Alternative metrics for assessing clinical benefit with immunotherapy in oncology

References

• Ascierto PA, Marincola FM. What have we learned from cancer immunotherapy in the last 3 years? J Transl Med 2014; 12:141; PMID:24886164; https://doi.org/10.1186/1479-5876-12-141
   PubMed Web of Science ®Google Scholar
• Chen TT. Statistical issues and challenges in immuno-oncology. J Immunother Cancer 2013; 1:18; PMID:24829754; https://doi.org/10.1186/2051-1426-1-18
   PubMedGoogle Scholar
• Antonia SJ, Larkin J, Ascierto PA. Immuno-oncology combinations: a review of clinical experience and future prospects. Clin Cancer Res 2014; 20:6258-68; PMID:25341541; https://doi.org/10.1158/1078-0432.CCR-14-1457
   PubMed Web of Science ®Google Scholar
• Hodi FS, O'Day SJ, McDermott DF, Weber RW, Sosman JA, Haanen JB, Gonzalez R, Robert C, Schadendorf D, Hassel JC, et al. Improved survival with ipilimumab in patients with metastatic melanoma. N Engl J Med 2010; 363:711-23; PMID:20525992; https://doi.org/10.1056/NEJMoa1003466
   PubMed Web of Science ®Google Scholar
• Robert C, Thomas L, Bondarenko I, O'Day S, Weber J, Garbe C, Lebbe C, Baurain JF, Testori A, Grob JJ, et al. Ipilimumab plus dacarbazine for previously untreated metastatic melanoma. N Engl J Med 2011; 364:2517-26; PMID:21639810; https://doi.org/10.1056/NEJMoa1104621
   PubMed Web of Science ®Google Scholar
• FDA. Opdivo (nivolumab) injection, for intravenous use. 2015.
   Google Scholar
• FDA. Yervoy (ipilimumab) injection, for intravenous infusion. 2015.
   Google Scholar
• FDA. Keytruda (pembrolizumab) for injection, for intravenous use. 2015.
   Google Scholar
• Research CfDEa. Summary review: Ipilimumab. 2011.
   Google Scholar
• Schadendorf D, Hodi FS, Robert C, Weber JS, Margolin K, Hamid O, Patt D, Chen TT, Berman DM, Wolchok JD. Pooled analysis of long-term survival data from Phase II and Phase III trials of ipilimumab in unresectable or metastatic melanoma. J Clin Oncol 2015; 33:1889-94; PMID:25667295; https://doi.org/10.1200/JCO.2014.56.2736
   PubMed Web of Science ®Google Scholar
• EMA. Nivolumab Summary of Product Characteristics. 2015.
   Google Scholar
• Davies A, Briggs A, Schneider J, Levy A, Ebeid O, Wagner S. The ends justify the mean: Outcome measures for estimating the value of new cancer therapies. Health Out Res Med 2012; 3:e25-e36; https://doi.org/10.1016/j.ehrm.2012.01.001
   Google Scholar
• Johnson P, Greiner W, Al-Dakkak I, Wagner S. Which metrics are appropriate to describe the value of new cancer therapies? Biomed Res Int 2015; 2015:865101; PMID:26161418
   PubMed Web of Science ®Google Scholar
• Joulain F, Proskorovsky I, Allegra C, Tabernero J, Hoyle M, Iqbal SU, Van Cutsem E. Mean overall survival gain with aflibercept plus FOLFIRI vs placebo plus FOLFIRI in patients with previously treated metastatic colorectal cancer. Br J Cancer 2013; 109:1735-43; PMID:24045663; https://doi.org/10.1038/bjc.2013.523
   PubMed Web of Science ®Google Scholar
• McDermott D, Lebbe C, Hodi FS, Maio M, Weber JS, Wolchok JD, Thompson JA, Balch CM. Durable benefit and the potential for long-term survival with immunotherapy in advanced melanoma. Cancer Treat Rev 2014; 40:1056-64; PMID:25060490; https://doi.org/10.1016/j.ctrv.2014.06.012
   PubMed Web of Science ®Google Scholar
• Robert C, Long GV, Brady B, Dutriaux C, Maio M, Mortier L, Hassel JC, Rutkowski P, McNeil C, Kalinka-Warzocha E, et al. Nivolumab in previously untreated melanoma without BRAF mutation. N Engl J Med, 2014:320-30.
   PubMed Web of Science ®Google Scholar
• Robert C, Schachter J, Long GV, Arance A, Grob JJ, Mortier L, Daud A, Carlino MS, McNeil C, Lotem M, et al. Pembrolizumab versus ipilimumab in advanced melanoma. N Engl J Med, 2015; 372:2521-32; PMID:25891173; https://doi.org/10.1056/NEJMoa1503093
   PubMed Web of Science ®Google Scholar
• Cook RJ, Sackett DL. The number needed to treat: a clinically useful measure of treatment effect. BMJ 1995; 310:452-4; PMID:7873954; https://doi.org/10.1136/bmj.310.6977.452
   PubMed Web of Science ®Google Scholar
• Attia J, Page J, Heller RF, Dobson AJ. Impact numbers in health policy decisions. J Epidemiol Comm Health 2002; 56:600-5; PMID:12118051; https://doi.org/10.1136/jech.56.8.600
   PubMed Web of Science ®Google Scholar
• Glimelius B, Hoffman K, Graf W, P+Ñhlman L, Sj+Âd+®n P. Quality of life during chemotherapy in patients with symptomatic advanced colorectal cancer. Cancer 1994; 73:556-62; PMID:8299077; https://doi.org/10.1002/1097-0142(19940201)73:3%3c556::AID-CNCR2820730310%3e3.0.CO;2-8
   PubMed Web of Science ®Google Scholar
• Clarke JM, Wang X, Ready NE. Surrogate clinical endpoints to predict overall survival in non-small cell lung cancer trials-are we in a new era? Transl Lung Cancer Res 2015; 4:804. PMID:26798592.
