(3.238.96.184) 您好!臺灣時間:2021/05/08 20:32
字體大小: 字級放大   字級縮小   預設字形  
回查詢結果

詳目顯示:::

: 
twitterline
研究生:陳祈安
研究生(外文):Chi-An Chen
論文名稱:臨床試驗上調整與選擇病人母體的統計方法
論文名稱(外文):Adaptive Enrichment Design in Clinical Trials
指導教授:黃郁芬黃郁芬引用關係蕭金福蕭金福引用關係
指導教授(外文):Yufen HuangChin Fu Hsiao
口試委員:蕭金福鄒小蕙
口試委員(外文):Chin Fu HsiaoHsiao-Hui Sophie Tsou
口試日期:2011-06-21
學位類別:碩士
校院名稱:國立中正大學
系所名稱:數理統計研究所
學門:數學及統計學門
學類:統計學類
論文種類:學術論文
論文出版年:2011
畢業學年度:99
語文別:英文
論文頁數:44
中文關鍵詞:臨床試驗生物標記
外文關鍵詞:Adaptive design
相關次數:
  • 被引用被引用:0
  • 點閱點閱:332
  • 評分評分:系統版面圖檔系統版面圖檔系統版面圖檔系統版面圖檔系統版面圖檔
  • 下載下載:41
  • 收藏至我的研究室書目清單書目收藏:0
Abstract:
In recent years, with the advancement observation in human genetic studies, the clinical trial community increasingly recognizes that the homogeneous patients may be heterogeneous at the genomic level. Such genomic technologies manage to bring along some possible avenues for developing genomic biomarkers to predict a genomically responsive patient subset that may have higher likelihood of benefiting from treatments. Additional genomic information can generate an appealing conceptual framework from the patient’s perspective in addressing the needs stemmed from personalized medicine during well-controlled clinical trials. There are many perceived benefits of personalized medicine that are based on the notion of being genomically proactive in disease identification, prevention and even better-stopping the recurrence all together.

The utility of personalized medicine in clinical trial can be well characterized through adaptive patient enrichment design. The adaptive enrichment approaches permit assessment of treatment effect that may be applicable to specific patient sets due to heterogeneous patient characteristics and/or different responses to the treatment. In this thesis, we adopt a weighted z-test to generate a test procedure, and the conditional power as our enrichment criterion and provide the type I error rate, power performance and sample size which are needed in respect to different weight t.

Keywords: adaptive design, conditional power, enrichment design, genomic biomarker, weighted z-test

Contents
1. Introduction 1
2. The Problem Under Study 2
3. Statistical Method in a Simple Scenario 4
3.1 Decision Rule Issue 4
3.2 Test Statistic Issue 5
3.3 Interim Assessment 6
3.4 Type I Error Issue 8
3.5 Power Calculation 10
4. Statistical method in Scenario II 11
4.1 Decision Rule Issue 11
4.2 Sample Size & Critical Value Adjustment: 13
5. Estimation of Treatment Effect Size 18
6. Simulation and Some Observed Properties 20
6.1 For Scenario I 21
6.2 For Scenario II 29
6.3 Estimation of Treatment Effect 35
7. Conclusion and Discussion 38
References 39

References
[1] Jack Bowden and Ekkehard Glimm. Unbiased estimation of selected
treatment means in two-stage trials. Biometrical Journal, 50:515–527,2008.
[2] Shein-Chung Chow and Mark Chang. Adaptive Design Methods in Clin-
ical Trials. Chapman and Hall/CRC, 2007.
[3] Lu Cui, H. M. James Hung, and Sue-Jane Wang. Modification of sample
size in group sequential clinical trials. Biometrics, Vol. 55, No. 3:853–857,1999.
[4] FDA. Guide for Industry. 2010.
[5] K. K. Gordon Lan, Yuhwen Soo, Cynthia Siu, and Mey Wang. The use of weighted z-tests in medical research. Journal of Biopharmaceutical Statistics, 15:625–639, 2005.
[6] Gang Li, Weichung J. Shih, Tailiang Xie, and Jiang Lu. A sample size adjustment procedure for clinical trials based on conditional power. Biostatistics, 3,2:277–287, 2002.
[7] Sue-Jane Wang, H. M. James Hung, and Robert T. ONeill. Adaptive patient enrichment designs in therapeutic trials. Biometrical Journal, 51:358–374, 2009.
[8] Sue-Jane Wang, Robert T. ONeill, and H.M.James Hung. Approaches to evaluation of treatment effect in randomized clinical trials with genomic
subset. Pharmaceutical Statistics, 6:227–244, 2007.
[9] Peter Bauer and Franz Koenig. The reassessment of trial perspectives
from interim data - critical view. Statistics in Medicine, 25:23–36, 2006.


QRCODE
 
 
 
 
 
                                                                                                                                                                                                                                                                                                                                                                                                               
第一頁 上一頁 下一頁 最後一頁 top
系統版面圖檔 系統版面圖檔