Simulation-Guided Planning of a Target Trial Emulated Cluster Randomized Trial for Mass Small-Quantity Lipid Nutrient Supplementation Combined with Expanded Program on Immunization in Rural Niger
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arXiv
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| Autores principales: | , , , , , |
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| Formato: | Preprint |
| Publicado: |
2025
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| _version_ | 1866909964011831296 |
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| author | Alam, Shomoita Dyrkton, Nathaniel Shepherd, Susan Sana, Ibrahim Phelan, Kevin Park, Jay JH |
| author_facet | Alam, Shomoita Dyrkton, Nathaniel Shepherd, Susan Sana, Ibrahim Phelan, Kevin Park, Jay JH |
| contents | Background: Target trial emulation (TTE) that applies trial design principles to improve the analysis of non-randomized studies is increasingly being used. Applications of TTE to emulate cluster randomized trials (RCTs) have been limited. This study explored how to integrate simulation-guided design into the TTE framework to inform planning of a non-randomized cluster trial. Methods: We performed simulations to prospectively plan data collection of a non-randomized study emulating a village-level cluster RCT when cluster-randomization was infeasible. The planned study will assess the impact of mass distribution of nutritional supplements embedded within an existing immunization program to improve pentavalent vaccination rates among children 12-24 months old in Niger. The design included covariate-constrained random selection of villages for outcome ascertainment at follow-up. Simulations used baseline census data on pentavalent vaccination rates and cluster-level covariates to compare the type I error rate and power of four statistical methods: beta-regression; quasi-binomial regression; inverse probability of treatment weighting (IPTW); and naive Wald test. Results: Of the four analytic methods considered, only IPTW and beta-regression controlled the type I error rate at 0.05, but IPTW yielded poor statistical power. Beta-regression that showed adequate statistical power was chosen as our primary analysis. Conclusions: Adopting simulation-guided design principles within TTE can enable robust planning of a group-level non-randomized study emulating a cluster RCT. Lessons from this study also apply to TTE planning of individually-RCTs. |
| format | Preprint |
| id |
arxiv_https___arxiv_org_abs_2510_19077 |
| institution | arXiv |
| publishDate | 2025 |
| record_format | arxiv |
| spellingShingle | Simulation-Guided Planning of a Target Trial Emulated Cluster Randomized Trial for Mass Small-Quantity Lipid Nutrient Supplementation Combined with Expanded Program on Immunization in Rural Niger Alam, Shomoita Dyrkton, Nathaniel Shepherd, Susan Sana, Ibrahim Phelan, Kevin Park, Jay JH Applications Methodology Background: Target trial emulation (TTE) that applies trial design principles to improve the analysis of non-randomized studies is increasingly being used. Applications of TTE to emulate cluster randomized trials (RCTs) have been limited. This study explored how to integrate simulation-guided design into the TTE framework to inform planning of a non-randomized cluster trial. Methods: We performed simulations to prospectively plan data collection of a non-randomized study emulating a village-level cluster RCT when cluster-randomization was infeasible. The planned study will assess the impact of mass distribution of nutritional supplements embedded within an existing immunization program to improve pentavalent vaccination rates among children 12-24 months old in Niger. The design included covariate-constrained random selection of villages for outcome ascertainment at follow-up. Simulations used baseline census data on pentavalent vaccination rates and cluster-level covariates to compare the type I error rate and power of four statistical methods: beta-regression; quasi-binomial regression; inverse probability of treatment weighting (IPTW); and naive Wald test. Results: Of the four analytic methods considered, only IPTW and beta-regression controlled the type I error rate at 0.05, but IPTW yielded poor statistical power. Beta-regression that showed adequate statistical power was chosen as our primary analysis. Conclusions: Adopting simulation-guided design principles within TTE can enable robust planning of a group-level non-randomized study emulating a cluster RCT. Lessons from this study also apply to TTE planning of individually-RCTs. |
| title | Simulation-Guided Planning of a Target Trial Emulated Cluster Randomized Trial for Mass Small-Quantity Lipid Nutrient Supplementation Combined with Expanded Program on Immunization in Rural Niger |
| topic | Applications Methodology |
| url | https://arxiv.org/abs/2510.19077 |