Integrating real world data and clinical trial results using survival data reconstruction and marginal moment-balancing weights

Getz, Kylie; Mamtani, Ronac; Hubbard, Rebecca A.

doi:10.1080/10543406.2021.1998097

Cited by 3 publications

(3 citation statements)

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“…By balancing patient characteristics between trial and routine-care populations, MAIC-weighted analyses can identify survival differences between the trial and routine-care settings that confounding may obscure. 9 In our analysis, we observed no difference in survival after applying MAIC weights, indicating the lack of survival differences in the unweighted analysis was not due to confounding by patient characteristics. The similar survival outcomes before and after weighting may also reflect the strict indication for ICI therapy, that is, for aUC patients unfit for standard platinum-chemotherapy.…”

Section: Resultsmentioning

confidence: 49%

“…In this study, no meaningful differences in ICI treatment outcomes were observed between the pivotal trial cohort and a U.S.‐based real‐world cohort (12‐, 24‐month survival probability: 0.47 vs. 0.45, 0.31 vs. 0.31, respectively) before or after adjusting for cohort differences using MAIC weighting. By balancing patient characteristics between trial and routine‐care populations, MAIC‐weighted analyses can identify survival differences between the trial and routine‐care settings that confounding may obscure 9 . In our analysis, we observed no difference in survival after applying MAIC weights, indicating the lack of survival differences in the unweighted analysis was not due to confounding by patient characteristics.…”

Section: Discussionmentioning

confidence: 56%

“…Median survival was 9 months (95% CI [7][8][9][10][11] in the unweighted EHR cohort, 9 months (95% CI 7-16) in the weighted EHR cohort, and 11.3 months (95% CI 9.7-13.1) in the trial cohort (Figure 1). The log-rank test of the difference in overall survival in the trial and EHR cohorts was not statistically significant.…”

Section: Resultsmentioning

confidence: 99%

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Comparing survival in trial‐ versus routine‐care advanced urothelial cancer patients on immune checkpoint blockade

Wang,

Hubbard,

Mamtani

2024

Pharmacoepidemiology and Drug

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PurposeAlthough recent trials involving first‐line immune checkpoint inhibitors have expanded treatment options for patients with advanced urothelial carcinoma (aUC) who are ineligible for standard cisplatin‐based chemotherapy, there exists limited evidence for whether trial efficacy translates into real‐world effectiveness for patients seen in routine care. This retrospective cohort study compares differences in overall survival (OS) between KEYNOTE‐052 trial participants and routine‐care patients receiving first‐line pembrolizumab monotherapy.MethodsA routine‐care patient cohort was constructed from the Flatiron Health database using trial eligibility criteria and was weighted to balance EHR and trial patient characteristics using matching‐adjusted indirect comparisons.ResultsThe routine‐care cohort was older, more likely to be female, and more often cisplatin‐ineligible due to renal dysfunction. ECOG performance status was comparable between the cohorts. Median OS was 9 months (95% CI 7–16) in the weighted routine‐care cohort and 11.3 months (9.7–13.1) in the trial cohort. No significant differences between the Kaplan–Meier OS curves were detected (p = 0.76). Survival probabilities were similar between the weighted routine‐care and trial cohorts at 12‐, 24‐, and 36‐ months (0.45 vs. 0.47, 0.31 vs. 0.31, 0.26 vs. 0.23, respectively). Notably, routine care patients had modestly lower survival at 3 months compared to trial participants (0.69 vs. 0.83, respectively).ConclusionOur results provide reassurance that cisplatin‐ineligible aUC patients receiving first‐line immunotherapy in routine care experience similar benefits to those observed in trial patients.

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Section: Resultsmentioning

confidence: 49%

Section: Discussionmentioning

confidence: 56%