preference: An R Package for Two-Stage Clinical Trial Design Accounting for Patient Preference

2020
The consideration of a patient's treatment preference may be essential in determining how a patient will respond to a particular treatment. While traditional clinical trials are unable to capture these effects, the two-stage randomized preference design provides an important tool for researchers seeking to understand the role of patient preferences. In addition to the treatment effect, these designs seek to estimate the role of preferences through testing of selection and preference effects. The R package preference facilitates the use of two-stage clinical trials by providing the necessary tools to design and analyze these studies. To aid in the design, functions are provided to estimate the required sample size and to estimate the study power when a sample size is fixed. In addition, analysis functions are provided to determine the significance of each effect using either raw data or summary statistics. The package is able to incorporate either an unstratified or stratified preference design. The functionality of the package is demonstrated using data from a study evaluating two management methods in women found to have an atypical Pap smear.
JOURNAL OF STATISTICAL SOFTWARE
页码:1-16|卷号:94|期号:CN2
ISSN:1548-7660
来源机构
Yale University
收录类型
SSCI
发表日期
2020
学科领域
循证社会科学-方法
国家
美国
语种
英语
DOI
10.18637/jss.v094.c02
其他关键词
RANDOMIZED-TRIAL; SELECTION
资助机构
Patient-Centered Outcomes Research Institute (PCORI) AwardPatient-Centered Outcomes Research Institute - PCORI [ME-1511-32832]; Yale's CTSA Award [Ul1TR001863]
资助信息
This work was partially supported through a Patient-Centered Outcomes Research Institute (PCORI) Award (ME-1511-32832) and Yale's CTSA Award (Ul1TR001863). We would also like to thank the IMAP team for sharing their data to demonstrate this package.
被引频次(WOS)
0
被引更新日期
2022-01
关键词
two-stage clinical trials preference sample size