메뉴 건너뛰기




Volumn 36, Issue 8, 2017, Pages 1334-1360

Tutorial on statistical considerations on subgroup analysis in confirmatory clinical trials

Author keywords

negative subgroup treatment effect; supportive subgroup findings; targeted subgroup; treatment by subgroup interaction

Indexed keywords

CLINICAL TRIAL; HUMAN; STATISTICAL MODEL; ANALOGS AND DERIVATIVES; CEREBROVASCULAR ACCIDENT; HEART INFARCTION; MORTALITY; PROCEDURES; RANDOMIZED CONTROLLED TRIAL (TOPIC); REPRODUCIBILITY; SEPSIS; STATISTICAL ANALYSIS; STATISTICS; SURVIVAL ANALYSIS; TREATMENT OUTCOME;

EID: 85005929980     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.7167     Document Type: Article
Times cited : (49)

References (56)
  • 3
    • 26844457534 scopus 로고    scopus 로고
    • The distinctive nature of HER2-positive breast cancers
    • Burstein HJ. The distinctive nature of HER2-positive breast cancers. New England Journal of Medicine 2005; 353:1652–1654.
    • (2005) New England Journal of Medicine , vol.353 , pp. 1652-1654
    • Burstein, H.J.1
  • 4
    • 0034466679 scopus 로고    scopus 로고
    • Debate: Subgroup analyses in clinical trials: fun to look at – but don't believe them!
    • Sleight P. Debate: Subgroup analyses in clinical trials: fun to look at – but don't believe them! Current Controlled Trials in Cardiovascular Medicine 2000; 1:25–27.
    • (2000) Current Controlled Trials in Cardiovascular Medicine , vol.1 , pp. 25-27
    • Sleight, P.1
  • 5
    • 33645895554 scopus 로고    scopus 로고
    • The challenge of subgroup analyses – reporting without distorting
    • Lagakos SW. The challenge of subgroup analyses – reporting without distorting. New England Journal of Medicine 2006; 354:1667–1669.
    • (2006) New England Journal of Medicine , vol.354 , pp. 1667-1669
    • Lagakos, S.W.1
  • 7
    • 85014372950 scopus 로고    scopus 로고
    • (accessed 20 February 2015)
    • Alimta (pemetrexed for injection) label, 2013. Available at: http://www.accessdata.fda.gov/drugsatfda_docs/label/2013/021462s045lbl.pdf (accessed 20 February 2015)
    • (2013) Alimta (pemetrexed for injection) label
  • 8
    • 85014352060 scopus 로고    scopus 로고
    • (accessed 20 February 2015)
    • Valcyte (valganciclovir hydrochloride) label, 2013. Available at: http://www.accessdata.fda.gov/drugsatfda_docs/label/2013/021304s010,022257s004lbl.pdf (accessed 20 February 2015)
    • (2013) Valcyte (valganciclovir hydrochloride) label
  • 9
    • 33846447795 scopus 로고    scopus 로고
    • The probability of observing negative subgroup results when the treatment effect is positive and homogenous across all subgroups
    • Li Z, Chuang-Stein C, Hoseyni C. The probability of observing negative subgroup results when the treatment effect is positive and homogenous across all subgroups. Drug Information Journal 2007; 41:47–56.
    • (2007) Drug Information Journal , vol.41 , pp. 47-56
    • Li, Z.1    Chuang-Stein, C.2    Hoseyni, C.3
  • 10
    • 77953682084 scopus 로고    scopus 로고
    • Regional differences in multinational clinical trials: anticipating chance variation
    • Marschner I. Regional differences in multinational clinical trials: anticipating chance variation. Clinical Trials 2010; 7:147–156.
    • (2010) Clinical Trials , vol.7 , pp. 147-156
    • Marschner, I.1
  • 11
    • 85014390816 scopus 로고    scopus 로고
    • Code of Federal Regulation, 21CFR314.126,, (accessed 20 February 2015)
    • Food and Drug Administration. Health Human Services, Code of Federal Regulation, 21CFR314.126, 2013. Available at: http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?fr=314.126 (accessed 20 February 2015)
    • (2013) Health Human Services
  • 12
    • 84865693205 scopus 로고    scopus 로고
    • (accessed 28 January 2015)
    • Food and Drug Administration Safety and Innovation Act (FDASIA), 2012. Available at: http://www.fda.gov/RegulatoryInformation/Legislation/SignificantAmendmentstotheFDCAct/FDASIA/ucm20027187.htm (accessed 28 January 2015).
