메뉴 건너뛰기




Volumn 7, Issue 1, 2010, Pages 33-47

Clinical trial designs for evaluating the medical utility of prognostic and predictive biomarkers in oncology

Author keywords

Adaptive design; Biomarker; Clinical trial design; Predictive; Prognostic; Validation

Indexed keywords

ANTINEOPLASTIC AGENT; BIOLOGICAL MARKER; TRASTUZUMAB;

EID: 74049098972     PISSN: 17410541     EISSN: 1744828X     Source Type: Journal    
DOI: 10.2217/pme.09.49     Document Type: Review
Times cited : (142)

References (47)
  • 1
    • 58149154725 scopus 로고    scopus 로고
    • Validation of analytical methods for biomarkers employed in drug development
    • Chau CH, Rixe O, McLeod H, Figg WD: Validation of analytical methods for biomarkers employed in drug development. Clin. Cancer Res. 14(19), 5967-5976 (2008).
    • (2008) Clin. Cancer Res , vol.14 , Issue.19 , pp. 5967-5976
    • Chau, C.H.1    Rixe, O.2    McLeod, H.3    Figg, W.D.4
  • 3
    • 0033936550 scopus 로고    scopus 로고
    • Time dependent ROC curves for censored survival data and a diagnostic marker
    • Heagerty PJ, Lumley T, Pepe MS: Time dependent ROC curves for censored survival data and a diagnostic marker. Biometrics 56, 337-344 (2004).
    • (2004) Biometrics , vol.56 , pp. 337-344
    • Heagerty, P.J.1    Lumley, T.2    Pepe, M.S.3
  • 4
    • 33846978784 scopus 로고    scopus 로고
    • Critical review of published microarray studies for cancer outcome and guidelines on statistical ana lysis and reporting
    • Dupuy A, Simon R: Critical review of published microarray studies for cancer outcome and guidelines on statistical ana lysis and reporting. J. Natl Cancer Inst. 99, 147-157 (2007).
    • (2007) J. Natl Cancer Inst , vol.99 , pp. 147-157
    • Dupuy, A.1    Simon, R.2
  • 5
    • 0037245343 scopus 로고    scopus 로고
    • Pitfalls in the use of DNA microarray data for diagnostic and prognostic classification
    • Simon R, Radmacher MD, Dobbin K, McShane LM: Pitfalls in the use of DNA microarray data for diagnostic and prognostic classification. J. Natl Cancer Inst. 95, 14-18 (2003).
    • (2003) J. Natl Cancer Inst , vol.95 , pp. 14-18
    • Simon, R.1    Radmacher, M.D.2    Dobbin, K.3    McShane, L.M.4
  • 6
    • 25144494760 scopus 로고    scopus 로고
    • Prediction error estimation: A comparison of resampling methods
    • Molinaro AM, Simon R, Pfeiffer RM: Prediction error estimation: a comparison of resampling methods. Bioinformatics 21(15), 3301-3307 (2005).
    • (2005) Bioinformatics , vol.21 , Issue.15 , pp. 3301-3307
    • Molinaro, A.M.1    Simon, R.2    Pfeiffer, R.M.3
  • 8
    • 3543094382 scopus 로고    scopus 로고
    • Perspectives and challenges of clinical pharmacogenomics in cancer
    • Pusztai L: Perspectives and challenges of clinical pharmacogenomics in cancer. Pharmacogenomics 5(5), 451-454 (2004).
    • (2004) Pharmacogenomics , vol.5 , Issue.5 , pp. 451-454
    • Pusztai, L.1
  • 9
    • 0028318390 scopus 로고
    • Statistical aspects of prognostic factor studies in oncology
    • Simon R, Altman DG: Statistical aspects of prognostic factor studies in oncology. Br. J. Cancer 69, 979-985 (1994).
