Subgroup discovery in non-inferiority trials

被引:0
|
作者
Fazzari, Melissa J. [1 ]
Kim, Mimi Y. [1 ]
机构
[1] Albert Einstein Coll Med, Dept Epidemiol & Populat, Div Biostat, 1300 Morris Pk Ave,Block Bldg,Room 314, Bronx, NY 10461 USA
基金
美国国家卫生研究院;
关键词
non-inferiority trials; random forests; regression trees; subgroup analysis; ACUPUNCTURE;
D O I
10.1002/sim.9118
中图分类号
Q [生物科学];
学科分类号
07 ; 0710 ; 09 ;
摘要
Approaches and guidelines for performing subgroup analysis to assess heterogeneity of treatment effect in clinical trials have been the topic of numerous papers in the statistical and clinical literature, but have been discussed predominantly in the context of conventional superiority trials. Concerns about treatment heterogeneity are the same if not greater in non-inferiority (NI) trials, especially since overall similarity between two treatment arms in a successful NI trial could be due to the existence of qualitative interactions that are more likely when comparing two active therapies. Even in unsuccessful NI trials, subgroup analyses can yield important insights about the potential reasons for failure to demonstrate non-inferiority of the experimental therapy. Recent NI trials have performed a priori subgroup analyses using standard statistical tests for interaction, but there is increasing interest in more flexible machine learning approaches for post-hoc subgroup discovery. The performance and practical application of such methods in NI trials have not been systematically explored, however. We considered the Virtual Twin method for the NI setting, an algorithm for subgroup identification that combines random forest with classification and regression trees, and conducted extensive simulation studies to examine its performance under different NI trial conditions and to devise decision rules for selecting the final subgroups. We illustrate the utility of the method with data from a NI trial that was conducted to compare two acupuncture treatments for chronic musculoskeletal pain.
引用
收藏
页码:5174 / 5187
页数:14
相关论文
共 50 条
  • [1] Non-Inferiority Trials
    不详
    [J]. MEDICAL LETTER ON DRUGS AND THERAPEUTICS, 2011, 53 (1355): : 1 - 1
  • [2] Non-inferiority trials
    Elie, Caroline
    Touze, Emmanuel
    [J]. SANG THROMBOSE VAISSEAUX, 2012, 24 (02): : 93 - 99
  • [3] Non-inferiority and equivalence trials
    Ranstam, J.
    Cook, J. A.
    [J]. BRITISH JOURNAL OF SURGERY, 2017, 104 (11) : 1578 - 1579
  • [4] The Non-Inferiority Complex: What Do Non-Inferiority Trials Tell Us?
    Assimon, Magdalene M.
    Cutter, Gary R.
    Bargman, Joanne M.
    [J]. JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, 2022, 33 (04): : 674 - 676
  • [5] Introduction to randomized trials: Non-inferiority trials
    Simon, E. -G.
    Fouche, C. -J.
    Perrotin, F.
    [J]. GYNECOLOGIE OBSTETRIQUE & FERTILITE, 2012, 40 (09): : 554 - 556
  • [6] Equivalence and Non-inferiority Trials of CAM
    Ernst, Edzard
    [J]. EVIDENCE-BASED COMPLEMENTARY AND ALTERNATIVE MEDICINE, 2004, 1 (01) : 9 - 10
  • [7] Understanding non-inferiority trials: an introduction
    Brasher, Penelope M. A.
    Dobson, Gary
    [J]. CANADIAN JOURNAL OF ANESTHESIA-JOURNAL CANADIEN D ANESTHESIE, 2014, 61 (05): : 389 - 392
  • [8] Non-inferiority trials in surgical oncology
    Fueglistaler, Philipp
    Adamina, Michel
    Guller, Ulrich
    [J]. ANNALS OF SURGICAL ONCOLOGY, 2007, 14 (05) : 1532 - 1539
  • [9] Is there a danger of “biocreep” with non-inferiority trials?
    Primrose Beryl
    Werner Vach
    [J]. Trials, 12 (Suppl 1)
  • [10] Anger over non-inferiority trials
    不详
    [J]. NATURE REVIEWS DRUG DISCOVERY, 2006, 5 (10) : 808 - 809