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Cost-effectiveness in clinical trials: using multiple imputation to deal with incomplete cost data
Andrea Burton1, Lucinda Jane Billingham, Stirling Bryan
1Cancer Research UK Clinical Trials Unit, University of Birmingham, Birmingham, UK. andrea.burton@warwick.ac.uk.
Multiple imputation (MI) is recommended for cost-effectiveness analysis when patient costs are missing. Complete case analysis may yield biased results, impacting the assessment of chemotherapy cost-effectiveness in advanced non-small cell lung cancer.
Area of Science:
- Health Economics
- Clinical Trial Analysis
- Oncology
Background:
- Cost-effectiveness is crucial in clinical trials, necessitating resource use data collection.
- A Cancer Research UK trial for advanced non-small cell lung cancer compared chemotherapy (CT) with palliative care.
- Missing cost data for some patients in the trial required addressing data gaps.
Purpose of the Study:
- To evaluate the cost-effectiveness of chemotherapy versus palliative care in advanced non-small cell lung cancer.
- To address missing cost data in a clinical trial setting.
- To compare the results of multiple imputation with complete case analysis.
Main Methods:
- Multiple imputation (MI) was employed to estimate missing individual cost components.
- Total costs were calculated for all patients using imputed data.
- Cost-effectiveness was assessed for all patients and compared to a complete case analysis.
Main Results:
- MI indicated a high probability of CT being cost-effective (over £20,000 per life-year gained).
- Complete case analysis suggested CT was not cost-effective at any reasonable willingness to pay.
- MI results contrasted sharply with complete case analysis findings.
Conclusions:
- Cost-effectiveness analysis using only complete cases can produce biased outcomes.
- Multiple imputation is recommended for handling missing cost data in clinical trials.
- Accurate cost-effectiveness assessments require addressing missing data rigorously.
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