Novel Bayesian Designs for Dose-Finding Trials: An Overview, Opinions, and Some Examples скачать в хорошем качестве

Novel Bayesian Designs for Dose-Finding Trials: An Overview, Opinions, and Some Examples

We are pleased to announce an exciting seminar on clinical trial innovation as part of the ISBS Webinar Series. The seminar, titled " Novel Bayesian Designs for Dose-Finding Trials: An Overview, Opinions, and Some Examples" will be held on June 6, 2025, 11am-12pm EDT, featuring distinguished speaker Prof. Peter Thall from University of Texas MD Anderson Cancer Center. Abstract: There is widespread agreement that conventional dose-finding designs often select unsafe or ineffective doses, and fail to optimize progression-free survival time, overall survival time, or remission duration. In many medical settings, however, it may be unclear how to structure a trial design. This talk first will provide a brief history of dose-finding designs for early phase clinical trials. A table with real world examples that explains flaws of conventional dose-finding designs in each of five indications will be presented, giving a solution for each flaw. General recommendations for trial design then will be given. When feasible and ethical, a dose-finding design should (1) account for long-term outcomes, (2) include screening rules that drop unsafe or ineffective doses, (3) enroll an adequate sample size, and (4) randomize patients among doses. By incorporating one or more of the recommended features, a design improves the likelihood that a selected dose or schedule will be optimal, benefit future patients, and obtain regulatory approval. Three designs that include one or more of these features will be reviewed. The first design randomized patients among two cell therapy doses and standard of care for COVID-19 induced acute respiratory distress syndrome. The second design is a new strategy that generalizes phase 1-2 by identifying a set of candidate doses, rather than one dose, randomizing additional patients among the candidates, and selecting an optimal dose to maximize progression-free survival over longer follow-up. The third design is for a randomized two-stage phase 1-2 basket trial that optimizes doses in multiple Indications.