Journal Article

Transporting observational study results to a target population of interest using inverse odds of participation weighting

Jreich, Rana, Desai, Manisha, Meng, Zhaoling, Chandross, Karen J., Kapphahn, Kris, Ling, Albee Y., Montez-Rath, Maria E.

2022

Overview

Inverse odds of participation weighting (IOPW) has been proposed to transport clinical trial findings to target populations of interest when the distribution of treatment effect modifiers differs between trial and target populations. We set out to apply IOPW to transport results from an observational study to a target population of interest. We demonstrated the feasibility of this idea with a real-world example using a nationwide electronic health record derived de-identified database from Flatiron Health. First, we conducted an observational study that carefully adjusted for confounding to estimate the treatment effect of fulvestrant plus palbociclib relative to letrozole plus palbociclib as a second-line therapy among estrogen receptor (ER)-positive, human epidermal growth factor receptor (HER2)-negative metastatic breast cancer patients. Second, we transported these findings to the broader cohort of patients who were eligible for a first-line therapy. The interpretation of the findings and validity of such studies, however, rely on the extent that causal inference assumptions are met.

• Publisher: Public Library of Science,Public Library of Science (PLoS)
• Subject: Antineoplastic Combined Chemotherapy Protocols - therapeutic use / Bias / Breast cancer / Breast Neoplasms - pathology / Cancer therapies / Clinical trials / Data entry / Decision-making / Electronic health records / Electronic medical records / Epidermal growth factor / ErbB-2 protein / Estrogen receptors / Estrogens / Evaluation / Female / Fulvestrant / Growth factors / Health aspects / Humans / Information management / Letrozole - therapeutic use / Medical diagnosis / Medical practices / Medicine and Health Sciences / Metastases / Metastasis / Missing data / Observational studies / Patients / Piperazines - therapeutic use / Population studies / Populations / Pyridines - therapeutic use / Receptor, ErbB-2 - metabolism / Receptors / Receptors, Estrogen - metabolism / Regulatory approval / Research and Analysis Methods / Weighting