References
- Hartley-Brown MA, Sullivan DM, Baz R. State-of-the-art management of complications of myeloma and its treatment. Adv Hematol. 2010;2010:343089.
- World Health Organization. International Agency for Cancer Research. Global Cancer Observatory. 2018. [cited 2018 Oct 31]. available from: http://gco.iarc.fr/
- Terpos E, Morgan G, Dimopoulos MA, et al. International Myeloma Working Group recommendations for the treatment of multiple myeloma-related bone disease. JCO. 2013;31:2347–2357.
- Hameed A, Brady JJ, Dowling P, et al. Bone disease in multiple myeloma: pathophysiology and management. Cancer Growth Metastas. 2014;7:33–42.
- Terpos E, Christoulas D, Gavriatopoulou M, et al. Mechanisms of bone destruction in multiple myeloma. Eur J Cancer Care. 2017;26:e12761.
- von Moos R, Strasser F, Gillessen S, et al. Metastatic bone pain: treatment options with an emphasis on bisphosphonates. Support Care Cancer. 2008;16:1105–1115.
- Cocks K, Cohen D, Wisloff F, et al. An international field study of the reliability and validity of a disease-specific questionnaire module (the QLQ-MY20) in assessing the quality of life of patients with multiple myeloma. Eur J Cancer. 2007;43:1670–1678.
- Terpos E, Berenson J, Cook RJ, et al. Prognostic variables for survival and skeletal complications in patients with multiple myeloma osteolytic bone disease. Leukemia. 2010;24:1043–1049.
- Costa L, Badia X, Chow E, et al. Impact of skeletal complications on patients' quality of life, mobility, and functional independence. Support Care Cancer. 2008;16:879–889.
- Ashcroft J, Duran I, Hoefeler H, et al. Healthcare resource utilisation associated with skeletal-related events in European patients with multiple myeloma: results from a prospective, multinational, observational study. Eur J Haematol. 2018;100:479–487.
- Nash Smyth E, Conti I, Wooldridge JE, et al. Frequency of skeletal-related events and associated healthcare resource use and costs in US patients with multiple myeloma. J Med Econ. 2016;19:477–486.
- Bhowmik D, Hines DM, Intorcia M, et al. Economic burden of skeletal-related events in patients with multiple myeloma: analysis of US commercial claims database. J Med Econ. 2018;21:622–628.
- Bhowmik D, Song X, Intorcia M, et al. Examination of burden of skeletal-related events in patients naive to denosumab and intravenous bisphosphonates therapy in bone metastases from solid tumors population. Curr Med Res Opin. 2019;35:513–523.
- Nishida H. Bone-targeted agents in multiple myeloma. Hematol Rep. 2018;10:7401.
- Coleman R, Body JJ, Aapro M, et al. Bone health in cancer patients: ESMO clinical practice guidelines. Ann Oncol. 2014;25:iii124–iii137.
- Terpos E, Kleber M, Engelhardt M, et al. European Myeloma Network guidelines for the management of multiple myeloma-related complications. Haematologica. 2015;100:1254–1266.
- European Medicines Agency. XGEVA (denosumab) summary of product characteristics. 2018. [cited 2018 Nov 29]. Available from: https://www.ema.europa.eu/documents/product-information/xgeva-epar-product-information_en.pdf
- European Medicines Agency. Zometa (zoledronic acid) summary of product characteristics. 2018. [cited 2018 Nov 29]. Available from: https://www.ema.europa.eu/documents/product-information/zometa-epar-product-information_en.pdf
- Moreau P, San Miguel J, Sonneveld P, et al. Multiple myeloma: ESMO clinical practice guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2017;28:iv52–iv61.
- Raje N, Terpos E, Willenbacher W, et al. Denosumab versus zoledronic acid in bone disease treatment of newly diagnosed multiple myeloma: an international, double-blind, double-dummy, randomised, controlled, phase 3 study. Lancet Oncol. 2018;19:370–381.
- Mhaskar R, Kumar A, Miladinovic B, et al. Bisphosphonates in multiple myeloma: an updated network meta-analysis. Cochrane Database Syst Rev. 2017;12:CD003188.
- Baron R, Ferrari S, Russell RG. Denosumab and bisphosphonates: different mechanisms of action and effects. Bone. 2011;48:677–692.
- Qian Y, Bhowmik D, Bond C, et al. Renal impairment and use of nephrotoxic agents in patients with multiple myeloma in the clinical practice setting in the United States. Cancer Med. 2017;6:1523–1530.
