In this paper we propose a general class of covariate-adjusted response-adaptive (CARA) designs based on a new functional urn model. We prove strong consistency concerning the functional urn proportion and the proportion of subjects assigned to the treatment groups, in the whole study and for each covariate profile, allowing the distribution of the responses conditioned on covariates to be estimated nonparametrically. In addition, we establish joint central limit theorems for the above quantities and the sufficient statistics of features of interest, which allow to construct procedures to make inference on the conditional response distributions. These results are then applied to typical situations concerning Gaussian and binary responses.

Nonparametric covariate-adjusted response-adaptive design based on a functional urn model / G. Aletti, A. Ghiglietti, W.F. Rosenberger. - In: ANNALS OF STATISTICS. - ISSN 0090-5364. - 46:6B(2018 Dec), pp. 3838-3866. [10.1214/17-AOS1677]

Nonparametric covariate-adjusted response-adaptive design based on a functional urn model

G. Aletti
Primo
;
A. Ghiglietti
;
2018

Abstract

In this paper we propose a general class of covariate-adjusted response-adaptive (CARA) designs based on a new functional urn model. We prove strong consistency concerning the functional urn proportion and the proportion of subjects assigned to the treatment groups, in the whole study and for each covariate profile, allowing the distribution of the responses conditioned on covariates to be estimated nonparametrically. In addition, we establish joint central limit theorems for the above quantities and the sufficient statistics of features of interest, which allow to construct procedures to make inference on the conditional response distributions. These results are then applied to typical situations concerning Gaussian and binary responses.
clinical trials; covariate-adjusted analysis; inference; large sample theory; personalized medicine; randomization
Settore MAT/06 - Probabilita' e Statistica Matematica
Settore SECS-S/01 - Statistica
dic-2018
2017
Centro di Ricerca Interdisciplinare su Modellistica Matematica, Analisi Statistica e Simulazione Computazionale per la Innovazione Scientifica e Tecnologica ADAMSS
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2434/460772
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