7 citations · 13 across the 4 of their papers we have counts for
4 papers
Applying the Estimand and Target Trial frameworks to external control analyses using observational data: a case study in the solid tumor setting
Letizia Polito, Qixing Liang, Navdeep Pal +5
In causal inference, the correct formulation of the scientific question of interest is a crucial step. Here we apply the estimand framework to a comparison of the outcomes of patie…
Weighted Approach for Estimating Effects in Principal Strata with Missing Data for a Categorical Post-Baseline Variable in Randomized Controlled Trials
Shengchun Kong, Dominik Heinzmann, Sabine Lauer +1
This research was motivated by studying anti-drug antibody (ADA) formation and its potential impact on long-term benefit of a biologic treatment in a randomized controlled trial, i…
A pragmatic adaptive enrichment design for selecting the right target population for cancer immunotherapies
Anh Nguyen Duc, Dominik Heinzmann, Claude Berge +1
One of the challenges in the design of confirmatory trials is to deal with uncertainties regarding the optimal target population for a novel drug. Adaptive enrichment designs (AED)…
Integrating Phase 2 into Phase 3 based on an Intermediate Endpoint While Accounting for a Cure Proportion -- with an Application to the Design of a Clinical Trial in Acute Myeloid Leukemia
Kaspar Rufibach, Dominik Heinzmann, Annabelle Monnet
For a trial with primary endpoint overall survival for a molecule with curative potential, statistical methods that rely on the proportional hazards assumption may underestimate th…