title: Comparison of single-arm vs. randomized phase II clinical trials: a Bayesian approach.
abstract: Single-arm studies are typically used in phase II of clinical trials, whose main objective is to determine whether a new treatment warrants further testing in a randomized phase III trial. The introduction of randomization in phase II, to avoid the limits of studies based on historical controls, is a critical issue widely debated in the recent literature. We use a Bayesian approach to compare single-arm and randomized studies, based on a binary response variable, in terms of their abilities of reaching the correct decision about the new treatment, both when it performs better than the standard one and when it is less effective. We evaluate how the historical control rate, the total sample size, and the elicitation of the prior distributions affect the decision about which study performs better.
authors.affiliation: a Dipartimento di Scienze Statistiche , Sapienza Università di Roma , Roma , Italia.
authors.forename: Valeria
authors.initials: V
authors.lastname: Sambucini
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use; D001499:Bayes Theorem; D017322:Clinical Trials, Phase II as Topic / Q000379:methods / Q000706:statistics & numerical data*; D003198:Computer Simulation; D003627:Data Interpretation, Statistical; D023381:Endpoint Determination; D016032:Randomized Controlled Trials as Topic / Q000379:methods / Q000706:statistics & numerical data*; D018401:Sample Size; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents
keywords: Bayesian approach; historical control rate; phase II clinical trials; randomization