Validation of biomarkers to predict response to immunotherapy in cancer: Volume II - clinical validation and regulatory considerations

Journal for ImmunoTherapy of Cancer

Volumn 4, Issue 1, 2016, Pages

  Dobbin, Kevin K ^a   Cesano, Alessandra ^b   Alvarez, John ^c   Hawtin, Rachael ^d   Janetzki, Sylvia ^e   Kirsch, Ilan ^f   Masucci, Giuseppe V ^g   Robbins, Paul B ^h   Selvan, Senthamil R ⁱ   Streicher, Howard Z ^j   Zhang, Jenny ^k   Butterfield, Lisa H ^l   Thurin, Magdalena ^j  

^a UNIVERSITY OF GEORGIA   (United States)

^b NANOSTRING TECHNOLOGIES   (United States)

^c JANSSEN RESEARCH AND DEVELOPMENT   (Belgium)

^d Nodality Inc   (United States)

^e ZellNet Consulting Inc   (United States)

^f ADAPTIVE BIOTECHNOLOGIES   (United States)

^g KAROLINSKA INSTITUTE   (Sweden)

^h PFIZER INC   (United States)

ⁱ Omni Array Biotechnology LLC   (United States)

^j NATIONAL CANCER INSTITUTE   (United States)

^k Department of Scientific Support and Applications Development   (United States)

^l UNIVERSITY OF PITTSBURGH CANCER INSTITUTE   (United States)

more..

Author keywords

Assay; Biomarker; Cancer; Immunotherapy; Regulatory; Validation

Indexed keywords

BIOLOGICAL MARKER; CD274 PROTEIN, HUMAN; IMMUNOLOGICAL ANTINEOPLASTIC AGENT; PROGRAMMED DEATH 1 LIGAND 1; TUMOR MARKER;

AREA UNDER THE CURVE; CANCER IMMUNOTHERAPY; DIAGNOSTIC VALUE; DRUG APPROVAL; HAZARD RATIO; HUMAN; PATIENT SELECTION; PHASE 3 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; PROCESS DEVELOPMENT; PROGRAM APPROPRIATENESS; RANDOMIZED CONTROLLED TRIAL (TOPIC); RECEIVER OPERATING CHARACTERISTIC; REVIEW; RISK FACTOR; SENSITIVITY AND SPECIFICITY; STUDY DESIGN; SYSTEMATIC ERROR; TREATMENT RESPONSE; VALIDATION PROCESS; ANTAGONISTS AND INHIBITORS; BIOASSAY; EUROPEAN UNION; FOOD AND DRUG ADMINISTRATION; IMMUNOTHERAPY; MOLECULARLY TARGETED THERAPY; MORTALITY; NEOPLASM; PROCEDURES; PROGNOSIS; REPRODUCIBILITY; STANDARDS; TREATMENT OUTCOME; UNITED STATES;

ANTINEOPLASTIC AGENTS, IMMUNOLOGICAL; B7-H1 ANTIGEN; BIOLOGICAL ASSAY; BIOMARKERS, TUMOR; EUROPEAN UNION; HUMANS; IMMUNOTHERAPY; MOLECULAR TARGETED THERAPY; NEOPLASMS; PROGNOSIS; REPRODUCIBILITY OF RESULTS; ROC CURVE; TREATMENT OUTCOME; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84998773662     PISSN: None     EISSN: 20511426     Source Type: Journal    
DOI: 10.1186/s40425-016-0179-0     Document Type: Review

References (36)