    • (1994) Br. J. Cancer , vol.69 , pp. 979-985
    • Simon, R.1    Altman, D.G.2
  • 10
    • 33745641883 scopus 로고    scopus 로고
    • Uses and abuses of tumor markers in the diagnosis, monitoring and treatment of primary and metastatic breast cancer
    • Henry NL, Hayes DF: Uses and abuses of tumor markers in the diagnosis, monitoring and treatment of primary and metastatic breast cancer. Oncologist 11, 541-552 (2006).
    • (2006) Oncologist , vol.11 , pp. 541-552
    • Henry, N.L.1    Hayes, D.F.2
  • 11
    • 19944422061 scopus 로고    scopus 로고
    • A multigene assay to predict recurrence of tamoxifen-treated, node-negative breast cancer
    • Paik S, Shak S, Tang G et al.: A multigene assay to predict recurrence of tamoxifen-treated, node-negative breast cancer. N. Engl. J. Med. 351, 2817-2826 (2004).
    • (2004) N. Engl. J. Med , vol.351 , pp. 2817-2826
    • Paik, S.1    Shak, S.2    Tang, G.3
  • 12
    • 74049124050 scopus 로고    scopus 로고
    • ■ Provides a good case study of the development of an effective prognostic biomarker
    • ■ Provides a good case study of the development of an effective prognostic biomarker.
  • 13
    • 34447132081 scopus 로고    scopus 로고
    • Development and clinical utility of a 21-gene recurrence score prognostic assay in patients with early breast cancer treated with tamoxifen
    • Paik S: Development and clinical utility of a 21-gene recurrence score prognostic assay in patients with early breast cancer treated with tamoxifen. Oncologist 12, 631-635 (2007).
    • (2007) Oncologist , vol.12 , pp. 631-635
    • Paik, S.1
  • 14
    • 2342571696 scopus 로고    scopus 로고
    • Limitations of the odds ratio in guaging the performance of a diagnostic, prognostic or screening marker
    • Pepe MS, Janes H, Longton G, Leisenring W, Newcomb P: Limitations of the odds ratio in guaging the performance of a diagnostic, prognostic or screening marker. Am. J. Epidemiol. 159, 882-890 (2004).
    • (2004) Am. J. Epidemiol , vol.159 , pp. 882-890
    • Pepe, M.S.1    Janes, H.2    Longton, G.3    Leisenring, W.4    Newcomb, P.5
  • 15
    • 74049101891 scopus 로고    scopus 로고
    • ■ Important demonstration of the distinction between measures of association and measures of predictive accuracy
    • ■ Important demonstration of the distinction between measures of association and measures of predictive accuracy.
  • 16
    • 18944384502 scopus 로고    scopus 로고
    • When is a genomic classifier ready for prime time?
    • Simon R: When is a genomic classifier ready for prime time? Nat. Clin. Pract. Oncol. 1(1), 2-3 (2004).
    • (2004) Nat. Clin. Pract. Oncol , vol.1 , Issue.1 , pp. 2-3
    • Simon, R.1
  • 17
    • 26444543829 scopus 로고    scopus 로고
    • Clinical trial designs for prospective validation of biomarkers
    • Mandrekar S, Grothey A, Goetz M, Sargent D: Clinical trial designs for prospective validation of biomarkers. Am. J. Pharmacogenomics 5(5), 317-325 (2005).
    • (2005) Am. J. Pharmacogenomics , vol.5 , Issue.5 , pp. 317-325
    • Mandrekar, S.1    Grothey, A.2    Goetz, M.3    Sargent, D.4
  • 18
    • 51649085832 scopus 로고    scopus 로고
    • Randomized Phase III clinical trial designs for targeted agents
    • Hoering A, Leblanc M, Crowley J: Randomized Phase III clinical trial designs for targeted agents. Clin. Cancer Res. 14(14), 4358-4367 (2008).