   PubMed Web of Science ®Google Scholar
• Wolchok JD, Hoos A, O'Day S, Weber JS, Hamid O, Lebbe C, Maio M, Binder M, Bohnsack O, Nichol G, et al. Guidelines for the evaluation of immune therapy activity in solid tumors: Immune-related response criteria. Clin Cancer Res 2009; 15:7412-20; PMID:19934295; https://doi.org/10.1158/1078-0432.CCR-09-1624
   PubMed Web of Science ®Google Scholar
• Bohnsack O, Hoos A, Ludajic K. Adaptation and modification of the immune related response criteria (IRRC): IrRECIST. ASCO, 2014:e22121.
   Google Scholar
• Nishino M. Immune-related response evaluations during immune-checkpoint inhibitor therapy: Establishing a "common language" for the new arena of cancer treatment. J Immunother Cancer 2016; 4:30; PMID:27330803; https://doi.org/10.1186/s40425-016-0134-0
   PubMed Web of Science ®Google Scholar
• Hjorth-Hansen H, Stenke L, Soderlund S, Dreimane A, Ehrencrona H, Gedde-Dahl T, Gjertsen BT, Höglund M, Koskenvesa P, Lotfi K, et al. Dasatinib induces fast and deep responses in newly diagnosed chronic myeloid leukaemia patients in chronic phase: clinical results from a randomised phase-2 study (NordCML006). Eur J Haematol 2015; 94:243-50; PMID:25082346; https://doi.org/10.1111/ejh.12423
   PubMed Web of Science ®Google Scholar
• Stewart AK, Rajkumar SV, Dimopoulos MA, Masszi T, Spicka I, Oriol A, Hájek R, Rosiñol L, Siegel DS, Mihaylov GG, et al. Carfilzomib, lenalidomide, and dexamethasone for relapsed multiple myeloma. N Engl J Med 2015; 372:142-52; PMID:25482145; https://doi.org/10.1056/NEJMoa1411321
   PubMed Web of Science ®Google Scholar
• Lonial S, Dimopoulos M, Palumbo A, White D, Grosicki S, Spicka I, Walter-Croneck A, Moreau P, Mateos MV, Magen H, et al. Elotuzumab therapy for relapsed or refractory multiple myeloma. N Engl J Med 2015; 373(7):621-31; PMID:26035255; https://doi.org/10.1056/NEJMoa1505654
   PubMed Web of Science ®Google Scholar
• Drummond MF, Sculpher MJ, Torrance GW, O'Brien BJ, Stoddart GL. Methods for the economic evaluation of health care programmes. Oxford: Oxford University Press, 2005.
   Google Scholar
• PBAC. Guidelines for preparing submissions to the Pharmaceutical Benefits Advisory Committee (Version 4.4). 2013.
   Google Scholar
• CADTH. HTA: Addendum to CADTH's guidelines for the economic evaluation of health technologies: specific guidance for oncology projects. 2009.
   Google Scholar
• CADTH. Guidelines for the economic evaluation of health technologies: Canada, 3rd edition. 2006.
   Google Scholar
• NICE. Guide to the methods of technology appraisal. 2013.
   Google Scholar
• Ribas A, Kefford R, Marshall MA, Punt CJ, Haanen JB, Marmol M, Garbe C, Gogas H, Schachter J, Linette G, et al. Phase III randomized clinical trial comparing tremelimumab with standard-of-care chemotherapy in patients with advanced melanoma. J Clin Oncol 2013; 31:616-22; PMID:23295794; https://doi.org/10.1200/JCO.2012.44.6112
   PubMed Web of Science ®Google Scholar
• Marshall MA, Ribas A, Huang B. Evaluation of baseline serum C-reactive protein (CRP) and benefit from tremelimumab compared to chemotherapy in first-line melanoma. Journal of Clinical Oncology, ASCO Annual Meeting Abstracts, 2010:2609-; ; https://doi.org/10.1200/jco.2010.28.15_suppl.2609
   Google Scholar
• Signorovitch JE, Sikirica V, Erder MH, Xie J, Lu M, Hodgkins PS, Betts KA, Wu EQ. Matching-adjusted indirect comparisons: A new tool for timely comparative effectiveness research. Value Health 2012; 15:940-7; PMID:22999145; https://doi.org/10.1016/j.jval.2012.05.004
   PubMed Web of Science ®Google Scholar