Is Individual-Patient Data Essential for Transportability, or Can Aggregated Data Suffice?
Author(s)
Thuresson PO1, Mpofu P2, Sanglier T3, Tchakoute C4, Horne E5, Pittell H2, Zhang Q2, Clunie-O'Connor C5, Sujenthiran A5, Adamson B2
1F. Hoffmann-La Roche Ltd, Basel, BS, Switzerland, 2Flatiron Health, New York, NY, USA, 3F. Hoffmann-La Roche Ltd, Basel, Switzerland, 4Roche/Genentech Inc., South San Francisco, CA, USA, 5Flatiron Health UK, London, UK
OBJECTIVES: When assessing evidence transportability between two settings, individual patient-level data (IPD) are usually available in only one. As a result, researchers use matching-adjusted indirect comparison (MAIC) to perform population adjustment. The presence of IPD in both settings provides a valuable opportunity to evaluate the extent to which MAIC weighting preserves the results from using IPD-based inverse-odds weighting (IO).
METHODS: We conducted a transportability study to compare the overall survival of individuals with HER2+ metastatic breast cancer (mBC) in the UK and the US between 2014 to 2023. In this study, IPD from the UK were obtained from the ESTHER registry and represented the target population. IPD from the US came from an EHR-derived de-identified mBC database and represented the study population. We performed population adjustment using inverse odds weighting and MAIC after summarizing UK IPD into aggregated data (AD).
RESULTS: The US cohort consisted of 907 individuals with a median age of 61 years, while the UK cohort included 248 individuals with a median age of 57. The restricted mean survival time (RMST) at 60 months was as follows: 41.7 months (se: 1.3) for the UK cohort; 40.0 months (se: 0.7) for the unweighted US cohort; 41.5 months (se, 0.8) for the US cohort after IO; and 41.3 months (se: 1.1) for the US cohort after MAIC. Compared to the UK, the hazard ratio estimates were 1.06 (0.88, 1.27) for the unweighted US cohort; 0.98 (0.79, 1.20) IO-weighted US cohort, and 1.00 (0.79, 1.27) for MAIC-weighted US cohort.
CONCLUSIONS: The point estimates obtained from population adjustment using MAIC were similar to those from IO. However, as expected, the MAIC estimates showed slightly greater variability. These findings provide support for using aggregated data to assess evidence transportability when individual patient-level data are unavailable for people with HER2+ mBC.
Conference/Value in Health Info
Value in Health, Volume 27, Issue 12, S2 (December 2024)
Code
RWD68
Topic
Organizational Practices, Real World Data & Information Systems
Topic Subcategory
Reproducibility & Replicability
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology