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Author:

Wang, Weizhen (Wang, Weizhen.) | Yin, Huan (Yin, Huan.) | Zhang, Zhongzhan (Zhang, Zhongzhan.) (Scholars:张忠占)

Indexed by:

Scopus SCIE PubMed

Abstract:

To assess the effectiveness of a treatment in phase II clinical trial for cancer study, an adaptive multi-stage design, especially a two-stage one, is commonly used. This type of design allows an on-going study to end early for the participant's safety and design's efficiency. Since a large value of the response rate p for the treatment is wanted, lower one-sided confidence intervals for p are of interest. Due to the limited sample size, exact intervals with a guaranteed confidence level are derived using a rank function that is based on the Clopper-Pearson lower confidence limit. When the sample size in stage 2 is a constant, two kinds of smallest intervals are constructed with or without using the sufficiency principle. The proposed intervals outperform the existing exact intervals, and the intervals not based on minimal sufficient statistic are recommended for practice due to their small expected lengths. When the sample size in stage 2 varies, the smallest interval is also proposed.

Keyword:

lower one-sided confidence limit Clopper-Pearson interval coverage probability phase II clinical trial sufficiency principle

Author Community:

  • [ 1 ] [Wang, Weizhen]Beijing Univ Technol, Coll Appl Sci, Beijing, Peoples R China
  • [ 2 ] [Yin, Huan]Beijing Univ Technol, Coll Appl Sci, Beijing, Peoples R China
  • [ 3 ] [Zhang, Zhongzhan]Beijing Univ Technol, Coll Appl Sci, Beijing, Peoples R China
  • [ 4 ] [Wang, Weizhen]Wright State Univ, Dept Math & Stat, Dayton, OH 45435 USA

Reprint Author's Address:

  • [Wang, Weizhen]Wright State Univ, Dept Math & Stat, Dayton, OH 45435 USA

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Source :

STATISTICAL METHODS IN MEDICAL RESEARCH

ISSN: 0962-2802

Year: 2019

Issue: 6

Volume: 29

Page: 1682-1699

2 . 3 0 0

JCR@2022

ESI Discipline: MATHEMATICS;

ESI HC Threshold:54

JCR Journal Grade:3

Cited Count:

WoS CC Cited Count: 4

SCOPUS Cited Count: 4

ESI Highly Cited Papers on the List: 0 Unfold All

WanFang Cited Count:

Chinese Cited Count:

30 Days PV: 0

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