Jonathan K Y Lai, Jonathan A Weiss
Priority service needs and receipt across the lifespan for individuals with autism spectrum disorder
Autism research : official journal of the International Society for Autism Research
coronavirus disease 2019 pandemic highlighted need conduct efficient randomized clinical trials interim monitoring guidelines efficacy futility several randomized coronavirus disease 2019 trials including multiplatform randomized clinical trial mprct used bayesian guidelines belief would lead quicker efficacy futility decisions traditional frequentist guidelines spending functions conditional power explore belief using intuitive interpretation bayesian methods translating prior opinion treatment effect imaginary prior data imaginary observations combined actual observations trial make conclusions using approach show bayesian efficacy boundary used mprct actually quite similar frequentist pocock boundary mprcts efficacy monitoring guideline considered stopping given observed data greater 99 probability treatment effective odds ratio greater 1 mprcts futility monitoring guideline considered stopping given observed data greater 95 probability treatment less 20 effective odds ratio less 12 mprct used normal prior distribution thought supplementing actual patients data imaginary patients data explore effects varying probability thresholds priortoactual patient ratio mprct compare resulting bayesian efficacy monitoring guidelines wellknown frequentist pocock obrienfleming efficacy guidelines also contrast bayesian futility guidelines traditional 20 conditional power futility guideline bayesian efficacy futility monitoring boundary using neutral weakly informative prior distribution fixed probability threshold interim analyses aggressive commonly used obrienfleming efficacy boundary coupled 20 conditional power threshold futility tradeoff aggressive boundaries tend stop trials earlier incur loss power interestingly bayesian efficacy boundary 99 probability threshold similar classic pocock efficacy boundary pandemic quickly weeding ineffective treatments identifying effective treatments paramount aggressive monitoring may preferred conservative approaches obrienfleming boundary accomplished either bayesian frequentist methods