Cost-Effectiveness of Trimodal Therapy and Radical Cystectomy for MIBC in the United States - Expert Commentary
A recent study compares the cost-effectiveness of TMT versus RC from a Medicare perspective. The model used data from a 2023 multicenter retrospective study comparing matched patients treated from 2005 to 2017. The index patient was 71 years old with clinical stage T2-4aN0M0 MIBC, solitary tumor <7 cm, no/unilateral hydronephrosis, adequate bladder function, and absence of multifocal carcinoma in situ. Patients unfit for RC, radiation, or cisplatin were excluded. TMT consisted of two TURBTs plus weekly cisplatin (40 mg/m²) for 6 doses during 64 Gy radiation therapy over 6.5 weeks. RC included urinary diversion and bilateral pelvic lymphadenectomy. The model incorporated health states for progression patterns, treatment toxicities, and death, using 3-month cycles with 5-year primary analysis and 10-year sensitivity analysis. Primary outcomes were quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICER) using $100,000 per QALY willingness-to-pay threshold. Costs were estimated from a Medicare perspective in 2021 dollars with 3% annual discounting.
At 5 years, TMT cost $30,525 more than RC ($71,014 vs $40,489). TMT provided slightly higher QALYs (3.94 vs 3.87) due to reduced acute and chronic toxicity disutility. However, TMT was not cost-effective with ICER of $464,291 per QALY. At 10 years, TMT remained more expensive ($85,137 vs $49,570) and effective (6.61 vs 6.49 QALYs) but not cost-effective (ICER $308,638 per QALY). One-way sensitivity analyses showed TMT would become cost-effective if: (1) TMT costs decreased to <$17,605 (base case $41,777), or (2) TMT achieved 11.6% absolute improvement in metastasis-free survival versus RC. Monte Carlo simulations found RC cost-effective in 87% of cases at 5 years and 79% at 10 years. The model was most sensitive to initial treatment costs and long-term toxicity disutility. Two-way analyses couldn't identify toxicity combinations making TMT cost-effective, emphasizing treatment cost impact over salvage cystectomy rates.
Despite TMT's quality-of-life advantages, high treatment costs prevented cost-effectiveness at standard thresholds. Canadian data showing minimal cost differences between treatments resulted in TMT cost-effectiveness (ICER $14,270 per QALY), highlighting policy intervention potential. Study limitations include reliance on retrospective data with inherent selection bias, uncertainty in long-term TMT complications, and does not account for recent combination therapies like enfortumab vedotin-pembrolizumab. The model assumed equivalent neoadjuvant chemotherapy rates and uniform TMT protocols.
This study highlights the importance of considering the different costs of definitive therapeutic interventions in patients with MIBC. Individualized counseling for patients regarding long-term efficacy and toxicity tradeoffs is critical to guide treatment decisions.
Written by: Bishoy M. Faltas, MD, Chief Research Officer, Englander Institute for Precision Medicine, Gellert Family - John P. Leonard, MD, Research Scholar, Associate Professor of Medicine, Cell and Developmental Biology, Weill Cornell Medicine, New York- Presbyterian Hospital, NY
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