    • (2008) Clin. Cancer Res , vol.14 , Issue.14 , pp. 4358-4367
    • Hoering, A.1    Leblanc, M.2    Crowley, J.3
  • 19
    • 67651030311 scopus 로고    scopus 로고
    • Clinical trial designs for predictive biomarker validation: One size does not fit all
    • Mandrekar S, Sargent D: Clinical trial designs for predictive biomarker validation: one size does not fit all. J. Biopharm. Stat. 19, 530-542 (2009).
    • (2009) J. Biopharm. Stat , vol.19 , pp. 530-542
    • Mandrekar, S.1    Sargent, D.2
  • 20
    • 33750601244 scopus 로고    scopus 로고
    • Gene signature evaluation as a prognostic tool: Challenges in the design of the MINDACT trial
    • Bogaerts J, Cardoso F, Buyse M et al.: Gene signature evaluation as a prognostic tool: challenges in the design of the MINDACT trial. Nat. Clin. Pract. Oncol. 3(10), 540-551 (2006).
    • (2006) Nat. Clin. Pract. Oncol , vol.3 , Issue.10 , pp. 540-551
    • Bogaerts, J.1    Cardoso, F.2    Buyse, M.3
  • 21
    • 74049085664 scopus 로고    scopus 로고
    • ■ Good review of the design considerations for a prospective trial to validate a prognostic biomarker
    • ■ Good review of the design considerations for a prospective trial to validate a prognostic biomarker.
  • 22
    • 39149113165 scopus 로고    scopus 로고
    • Development of the 21-gene assay and its application in clinical practice and clinical trials
    • Sparano JA, Paik S: Development of the 21-gene assay and its application in clinical practice and clinical trials. J. Clin. Oncol. 26(5), 721-728 (2008).
    • (2008) J. Clin. Oncol , vol.26 , Issue.5 , pp. 721-728
    • Sparano, J.A.1    Paik, S.2
  • 23
    • 70449420123 scopus 로고    scopus 로고
    • Use of archived specimens in evaluation of prognostic and predictive biomarkers
    • Simon RM, Paik S, Hayes DF: Use of archived specimens in evaluation of prognostic and predictive biomarkers. Natl Cancer Inst. 101(21), 1446-1452 (2009).
    • (2009) Natl Cancer Inst , vol.101 , Issue.21 , pp. 1446-1452
    • Simon, R.M.1    Paik, S.2    Hayes, D.F.3
  • 24
    • 54049129625 scopus 로고    scopus 로고
    • Pivotal evaluation of the accuracy of a classification biomarker: The probe study design
    • Pepe MS, Feng Z, Janes H, Bossuyt PM, Potter J: Pivotal evaluation of the accuracy of a classification biomarker: the probe study design. J. Natl Cancer Inst. 100, 432-438 (2008).
    • (2008) J. Natl Cancer Inst , vol.100 , pp. 432-438
    • Pepe, M.S.1    Feng, Z.2    Janes, H.3    Bossuyt, P.M.4    Potter, J.5
  • 25
    • 74049162235 scopus 로고    scopus 로고
    • ■ Important paper on an improved approach to the design of studies for the evaluation of biomarkers used for early disease detection
    • ■ Important paper on an improved approach to the design of studies for the evaluation of biomarkers used for early disease detection.
  • 26
    • 27244446214 scopus 로고    scopus 로고
    • Prognostic and predictive factors revisited
    • Hayes DF: Prognostic and predictive factors revisited. Breast 14, 493-499 (2005).
    • (2005) Breast , vol.14 , pp. 493-499
    • Hayes, D.F.1
  • 27
    • 38049103907 scopus 로고    scopus 로고
    • HER2 status and efficacy of adjuvant anthracyclines in early breast cancer: A pooled ana lysis of randomized clinical trials
    • Gennari A, Sormani MP, Pronzato P et al.: HER2 status and efficacy of adjuvant anthracyclines in early breast cancer: a pooled ana lysis of randomized clinical trials. J. Natl Cancer Inst. 100(1), 14-20 (2008).