    • (2012) Food and Drug Administration Safety and Innovation Act (FDASIA)
  • 13
    • 85014357948 scopus 로고    scopus 로고
    • E9 Statistical Principles for Clinical Trials (ICH E9 guidance),, (accessed 28 January 2015)
    • International Conference on Harmonization International Conference on Harmonization (ICH) Guidance, E9 Statistical Principles for Clinical Trials (ICH E9 guidance), 1998. Available at: http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E9/Step4/E9_Guideline.pdf (accessed 28 January 2015).
    • (1998) International Conference on Harmonization International Conference on Harmonization (ICH) Guidance
  • 18
    • 84911962987 scopus 로고    scopus 로고
    • Multiplicity and replicability: two sides of the same coin
    • Bretz F, Westfall PH. Multiplicity and replicability: two sides of the same coin. Pharmaceutical Statistics 2014; 13:343–344.
    • (2014) Pharmaceutical Statistics , vol.13 , pp. 343-344
    • Bretz, F.1    Westfall, P.H.2
  • 20
    • 84891752239 scopus 로고    scopus 로고
    • Reliably basing conclusions on subgroups of randomized clinical trials
    • Koch A, Framke T. Reliably basing conclusions on subgroups of randomized clinical trials. Journal of Biopharmaceutical Statistics 2014; 24:42–57.
    • (2014) Journal of Biopharmaceutical Statistics , vol.24 , pp. 42-57
    • Koch, A.1    Framke, T.2
  • 23
    • 0030590746 scopus 로고    scopus 로고
    • A randomised, blinded, trial of clopidogrel versus aspirin in patients at risk of ischaemic events (CAPRIE). CAPRIE Steering Committee
    • CAPRIE Steering Committee. A randomised, blinded, trial of clopidogrel versus aspirin in patients at risk of ischaemic events (CAPRIE). CAPRIE Steering Committee. Lancet 1996; 348(9038):1329–1339.
    • (1996) Lancet , vol.348 , Issue.9038 , pp. 1329-1339
  • 24
    • 78049293287 scopus 로고    scopus 로고
    • No inconsistent trial assessments by NICE and IQWiG: different assessment goals may lead to different assessment results regarding subgroup analyses
    • Bender R, Koch A, Skipka G, Kaiser T, Lange S. No inconsistent trial assessments by NICE and IQWiG: different assessment goals may lead to different assessment results regarding subgroup analyses. Journal of Clinical Epidemiology 2010; 63:1305–1307.
    • (2010) Journal of Clinical Epidemiology , vol.63 , pp. 1305-1307
    • Bender, R.1    Koch, A.2    Skipka, G.3    Kaiser, T.4    Lange, S.5
  • 25
    • 78049249374 scopus 로고    scopus 로고
    • Inconsistent trial assessments by the National Institute for Health and Clinical Excellence and IQWiG: standards for the performance and interpretation of subgroup analyses are needed
    • Hasford J, Bramlag P, Koch GG, Lehmacher W, Einhäupl K, Rothwell PM. Inconsistent trial assessments by the National Institute for Health and Clinical Excellence and IQWiG: standards for the performance and interpretation of subgroup analyses are needed. Journal of Clinical Epidemiology 2010; 63:1298–1304.
    • (2010) Journal of Clinical Epidemiology , vol.63 , pp. 1298-1304
    • Hasford, J.1    Bramlag, P.2    Koch, G.G.3    Lehmacher, W.4    Einhäupl, K.5    Rothwell, P.M.6
  • 26
    • 4644225190 scopus 로고    scopus 로고
    • Doxil study 30–49 investigators. Long-term survival advantage for women treated with pegylated liposomal doxorubicin compared with topotecan in a phase 3 randomized study of recurrent and refractory epithelial ovarian cancer
    • Gordon AN, Tonda M, Sun S, Rackoff W. Doxil study 30–49 investigators. Long-term survival advantage for women treated with pegylated liposomal doxorubicin compared with topotecan in a phase 3 randomized study of recurrent and refractory epithelial ovarian cancer. Gynecologic Oncology 2004; 95(1):1–8.
