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

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

Research output: Contribution to journalArticlepeer-review


Background: 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). Methods: 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. Results: 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. Conclusions: 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.

Original languageEnglish (US)
Pages (from-to)732.e9-732.e16
JournalUrologic Oncology: Seminars and Original Investigations
Issue number10
StatePublished - Oct 2021
Externally publishedYes


  • Cost effectiveness analysis
  • Immunotherapy
  • Muscle invasive bladder cancer
  • Neoadjuvant
  • Urothelial carcinoma

ASJC Scopus subject areas

  • Oncology
  • Urology


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