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

PLoS One. 2022 Dec 15;17(12):e0278842. doi: 10.1371/journal.pone.0278842. eCollection 2022.

Abstract

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.

Publication types

  • Observational Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Breast Neoplasms* / pathology
  • Female
  • Humans
  • Letrozole / therapeutic use
  • Piperazines / therapeutic use
  • Pyridines / therapeutic use
  • Receptor, ErbB-2* / metabolism
  • Receptors, Estrogen / metabolism

Substances

  • Receptor, ErbB-2
  • Letrozole
  • Receptors, Estrogen
  • Piperazines
  • Pyridines

Grants and funding

"We are graciously funded by a Sanofi iDEA award (https://www.sanofi.us/en/innovation-andscience/partnering-initiatives/sanofi-idea-awards). While the project was funded by Sanofi based on a proposal developed by investigators at Stanford, the work in the manuscript reflects a scientific collaboration between investigators at Stanford and Sanofi. Investigators at Stanford and Sanofi codesigned the study and analysis. Investigators at Stanford independently carried out the analysis and both Stanford and Sanofi authors contributed to the preparation of the manuscript. The decision to publish was not influenced by Sanofi."