Odhadování efektu léčby v klinické studii se zkříženým designem a zbytkovými efekty
Název práce v češtině: | Odhadování efektu léčby v klinické studii se zkříženým designem a zbytkovými efekty |
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Název v anglickém jazyce: | Treatment effect estimation in the cross-over design with carry-over effects |
Akademický rok vypsání: | 2023/2024 |
Typ práce: | diplomová práce |
Jazyk práce: | |
Ústav: | Katedra pravděpodobnosti a matematické statistiky (32-KPMS) |
Vedoucí / školitel: | doc. Mgr. Michal Kulich, Ph.D. |
Řešitel: | skrytý - zadáno a potvrzeno stud. odd. |
Datum přihlášení: | 22.04.2024 |
Datum zadání: | 23.04.2024 |
Datum potvrzení stud. oddělením: | 23.04.2024 |
Zásady pro vypracování |
The goal of the thesis is to propose a new estimator of the treatment effect in a two-period cross-over study in the presence of a carry-over effect. The efficient method for treatment effect estimation assumes that there is no carry-over effect or at least that the carry-over effects of both treatments are the same. If this assumption is not true this estimator is biased but one can get an unbiased estimator either by fitting a regression model on correlated outcomes (GEE or LME model) or by analyzing the first period only. Both of these methods have low power and lack precision. The thesis will investigate various approaches to combining the more efficient but potentially biased estimator with one of the inefficient unbiased estimators, will assess their theoretical properties and illustrate their behavior in simulation studies.
The topic requires development and evaluation of an original idea that has not been considered in previously published literature. |
Seznam odborné literatury |
Senn S. (2002) Cross-Over Trials in Clinical research. John Wiley & Sons, Ltd, Chichester.
Helebrand F. (2023) Analysis of cross-over clinical trials in the presence of baseline measurements. Diplomová práce, MFF UK, Praha. Lavancier F. and Rochet P. (2016) A general procedure to combine estimators. Computational Statistics and Data Analysis, 94, 175-192. Senn S. (2006) Cross-over trials in Statistics in Medicine: The first 25 years. Statistics in Medicine, 25, 3430–3442. Senn S., D’Angelo G. and Potvin D. (2004) Carry-over in cross-over trials in bioequivalence: theoretical concerns and empirical evidence. Pharmaceutical Statistics, 3, 133–142. |