Effect of PD-L1 testing on the cost-effectiveness and budget impact of pembrolizumab for advanced urothelial carcinoma of the bladder in the United States.

Our purpose was to evaluate the effect of PD-L1 testing on the cost-effectiveness of pembrolizumab for second-line treatment of advanced urothelial carcinoma in the bladder from the U.S. societal perspective.

We developed a microsimulation model to compare 3 treatment strategies: (1) treat all patients with standard-of-care chemotherapy, (2) treat all patients with pembrolizumab, and (3) treat patients with PD-L1-positive tumors at a ≥1% expression threshold with pembrolizumab, and all others with standard-of-care chemotherapy. Additionally, we performed a budget impact analysis based on the projected number of urothelial carcinoma patients eligible for second-line pembrolizumab treatment.

Treating all patients with chemotherapy resulted in a mean cost of $17,232 and mean effect of 0.43 quality-adjusted life-years. The PD-L1 test strategy was the most efficient strategy, with an incremental cost-effectiveness ratio of $122,933/quality-adjusted life-year. Treating all patients with pembrolizumab resulted in an incremental cost-effectiveness ratio of $197,383/quality-adjusted life-year compared to the PD-L1 test strategy. The PD-L1 test strategy would produce an incremental budget impact of $14.9 million in the first year of use compared to chemotherapy, increasing to $16.5 million in the fifth year of use. Treating all patients with pembrolizumab would produce an incremental budget impact of $19.6 million compared to the PD-L1 test strategy in its first year of use, increasing to $20.9 million by year 5.

Pembrolizumab was not cost-effective in either strategy based on a $100,000/quality-adjusted life-year willingness-to-pay threshold. Using PD-L1 testing to select for patients who may have better associated outcomes may improve the affordability of pembrolizumab.

Urologic oncology. 2018 Dec 06 [Epub ahead of print]

Steven D Criss, Davis T Weaver, Deirdre F Sheehan, Richard J Lee, Pari V Pandharipande, Chung Yin Kong

Institute for Technology Assessment, Massachusetts General Hospital, Boston, MA., Harvard Medical School, Boston, MA; Department of Medicine, Massachusetts General Hospital, Boston, MA., Institute for Technology Assessment, Massachusetts General Hospital, Boston, MA; Harvard Medical School, Boston, MA; Department of Radiology, Massachusetts General Hospital, Boston, MA., Institute for Technology Assessment, Massachusetts General Hospital, Boston, MA; Harvard Medical School, Boston, MA. Electronic address: .

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