    • (2004) Gynecologic Oncology , vol.95 , Issue.1 , pp. 1-8
    • Gordon, A.N.1    Tonda, M.2    Sun, S.3    Rackoff, W.4
  • 27
    • 84891765135 scopus 로고    scopus 로고
    • Standardization for subgroup analysis in randomized controlled trials
    • Varadhan R, Wang SJ. Standardization for subgroup analysis in randomized controlled trials. Journal of Biopharmaceutical Statistics 2014; 24(1):154–167.
    • (2014) Journal of Biopharmaceutical Statistics , vol.24 , Issue.1 , pp. 154-167
    • Varadhan, R.1    Wang, S.J.2
  • 29
    • 0022072706 scopus 로고
    • Testing for qualitative interaction between treatment effects and patient subsets
    • Gail M, Simon R. Testing for qualitative interaction between treatment effects and patient subsets. Biometrics 1985; 41:361–372.
    • (1985) Biometrics , vol.41 , pp. 361-372
    • Gail, M.1    Simon, R.2
  • 30
    • 0002255973 scopus 로고
    • Statistical aspects of cancer trials
    • In, Halnan KE, (ed)., Chapman and Hall, London, UK
    • Peto R. Statistical aspects of cancer trials. In Treatment of Cancer, Halnan KE (ed). Chapman and Hall: London, UK, 1982; 867–871.
    • (1982) Treatment of Cancer , pp. 867-871
    • Peto, R.1
  • 31
    • 1842453957 scopus 로고    scopus 로고
    • Subgroup analyses in randomized trials: risks of subgroup-specific analyses; power and sample size for the interaction test
    • Brookes ST, Whitely E, Egger M, Smith GD, Mulheran PA, Peters TJ. Subgroup analyses in randomized trials: risks of subgroup-specific analyses; power and sample size for the interaction test. Journal of Clinical Epidemiology 2004; 57:229–236.
    • (2004) Journal of Clinical Epidemiology , vol.57 , pp. 229-236
    • Brookes, S.T.1    Whitely, E.2    Egger, M.3    Smith, G.D.4    Mulheran, P.A.5    Peters, T.J.6
  • 32
    • 84944151465 scopus 로고    scopus 로고
    • Statistical perspectives on subgroup analysis: testing for heterogeneity and evaluating error rate for the complementary subgroup
    • Alosh M, Huque MF, Koch GG. Statistical perspectives on subgroup analysis: testing for heterogeneity and evaluating error rate for the complementary subgroup. Journal of Biopharmaceutical Statistics 2015; 25:1161–1178.
    • (2015) Journal of Biopharmaceutical Statistics , vol.25 , pp. 1161-1178
    • Alosh, M.1    Huque, M.F.2    Koch, G.G.3
  • 33
    • 84889820624 scopus 로고    scopus 로고
    • For the society for clinical trials (SCT) multi-regional clinical trial consistency working group. Graphical assessment of consistency in treatment effect among countries in multi-regional clinical trials
    • Chen J, Zheng H, Quan H, Li G, Gallo P, Ouyang SP, Binkowitz B, Ting N, Tanaka YX, Luo Ibia E. For the society for clinical trials (SCT) multi-regional clinical trial consistency working group. Graphical assessment of consistency in treatment effect among countries in multi-regional clinical trials. Clinical Trials 2013; 10:842–851.
    • (2013) Clinical Trials , vol.10 , pp. 842-851
    • Chen, J.1    Zheng, H.2    Quan, H.3    Li, G.4    Gallo, P.5    Ouyang, S.P.6    Binkowitz, B.7    Ting, N.8    Tanaka, Y.X.9    Luo Ibia, E.10
  • 34
    • 84921626814 scopus 로고    scopus 로고
    • Methods for exploring treatment effect heterogeneity in subgroup analysis: an application to global clinical trials
    • Schou IM, Marschner IC. Methods for exploring treatment effect heterogeneity in subgroup analysis: an application to global clinical trials. Pharmaceutical Statistics 2015; 14:44–55.