    • (2008) J. Natl Cancer Inst , vol.100 , Issue.1 , pp. 14-20
    • Gennari, A.1    Sormani, M.P.2    Pronzato, P.3
  • 28
    • 35248895346 scopus 로고    scopus 로고
    • HER2 and response to paclitaxel in node-positive breast cancer
    • Hayes DF, Thor AD, Dressler LG et al.: HER2 and response to paclitaxel in node-positive breast cancer. N. Engl. J. Med. 357, 1496-1506 (2007).
    • (2007) N. Engl. J. Med , vol.357 , pp. 1496-1506
    • Hayes, D.F.1    Thor, A.D.2    Dressler, L.G.3
  • 29
    • 42649145667 scopus 로고    scopus 로고
    • Wild-type KRAS is required for panitumumab efficacy in patients with metastatic colorectal cancer
    • Amado RG, Wolf M, Peeters M et al.: Wild-type KRAS is required for panitumumab efficacy in patients with metastatic colorectal cancer. J. Clin. Oncol. 26(10), 1626-1634 (2008).
    • (2008) J. Clin. Oncol , vol.26 , Issue.10 , pp. 1626-1634
    • Amado, R.G.1    Wolf, M.2    Peeters, M.3
  • 30
    • 54949085398 scopus 로고    scopus 로고
    • Kras mutations and benefit from cetuximab in advanced colorectal cancer
    • Karapetis CS, Khambata-Ford S, Jonker DJ et al.: Kras mutations and benefit from cetuximab in advanced colorectal cancer. N. Engl. J. Med. 359(17), 1757-1765 (2008).
    • (2008) N. Engl. J. Med , vol.359 , Issue.17 , pp. 1757-1765
    • Karapetis, C.S.1    Khambata-Ford, S.2    Jonker, D.J.3
  • 31
    • 33847147313 scopus 로고    scopus 로고
    • American Society of Clinical Oncology/College of American Pathologists guideline recommendations for human epidermal growth factor receptor 2 testing in breast cancer
    • Wolff AC, Hammond EH, Schwartz JN et al.: American Society of Clinical Oncology/College of American Pathologists guideline recommendations for human epidermal growth factor receptor 2 testing in breast cancer. J. Clin. Oncol. 25(1), 118-145 (2007).
    • (2007) J. Clin. Oncol , vol.25 , Issue.1 , pp. 118-145
    • Wolff, A.C.1    Hammond, E.H.2    Schwartz, J.N.3
  • 32
    • 41649102043 scopus 로고    scopus 로고
    • The cancer biomarker problem
    • Sawyers CL: The cancer biomarker problem. Nature 452, 548-552 (2008).
    • (2008) Nature , vol.452 , pp. 548-552
    • Sawyers, C.L.1
  • 33
    • 74049122724 scopus 로고    scopus 로고
    • ■■ Very useful review of the importance and challenges in the development of predictive biomarkers
    • ■■ Very useful review of the importance and challenges in the development of predictive biomarkers.
  • 34
    • 6044278144 scopus 로고    scopus 로고
    • Evaluating the efficiency of targeted designs for randomized clinical trials
    • Simon R, Maitournam A: Evaluating the efficiency of targeted designs for randomized clinical trials. Clin. Cancer Res. 10, 6759-6763 (2005).
    • (2005) Clin. Cancer Res , vol.10 , pp. 6759-6763
    • Simon, R.1    Maitournam, A.2
  • 35
    • 74049109915 scopus 로고    scopus 로고
    • ■■ Quantitative evaluation of the efficiency of targeted enrichment designs
    • ■■ Quantitative evaluation of the efficiency of targeted enrichment designs.
  • 36
    • 33744828667 scopus 로고    scopus 로고
    • Evaluating the efficiency of targeted designs for randomized clinical trials: Supplement and correction
    • Simon R, Maitournam A: Evaluating the efficiency of targeted designs for randomized clinical trials: supplement and correction. Clin. Cancer Res. 12, 3229 (2006).
