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Whole population cohorts versus sampled comparators designs for evaluating health and educational outcomes of children with inborn rare conditions: a simulation study
2026-01-11
pediatrics
Title + abstract only
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BackgroundLinked administrative data covering whole populations are fundamental resources for longitudinal studies of children with rare conditions (cases) and unaffected peers (comparators). Data minimisation regulations sometimes limit the number of comparators per case (sampled comparators, SC), with unknown impact on study findings. MethodsUsing Monte Carlo draws, we simulated 100 000 children with and without an exemplar condition, congenital hypothyroidism (CHT), with covariates (sex and ...
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