- Dimopoulos MA, Sonneveld P, Leung N, et al. International Myeloma Working Group recommendations for the diagnosis and management of myeloma-related renal impairment. JCO. 2016;34:1544–1557.
- Anderson K, Ismaila N, Flynn PJ, et al. Role of bone-modifying agents in multiple myeloma: American Society of Clinical Oncology clinical practice guideline update. JCO. 2018;36:812.
- National Comprehensive Cancer Network (NCCN). NCCN guidelines. Multiple myeloma (version 2, 2019). 2018. [cited 2018 Nov 30]. Available from: https://www.nccn.org/professionals/physician_gls/PDF/myeloma.pdf
- Lipton A, Fizazi K, Stopeck AT, et al. Superiority of denosumab to zoledronic acid for prevention of skeletal-related events: a combined analysis of 3 pivotal, randomised, phase 3 trials. Eur J Cancer. 2012;48:3082–3092.
- Raje NS, Bhatta S, Terpos E. Role of the RANK/RANKL pathway in multiple myeloma. Clin Cancer Res. 2019;25:12–20.
- Calabro GE, La Torre G, de Waure C, et al. Disinvestment in healthcare: an overview of HTA agencies and organizations activities at European level. BMC Health Serv Res. 2018;18:148.
- Garrison LP, Jr, Kamal-Bahl S, Towse A. Toward a broader concept of value: identifying and defining elements for an expanded cost-effectiveness analysis. Value Health. 2017;20:213–216.
- Raje N, Roodman GD, Willenbacher W, et al. A cost-effectiveness analysis of denosumab for the prevention of skeletal-related events in patients with multiple myeloma in the United States of America. J Med Econ. 2018;21:525–536.
- Hechmati G, Cure S, Gouepo A, et al. Cost of skeletal-related events in European patients with solid tumours and bone metastases: data from a prospective multinational observational study. J Med Econ. 2013;16:691–700.
- Cristino J, Finek J, Jandova P, et al. Cost-effectiveness of denosumab versus zoledronic acid for preventing skeletal-related events in the Czech Republic. J Med Econ. 2017;20:799–812.
- Henk HJ, Teitelbaum A, Perez JR, et al. Persistency with zoledronic acid is associated with clinical benefit in patients with multiple myeloma. Am J Hematol. 2012;87:490–495.
- Statistik Austria. Life tables. 2017. [cited 2018 Nov 20]. Available from: http://www.statistik.at/web_de/statistiken/menschen_und_gesellschaft/bevoelkerung/sterbetafeln/index.html
- European Health and Life Expectancy Information System. Life tables – Greece. 2015. [cited 2018 Nov 20]. Available from: http://www.eurohex.eu
- National Institute for Statistics (Italy). Life tables by single age group and gender. 2016. [cited 2018 Nov 20]. Available from: http://demo.istat.it/tvm2016/
- Statistics Belgium (STATBEL). Life expectancy and life tables. 2017. [cited 2018 Nov 30]. Available from: https://statbel.fgov.be/en/themes/population/life-expectancy-and-life-tables#panel-12
- Matza LS, Chung K, Van Brunt K, et al. Health state utilities for skeletal-related events secondary to bone metastases. Eur J Health Econ. 2014;15:7–18.
- Stopeck A, Rader M, Henry D, et al. Cost-effectiveness of denosumab vs zoledronic acid for prevention of skeletal-related events in patients with solid tumors and bone metastases in the United States. J Med Econ. 2012;15:712–723.
- Matza LS, Cong Z, Chung K, et al. Utilities associated with subcutaneous injections and intravenous infusions for treatment of patients with bone metastases. Patient Prefer Adher. 2013;7:855–865.
- van Agthoven M, Segeren CM, Buijt I, et al. A cost-utility analysis comparing intensive chemotherapy alone to intensive chemotherapy followed by myeloablative chemotherapy with autologous stem-cell rescue in newly diagnosed patients with stage II/III multiple myeloma; a prospective randomised phase III study. Eur J Cancer. 2004;40:1159–1169.
- Walter E, Zehetmayr S. Guidelines zur gesundheitsökonomischen evaluation konsensuspapier [Guidelines for health-economic evaluation consensus paper]. Wien Med Wochenschr. 2006;156:628–632. [German].
- Cleemput I, Neyt M, Van de Sande S, et al. Belgian guidelines for economic evaluations and budget impact analyses. 2nd ed. Health Technology Assessment. Brussels (Belgium): Belgian Healthcare Knowledge Centre (KCE). 2012. KCE Report 183C. D/2012/10.273/54.
