Bayesian methods for consensus and evidence in clinical trials

Anno
2020
Proponente Stefania Gubbiotti - Professore Associato
Sottosettore ERC del proponente del progetto
PE1_14
Componenti gruppo di ricerca
Componente Categoria
Valeria Sambucini Componenti strutturati del gruppo di ricerca
Gianmarco Caruso Dottorando/Assegnista/Specializzando componente non strutturato del gruppo di ricerca
Pierpaolo Brutti Componenti strutturati del gruppo di ricerca
Fulvio De Santis Componenti strutturati del gruppo di ricerca
Abstract

In this project we consider the problem of sample size determination (SSD) under a Bayesian framework. We focus on experiments in which multiple priors are available that formalize pre-experimental information and uncertainty on model parameters. In this setup, prior-to-posterior analysis can lead to discordant final inferential conclusions.
In a recent contribution, Joseph and Belisle (2019) proposed a measure of discrepancy between credible intervals based on two conflicting priors and defined a sample size criterion aimed at guaranteeing posterior consensus between them. In this sense, this criterion ignores the achievement of success of the trial, that is usually thought in terms of evidence.
Our proposal is to introduce a method for SSD that simultaneously control the achievement of minimal evidence and consensus, measured by appropriate functions of the posterior distributions. In some preliminary works we have started considering efficacy and superiority trials involving binary outcomes.

The goal of the present project is to elaborate on this idea and to widen the scope of the existing methodology in several directions according to the following tasks.

TASK 1. Providing explicit and general results for trials with different goals (e.g. equivalence, non-inferiority) and with different outcomes (e.g. continuous, categorical or both, ¿).

TASK 2. Comparing the properties of several SSD criteria (e.g. based on different alternative measures of consensus).

TASK 3. Developing the code for the implementation of the proposed criteria in an R package supplemented with a Shiny app directly accessible to the final users.

ERC
PE1_14, PE1_13, LS7_4
Keywords:
INFERENZA STATISTICA, BIOSTATISTICA, INDAGINI CAMPIONARIE E DISEGNO DEGLI ESPERIMENTI, STATISTICA MEDICA, STATISTICA PER LA RICERCA SPERIMENTALE E TECNOLOGICA

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