Health care costs and resource utilization, including patient burden, associated with novel-agent-based treatment versus other therapies for multiple myeloma: findings using real-world claims data.

BACKGROUND . Treatment of multiple myeloma has dramatically improved with the introduction of bortezomib (BOR), thalidomide (THAL), and lenalidomide (LEN). Studies assessing health care costs, particularly economic burden on patients, are limited. We conducted a claims-based, retrospective analysis of total health care costs as well as patient burden (patient out-of-pocket costs and number of ambulatory/hospital visits) associated with BOR/THAL/LEN treatment versus other therapies (OTHER). METHODS. Treatment episodes starting between January 1, 2005 and September 30, 2010 were identified from the claims database of a large U.S. health plan. Health care costs and utilization were measured during 1 year after initiation and analyzed per treatment episode. Multivariate analyses were used to adjust for patient characteristics, comorbidities, and line of treatment. RESULTS A total of 4,836 treatment episodes were identified. Mean adjusted total costs were similar between BOR ($112,889) and OTHER ($111,820), but higher with THAL ($129,412) and LEN ($158,428). Mean adjusted patient out-of-pocket costs were also similar for BOR ($3,846) and OTHER ($3,900) but remained higher with THAL ($4,666) and LEN ($4,483). Mean adjusted rates of ambulatory visits were similar across therapies (BOR: 69.67; THAL: 66.31; LEN: 65.60; OTHER 69.42). CONCLUSIONS Adjusted analyses of real-world claims data show that total health care costs, as well as patient out-of-pocket costs, are higher with THAL/LEN treatment episodes than with BOR/OTHER therapies. Additionally, similar rates of ambulatory visits suggest that any perceived advantage in patient convenience of the orally administered drugs THAL/LEN is not supported by these data.

[1]  P. Wen,et al.  Management of treatment-emergent peripheral neuropathy in multiple myeloma , 2012, Leukemia.

[2]  O. Baser,et al.  Patient survival and healthcare utilization costs after diagnosis of triple-negative breast cancer in a United States managed care cancer registry , 2012, Current medical research and opinion.

[3]  A. Bosi,et al.  The Role of Bortezomib, Thalidomide and Lenalidomide in the Management of Multiple Myeloma , 2011, PharmacoEconomics.

[4]  L. Tanoue Cancer Statistics, 2011: The Impact of Eliminating Socioeconomic and Racial Disparities on Premature Cancer Deaths , 2012 .

[5]  W. Feng,et al.  Treatment Interruptions and Non-Adherence with Imatinib and Associated Healthcare Costs , 2012, PharmacoEconomics.

[6]  J. Dürig,et al.  Potent Anti-Leukemic Activity of a Novel Cdk9 Inhibitor in Preclinical CLL Mouse Models , 2011 .

[7]  Ruth E. Brown,et al.  Cost-Effectiveness of Lenalidomide in Multiple Myeloma Patients with 1 Prior Therapy in England and Wales, , 2011 .

[8]  P. Fayers,et al.  Thalidomide for previously untreated elderly patients with multiple myeloma: meta-analysis of 1685 individual patient data from 6 randomized clinical trials. , 2011, Blood.

[9]  D. Woulfe PKD is for dense granule secretion. , 2011, Blood.

[10]  A. Jemal,et al.  Cancer statistics, 2011 , 2011, CA: a cancer journal for clinicians.

[11]  J. Snowden,et al.  Guidelines for supportive care in multiple myeloma 2011 , 2011, British journal of haematology.

[12]  M. Dimopoulos,et al.  International Myeloma Working Group consensus approach to the treatment of multiple myeloma patients who are candidates for autologous stem cell transplantation. , 2011, Blood.

[13]  H. Goldschmidt,et al.  Treatment of relapsed and refractory multiple myeloma in the era of novel agents. , 2011, Cancer treatment reviews.

[14]  T. Szucs,et al.  Cost differences among treatment options for patients with refractory myeloma previously treated with high-dose chemotherapy and autologous stem-cell transplantation: An analysis from the U.S. and Swiss perspectives. , 2011, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[15]  D. Koleva,et al.  Healthcare costs of multiple myeloma: an Italian study. , 2011, European journal of cancer care.

[16]  M. Beksac,et al.  Multiple myeloma treatment strategies with novel agents in 2011: a European perspective. , 2011, The oncologist.

[17]  E. Zamagni,et al.  Multiple myeloma, venous thromboembolism, and treatment-related risk of thrombosis. , 2011, Seminars in thrombosis and hemostasis.

[18]  A. Palumbo,et al.  Shifts in the Therapeutic Paradigm for Patients Newly Diagnosed with Multiple Myeloma: Maintenance Therapy and Overall Survival , 2011, Clinical Cancer Research.

[19]  F. Fagnani,et al.  Management of relapsed or refractory multiple myeloma in French hospitals and estimation of associated direct costs: a multi‐centre retrospective cohort study , 2011, Journal of clinical pharmacy and therapeutics.

[20]  J. Möller,et al.  Cost-effectiveness of novel relapsed-refractory multiple myeloma therapies in Norway: lenalidomide plus dexamethasone vs bortezomib , 2011, Journal of medical economics.

[21]  M. Amonkar,et al.  Economic burden of resected squamous cell carcinoma of the head and neck in a US managed-care population , 2011, Journal of medical economics.