1. Hodi FS, O'Day SJ, McDermott DF, Weber RW, Sosman JA, Haanen JB, et al. Improved survival with ipilimumab in patients with metastatic melanoma. N Engl J Med. 2010;363(8):711-23. doi: 10.1056/NEJMoa1003466.
2. Garon EB, Rizvi NA, Hui R, Leighl N, Balmanoukian AS, Eder JP, et al. Pembrolizumab for the treatment of non-small-cell lung cancer. N Engl J Med. 2015;372(21):2018-28. doi: 10.1056/NEJMoa1501824.
3. Motzer RJ, Rini BI, McDermott DF, Redman BG, Kuzel TM, Harrison MR, et al. Nivolumab for metastatic renal cell carcinoma: results of a randomized phase II trial. J Clin Oncol. 2015;33(13):1430-7. doi: 10.1200/jco.2014.59.0703.
4. Powles T, Eder JP, Fine GD, Braiteh FS, Loriot Y, Cruz C, et al. MPDL3280A (anti-PD-L1) treatment leads to clinical activity in metastatic bladder cancer. Nature. 2014;515(7528):558-62. doi: 10.1038/nature13904.
5. Institute of Medicine. The national academies collection: reports funded by national institutes of health, transforming clinical research in the United States: challenges and opportunities: workshop summary. Washington DC: National Academies Press; 2010.
6. Institute of Medicine Committee on Patient Safety and Health Information Technology. Health IT and patient safety: building safer systems for better care. Washington DC: National Academies Press; 2011.
7. Ventana. PD-L1 (SP142 assay) specification sheet. 2016. http://www.accessdata.fda.gov/cdrh_docs/pdf16/P160002c.pdf. Accessed 6 Sept 2016.
8. Febbo PG, Ladanyi M, Aldape KD, De Marzo AM, Hammond ME, Hayes DF, et al. NCCN Task Force report: Evaluating the clinical utility of tumor markers in oncology. J Natl Compr Canc Netw. 2011;9 Suppl 5:S1-32. quiz S3.
9. Woodcock J. Assessing the clinical utility of diagnostics used in drug therapy. Clin Pharmacol Ther. 2010;88(6):765-73. doi: 10.1038/clpt.2010.230.
10. Borghaei H, Paz-Ares L, Horn L, Spigel DR, Steins M, Ready NE, et al. Nivolumab versus docetaxel in advanced nonsquamous Non-small-cell lung cancer. N Engl J Med. 2015;373(17):1627-39. doi: 10.1056/NEJMoa1507643.
11. Simon R. Roadmap for developing and validating therapeutically relevant genomic classifiers. J Clin Oncol. 2005;23(29):7332-41. doi: 10.1200/jco.2005.02.8712.
12. 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. 2003;95(1):14-8.
13. Montgomery SB, Sammeth M, Gutierrez-Arcelus M, Lach RP, Ingle C, Nisbett J, Guigo R, Dermitzakis ET. Transcriptome genetics using second generation sequencing in a Caucasian population. Nature. 2010;464(7289):773-7.
14. Pickrell JK, Marioni JC, Pai AA, Degner JF, Engelhardt BE, Nkadori E, Veyrieras JB, Stephens M, Gilad Y, Pritchard JK. Understanding mechanisms underlying human gene expression variation with RNA sequencing. Nature. 2010;464(7289):768-72.
15. Frazee AC, Langmead B, Leek JT. ReCount: A milti-experiment resource for analysis-ready RNA-seq gene count datasets. BMC Bioinformatics. 2011;12:449.
16. Tibshirani R. Regression shrinkage and selection via the lasso. J R Stat Soc Series B Stat Methodol. 1996;58:267-88.
17. Mandrekar SJ, Sargent DJ. Clinical trial designs for predictive biomarker validation: theoretical considerations and practical challenges. J Clin Oncol. 2009;27(24):4027-34. doi: 10.1200/jco.2009.22.3701.
18. Romond EH, Perez EA, Bryant J, Suman VJ, Geyer Jr CE, Davidson NE, et al. Trastuzumab plus adjuvant chemotherapy for operable HER2-positive breast cancer. N Engl J Med. 2005;353(16):1673-84. doi: 10.1056/NEJMoa052122.
19. Cappuzzo F, Ligorio C, Toschi L, Rossi E, Trisolini R, Paioli D, et al. EGFR and HER2 gene copy number and response to first-line chemotherapy in patients with advanced non-small cell lung cancer (NSCLC). J Thorac Oncol. 2007;2(5):423-9. doi: 10.1097/01.JTO.0000268676.79872.9b.
20. Freidlin B, McShane LM, Korn EL. Randomized clinical trials with biomarkers: design issues. J Natl Cancer Inst. 2010;102(3):152-60. doi: 10.1093/jnci/djp477.
21. Cobo M, Isla D, Massuti B, Montes A, Sanchez JM, Provencio M, et al. Customizing cisplatin based on quantitative excision repair cross-complementing 1 mRNA expression: a phase III trial in non-small-cell lung cancer. J Clin Oncol. 2007;25(19):2747-54. doi: 10.1200/jco.2006.09.7915.
