational studies

Peter C. Austin · Pharmaceutical Statistics · 2010 · 3.7K citations · Full text

Treatment Effect, Propensity-score Matching, Quasi-experiment +16

Real-World Evidence — What Is It and What Can It Tell Us?

Rachel E. Sherman, Steven A. Anderson, Gerald J. Dal Pan et al. · New England Journal of Medicine · 2016 · 1.9K citations

Healthcare Monitoring Systems, Engineering, Ehr Systems +24