    • (2015) Pharmaceutical Statistics , vol.14 , pp. 44-55
    • Schou, I.M.1    Marschner, I.C.2
  • 35
    • 79957529687 scopus 로고    scopus 로고
    • Chain procedure: a class of flexible closed testing procedures with clinical trial application
    • Millen B, Dmitrienko A. Chain procedure: a class of flexible closed testing procedures with clinical trial application. Statistics in Biopharmaceutical Research 2011; 3:14–30.
    • (2011) Statistics in Biopharmaceutical Research , vol.3 , pp. 14-30
    • Millen, B.1    Dmitrienko, A.2
  • 36
    • 84876969027 scopus 로고    scopus 로고
    • Multiplicity considerations for subgroup analysis subject to consistency constraints
    • Alosh M, Huque MF. Multiplicity considerations for subgroup analysis subject to consistency constraints. Biometrical Journal 2013; 55:444–462.
    • (2013) Biometrical Journal , vol.55 , pp. 444-462
    • Alosh, M.1    Huque, M.F.2
  • 37
    • 84888130933 scopus 로고    scopus 로고
    • A regulatory perspective on essential considerations in design and analysis of subgroups when correctly classified
    • Wang SJ, Hung J. A regulatory perspective on essential considerations in design and analysis of subgroups when correctly classified. Journal of Biopharmaceutical Statistics 2014; 24:19–41.
    • (2014) Journal of Biopharmaceutical Statistics , vol.24 , pp. 19-41
    • Wang, S.J.1    Hung, J.2
  • 38
    • 84891810976 scopus 로고    scopus 로고
    • An overview of statistical planning to address subgroups in confirmatory clinical trials
    • Koch GG, Schwartz T. An overview of statistical planning to address subgroups in confirmatory clinical trials. Journal of Biopharmaceutical Statistics 2014; 24:72–93.
    • (2014) Journal of Biopharmaceutical Statistics , vol.24 , pp. 72-93
    • Koch, G.G.1    Schwartz, T.2
  • 39
    • 85014380575 scopus 로고    scopus 로고
    • (accessed 28 January 2015)
    • Xigris label, 2007. Available at: http://www.accessdata.fda.gov/drugsatfda_docs/label/2007/125029s080LBL.pdf (accessed 28 January 2015).
    • (2007) Xigris label
  • 41
    • 0141883593 scopus 로고    scopus 로고
    • Discussion for “p-value adjustments for subgroup analyses
    • Koch GG. Discussion for “p-value adjustments for subgroup analyses.”. Journal of Biopharmaceutical Statistics 1997; 7(2):323–331.
    • (1997) Journal of Biopharmaceutical Statistics , vol.7 , Issue.2 , pp. 323-331
    • Koch, G.G.1
  • 42
    • 61749083299 scopus 로고    scopus 로고
    • A flexible strategy for testing subgroups and overall population
    • Alosh M, Huque MF. A flexible strategy for testing subgroups and overall population. Statistics in Medicine 2009; 28:3–23.
    • (2009) Statistics in Medicine , vol.28 , pp. 3-23
    • Alosh, M.1    Huque, M.F.2
  • 44
    • 78449283356 scopus 로고    scopus 로고
    • Enrichment of clinical study populations. State of the art
    • Temple R. Enrichment of clinical study populations. State of the art. Nature Publishing Group 2010; 88(6):774–778.
    • (2010) Nature Publishing Group , vol.88 , Issue.6 , pp. 774-778
    • Temple, R.1
  • 45
    • 27744537954 scopus 로고    scopus 로고
    • Adaptive signature design: an adaptive clinical trial design for generating and prospectively testing a gene expression signature for sensitive patients
    • Freidlin B, Simon R. Adaptive signature design: an adaptive clinical trial design for generating and prospectively testing a gene expression signature for sensitive patients. Clinical Cancer Research 2005; 11:7872–7878.
    • (2005) Clinical Cancer Research , vol.11 , pp. 7872-7878
    • Freidlin, B.1    Simon, R.2
  • 46
    • 66349125688 scopus 로고    scopus 로고
    • Adaptive patient enrichment designs in therapeutic trials
    • Wang SJ, Hung HM, O'Neill RT. Adaptive patient enrichment designs in therapeutic trials. Biometrical Journal 2009; 51:358–374.