- National Institute for Health and Care Excellence (United Kingdom). Processing and methods guidance [PMG9]. Guide to the methods of technology appraisal. 2013. [cited 2018 Nov 20]. Available from: https://www.nice.org.uk/process/pmg9/resources/guide-to-the-methods-of-technology-appraisal-2013-pdf-2007975843781
- Capri S, Ceci A, Terranova L, et al. Guidelines for economic evaluations in Italy: recommendations from the Italian Group for Pharmacoeconomic Studies. Drug Info J. 2001;35:189–201.
- Warenverzeichnis (Austria). Official list price of drugs. 2018. [cited 2018 Nov 20]. Available from: https://warenverzeichnis.apoverlag.at/
- National Institute for Health and Disability Insurance – INAMI–RIZIV (Belgium). Reimbursed pharmaceutical drugs: list and reference files. 2018. [cited 2018 Nov 20]. Available from: https://www.inami.fgov.be/fr/themes/cout-remboursement/par-mutualite/medicament-produits-sante/remboursement/specialites/Pages/specialites-pharmaceutiques-remboursables-listes-fichiers-reference.aspx#.WRA3UlWLS70
- Ministry of Health (Greece). Corrected price list for medicines for human use, November 2017. 2018. [cited 2018 Nov 20]. Available from: http://www.moh.gov.gr/articles/times-farmakwn/deltia-timwn/5313-laquo-diorthwtiko-deltio-timwn-farmakwn-anthrwpinhs-xrhshs-noembrioy-2017-raquo
- Ministry of Health (Greece). Decree 1702/B/01.08. 2011. [cited 2018 Nov 20]. Available from: http://www.moh.gov.gr/articles/ken-eswteriko/710-ypoyrgikes-apofaseis-egkyklioi-g-g?fdl=2431
- Codifa (Italy). 2018. [cited 2018 Nov 28]. Available from: https://www.codifa.it/
- Official Gazette of the Italian Republic. General series. Ordinary supplement. 2013. [cited 2018 Nov 30]. Available from: http://www.gazzettaufficiale.it/
- Pereira J, Body JJ, Gunther O, et al. Cost of skeletal complications from bone metastases in six European countries. J Med Econ. 2016;19:611–618.
- Body JJ, Chevalier P, Gunther O, et al. The economic burden associated with skeletal-related events in patients with bone metastases secondary to solid tumors in Belgium. J Med Econ. 2013;16:539–546.
- Statistics Belgium (STATBEL). Consumer price index and health index. 2017. [cited 2018 Nov 20]. Available from: https://statbel.fgov.be/en/open-data/consumer-price-index-and-health-index
- European Central Bank. Euro reference exchange rate: Swiss Franc (CHF). Average: 1 January 2017 to 31 December 2017. [cited 2018 Nov 20]. Available from: https://www.ecb.europa.eu/stats/policy_and_exchange_rates/euro_reference_exchange_rates/html/eurofxref-graph-chf.en.html
- European Commission. Eurostat. Gross domestic product at market prices. 2017. [cited 2018 Nov 2]. Available from: https://ec.europa.eu/eurostat/tgm/table.do?tab=table&init=1&language=en&pcode=tec00001&plugin=0&tableSelection=1
- European Network for Health Technology Assessment. Methods for health economic evaluations – a guideline based on current practices in Europe. 2015. [cited 2018 Nov 5]. Available from: https://www.eunethta.eu/wp-content/uploads/2018/03/Methods_for_health_economic_evaluations.pdf
- Marseille E, Larson B, Kazi DS, et al. Thresholds for the cost-effectiveness of interventions: alternative approaches. Bull WHO. 2015;93:118–124.
- Hutubessy R, Chisholm D, Edejer TT. Cost Eff Resour Alloc. 2003;1:8.
- Robinson LA, Hammitt JK, Chang AY, et al. Understanding and improving the one and three times GDP per capita cost-effectiveness thresholds. Health Policy Plan. 2017;32:141–145.
- Orphanet. Orphanet report series: rare diseases collection. Rare disease registries in Europe. 2018. [cited 2018 Nov 8]. Available from: https://www.orpha.net/orphacom/cahiers/docs/GB/Registries.pdf
- Nicod E, Annemans L, Bucsics A, et al. HTA programme response to the challenges of dealing with orphan medicinal products: process evaluation in selected European countries. Health Policy. 2019;123:140–151.
