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Noninferiority testing in cost-minimization studies: Practical issues concerning power analysis.
Mark M Span1, Elisabeth M TenVergert, Christian S van der Hilst
1Office of Medical Technology Assessment, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands. M.M.Span@rug.nl
Summary
For cost-minimization studies, defining the equivalence limit (delta) as an absolute difference may obscure results. Using a proportional delta is more aligned with clinical practice and avoids inflated values, ensuring valid noninferiority testing.
Area of Science:
- Biostatistics
- Health Economics
- Clinical Trial Design
Background:
- Cost-minimization studies require establishing noninferiority of clinical effects.
- Dichotomous clinical endpoints are common in these studies.
- The equivalence limit (delta) and statistical power are critical factors.
Purpose of the Study:
- To investigate the relationship between endpoint proportions, delta, and power in cost-minimization studies.
- To explore absolute and relative formulations of delta in hypothesis testing.
- To provide guidance on appropriate noninferiority testing methodologies.
Main Methods:
- Sensitivity analysis was conducted to assess the impact of predicted proportions and delta on power.
- The study examined different patterns of results based on the formulation of delta.
- Practical relevance within the cost-minimization framework was discussed.
Main Results:
- Sensitivity analyses revealed distinct patterns depending on the null-hypothesis formulation.
- Expressing delta as an absolute difference led to rapid power increases with smaller-than-expected sample proportions.
- Using a proportional delta resulted in persistently low power at small sample proportions.
Conclusions:
- Accurate methodology for noninferiority testing is crucial for valid cost-minimization study results.
- Defining delta using absolute differences can obscure outcomes.
- Expressing delta as a proportion of usual treatment effectiveness is more clinically relevant and avoids inflated delta values.
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