    • (2009) Biometrical Journal , vol.51 , pp. 358-374
    • Wang, S.J.1    Hung, H.M.2    O'Neill, R.T.3
  • 47
    • 34648840242 scopus 로고    scopus 로고
    • Approaches to evaluation of treatment effect in randomized clinical trials with genomic subset
    • Wang SJ, O'Neill RT, Hung HM. Approaches to evaluation of treatment effect in randomized clinical trials with genomic subset. Pharmaceutical Statistics 2007; 6:227–244.
    • (2007) Pharmaceutical Statistics , vol.6 , pp. 227-244
    • Wang, S.J.1    O'Neill, R.T.2    Hung, H.M.3
  • 49
    • 79957438038 scopus 로고    scopus 로고
    • Design and analysis considerations in clinical trials with a sensitive subpopulation
    • Zhao YD, Dmitrienko A, Tamura R. Design and analysis considerations in clinical trials with a sensitive subpopulation. Statistics in Biopharmaceutical Research 2010; 2:72–83.
    • (2010) Statistics in Biopharmaceutical Research , vol.2 , pp. 72-83
    • Zhao, Y.D.1    Dmitrienko, A.2    Tamura, R.3
  • 50
    • 34547529442 scopus 로고    scopus 로고
    • A method for testing a prespecified subgroup in clinical trials
    • Song Y, Chi GY. A method for testing a prespecified subgroup in clinical trials. Statistics in Medicine 2007; 26:3535–3549.
    • (2007) Statistics in Medicine , vol.26 , pp. 3535-3549
    • Song, Y.1    Chi, G.Y.2
  • 51
    • 84876440727 scopus 로고    scopus 로고
    • Testing each hypothesis marginally at alpha while still controlling FWER: how and when
    • Li J. Testing each hypothesis marginally at alpha while still controlling FWER: how and when. Statistics in Medicine 2013; 32:1730–1738.
    • (2013) Statistics in Medicine , vol.32 , pp. 1730-1738
    • Li, J.1
  • 52
    • 15944368765 scopus 로고    scopus 로고
    • A fixed-sequence Bonferroni procedure for testing multiple endpoints
    • Wiens B. A fixed-sequence Bonferroni procedure for testing multiple endpoints. Pharmaceutical Statistics 2003; 2:211–215.
    • (2003) Pharmaceutical Statistics , vol.2 , pp. 211-215
    • Wiens, B.1
  • 53
    • 60749089513 scopus 로고    scopus 로고
    • A graphical approach to sequentially rejective multiple testing procedures
    • Bretz F, Maurer W, Brannath W, Posch M. A graphical approach to sequentially rejective multiple testing procedures. Statistics in Medicine 2009; 28:586–604.
    • (2009) Statistics in Medicine , vol.28 , pp. 586-604
    • Bretz, F.1    Maurer, W.2    Brannath, W.3    Posch, M.4
  • 54
    • 64549084568 scopus 로고    scopus 로고
    • A recycling framework for the construction of Bonferroni-based multiple tests
    • Burman CF, Sonesson C, Guilbaud O. A recycling framework for the construction of Bonferroni-based multiple tests. Statistics in Medicine 2009; 28:739–761.
    • (2009) Statistics in Medicine , vol.28 , pp. 739-761
    • Burman, C.F.1    Sonesson, C.2    Guilbaud, O.3
  • 55
    • 84873817347 scopus 로고    scopus 로고
    • A statistical framework for decision making in confirmatory multi population tailoring clinical trials
    • Millen B, Dmitrienko A, Ruberg S, Shen L. A statistical framework for decision making in confirmatory multi population tailoring clinical trials. Drug Information Journal 2012; 46:647–656.
    • (2012) Drug Information Journal , vol.46 , pp. 647-656
    • Millen, B.1    Dmitrienko, A.2    Ruberg, S.3    Shen, L.4
  • 56
    • 35348821173 scopus 로고    scopus 로고
    • A flexible fixed-sequence testing method for hierarchically ordered correlated endpoints in clinical trials
    • Huque MF, Alosh M. A flexible fixed-sequence testing method for hierarchically ordered correlated endpoints in clinical trials. Journal of Statistical Planning and Inference 2008; 138:321–335.
    • (2008) Journal of Statistical Planning and Inference , vol.138 , pp. 321-335
    • Huque, M.F.1    Alosh, M.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.