Skip to Main content Skip to Navigation
Theses

Méthodes d'aide à la décision thérapeutique dans les cas des maladies rares : intérêt des méthodes bayésiennes et application à la maladie de Horton

Abstract : In recent years, scientists have difficulties to study rare diseases by conventional methods, because the sample size needed in such studies to meet a conventional frequentist power is not adapted to the number of available patients. After systemically searching in literature and characterizing different methods used in the contest of rare diseases, we remarked that most of the proposed methods are deterministic and are globally unsatisfactory because it is difficult to correct the insufficient statistical power.More attention has been placed on Bayesian models which through a prior distribution combined with a current study enable to draw decisionsfrom a posterior distribution. Determination of the prior distribution in a Bayesian model is challenging, we will describe the process of determining the prior including the possibility of considering information from some historical controlled trials and/or data coming from other studies sufficiently close to the subject of interest.First, we describe a Bayesian model that aims to test the hypothesis of the non-inferiority trial based on the hypothesis that methotrexate is more effective than corticosteroids alone.On the other hand, our work rests on the use of the epsilon-contamination method, which is based on contaminating an a priori not entirely satisfactory by a series of distributions drawn from information on other studies sharing close conditions,treatments or even populations. Contamination is a way to include the proximity of information provided bythese studies.
Document type :
Theses
Complete list of metadatas

Cited literature [102 references]  Display  Hide  Download

https://tel.archives-ouvertes.fr/tel-01921936
Contributor : Abes Star :  Contact
Submitted on : Wednesday, November 14, 2018 - 11:03:05 AM
Last modification on : Tuesday, September 8, 2020 - 5:21:48 AM
Long-term archiving on: : Friday, February 15, 2019 - 1:33:08 PM

File

EL_HAJJ_2017_archivage.pdf
Version validated by the jury (STAR)

Identifiers

  • HAL Id : tel-01921936, version 1

Collections

Citation

Paméla El Hajj. Méthodes d'aide à la décision thérapeutique dans les cas des maladies rares : intérêt des méthodes bayésiennes et application à la maladie de Horton. Méthodologie [stat.ME]. Université Montpellier, 2017. Français. ⟨NNT : 2017MONTS037⟩. ⟨tel-01921936⟩

Share

Metrics

Record views

102

Files downloads

1207