Non-adherence in non-inferiority trials: pitfalls and recommendations

被引：54

作者：

Mo, Yin ^{[1
,2
,3
,4
]}

Lim, Cherry ^{[1
,4
]}

Watson, James A. ^{[1
,4
]}

White, Nicholas J. ^{[1
,4
]}

Cooper, Ben S. ^{[1
,4
]}

机构：

[1] Mahidol Univ, Fac Trop Med, Mahidol Oxford Trop Med Res Unit, Bangkok, Thailand

[2] Natl Univ Singapore Hosp, Univ Med Cluster, Singapore, Singapore

[3] Natl Univ Singapore, Dept Med, Singapore, Singapore

[4] Univ Oxford, Nuffield Dept Med, Ctr Trop Med, Oxford OX3 7BN, England

来源：

BMJ-BRITISH MEDICAL JOURNAL | 2020年 / 370卷

基金：

英国惠康基金; 英国医学研究理事会;

关键词：

CAUSAL; DURATION; EFFICACY; CARE;

D O I：

10.1136/bmj.m2215

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Non-adherence in non-inferiority trials can affect treatment effect estimates and often increases the chance of claiming non-inferiority under the standard intention-to-treat analysis. This article discusses the implications of different patterns of non-adherence in non-inferiority trials and offers practical recommendations for trial design, alternative analysis strategies, and outcome reporting to reduce bias in treatment estimates and improve transparency in reporting.

引用

页数：10

共 38 条

[1] Do non-inferiority trials of reduced intensity therapies show reduced effects? A descriptive analysis [J].

Aberegg, Scott K. ;

Hersh, Andrew M. ;

Samore, Matthew H. .

BMJ OPEN, 2018, 8 (03)

[2] Deviation from intention to treat analysis in randomised trials and treatment effect estimates: meta-epidemiological study [J].

Abraha, Iosief ;

Cherubini, Antonio ;

Cozzolino, Francesco ;

De Florio, Rita ;

Luchetta, Maria Laura ;

Rimland, Joseph M. ;

Folletti, Ilenia ;

Marchesi, Mauro ;

Germani, Antonella ;

Orso, Massimiliano ;

Eusebi, Paolo ;

Montedori, Alessandro .

BMJ-BRITISH MEDICAL JOURNAL, 2015, 350

[3] Percutaneous coronary intervention in stable angina (ORBITA): a double-blind, randomised controlled trial [J].

Al-Lamee, Rasha ;

Thompson, David ;

Dehbi, Hakim-Moulay ;

Sen, Sayan ;

Tang, Kare ;

Davies, John ;

Keeble, Thomas ;

Mielewczik, Michael ;

Kaprielian, Raffi ;

Malik, Iqbal S. ;

Nijjer, Sukhjinder S. ;

Petraco, Ricardo ;

Cook, Christopher ;

Ahmad, Yousif ;

Howard, James ;

Baker, Christopher ;

Sharp, Andrew ;

Gerber, Robert ;

Talwar, Suneel ;

Assomull, Ravi ;

Mayet, Jamil ;

Wensel, Roland ;

Collier, David ;

Shun-Shin, Matthew ;

Thom, Simon A. ;

Davies, Justin E. ;

Francis, Darrel P. .

LANCET, 2018, 391 (10115) :31-40

[4] Committee for medicinal products for human use (CHMP) guideline on the choice of the non-inferiority margin [J].

不详 .

STATISTICS IN MEDICINE, 2006, 25 (10) :1628-1638

[5]

[Anonymous], 2015, BMJ

[6]

[Anonymous], J AM STAT ASS

[7]

[Anonymous], 2010, Guidance for Industry: Non-Inferiority Clinical Trials

[8]

[Anonymous], 2016, NON CLIN TRIALS

[9] Role of feasibility and pilot studies in randomised controlled trials: a cross-sectional study [J].

Blatch-Jones, Amanda Jane ;

Pek, Wei ;

Kirkpatrick, Emma ;

Ashton-Key, Martin .

BMJ OPEN, 2018, 8 (09)

[10] Weekends-off efavirenz-based antiretroviral therapy in HIV-infected children, adolescents, and young adults (BREATHER): a randomised, open-label, non-inferiority, phase 2/3 trial [J].

Butler, Karina ;

Turkova, Anna ;

Inshaw, Jamie ;

Compagnucci, Alexandra ;

Kenny, Julia ;

Saidi, Yacine ;

Musiime, Victor ;

Nanduudu, Annett ;

Cressey, Tim R. ;

Chalermpantmetagul, Suwalai ;

Scott, Karen ;

Harper, Lynda ;

Montero, Sam ;

Riault, Yoann ;

Bunupuradah, Torsak ;

Volokha, Alla ;

Flynn, Patricia M. ;

Bologna, Rosa ;

Kizito, Hilda ;

Ramos, Jose T. ;

Nastouli, Eleni ;

Klein, Nigel ;

Giaquinto, Carlo ;

Ford, Deborah ;

Babiker, Abdel ;

Gibb, Diana M. .

LANCET HIV, 2016, 3 (09) :E421-E430

← 1 2 3 4 →