6 papers
Assessing covariate-adjusted risk differences in small-sample clinical trials
Martin Schnuerch, Alex Ocampo, Klaus Kähler Holst +1
Binary endpoints are common in clinical trials and conditional odds ratios have traditionally been used to assess treatment effects. However, the interpretation of odds ratios is d…
Improving Variance Estimation for Covariate Adjustment with Binary Outcomes
Kaitlyn Lee, Alex Ocampo, Courtney Schiffman +3
Covariate adjustment is a general method for improving precision when estimating treatment effects in randomized trials and is recommended by the FDA in its 2023 guidance when base…
Unified implementation and comparison of Bayesian shrinkage methods for treatment effect estimation in subgroups
Marcel Wolbers, Miriam Pedrera Gómez, Alex Ocampo +1
Evaluating treatment effect heterogeneity across patient subgroups is a fundamental aspect of clinical trial analysis. Yet, these analyses have inherent limitations due to small sa…
Statistical Methodology Groups in the Pharmaceutical Industry
Jenny Devenport, Tobias Mielke, Mouna Akacha +10
Research and Development is the largest budget position in the pharmaceutical industry, with clinical trials being a critical, yet costly and time-consuming component to inform dec…
Revealing the Truth: Calculating True Values in Causal Inference Simulation Studies via Gaussian Quadrature
Alex Ocampo, Enrico Giudice, Zachary R. McCaw +1
Simulation studies are used to understand the properties of statistical methods. A key luxury in many simulation studies is knowledge of the true value (i.e. the estimand) being ta…
Simplifying Causal Mediation Analysis for Time-to-Event Outcomes using Pseudo-Values
Alex Ocampo, Enrico Giudice, Dieter A. Häring +3
Mediation analysis for survival outcomes is challenging. Most existing methods quantify the treatment effect using the hazard ratio (HR) and attempt to decompose the HR into the di…