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024 7 _ |a 10.1002/bimj.70028
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024 7 _ |a 0323-3847
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024 7 _ |a 1521-4036
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037 _ _ |a DKFZ-2024-02737
041 _ _ |a English
082 _ _ |a 570
100 1 _ |a Edelmann, Dominic
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245 _ _ |a The Progression-Free-Survival Ratio in Molecularly Aided Tumor Trials: A Critical Examination of Current Practice and Suggestions for Alternative Methods.
260 _ _ |a Berlin
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|b Wiley-VCH
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520 _ _ |a The progression-free-survival ratio is a popular endpoint in oncology trials, which is frequently applied to evaluate the efficacy of molecularly targeted treatments in late-stage patients. Using elementary calculations and simulations, numerous shortcomings of the current methodology are pointed out. As a remedy to these shortcomings, an alternative methodology is proposed, using a marginal Cox model or a marginal accelerated failure time model for clustered time-to-event data. Using comprehensive simulations, it is shown that this methodology outperforms existing methods in settings where the intrapatient correlation is low to moderate. The performance of the model is further demonstrated in a real data example from a molecularly aided tumor trial. Sample size considerations are discussed.
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650 _ 7 |a Weibull distribution
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650 _ 7 |a accelerated failure time model
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650 _ 7 |a growth modulation index
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650 _ 7 |a marginal model
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650 _ 7 |a paired time‐to‐event data
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650 _ 7 |a progression‐free‐survival ratio
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650 _ 2 |a Humans
|2 MeSH
650 _ 2 |a Biometry: methods
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650 _ 2 |a Neoplasms: mortality
|2 MeSH
650 _ 2 |a Clinical Trials as Topic: methods
|2 MeSH
650 _ 2 |a Progression-Free Survival
|2 MeSH
650 _ 2 |a Molecular Targeted Therapy: methods
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650 _ 2 |a Models, Statistical
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650 _ 2 |a Proportional Hazards Models
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700 1 _ |a Terzer, Tobias
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700 1 _ |a Horak, Peter
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700 1 _ |a Schlenk, Richard
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700 1 _ |a Benner, Axel
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773 _ _ |a 10.1002/bimj.70028
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|t Biometrical journal
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