Estimation of treatment effect among treatment responders with a time-to-event endpoint

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Standard

Estimation of treatment effect among treatment responders with a time-to-event endpoint. / Nordland, Andreas; Martinussen, Torben.

In: Scandinavian Journal of Statistics, 2024.

Research output: Contribution to journal › Journal article › Research › peer-review

Harvard

Nordland, A & Martinussen, T 2024, 'Estimation of treatment effect among treatment responders with a time-to-event endpoint', Scandinavian Journal of Statistics. https://doi.org/10.1111/sjos.12706

APA

Nordland, A., & Martinussen, T. (2024). Estimation of treatment effect among treatment responders with a time-to-event endpoint. Scandinavian Journal of Statistics. https://doi.org/10.1111/sjos.12706

Vancouver

Nordland A, Martinussen T. Estimation of treatment effect among treatment responders with a time-to-event endpoint. Scandinavian Journal of Statistics. 2024. https://doi.org/10.1111/sjos.12706

Author

Nordland, Andreas ; Martinussen, Torben. / Estimation of treatment effect among treatment responders with a time-to-event endpoint. In: Scandinavian Journal of Statistics. 2024.

Bibtex

@article{e0f5f10d4b964e4f830d6f9d8b76859c,
title = "Estimation of treatment effect among treatment responders with a time-to-event endpoint",
abstract = "In a placebo-controlled clinical study one may calculate the average treatment effect to convey the effect of the active treatment on some outcome. However, if it is speculated that the treatment only has an effect if the patient responds to the treatment defined by a certain biomarker response, then it is arguably more relevant to estimate the treatment effect among such responders. We present such a causal parameter that is based on principal stratification and is identified under the exclusion of a treatment effect among the non-responders. We focus on time-;to-event outcomes allowing for right censoring, and construct a doubly robust and efficient estimator based on the associated efficient influence function. The properties of the estimator are showcased in a simulation study and the methodology is applied to the Leader trial investigating the effect of liraglutide on the occurrence of cardiovascular events.",
author = "Andreas Nordland and Torben Martinussen",
year = "2024",
doi = "10.1111/sjos.12706",
language = "English",
journal = "Scandinavian Journal of Statistics",
issn = "0303-6898",
publisher = "Wiley-Blackwell",

}

RIS

TY - JOUR

T1 - Estimation of treatment effect among treatment responders with a time-to-event endpoint

AU - Nordland, Andreas

AU - Martinussen, Torben

PY - 2024

Y1 - 2024

N2 - In a placebo-controlled clinical study one may calculate the average treatment effect to convey the effect of the active treatment on some outcome. However, if it is speculated that the treatment only has an effect if the patient responds to the treatment defined by a certain biomarker response, then it is arguably more relevant to estimate the treatment effect among such responders. We present such a causal parameter that is based on principal stratification and is identified under the exclusion of a treatment effect among the non-responders. We focus on time-;to-event outcomes allowing for right censoring, and construct a doubly robust and efficient estimator based on the associated efficient influence function. The properties of the estimator are showcased in a simulation study and the methodology is applied to the Leader trial investigating the effect of liraglutide on the occurrence of cardiovascular events.

AB - In a placebo-controlled clinical study one may calculate the average treatment effect to convey the effect of the active treatment on some outcome. However, if it is speculated that the treatment only has an effect if the patient responds to the treatment defined by a certain biomarker response, then it is arguably more relevant to estimate the treatment effect among such responders. We present such a causal parameter that is based on principal stratification and is identified under the exclusion of a treatment effect among the non-responders. We focus on time-;to-event outcomes allowing for right censoring, and construct a doubly robust and efficient estimator based on the associated efficient influence function. The properties of the estimator are showcased in a simulation study and the methodology is applied to the Leader trial investigating the effect of liraglutide on the occurrence of cardiovascular events.

U2 - 10.1111/sjos.12706

DO - 10.1111/sjos.12706

M3 - Journal article

JO - Scandinavian Journal of Statistics

JF - Scandinavian Journal of Statistics

SN - 0303-6898

ER -

ID: 381151751