Treatment-free interval as a metric of patient experience and a health outcome of value for advanced multiple myeloma: the case for the histone deacetylase inhibitor panobinostat, a next-generation novel agent

References

• IMS Health [Internet]. Parsippany (NJ). [cited 2017 Sep 1]. Available from: http://www.imshealth.com/en/thought-leadership/quintilesims-institute/reports/ims-health-finds-global-cancer-drug-spending-crossed-$100-billion-threshold-in-2014-article
   Google Scholar
• Guo J, Pandey S, Doyle J, et al. A review of quantitative risk–benefit methodologies for assessing drug safety and efficacy—report of the ISPOR Risk–Benefit Management Working Group. Value Health. 2010;13(5):657–666.
   PubMed Web of Science ®Google Scholar
• Burnette BL, Dispenzieri A, Kumar S, et al. Treatment trade-offs in myeloma: a survey of consecutive patients about contemporary maintenance strategies. Cancer. 2013;119(24):4308–4315.
   PubMed Web of Science ®Google Scholar
• Muhlbacher AC, Lincke HJ, Nubling M. Evaluating patients’ preferences for multiple myeloma therapy, a discrete-choice-experiment. Psychosoc Med. 2008 Dec 19;5:Doc10.
   PubMedGoogle Scholar
• Acaster S, Gaugris S, Velikova G, et al. Impact of the treatment-free interval on health-related quality of life in patients with multiple myeloma: a UK cross-sectional survey. Support Care Cancer. 2013 Feb;21(2):599–607.
   PubMed Web of Science ®Google Scholar
• Farydak [package insert] [Internet]. East Hanover (NJ): Novartis; 2015 Feb. Available from: https://www.pharma.us.novartis.com/sites/www.pharma.us.novartis.com/files/farydak.pdf
   Google Scholar
• EMA. First HDAC inhibitor for multiple myeloma recommended for approval in EU [Internet]. 2015 Jun 26. Available from: http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2015/06/news_detail_002353.jsp&mid=WC0b01ac058004d5c1
   Google Scholar
• Food and Drug Administration. Guidance for industry patient-reported outcome measures: use in medical product development to support labeling claims [Internet]. [cited 2017 Sep 1]. Available from: http://www.fda.gov/downloads/Drugs/Guidances/UCM193282.pdf
   Google Scholar
• Eng KH, Hanlon BM, Bradley WH, et al. Prognostic factors modifying the treatment-free interval in recurrent ovarian cancer. Gynecol Oncol. 2015 Nov;139(2):228–235.
   PubMed Web of Science ®Google Scholar
• Richardson PG, Moreau P, Laubach JP, et al. Deacetylase inhibitors as a novel modality in the treatment of multiple myeloma. Pharmacol Res. 2016 Nov 21;pii:S1043-6618(16)31192-6.
   Google Scholar
• Gelber RD, Goldhirsch A, Cole BF, et al. A quality-adjusted time without symptoms or toxicity (Q-TWiST) analysis of adjuvant radiation therapy and chemotherapy for resectable rectal cancer. J Natl Cancer Inst. 1996;88(15):1039–1045.
   PubMedGoogle Scholar
• San Miguel JF, Hungria VTM, Yoon SS, et al. Panobinostat plus bortezomib and dexamethasone versus placebo plus bortezomib and dexamethasone in patients with relapsed or relapsed and refractory multiple myeloma: a multicenter, randomized, double-blind phase 3 trial. Lancet Oncol. 2014;15(11):1195–1206.
   PubMed Web of Science ®Google Scholar
• Moreau P, San Miguel J, Ludwig H, et al. Multiple myeloma: ESMO clinical practice guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2013;24(Suppl 6):vi133–vi137.
   PubMed Web of Science ®Google Scholar
• NCCN clinical practice guidelines in oncology (NCCN guidelines®). Multiple Myeloma. Version 2. 2017 – [2016 Oct 28].
   Google Scholar
• Jagganath S, Roy A, Kish J, et al. Patterns of utilization of bortezomib retreatment in patients with relapsed and/or refractory multiple myeloma who received ≥ 3 lines of therapy: analysis of physician chart data from US Community oncology practices. Presented at: 5th Annual Conference of the Association for Value-Based Cancer Care; 2015 May 3–6; Washington, DC.
   Google Scholar
• Harousseau JL, Palumbo A, Richardson PG, et al. Superior outcomes associated with complete response in newly diagnosed multiple myeloma patients treated with nonintensive therapy: analysis of the phase 3 VISTA study of bortezomib plus melphalan-prednisone versus melphalan-prednisone. Blood. 2010 Nov 11;116(19):3743–3750.