    • (2006) Clin. Cancer Res , vol.12 , pp. 3229
    • Simon, R.1    Maitournam, A.2
  • 37
    • 13644270099 scopus 로고    scopus 로고
    • On the efficiency of targeted clinical trials
    • Maitournam A, Simon R: On the efficiency of targeted clinical trials. Stat. Med. 24, 329-339 (2005).
    • (2005) Stat. Med , vol.24 , pp. 329-339
    • Maitournam, A.1    Simon, R.2
  • 38
    • 58149151278 scopus 로고    scopus 로고
    • Using genomics in clinical trial design
    • Simon R: Using genomics in clinical trial design. Clin. Cancer Res. 14, 5984-5993 (2008).
    • (2008) Clin. Cancer Res , vol.14 , pp. 5984-5993
    • Simon, R.1
  • 39
    • 74049163974 scopus 로고    scopus 로고
    • ■■ Provides a roadmap for the prospective validation of predictive biomarkers
    • ■■ Provides a roadmap for the prospective validation of predictive biomarkers.
  • 40
    • 67650440606 scopus 로고    scopus 로고
    • Designs and adaptive ana lysis plans for pivotal clinical trials of therapeutics and companion diagnostics
    • Simon R: Designs and adaptive ana lysis plans for pivotal clinical trials of therapeutics and companion diagnostics. Expert Rev. Mol. Diagn. 2(6), 721-729 (2008).
    • (2008) Expert Rev. Mol. Diagn , vol.2 , Issue.6 , pp. 721-729
    • Simon, R.1
  • 41
    • 56949103832 scopus 로고    scopus 로고
    • Use of genomic signatures in therapeutics development
    • Simon R, Wang SJ: Use of genomic signatures in therapeutics development. Pharmacogenomics J. 6, 1667-1173 (2006).
    • (2006) Pharmacogenomics J , vol.6 , pp. 1667-1173
    • Simon, R.1    Wang, S.J.2
  • 42
    • 34648840242 scopus 로고    scopus 로고
    • Approaches to evaluation of treatment effect in randomized clinical trials with genomic subset
    • Wang SJ, O'Neill RT, Hung HMJ: Approaches to evaluation of treatment effect in randomized clinical trials with genomic subset. Pharm. Stat. 6, 227-244 (2007).
    • (2007) Pharm. Stat , vol.6 , pp. 227-244
    • Wang, S.J.1    O'Neill, R.T.2    Hung, H.M.J.3
  • 43
    • 74049118228 scopus 로고    scopus 로고
    • A threshold sample-enrichment approach in a clinical trial with heterogeneous subpopulations
    • In press
    • Liu A, Li Q, Yu KF, Yuan VW: A threshold sample-enrichment approach in a clinical trial with heterogeneous subpopulations. Stat. Med. (2009) (In press).
    • (2009) Stat. Med
    • Liu, A.1    Li, Q.2    Yu, K.F.3    Yuan, V.W.4
  • 44
    • 34447264769 scopus 로고    scopus 로고
    • Biomarker adaptive threshold design: A procedure for evaluating treatment with possible biomarker-defined subset effect
    • Jiang W, Freidlin B, Simon R: Biomarker adaptive threshold design: a procedure for evaluating treatment with possible biomarker-defined subset effect. J. Natl Cancer Inst. 99, 1036-1043 (2007).
    • (2007) J. Natl Cancer Inst , vol.99 , pp. 1036-1043
    • Jiang, W.1    Freidlin, B.2    Simon, R.3
  • 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. Clin. Cancer Res. 11, 7872-7878 (2005).
    • (2005) Clin. Cancer Res , vol.11 , pp. 7872-7878
    • Freidlin, B.1    Simon, R.2
  • 46
    • 34547529442 scopus 로고    scopus 로고
    • A method for testing a prespecified subgroup in clinical trials
    • Song Y, Chi GYH: A method for testing a prespecified subgroup in clinical trials. Stat. Med. 26, 3535-3549 (2007).
    • (2007) Stat. Med , vol.26 , pp. 3535-3549
    • Song, Y.1    Chi, G.Y.H.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.