[22]  M. Baccarani,et al.  Bortezomib with thalidomide plus dexamethasone compared with thalidomide plus dexamethasone as induction therapy before, and consolidation therapy after, double autologous stem-cell transplantation in newly diagnosed multiple myeloma: a randomised phase 3 study , 2010, The Lancet.

[23]  M. Boccadoro,et al.  Bortezomib-melphalan-prednisone-thalidomide followed by maintenance with bortezomib-thalidomide compared with bortezomib-melphalan-prednisone for initial treatment of multiple myeloma: a randomized controlled trial. , 2010, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[24]  J. Hornberger,et al.  The cost‐effectiveness of bortezomib in relapsed/refractory multiple myeloma: Swedish perspective , 2010, European journal of haematology.

[25]  B. Barlogie,et al.  Renal impairment in patients with multiple myeloma: a consensus statement on behalf of the International Myeloma Working Group. , 2010, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[26]  J. Miguel,et al.  A Phase III PETHEMA/GEM Study of Induction Therapy Prior Autologous Stem Cell Transplantation (ASCT) In Multiple Myeloma: Superiority of VTD (Bortezomib/Thalidomide/Dexamethasone) Over TD and VBMCP/VBAD Plus Bortezomib , 2010 .

[27]  D. Esseltine,et al.  The Cost-Effectiveness of Bortezomib Plus Melphalan and Prednisone Versus Lenalidomide Plus Melphalan and Prednisone with Continuous Lenalidomide Maintenance Treatment for the Initial Treatment of Multiple Myeloma In the United States , 2010 .

[28]  M. Kersten,et al.  HOVON-65/GMMG-HD4 Randomized Phase III Trial Comparing Bortezomib, Doxorubicin, Dexamethasone (PAD) Vs VAD Followed by High-Dose Melphalan (HDM) and Maintenance with Bortezomib or Thalidomide In Patients with Newly Diagnosed Multiple Myeloma (MM) , 2010 .

[29]  M. Mohty,et al.  Bortezomib plus dexamethasone is superior to vincristine plus doxorubicin plus dexamethasone as induction treatment prior to autologous stem-cell transplantation in newly diagnosed multiple myeloma: results of the IFM 2005-01 phase III trial. , 2010, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[30]  M. Ji,et al.  Repression of Id2 expression by Gfi-1 is required for B-cell and myeloid development. , 2010, Blood.

[31]  I. Higginson,et al.  Cost–effectiveness of lenalidomide in multiple myeloma , 2010, Expert review of pharmacoeconomics & outcomes research.

[32]  Xiaodong Yang,et al.  Mir-886-3p Contributes to the Regulation of the hematopoietic microenvironment by down-Regulating SDF-1α (CXCL12). , 2009 .

[33]  D. Esseltine,et al.  The Cost-Effectiveness of Bortezomib for the Initial Treatment of Multiple Myeloma in the United States. , 2009 .

[34]  J. Vose,et al.  NCCN clinical practice guidelines in oncology: multiple myeloma. , 2009, Journal of the National Comprehensive Cancer Network : JNCCN.

[35]  R. Kyle,et al.  Treatment of multiple myeloma: a comprehensive review. , 2009, Clinical lymphoma & myeloma.

[36]  M. Dimopoulos,et al.  Improved survival of patients with multiple myeloma after the introduction of novel agents and the applicability of the International Staging System (ISS): an analysis of the Greek Myeloma Study Group (GMSG) , 2009, Leukemia.

[37]  H. Brenner,et al.  Expected long-term survival of patients diagnosed with multiple myeloma in 2006–2010 , 2008, Haematologica.

[38]  M. Dimopoulos,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 , 2009, Leukemia.

[39]  Wei Chen,et al.  International Myeloma Working Group guidelines for the management of multiple myeloma patients ineligible for standard high-dose chemotherapy with autologous stem cell transplantation , 2009, Leukemia.

[40]  Bin Jiang,et al.  Bortezomib plus melphalan and prednisone for initial treatment of multiple myeloma. , 2008, The New England journal of medicine.

[41]  Lisa C. Smith,et al.  Management of side effects of novel therapies for multiple myeloma: consensus statements developed by the International Myeloma Foundation's Nurse Leadership Board. , 2008, Clinical journal of oncology nursing.

[42]  D. Dingli,et al.  Improved survival in multiple myeloma and the impact of novel therapies. , 2008, Blood.

[43]  H. Brenner,et al.  Recent major improvement in long-term survival of younger patients with multiple myeloma. , 2008, Blood.

[44]  D. Esseltine,et al.  A Budget Impact Model Comparing Resource Utilization of Four Approved Therapies for Multiple Myeloma (MM) in the US. , 2007 .

[45]  Anthony Boral,et al.  Extended follow-up of a phase 3 trial in relapsed multiple myeloma: final time-to-event results of the APEX trial. , 2007, Blood.

[46]  E. Feuer,et al.  SEER Cancer Statistics Review, 1975-2003 , 2006 .

[47]  A. Basu,et al.  Estimating marginal and incremental effects on health outcomes using flexible link and variance function models. , 2005, Biostatistics.

[48]  Terry M Therneau,et al.  Review of 1027 patients with newly diagnosed multiple myeloma. , 2003, Mayo Clinic proceedings.

[49]  J. T. Wulu,et al.  Regression analysis of count data , 2002 .

[50]  N. Dubrawsky Cancer statistics , 1989, CA: a cancer journal for clinicians.