TY - JOUR
T1 - Cost-effectiveness of Pomalidomide, Carfilzomib, and Daratumumab for the Treatment of Patients with Heavily Pretreated Relapsed–refractory Multiple Myeloma in the United States
AU - Pelligra, Christopher G.
AU - Parikh, Kejal
AU - Guo, Shien
AU - Chandler, Conor
AU - Mouro, Jorge
AU - Abouzaid, Safiya
AU - Ailawadhi, Sikander
N1 - Funding Information:
K. Parikh, J. Mouro, and S. Abouzaid are employees and shareholders of Celgene Corporation. C.G. Pelligra, S. Guo, and C. Chandler, are salaried employees of Evidera, which provides consulting and research services to pharmaceutical and other such companies. Evidera received funding from Celgene Corporation to conduct the study and develop the manuscript. S. Ailawadhi has received consultant's fees from Takeda, Novartis, and Amgen, and has received research support from Pharmacyclics. The authors have indicated that they have no other conflicts of interest with regard to the content of this article.
Funding Information:
This study was funded by Celgene Corporation (Summit, New Jersey).
Publisher Copyright:
© 2017 The Authors
PY - 2017/10
Y1 - 2017/10
N2 - Purpose Pomalidomide plus low-dose dexamethasone (POM-d), daratumumab monotherapy (DARA), and carfilzomib monotherapy (CAR) have been approved for use in the treatment of patients with heavily pretreated relapsed–refractory multiple myeloma (RRMM) in the US, based on findings from the MM-002, SIRIUS, and PX-171-003-A1 studies, respectively. The objective of this study was to assess the cost-effectiveness of POM-d, DARA, and CAR in this patient population from a US payer's perspective. Methods A cost-effectiveness model was developed to estimate the cost and health outcomes over a 3-year time horizon in 3 health states: progression-free, post-progression, and death. The main efficacy data source was a matching-adjusted indirect comparison using data from the aforementioned studies. Direct medical costs were considered, including: treatment acquisition and administration (initial line and subsequent line), pre- and post-medication, prophylaxis treatment, adverse event management, and health care resource utilization. Sensitivity analyses were conducted. A scenario analysis that assumed equal efficacy across all 3 treatments was conducted. Costs, life-years, and quality-adjusted life-years were estimated and discounted at 3% per annum. Findings Over 3 years, the use of POM-d was associated with similar life-years and quality-adjusted life-years gained compared with DARA and CAR (incremental: life-years, +0.02 and +0.07, respectively; quality-adjusted life-years, +0.01 and +0.05), and with a cost less than that of DARA (–$8,919) and similar to that of CAR (–$195). Sensitivity analyses illustrated that the results were sensitive to progression-free survival, treatment duration, and drug costs. An equal efficacy scenario resulted in cost-savings relative to those of both DARA and CAR (–$11,779 and –$12,595). Implications POM-d may be a cost-effective treatment option relative to DARA or CAR in heavily pretreated patients with RRMM in the US.
AB - Purpose Pomalidomide plus low-dose dexamethasone (POM-d), daratumumab monotherapy (DARA), and carfilzomib monotherapy (CAR) have been approved for use in the treatment of patients with heavily pretreated relapsed–refractory multiple myeloma (RRMM) in the US, based on findings from the MM-002, SIRIUS, and PX-171-003-A1 studies, respectively. The objective of this study was to assess the cost-effectiveness of POM-d, DARA, and CAR in this patient population from a US payer's perspective. Methods A cost-effectiveness model was developed to estimate the cost and health outcomes over a 3-year time horizon in 3 health states: progression-free, post-progression, and death. The main efficacy data source was a matching-adjusted indirect comparison using data from the aforementioned studies. Direct medical costs were considered, including: treatment acquisition and administration (initial line and subsequent line), pre- and post-medication, prophylaxis treatment, adverse event management, and health care resource utilization. Sensitivity analyses were conducted. A scenario analysis that assumed equal efficacy across all 3 treatments was conducted. Costs, life-years, and quality-adjusted life-years were estimated and discounted at 3% per annum. Findings Over 3 years, the use of POM-d was associated with similar life-years and quality-adjusted life-years gained compared with DARA and CAR (incremental: life-years, +0.02 and +0.07, respectively; quality-adjusted life-years, +0.01 and +0.05), and with a cost less than that of DARA (–$8,919) and similar to that of CAR (–$195). Sensitivity analyses illustrated that the results were sensitive to progression-free survival, treatment duration, and drug costs. An equal efficacy scenario resulted in cost-savings relative to those of both DARA and CAR (–$11,779 and –$12,595). Implications POM-d may be a cost-effective treatment option relative to DARA or CAR in heavily pretreated patients with RRMM in the US.
KW - cost effectiveness
KW - economic models
KW - immunomodulation
KW - immunotherapy
KW - multiple myeloma
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U2 - 10.1016/j.clinthera.2017.08.010
DO - 10.1016/j.clinthera.2017.08.010
M3 - Article
C2 - 28967482
AN - SCOPUS:85030168921
SN - 0149-2918
VL - 39
SP - 1986-2005.e5
JO - Clinical Therapeutics
JF - Clinical Therapeutics
IS - 10
ER -