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A Cautionary Tale on Integrating Studies with Disparate Outcome Measures for Causal Inference

arXiv 2505.11014 ↗First posted 16 May 2025stat.ME cs.LG econ.EMcited by 0

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Abstract (arXiv)

Data integration approaches are increasingly used to enhance the efficiency and generalizability of studies. However, a key limitation of these methods is the assumption that outcome measures are identical across datasets -- an assumption that often does not hold in practice. Consider the following opioid use disorder (OUD) studies: the XBOT trial and the POAT study, both evaluating the effect of medications for OUD on withdrawal symptom severity (not the primary outcome of either trial). While XBOT measures withdrawal severity using the subjective opiate withdrawal scale, POAT uses the clinical opiate withdrawal scale. We analyze this realistic yet challenging setting where outcome measures differ across studies and where neither study records both types of outcomes. Our paper studies whether and when integrating studies with disparate outcome measures leads to efficiency gains. We introduce three sets of assumptions -- with varying degrees of strength -- linking both outcome measures. Our theoretical and empirical results highlight a cautionary tale: integration can improve asymptotic efficiency only under the strongest assumption linking the outcomes. However, misspecification of this assumption leads to bias. In contrast, a milder assumption may yield finite-sample efficiency gains, yet these benefits diminish as sample size increases. We illustrate these trade-offs via a case study integrating the XBOT and POAT datasets to estimate the comparative effect of two medications for opioid use disorder on withdrawal symptoms. By systematically varying the assumptions linking the SOW and COW scales, we show potential efficiency gains and the risks of bias. Our findings emphasize the need for careful assumption selection when fusing datasets with differing outcome measures, offering guidance for researchers navigating this common challenge in modern data integration.

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References · 14

  1. 2021Discussion on "Bayesian Regression Tree Models for Causal Inference: Regularization, Confounding, and Heterogeneous Effects" by Hahn, Murray and Carvalhocited by 77
  2. 2018Removing Hidden Confounding by Experimental Groundingcited by 64
  3. 2021A Review of Generalizability and Transportabilitycited by 37
  4. 2015The Influence Function of Semiparametric Estimatorscited by 36
  5. 2018Combining multiple observational data sources to estimate causal effectscited by 29
  6. 2020Combining Observational and Experimental Datasets Using Shrinkage Estimatorscited by 28
  7. 2023Methods for Integrating Trials and Non-Experimental Data to Examine Treatment Effect Heterogeneitycited by 10
  8. 2022Combining Experimental and Observational Data for Identification and Estimation of Long-Term Causal Effectscited by 7
  9. 2023Prognostic Adjustment with Efficient Estimators to Unbiasedly Leverage Historical Data in Randomized Trialscited by 6
  10. 2024Who Are We Missing? A Principled Approach to Characterizing the Underrepresented Populationcited by 6
  11. 2021Efficient Estimation Under Data Fusioncited by 4
  12. 2024Towards Generalizing Inferences from Trials to Target Populationscited by 4
2 more
  1. 2023Combining experimental and observational data through a power likelihoodcited by 3
  2. 2023Improving efficiency in transporting average treatment effectscited by 2

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