- Institute for Clinical or Economic Review (ICER). Assessing the effectiveness and value of drugs for rare conditions: a technical brief for the ICER orphan drug assessment & pricing summit. 2017. [cited 2018 Nov 5]. Available from: https://icer-review.org/wp-content/uploads/2017/02/ICER_Assessing-the-Value-of-Drugs-for-Rare-Conditions_051017.pdf
- National Institute for Health and Care Excellence (United Kingdom). NICE and NHS England consultation on changes to the arrangements for evaluating and funding drugs and other health technologies assessed through NICE’s technology appraisal and highly specialised technologies programmes. 2017. [cited 2018 Nov 5]. Available from: https://www.nice.org.uk/Media/Default/About/what-we-do/NICE-guidance/NICE-technology-appraisals/board-paper-TA-HST-consultation-mar-17-HST-only.pdf
- Raab MS, Cavo M, Delforge M, et al. Multiple myeloma: practice patterns across Europe. Br J Haematol. 2016;175:66–76.
- Kim C, Hernandez RK, Cyprien L, et al. Patterns of bisphosphonate treatment among patients with multiple myeloma treated at oncology clinics across the USA: observations from real-world data. Support Care Cancer. 2018;26:2833–2841.
- Lebret T, Casas A, Cavo M, et al. The use of bisphosphonates in the management of bone involvement from solid tumours and haematological malignancies – a European survey. Eur J Cancer Care. 2017;26:e12490.
- Allen N, Liberti L, Walker SR, et al. A comparison of reimbursement recommendations by European HTA agencies: is there opportunity for further alignment? Front Pharmacol. 2017;8:384.
- European Commission. Mapping of HTAs methodologies in EU and Norway. 2017. [cited 2018 Nov 5]. Available from: https://ec.europa.eu/health/sites/health/files/technology_assessment/docs/2018_mapping_methodologies_en.pdf
- Yfantopoulos J, Christopoulou A, Hatzikou M, et al. The importance of economic evaluation in healthcare decision-making – a case of denosumab versus zoledronic acid from Greece. Third-party payer perspective. Forum Clin Oncol. 2013;4:25–31.
- Koo K, Lam K, Mittmann N, et al. Comparing cost-effectiveness analyses of denosumab versus zoledronic acid for the treatment of bone metastases. Support Care Cancer. 2013;21:1785–1791.
- Dellis A, Papatsoris A. Cost-effectiveness of denosumab as a bone protective agent for patients with castration resistant prostate cancer. Expert Rev Pharmacoecon Outcomes Res. 2016;16:5–10.
- Shapiro CL, Moriarty JP, Dusetzina S, et al. Cost-effectiveness analysis of monthly zoledronic acid, zoledronic acid every 3 months, and monthly denosumab in women with breast cancer and skeletal metastases: CALGB 70604 (Alliance). JCO. 2017;35:3949–3955.
- Snedecor SJ, Carter JA, Kaura S, et al. Denosumab versus zoledronic acid for treatment of bone metastases in men with castration-resistant prostate cancer: a cost-effectiveness analysis. J Med Econ. 2013;16:19–29.
- Snedecor SJ, Carter JA, Kaura S, et al. Cost-effectiveness of denosumab versus zoledronic acid in the management of skeletal metastases secondary to breast cancer. Clin Ther. 2012;34:1334–1349.
- Ford J, Cummins E, Sharma P, et al. Systematic review of the clinical effectiveness and cost-effectiveness, and economic evaluation, of denosumab for the treatment of bone metastases from solid tumours. Health Technol Assess. 2013;17:1–386.
- Xie J, Diener M, Sorg R, et al. Cost-effectiveness of denosumab compared with zoledronic acid in patients with breast cancer and bone metastases. Clin Breast Cancer. 2012;12:247–258.
- Xie J, Namjoshi M, Wu EQ, et al. Economic evaluation of denosumab compared with zoledronic acid in hormone-refractory prostate cancer patients with bone metastases. JMCP. 2011;17:621–643.
- Carter JA, Botteman MF. Health-economic review of zoledronic acid for the management of skeletal-related events in bone-metastatic prostate cancer. Expert Rev Pharmacoecon Outcomes Res. 2012;12:425–437.
- Lothgren M, Ribnicsek E, Schmidt L, et al. Cost per patient and potential budget implications of denosumab compared with zoledronic acid in adults with bone metastases from solid tumours who are at risk of skeletal-related events: an analysis for Austria, Sweden and Switzerland. Eur J Hosp Pharm. 2013;20:227–231.
- Cavo M, Terpos E, Bargay J, et al. The multiple myeloma treatment landscape: international guideline recommendations and clinical practice in Europe. Expert Rev Hematol. 2018;11:219–237.
- Ramsey SD, Willke RJ, Glick H, et al. Cost-effectiveness analysis alongside clinical trials II – an ISPOR Good Research Practices Task Force report. Value Health. 2015;18:161–172.