Study type

Study topic

Other

Study topic, other

Disease/Epidemiology study

Study type

Not applicable

If ‘Not applicable’, further details on the study type

Statistical methodology study

If ‘other’, further details on the scope of the study

A statistical methodology study to evaluate External Comparator Arm (ECA) study results versus Randomised Controlled Trial (RCT) results
Population studied

Short description of the study population

N/A

Age groups

Adults (18 to < 46 years)
Adults (46 to < 65 years)
Adults (65 to < 75 years)
Adults (75 to < 85 years)
Adults (85 years and over)

Estimated number of subjects

0
Study design details

Main study objective

The aim of this study is to evaluate External Comparator Arm (ECA) study results versus RCT results and evaluate statistical methods and potential sources of bias in the oncology therapeutic area.

Data analysis plan

The primary analyses from the selected RCTs will be re-derived following the analysis specified in their respective study protocols and statistical analysis plans (SAPs). To emulate an ECA study, the data from the experimental arm of the RCT will then be used as if it was a SAT where it will be analysed together with the RWD using propensity score methods, such as inverse probability of treatment weighting, to account for the imbalance of potential confounders between the experimental treatment arm of the RCT and the external control arm. As it is expected that the RWD has a higher degree of missingness, multiple imputation will be used to derive a complete set of baseline covariates for each patient. Sensitivity analyses will be conducted to assess the robustness of the findings. In addition, simulation studies will be performed using realistic assumptions based on the selected RCTs and RWD sources.

Summary results

See link to publication (Study publications section).
Documents
Study publications
Rippin G, Sanz H, Hoogendoorn WE, Ballarini NM, Largent JA, Demas E, Postmus D,…