Amgen Statement on the Clinical and Economic Value of BLINCYTO® (Blinatumomab) in Treating Adults With Relapsed or Refractory Philadelphia Chromosome Negative B-Cell Precursor Acute Lymphoblastic Leukemia

Acute lymphoblastic leukemia (ALL) is an aggressive cancer of the blood and bone marrow, the spongy tissue inside bones where blood cells are made. The disease strikes with little warning, progresses rapidly, and is nearly always a medical emergency when the diagnosis is finally made. For adult patients with relapsed or refractory (R/R) ALL, median overall survival without treatment is just three to five months. Even with initially effective treatment which may include bone marrow transplantation, there are approximately 1,000 patients a year in the U.S. whose disease does not respond or has relapsed.

These patients have few treatment options and new approaches, including immunotherapy approaches, are actively being investigated. BLINCYTO® (blinatumomab) is indicated for the treatment of Philadelphia chromosome-negative (Ph-) R/R B-cell precursor ALL.

There is an ongoing debate regarding the value of treatments for rare diseases like Ph- R/R B-cell precursor ALL. Assessing the value of innovation is complex, and many methods can be utilized. Amgen believes that such assessments should aim to achieve a high level of transparency and place scientific rigor and patient interests at the center of the analysis. Often, value assessments rely upon a cost-effectiveness analysis based on quality-adjusted life-years (QALYs). QALY is a generic measure of disease burden, including both the quality and the quantity of life lived. It is used in economic evaluations to assess the value for money of medical interventions. This approach has not been widely endorsed for use in the U.S. and specific thresholds have not been defined for the U.S. 

As cost-effectiveness analyses are required by several health authorities around the world, Amgen has worked with experts in ALL to build a cost-effectiveness model to assess the value of BLINCYTO vs. chemotherapy in treating patients with Ph- R/R B-cell precursor ALL. The BLINCYTO Global Economic Model (B-GEM) used direct trial data from a randomized, open-label phase 3 study (TOWER) to derive the QALYs among the patients treated with BLINCYTO vs. chemotherapy. Direct medical costs including costs for medications, hospitalizations, and transplants were considered.

The B-GEM demonstrated that, in the U.S., the incremental cost per QALY gained from BLINCYTO versus chemotherapy was less than $180,000. This economic value is estimated to be in the lower band of $150,000 to $300,000 per QALY benchmarks cited as reasonable thresholds for cancer in the U.S.1 The B-GEM results will be subject to a peer-reviewed journal submission and suggest that even treatments for rare diseases like Ph- R/R B-cell precursor ALL can be cost-effective, but at thresholds that take into account the severe nature of cancer and pressing needs for additional treatments.


  1. 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)