Guidance for a causal comparative effectiveness analysis emulating a target trial based on big real world evidence: when to start statin treatment

Kuehne, Felicitas; Jahn, Beate; Conrads-Frank, Annette; Bundo, Marvin; Arvandi, Marjan; Endel, Florian; Popper, Niki; Endel, Gottfried; Urach, Christoph; Gyimesi, Michael; Murray, Eleanor; Danaei, Goodarz; Gaziano, Thomas A.; Pandya, Ankur; Siebert, Uwe

doi:10.2217/cer-2018-0103

Cited by 9 publications

(2 citation statements)

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“…With such data, causal inference methods could be used to derive causal statements, even in the presence of time-varying confounding [31][32][33] . Such longitudinal observational studies should be analyzed according to causal principles 34 .…”

Section: Discussionmentioning

confidence: 99%

Health-related quality of life, work productivity and costs related to patients with inflammatory bowel disease in Austria

Walter

Hausberger

Groß³

et al. 2020

Journal of Medical Economics

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Objective: Inflammatory-Bowel-Disease (IBD) is a lifelong illness with significant impact on health-related quality of life (HRQoL). The disease-burden causes work productivity impairment, such as sick-leave and restriction of leisure time activities. From a societal perspective, productivity loss often contributes significantly to the total costs. The aim of the study is to analyze the impact of disease-burden on work productivity, daily activities, and HRQoL. Methods: We performed a survey among Austrian patients with IBD to assess general questions about demography and disease pattern, impaired work productivity and reduced daily activities using the Work Productivity and Activity Impairment questionnaire and HRQoL utilizing the Short Inflammatory Bowel Disease Questionnaire (SIBDQ). Associations were assessed employing linear regressions. The questionnaire was circulated by the Austrian Crohn's Disease/Ulcerative Colitis Association. Results: In total, 510 patients with median age of 40 years completed the questionnaire. Patients had a median disease-duration of 9 years, 46% were treated with biologics and 64% were employed. The median SIBDQ score was 45 (16-69). Patients in self-assessed remission (n ¼ 319) and relapse (n ¼ 96) differed by 18 points on the SIBDQ score (p value < 0.0001). Mean work productivity reduction amounted to 7 h 17 min (95% CI 6:05-8:29) during the last 7 days. Work absenteeism accounted for 4 h 28 min (95% CI 3:25-5:31) and reduced productivity at the workplace (presenteeism) for 2 h 49 min (95% CI 2:31-3:07). During a relapse, 12 h 9 min were lost (p value < 0.0001). Productivity reduction correlated with HRQoL (r ¼ 0.3964). In total, 143 eper patient per week (95% CI 119-167) were lost (7,411 eper year). On average, patients lost 4 h (95% CI 3:44-4:16) of unpaid daily activities; 3 h 17 min the employed subgroup and 5 h 19 min the not employed. Conclusion: IBD leads to a substantial burden of disease, which is associated with indirect costs for society related to work ability reduction. HRQoL significantly influences loss of work productivity. Improving HRQoL should be a key factor to reduce disease burden and societal costs.

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Section: Discussionmentioning

confidence: 99%

Health-related quality of life, work productivity and costs related to patients with inflammatory bowel disease in Austria

Walter

Hausberger

Groß³

et al. 2020

Journal of Medical Economics

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“…17,75 Causal inference methods such as the g-methods exist that try to show causality rather than correlations and may be applicable in this setting. 21,23,25,[76][77][78][79][80][81][82][83][84][85][86][87][88][89][90][91][92] An important implication for the study design and planning of data collection is the fact that all these methods need detailed and valid information on the reasons for switching to control for the switching bias. Our analysis shows a novel decision-analytic approach in controlling for the switching bias even with limited information on switching behavior by integrating evidence from different sources.…”

Section: Discussionmentioning

confidence: 99%

Estimating Treatment-Switching Bias in a Randomized Clinical Trial of Ovarian Cancer Treatment: Combining Causal Inference with Decision-Analytic Modeling

et al. 2021

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Background Bevacizumab is efficacious in delaying ovarian cancer progression and controlling ascites. The ICON7 trial showed a significant benefit in overall survival for bevacizumab, whereas the GOG-218 trial did not. GOG-218 allowed control group patients to switch to bevacizumab upon progression, which may have biased the results. Lack of data on switching behavior prevented the application of g-methods to adjust for switching. The objective of this study was to apply decision-analytic modeling to estimate the impact of switching bias on causal treatment-effect estimates. Methods We developed a causal decision-analytic Markov model (CDAMM) to emulate the GOG-218 trial and estimate overall survival. CDAMM input parameters were based on data from randomized clinical trials and the published literature. Overall switching proportion was based on GOG-218 trial information, whereas the proportion switching with and without ascites was estimated using calibration. We estimated the counterfactual treatment effect that would have been observed had no switching occurred by denying switching in the CDAMM. Results The survival curves generated by the CDAMM matched well with the ones reported in the GOG-218 trial. The survival curve correcting for switching showed an estimated bias such that 79% of the true treatment effect could not be observed in the GOG-218 trial. Results were most sensitive to changes in the proportion progressing with severe ascites and mortality. Limitations We used a simplified model structure and based model parameters on published data and assumptions. Robustness of the CDAMM was tested and model assumptions transparently reported. Conclusions Medical-decision science methods may be merged with empirical methods of causal inference to integrate data from other sources where empirical data are not sufficient. We recommend collecting sufficient information on switching behavior when switching cannot be avoided.

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The role of causal inference in health services research II: a framework for causal inference

Moser

Puhan

Zwahlen

2020

Int J Public Health

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Guidance for a causal comparative effectiveness analysis emulating a target trial based on big real world evidence: when to start statin treatment

Cited by 9 publications

References 50 publications

Health-related quality of life, work productivity and costs related to patients with inflammatory bowel disease in Austria

Health-related quality of life, work productivity and costs related to patients with inflammatory bowel disease in Austria

Estimating Treatment-Switching Bias in a Randomized Clinical Trial of Ovarian Cancer Treatment: Combining Causal Inference with Decision-Analytic Modeling

The role of causal inference in health services research II: a framework for causal inference

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