   PubMed Web of Science ®Google Scholar
• Dimopoulos MA, Chen C, Spencer A, et al. Long-term follow-up on overall survival from the MM-009 and MM-010 phase III trials of lenalidomide plus dexamethasone in patients with relapsed or refractory multiple myeloma. Leukemia. 2009;23(11):2147–2152.
   PubMed Web of Science ®Google Scholar
• Richardson PG, Xie W, Jagannath S, et al. A phase 2 trial of lenalidomide, bortezomib, and dexamethasone in patients with relapsed and relapsed/refractory myeloma. Blood. 2014;123(10):1461–1469.
   PubMed Web of Science ®Google Scholar
• Siegel DS, Martin T, Wang M, et al. A phase 2 study of single-agent carfilzomib (PX-171-003-A1) in patients with relapsed and refractory multiple myeloma. Blood. 2012;120(14):2817–2825.
   PubMed Web of Science ®Google Scholar
• Lendvai N, Hilden P, Devlin S, et al. A phase 2 single-center study of carfilzomib 56 mg/m2 with or without low-dose dexamethasone in relapsed multiple myeloma. Blood. 2014;124(6):899–906.
   PubMed Web of Science ®Google Scholar
• Stewart AK, Rajkumar SV, Dimopoulos MA, et al. Carfilzomib, lenalidomide, and dexamethasone for relapsed multiple myeloma. N Engl J Med. 2015;372:142–152.
   PubMed Web of Science ®Google Scholar
• Pomalyst [prescribing information]. Summit (NJ): Celgene Corporation; 2015 Apr.
   Google Scholar
• Lonial S, Weiss BM, Usmani SZ, et al. Daratumumab monotherapy in patients with treatment-refractory multiple myeloma (SIRIUS): an open-label, randomised, phase 2 trial. Lancet. 2016;387(10027):1551–1560.
   PubMed Web of Science ®Google Scholar
• Empliciti (elotuzumab) [prescribing information]. Princeton (NJ): Bristol-Myers Squibb Company; 2015 Nov.
   Google Scholar
• Moreau P, Masszi T, Grzasko N, et al. Ixazomib, an investigational oral proteasome inhibitor (PI), in combination with lenalidomide and dexamethasone (IRd), significantly extends progression-free survival (PFS) for patients (Pts) with relapsed and/or refractory multiple myeloma (RRMM): the phase 3 Tourmaline-MM1 Study (NCT01564537). Poster presented at the American Society of Hematology 57th Annual Meeting & Exposition; 2015 Dec 5–8; Orlando, FL.
   Google Scholar
• Schnipper L, Davidson N, Wollins D, et al. American Society of Clinical Oncology statement: updating the American Society of Clinical Oncology Value Framework: revisions and reflections in response to comments received [Internet]. J Clin Oncol. 2016 [cited 2016 Jun 28];34:2925–2934. Available from: http://jco.ascopubs.org/content/early/2016/05/25/JCO.2016.68.2518.full.pdf+html
   PubMed Web of Science ®Google Scholar
• Petrucci MT, Finsinger P, Chisini M, et al. Subcutaneous bortezomib for multiple myeloma treatment: patients’ benefits. Patient Prefer Adherence. 2014 Jul;4(8):939–946.
   Web of Science ®Google Scholar
• Roy A, Kish J, Bloudek L, et al. Estimating the costs of therapy in patients with relapsed and/or refractory multiple myeloma: a model framework. Am Health Drug Ben. 2015 June;8(4):204–215.
   PubMed Web of Science ®Google Scholar
• Delforge M, Minuk L, Eisenmann JC. Health-related quality-of-life in patients with newly diagnosed multiple myeloma in the FIRST trial: lenalidomide plus low-dose dexamethasone versus melphalan, prednisone, thalidomide. Haematologica. 2015 Jun;100(6):826–833.
   PubMed Web of Science ®Google Scholar
• San Miguel JF, Hungria VT, Yoon SS, et al. Overall survival of patients with relapsed multiple myeloma treated with panobinostat or placebo plus bortezomib and dexamethasone (the PANORAMA 1 trial): a randomised, placebo-controlled, phase 3 trial. Lancet Haematology. 2016;3(11):e506–e515.
   PubMed Web of Science ®Google Scholar
• Cook R. Economic and clinical impact of multiple myeloma to managed care. J Manag Care Pharm. 2008 Sep;14(7 Suppl):19–25.
   PubMed Web of Science ®Google Scholar
• Sonneveld P, Verelst S, Lewis P, et al. Review of health-related quality of life data in multiple myeloma patients treated with novel agents. Leukemia. 2013;27:1959–1969.
   PubMed Web of Science ®Google Scholar