The impact of allocation bias on test decisions in clinical trials with multiple endpoints using multiple testing strategies
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| Názov: | The impact of allocation bias on test decisions in clinical trials with multiple endpoints using multiple testing strategies |
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| Autori: | Schoenen, Stefanie, Heussen, Nicole, Verbeeck, Johan, Hilgers, Ralf-Dieter |
| Prispievatelia: | Schoenen, Stefanie, Heussen, Nicole, VERBEECK, Johan, Hilgers, Ralf-Dieter |
| Zdroj: | BMC Med Res Methodol BMC Medical Research Methodology, Vol 24, Iss 1, Pp 1-14 (2024) BMC medical research methodology 24(1), 223 (2024). doi:10.1186/s12874-024-02335-x |
| Informácie o vydavateľovi: | Springer Science and Business Media LLC, 2024. |
| Rok vydania: | 2024 |
| Predmety: | Medicine (General), Šidák, Endpoint Determination, Type I error rate, All-or-none approach, Randomization, Allocation bias, R5-920, Bias, Humans, Multiple testing, Computer Simulation, Family-wise error rate, Randomized Controlled Trials as Topic, Clinical Trials as Topic, Multiple endpoints, Models, Statistical, Research, Intersection-union test, Research Design, Sample Size, Co-primary endpoints, Endpoint Determination/methods [MeSH], Algorithms [MeSH], Bias [MeSH], Humans [MeSH], Randomized Controlled Trials as Topic/methods [MeSH], Computer Simulation [MeSH], Research Design [MeSH], Endpoint Determination/statistics, Sample Size [MeSH], Clinical Trials as Topic/statistics, Clinical Trials as Topic/methods [MeSH], Models, Statistical [MeSH], Randomized Controlled Trials as Topic/statistics, Algorithms, SidakAll-or-none approach |
| Popis: | Background Considering multiple endpoints in clinical trials provide a more comprehensive understanding of treatment effects and may lead to increased power or reduced sample size, which may be beneficial in rare diseases. Besides the small sample sizes, allocation bias is an issue that affects the validity of these trials. We investigate the impact of allocation bias on testing decisions in clinical trials with multiple endpoints and offer a tool for selecting an appropriate randomization procedure (RP). Methods We derive a model for quantifying the effect of allocation bias depending on the RP in the case of two-arm parallel group trials with continuous multiple endpoints. We focus on two approaches to analyze multiple endpoints, either the Šidák procedure to show efficacy in at least one endpoint and the all-or-none procedure to show efficacy in all endpoints. Results To evaluate the impact of allocation bias on the test decision we propose a biasing policy for multiple endpoints. The impact of allocation on the test decision is measured by the family-wise error rate of the Šidák procedure and the type I error rate of the all-or-none procedure. Using the biasing policy we derive formulas to calculate these error rates. In simulations we show that, for the Šidák procedure as well as for the all-or-none procedure, allocation bias leads to inflation of the mean family-wise error and mean type I error, respectively. The strength of this inflation is affected by the choice of the RP. Conclusion Allocation bias should be considered during the design phase of a trial to increase validity. The developed methodology is useful for selecting an appropriate RP for a clinical trial with multiple endpoints to minimize allocation bias effects. |
| Druh dokumentu: | Article Other literature type |
| Popis súboru: | application/pdf |
| Jazyk: | English |
| ISSN: | 1471-2288 |
| DOI: | 10.1186/s12874-024-02335-x |
| DOI: | 10.18154/rwth-2024-09780 |
| Prístupová URL adresa: | https://pubmed.ncbi.nlm.nih.gov/39350102 https://doaj.org/article/8fab8cf0e83a4fc5995c99e19d13ea89 https://repository.publisso.de/resource/frl:6508534 http://hdl.handle.net/1942/44552 https://publications.rwth-aachen.de/record/995201 |
| Rights: | CC BY |
| Prístupové číslo: | edsair.doi.dedup.....64384d9bd479b12f5a40ac006a935bdc |
| Databáza: | OpenAIRE |
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Nájsť tento článok vo Web of Science | Abstrakt: | Background Considering multiple endpoints in clinical trials provide a more comprehensive understanding of treatment effects and may lead to increased power or reduced sample size, which may be beneficial in rare diseases. Besides the small sample sizes, allocation bias is an issue that affects the validity of these trials. We investigate the impact of allocation bias on testing decisions in clinical trials with multiple endpoints and offer a tool for selecting an appropriate randomization procedure (RP). Methods We derive a model for quantifying the effect of allocation bias depending on the RP in the case of two-arm parallel group trials with continuous multiple endpoints. We focus on two approaches to analyze multiple endpoints, either the Šidák procedure to show efficacy in at least one endpoint and the all-or-none procedure to show efficacy in all endpoints. Results To evaluate the impact of allocation bias on the test decision we propose a biasing policy for multiple endpoints. The impact of allocation on the test decision is measured by the family-wise error rate of the Šidák procedure and the type I error rate of the all-or-none procedure. Using the biasing policy we derive formulas to calculate these error rates. In simulations we show that, for the Šidák procedure as well as for the all-or-none procedure, allocation bias leads to inflation of the mean family-wise error and mean type I error, respectively. The strength of this inflation is affected by the choice of the RP. Conclusion Allocation bias should be considered during the design phase of a trial to increase validity. The developed methodology is useful for selecting an appropriate RP for a clinical trial with multiple endpoints to minimize allocation bias effects. |
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| ISSN: | 14712288 |
| DOI: | 10.1186/s12874-024-02335-x |