Amgen’s Response to Technology Assessments for Kyprolis® (Carfilzomib) in Multiple Myeloma

Mar. 31, 2016

At the same time as the pace of innovation in the fight against cancer has increased, so has the use of “technology assessments” to understand the economic value of innovation. As there is no single answer to the question of value, many different assessment frameworks have been developed, and analyses often reach divergent conclusions. Amgen thinks about value from a holistic, societal perspective with the patient at the center.

Treatment for multiple myeloma is a good example of the innovation Amgen and other companies have provided to patients. Ten years ago, patients with multiple myeloma lived two to three years from diagnosis. Today, thanks to the development of novel medicines, patients are living as long as seven to 10 years.i Scientific innovation has led to a significant change in the multiple myeloma treatment landscape over the past decade, changing the lives of patients and their families. At the same time, the various therapeutic options for relapsed or refractory multiple myeloma, including Amgen’s Kyprolis® (carfilzomib) are undergoing multiple technology assessments around the globe. Given the stakes for patients, Amgen believes that all economic reviews on the value of medicines should aim to achieve the highest level of transparency, strive for very broad stakeholder engagement, and place scientific rigor and patient interests at the center of the analysis.

Some of these technology assessments, however, express results in terms of dollars per quality-adjusted life-years (QALYs) gained relative to specific threshold values (cost-effectiveness), or in terms of payer budget impact. Amgen believes that thorough and balanced assessments should rely on direct data from rigorous comparative trials when available rather than using opaque methods to combine multiple, disparate trials to arrive at different estimates of efficacy, or make assumptions to create unrealistic “worst-case” scenarios. Results produced by independent organizations should be informed by experts, made fully transparent and available, and undergo complete and independent peer-review.

When it comes to relapsed or refractory multiple myeloma, and the impact Kyprolis has on patients with the disease, the Kyprolis Global Economic Model (K-GEM)ii provides a framework for considering value. It was co-developed with experts worldwide over a number of years, and has been used as the basis for a number of statutory Health Technology Assessment analyses around the world. The K-GEM incorporated data directly from a comparative Phase 3 trial (ASPIRE) in patients with relapsed multiple myeloma who had received one to three prior therapies. The ASPIRE trial showed that the addition of Kyprolis to lenalidomide and dexamethasone (KRd) resulted in a significant improvement (additional 8.6 months) in median progression-free survival (PFS) compared to lenalidomide plus dexamethasone (Rd) (26.3 months vs. 17.6 months, hazard ratio for progression or death= 0.69; 95 percent confidence interval [CI]: 0.57 to 0.83; p<0.0001).

The K-GEM used direct trial data from ASPIRE to derive QALYs for KRd compared to those treated with Rd alone in this lifetime model. The pre-progression costs for the multi-year KRd regimen are $356,041 with 30.8 percent ($109,779) of the pre-progression costs being attributable to Kyprolis and the remainder 69.2 percent ($246,262) being attributable to Rd and other concomitant medications. The economic value is estimated to be well below the benchmarks of $150,000 to $300,000 QALY cited as reasonable benchmarks for cancer in the U.S.iii

“The rigorous approach taken in the K-GEM, informed by clinical experts in myeloma, demonstrates the economic value of Kyprolis, with clear evidence of benefit to patients, and compelling value for patients, payers and society,” said myeloma expert Dr. Andrzej Jakubowiak.

Among technology assessments in the U.S., the American Society of Clinical Oncology (ASCO) uses a framework for comparing the relative clinical benefit, toxicity, and cost of treatment in the medical oncology setting.iv The National Comprehensive Cancer Network (NCCN) also publishes information on drug affordability in its clinical practice guidelines,v, vi which included multiple myeloma. Finally, the Institute for Clinical and Economic Review (ICER) recently published a final scoping document for its ongoing assessment.vii In all cases, but especially when direct modelling obtained from head to head trials is not employed, the analysis should fully disclose the assumptions and complex calculations used, and establish the validity of the assessment.

Amgen has engaged in balanced scientific discussions about the value of our treatments with health authorities worldwide for many years and we welcome these discussions. These assessments are transparent, include deep engagement with patient groups and health care professionals, and provide meaningful opportunities for scientific discussion and exchange. Transparency in analysis and engagement with patients and health care professionals are essential to assessing the value of innovative therapeutic technologies.


  1. (SEER. Surveillance, Epidemiology, and End Results Program. Stat Fact Sheets: Myeloma.
  2. Amgen data on file.
  3. 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)
  4. American Society of Clinical Oncology Statement: A Conceptual Framework to Assess the Value of Cancer Treatment Options
  5. NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) with NCCN Evidence Blocks
  6. NCCN Launches Evidence Blocks for its Guidelines:
  7. Treatment Options for Relapsed or Refractory Multiple Myeloma: Effectiveness, Value, and Value-Based Price Benchmarks, Draft Background and Scope