An Overview of Current Methods for Real-world Applications to Generalize or Transport Clinical Trial Findings to Target Populations of Interest.
Journal
Epidemiology (Cambridge, Mass.)
ISSN: 1531-5487
Titre abrégé: Epidemiology
Pays: United States
ID NLM: 9009644
Informations de publication
Date de publication:
01 09 2023
01 09 2023
Historique:
medline:
3
8
2023
pubmed:
31
5
2023
entrez:
31
5
2023
Statut:
ppublish
Résumé
It has been well established that randomized clinical trials have poor external validity, resulting in findings that may not apply to relevant-or target-populations. When the trial is sampled from the target population, generalizability methods have been proposed to address the applicability of trial findings to target populations. When the trial sample and target populations are distinct, transportability methods may be applied for this purpose. However, generalizability and transportability studies present challenges, particularly around the strength of their conclusions. We review and summarize state-of-the-art methods for translating trial findings to target populations. We additionally provide a novel step-by-step guide to address these challenges, illustrating principles through a published case study. When conducted with rigor, generalizability and transportability studies can play an integral role in regulatory decisions by providing key real-world evidence.
Identifiants
pubmed: 37255252
doi: 10.1097/EDE.0000000000001633
pii: 00001648-990000000-00144
pmc: PMC10392887
doi:
Types de publication
Review
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
627-636Informations de copyright
Copyright © 2023 The Author(s). Published by Wolters Kluwer Health, Inc.
Références
Clin Pharmacol Ther. 2020 Apr;107(4):817-826
pubmed: 31541454
Lancet. 2002 Jan 5;359(9300):57-61
pubmed: 11809203
BMC Med Res Methodol. 2013 Jul 16;13:92
pubmed: 23855337
Circulation. 2008 Sep 16;118(12):1294-303
pubmed: 18794402
Epidemiology. 2000 Sep;11(5):550-60
pubmed: 10955408
Int J Epidemiol. 2016 Feb;45(1):140-50
pubmed: 26772869
PLoS One. 2022 Dec 15;17(12):e0278842
pubmed: 36520950
Prev Sci. 2015 Apr;16(3):475-85
pubmed: 25307417
Stat Med. 2020 Jun 30;39(14):1999-2014
pubmed: 32253789
Commun Stat Simul Comput. 2022;51(8):4326-4348
pubmed: 36419543
J Am Med Inform Assoc. 2019 May 1;26(5):429-437
pubmed: 30869798
J R Stat Soc Ser A Stat Soc. 2018 Oct;181(4):1193-1209
pubmed: 30555215
Epidemiology. 2009 Nov;20(6):863-71
pubmed: 19806059
Am J Epidemiol. 2014 Oct 1;180(7):737-48
pubmed: 25190679
Addiction. 2016 Jul;111(7):1226-34
pubmed: 26825855
Stat Methods Med Res. 2021 May;30(5):1235-1248
pubmed: 33620006
Am J Epidemiol. 2019 Feb 1;188(2):438-443
pubmed: 30299451
Stat Med. 2009 Nov 10;28(25):3083-107
pubmed: 19757444
Stat Med. 2023 Jun 15;42(13):2029-2043
pubmed: 36847107
J Clin Epidemiol. 2019 Apr;108:54-63
pubmed: 30562543
Curr Med Res Opin. 2022 Aug;38(8):1431-1437
pubmed: 35699028
Stat Med. 2019 Jun 30;38(14):2573-2588
pubmed: 30883861
Am J Epidemiol. 2016 Nov 1;184(9):670-680
pubmed: 27737840
PLoS Clin Trials. 2006 May;1(1):e9
pubmed: 16871331
Am J Epidemiol. 2010 Jul 1;172(1):107-15
pubmed: 20547574
Clin Pharmacol Ther. 2019 May;105(5):1156-1163
pubmed: 30107034
J Acquir Immune Defic Syndr. 2016 Dec 1;73(4):482-488
pubmed: 27668804
Addiction. 2017 Jul;112(7):1210-1219
pubmed: 28191694
J R Stat Soc Ser A Stat Soc. 2001 Apr 1;174(2):369-386
pubmed: 24926156
Epidemiology. 2019 Jan;30(1):103-111
pubmed: 30252687
Epidemiology. 2011 May;22(3):368-77
pubmed: 21399502
Addict Behav. 2019 Jul;94:124-132
pubmed: 30415786
Eval Rev. 2017 Aug;41(4):357-388
pubmed: 27491758
Biometrics. 2019 Jun;75(2):685-694
pubmed: 30488513
Eval Rev. 2017 Oct;41(5):472-505
pubmed: 27402612
Stat Methods Med Res. 2019 Jan;28(1):3-19
pubmed: 28573919
Am J Epidemiol. 2017 Oct 15;186(8):1010-1014
pubmed: 28535275
Health Serv Res. 2014 Feb;49(1):284-303
pubmed: 23855598
Clin J Am Soc Nephrol. 2014 Jul;9(7):1328-35
pubmed: 24509298
J R Stat Soc Series B Stat Methodol. 2017 Nov;79(5):1509-1525
pubmed: 29375249
N Engl J Med. 2017 Mar 30;376(13):1203-1205
pubmed: 28355503
Am J Epidemiol. 2021 Oct 1;190(10):2075-2084
pubmed: 33972995
JAMA Netw Open. 2022 Nov 1;5(11):e2239874
pubmed: 36326765
Eur J Epidemiol. 2019 Aug;34(8):719-722
pubmed: 31218483
Nat Rev Drug Discov. 2011 Jul 01;10(7):495-506
pubmed: 21720406
Epidemiology. 2017 Jul;28(4):553-561
pubmed: 28346267
Res Soc Work Pract. 2018 Jul;28(5):532-537
pubmed: 30034203
J Res Educ Eff. 2016;9(1):103-127
pubmed: 27668031
Stat Med. 2015 Dec 10;34(28):3661-79
pubmed: 26238958
J Policy Anal Manage. 2013 Winter;32(1):107-121
pubmed: 25152557
Biometrics. 2023 Jun;79(2):1213-1225
pubmed: 34862966
Am J Epidemiol. 2018 Apr 1;187(4):817-827
pubmed: 29020193
J Clin Epidemiol. 2011 Nov;64(11):1198-207
pubmed: 21463926
PLoS One. 2021 Jul 1;16(7):e0253969
pubmed: 34197538
Stat Med. 2010 Feb 10;29(3):337-46
pubmed: 19960510
J R Stat Soc Ser C Appl Stat. 2022 Jun;71(3):669-697
pubmed: 35968541
PLoS One. 2018 Dec 11;13(12):e0208795
pubmed: 30533053
Commun Stat Theory Methods. 2023;52(16):5767-5798
pubmed: 37484707
N Engl J Med. 2015 Nov 26;373(22):2103-16
pubmed: 26551272