The effect of estimating prevalences on the population-wise error rate
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arXiv
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| Format: | Preprint |
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2023
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| _version_ | 1866912152539889664 |
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| author | Luschei, Remi Brannath, Werner |
| author_facet | Luschei, Remi Brannath, Werner |
| contents | The population-wise error rate (PWER) is a type I error rate for clinical trials with multiple target populations. In such trials, a treatment is tested for its efficacy in each population. The PWER is defined as the probability that a randomly selected, future patient will be exposed to an inefficient treatment based on the study results. It can be understood and computed as an average of strata-specific family wise error rates and involves the prevalences of these strata. A major issue of this concept is that the prevalences are usually unknown in practice, so that the PWER cannot be directly controlled. Instead, one could use an estimator based on the given sample, like their maximum-likelihood estimator under a multinomial distribution. In this article, we demonstrate through simulations that this does not substantially inflate the true PWER. We differentiate between the expected PWER, which is almost perfectly controlled, and study-specific values of the PWER which are conditioned on all subgroup sample sizes and vary within a narrow range. Thereby, we consider up to eight different overlapping populations and moderate to large sample sizes. In these settings, we also consider the maximum strata-wise family wise error rate, which is found to be, on average, at least bounded by twice the significance level used for PWER control. |
| format | Preprint |
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arxiv_https___arxiv_org_abs_2304_09988 |
| institution | arXiv |
| publishDate | 2023 |
| record_format | arxiv |
| spellingShingle | The effect of estimating prevalences on the population-wise error rate Luschei, Remi Brannath, Werner Methodology The population-wise error rate (PWER) is a type I error rate for clinical trials with multiple target populations. In such trials, a treatment is tested for its efficacy in each population. The PWER is defined as the probability that a randomly selected, future patient will be exposed to an inefficient treatment based on the study results. It can be understood and computed as an average of strata-specific family wise error rates and involves the prevalences of these strata. A major issue of this concept is that the prevalences are usually unknown in practice, so that the PWER cannot be directly controlled. Instead, one could use an estimator based on the given sample, like their maximum-likelihood estimator under a multinomial distribution. In this article, we demonstrate through simulations that this does not substantially inflate the true PWER. We differentiate between the expected PWER, which is almost perfectly controlled, and study-specific values of the PWER which are conditioned on all subgroup sample sizes and vary within a narrow range. Thereby, we consider up to eight different overlapping populations and moderate to large sample sizes. In these settings, we also consider the maximum strata-wise family wise error rate, which is found to be, on average, at least bounded by twice the significance level used for PWER control. |
| title | The effect of estimating prevalences on the population-wise error rate |
| topic | Methodology |
| url | https://arxiv.org/abs/2304.09988 |