22. Tang G, Shak S, Paik S, Anderson SJ, Costantino JP, Geyer Jr CE, et al. Comparison of the prognostic and predictive utilities of the 21-gene Recurrence Score assay and Adjuvant! for women with node-negative, ER-positive breast cancer: results from NSABP B-14 and NSABP B-20. Breast Cancer Res Treat. 2011;127(1):133-42. doi: 10.1007/s10549-010-1331-z.
23. Amado RG, Wolf M, Peeters M, Van Cutsem E, Siena S, Freeman DJ, et al. Wild-type KRAS is required for panitumumab efficacy in patients with metastatic colorectal cancer. J Clin Oncol. 2008;26(10):1626-34. doi: 10.1200/jco.2007.14.7116.
24. Hoos A, Eggermont AM, Janetzki S, Hodi FS, Ibrahim R, Anderson A, et al. Improved endpoints for cancer immunotherapy trials. J Natl Cancer Inst. 2010;102(18):1388-97. doi: 10.1093/jnci/djq310.
25. Janes H, Pepe MS, Bossuyt PM, Barlow WE. Measuring the performance of markers for guiding treatment decisions. Ann Intern Med. 2011;154(4):253-9. doi: 10.7326/0003-4819-154-4-201102150-00006.
26. U.S. Department of Health and Human Services, Food and Drug Administration, Center for Devices and Radiological Health, Center for Biologics Evaluation and Research. Guidance for Industry and FDA Staff In Vitro Diagnostic (IVD) Device Studies -Frequently Asked Questions. http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/ucm071230.pdf. Accessed 25 June 2010.
27. Draft Guidance for Industry, Food and Drug Administration Staff, and Clinical Laboratories. Framework for Regulatory Oversight of Laboratory Developed Tests (LDTs). US Food and Drug Administration. 2014. http://www.fda.gov/downloads/medicaldevices/deviceregulationandguidance/guidancedocuments/ucm416685.pdf. Accessed 3 Oct 2014.
28. Mansfield EA. FDA perspective on companion diagnostics: an evolving paradigm. Clin Cancer Res. 2014;20(6):1453-7. doi: 10.1158/1078-0432.ccr-13-1954.
29. US Food and Drug Administration. List of cleared or approved companion diagnostic devices (in vitro and imaging tools). 2015. http://www.fda.gov/MedicalDevices/ProductsandMedicalProcedures/InVitroDiagnostics/ucm301431.htm. Accessed 3 Oct 2016.
30. US Department of Health and Human Services, Food and Drug Administration Center for Devices and Radiological Health, Center for Biologics Evaluation and Research, Center for Drug Evaluation and Research. In Vitro Companion Diagnostic Devices Guidance for Industry and Food and Drug Administration Staff. http://www.fda.gov/downloads/medicaldevices/deviceregulationandguidance/guidancedocuments/ucm262327.pdf. Accessed 6 Aug 2014.
31. Olsen D, Jorgensen JT. Companion diagnostics for targeted cancer drugs - clinical and regulatory aspects. Front Oncol. 2014;4:105. doi: 10.3389/fonc.2014.00105.
32. U.S. Department Of Health And Human Services, Food and Drug Administration, Center for Devices and Radiological Health, Immunology Branch, Division of Clinical Laboratory Devices, Office of Device Evaluation. Guidance for Submission of Immunohistochemistry Applications to the FDA; Final Guidance for Industry. http://www.fda.gov/RegulatoryInformation/Guidances/ucm094002.htm. Accessed 3 June 1998.
33. Heger M. FDA Official Discusses Issues with Regulating NGS Dx Tests. GenomeWeb. 2012.
34. U.S. Department of Health and Human Services, Food and Drug Administration, Center for Devices and Radiological Health, Office of In Vitro Diagnostic Device Evaluation and Safety, Center for Biologic Evaluation and Research. Draft Guidance for Industry, Clinical Laboratories, and FDA Staff In Vitro Diagnostic Multivariate Index Assays. http://www.fda.gov/downloads/MedicalDevices/.../ucm071455.pdf. Accessed 26 July 2007.
35. Pignatti F, Ehmann F, Hemmings R, Jonsson B, Nuebling M, Papaluca-Amati M, et al. Cancer drug development and the evolving regulatory framework for companion diagnostics in the European union. Clin Cancer Res. 2014;20(6):1458-68. doi: 10.1158/1078-0432.ccr-13-1571.
36. Friedman JH, Hastie T, Tibshirani R. Regularization paths for generalized linear models via coordinate descent. J Stat Softw. 2010;33:1-22.