Amgen’s Statement on Cost Effectiveness of KYPROLIS® (carfilzomib) Compared to Velcade® (bortezomib) in Relapsed or Refractory Multiple Myeloma, Published in Expert Review of Hematology

Prior to 2012, patients with multiple myeloma, a uniformly fatal and incurable blood cancer, were primarily cycled between two types of novel treatments.1 In 2000, less than 35 percent of patients with multiple myeloma survived five years.2 Today there are more than a dozen approved medicines and patients are living twice as long with their disease (7-10 years).2, 3

Along with the increasing number of effective options for patients has come an increasing number of Value Frameworks and Technology Assessments to address the economic value of various innovations. Not surprisingly, the variable assessment methods used have generated dissimilar results. Amgen believes that assessment of value must thoughtfully combine the most rigorous data from both randomized trials and real-world sources, especially in disease areas like multiple myeloma that are rapidly evolving.  

On November 30, 2017, results from an analysis using the KYPROLIS Global Economic Model (K-GEM) were published in Expert Review of Hematology. Using data from the Phase 3 head-to-head ENDEAVOR trial, the K-GEM showed that in the U.S., KYPROLIS® (carfilzomib) in combination with dexamethasone (Kd) administered twice-weekly at 56 mg/m2, is cost-effective compared to Velcade® (bortezomib) and dexamethasone in patients with relapsed or refractory multiple myeloma. For patients treated with Kd, the analysis demonstrated an incremental cost-effectiveness ratio (ICER) of $121,828 per Quality-Adjusted Life Year (QALY) gained. Even taking into account an up to 85 percent discount in price for Velcade, the ICER was less than $150,000 per QALY for patients treated with the KYPROLIS regimen. Additionally, the ICER was even better for patients treated at first relapse ($114,793/QALY).

The researchers concluded that Kd provided significant value for patients with relapsed or refractory multiple myeloma when its cost per QALY gained is contrasted against willingness-to-pay estimates of $150,000 to $300,000 per QALY, which are cited as reasonable benchmarks for assessing the value of cancer treatments in the U.S.4

The K-GEM is a robust, multi-year, and multi-stakeholder framework used to consider value based on actual head-to-head trials.5 The K-GEM analysis favors the most recent data from head-to-head Phase 3 trials, and incorporates feedback from clinical experts, payers, and health economists worldwide to make realistic, generalizable and transparent assumptions.

As previously reported, ENDEAVOR was a superiority trial which showed patients treated with Kd achieved a median progression-free survival of 18.7 months compared to 9.4 months in those receiving Velcade and dexamethasone (Vd) (HR=0.53; 95 percent CI: 044, 0.65; p<0.0001), a 9.3 month improvement in the Kd arm.6 The study also met the key secondary endpoint of overall survival (OS), demonstrating that Kd offered patients a statistically significant 21 percent reduction in the risk of death and increased OS by 7.6 months compared to Vd (median OS 47.6 months for Kd versus 40.0 months for Vd, HR=0.79; p=0.01).7 The most common adverse events (greater than or equal to 20 percent) in the KYPROLIS arm were anemia, diarrhea, pyrexia, dyspnea, fatigue, hypertension, cough, insomnia, upper respiratory tract infection, peripheral edema, nausea, bronchitis, asthenia, back pain, thrombocytopenia and headache.8

Amgen believes that robust methodologies coupled with transparency in analysis and engagement with patients and healthcare professionals are essential to assessing the value of innovative therapeutic technologies. These data confirm that along with being a superior proteasome inhibitor, KYPROLIS is also the more cost-effective option versus Velcade for relapsed or refractory multiple myeloma patients.


  1. Warren JL, et al. Multiple myeloma treatment transformed: a population-based study of changes in initial management approaches in the United States. Journal of Clinical Oncology. 2013; 31(16): 1984-89.
  2. National Cancer Institute. Surveillance, Epidemiology, and End Results Program. 2016. Available at Accessed December 13, 2016.
  3. Multiple myeloma drug therapies. The MMRF. 2016. Available at Accessed December 13, 2016.
  4. Neumann PJ, Cohen JT, Weinstein MC. Updating cost-effectiveness — the curious resilience of the $50,000-per-QALY threshold. N Engl J Med. 2014; 371:796-797.
  5. Amgen data on file.
  6. Dimopoulos, M, et al. Carfilzomib and Dexamethasone Versus Bortezomib and Dexamethasone for Patients With Relapsed or Refractory Multiple myeloma (ENDEAVOR): A Randomised, Phase 3, Open-label, Multicentre study. Lancet Oncol. 2016; 17(1):27-38.
  7. Dimopoulos, M, et al. Carfilzomib or Bortezomib in Relapsed or Refractory Multiple Myeloma (ENDEAVOR): An Interim Overall Survival Analysis of an Open-label, Randomised, Phase 3 Trial. Lancet Oncol. 2017; 18(10):1327-1337.