Cost-effectiveness analysis of neoadjuvant immune checkpoint inhibition vs. cisplatin-based chemotherapy in muscle invasive bladder cancer.

Multiple single-arm clinical trials showed promising pathologic complete response rates with neoadjuvant immune checkpoint inhibitors (ICIs) in muscle-invasive bladder cancer. We conducted a cost-effectiveness analysis comparing neoadjuvant ICIs with cisplatin-based chemotherapy (CBC).

We applied a decision analytic simulation model with a health care payer perspective to compare neoadjuvant ICIs vs. CBC. For the primary analysis we compared pembrolizumab with ddMVAC. We performed a secondary analysis with gemcitabine/cisplatin as CBC and exploratory analyses with atezolizumab or nivolumab/ipilimumab as ICI. We input pathologic complete response rates from trials or meta-analysis and costs from average sales price. Outcomes of interest included costs, 2-year recurrence-free survival (RFS), and incremental cost-effectiveness ratio (ICER) of cost per 2-year RFS. A threshold analysis estimated a price reduction for ICI to be cost-effective and one-way and probabilistic sensitivity analyses were performed.

The incremental cost of pembrolizumab compared with ddMVAC was $8,041 resulting in an incremental improvement of 1.5% in 2-year RFS for an ICER of $522,143 per 2-year RFS. A 21% reduction in cost of pembrolizumab would render it more cost-effective with an ICER of $100,000 per 2-year RFS. GC required an 89% pembrolizumab cost reduction to achieve an ICER of $100,000 per 2-year RFS. Atezolizumab appeared to be more cost-effective than ddMVAC.

ICIs were not cost-effective as neoadjuvant therapies, except when atezolizumab was compared with ddMVAC. Randomized clinical trials, larger sample sizes and longer follow-up are required to better understand the value of ICIs as neoadjuvant treatments.

Urologic oncology. 2021 Mar 22 [Epub ahead of print]

Ali Raza Khaki, Yong Shan, Richard E Nelson, Sapna Kaul, John L Gore, Petros Grivas, Stephen B Williams

Division of Medical Oncology, Department of Medicine, University of Washington, Seattle Cancer Care Alliance, Fred Hutchinson Cancer Research Center, Seattle, WA; Division of Oncology, Department of Medicine, Stanford University, Palo Alto, CA., Division of Urology, Department of Surgery, The University of Texas Medical Branch, Galveston, TX., IDEAS Center, VA Salt Lake City Health Care System, Salt Lake City, UT; Division of Epidemiology, Department of Internal Medicine, University of Utah, Salt Lake City, UT., Department of Preventive Medicine and Community Health, The University of Texas Medical Branch, Galveston, TX., Department of Urology, University of Washington, Seattle Cancer Care Alliance, Seattle, WA., Division of Medical Oncology, Department of Medicine, University of Washington, Seattle Cancer Care Alliance, Fred Hutchinson Cancer Research Center, Seattle, WA., Division of Urology, Department of Surgery, The University of Texas Medical Branch, Galveston, TX. Electronic address: .