Bladder Cancer: Treatment Update and Managed Care Impact

With the introduction of the first gene therapy for bladder cancer and its associated high costs, management criteria around the specific patient population and indications will be key.

An estimated 82,290 adults in the U.S. will be diagnosed with bladder cancer in 2023.1 Urothelial carcinoma (UC) is the most common form of bladder cancer, accounting for about 95% of all bladder cancer cases.2 The rate of bladder cancer steadily declined an estimated 2% yearly from 2015 to 2019. About 90% of people with bladder cancer are over age 55, and the average age of those diagnosed is 73. It is the fourth most common cancer in men but is less common in women.3

Andrew J. Colby, R.Ph., MBA VP, Enterprise Pharmacy Solutions

Estimates state that about 16,710 deaths from bladder cancer will occur in the U.S. in 2023.1 While the death rate for bladder cancer decreased by over 2% annually in recent years, bladder cancer is the eighth most common cause of cancer death among men in the U.S.1 The five-year relative survival rate for bladder cancer is 77%, though this depends on various factors, including the cancer’s stage, the patient’s age and general health, and the effectiveness of the treatment plan.1 In cases where the bladder cancer has not spread beyond the inner layer of the bladder wall, about half of diagnoses, the five-year survival rate is 96%.1 The survival rate decreases as the cancer metastasizes to surrounding tissue and other organs.1

Treatment Landscape

Drug therapy for bladder cancer will vary depending on the stage and type of disease, but may include chemotherapy, immunotherapy, targeted therapy, or gene therapy. Common systemic chemotherapy regimens for bladder cancer include: cisplatin and gemcitabine, carboplatin and gemcitabine, MVAC (combination of methotrexate, vinblastine, doxorubicin, and cisplatin), dose-dense-MVAC, docetaxel or paclitaxel, and pemetrexed.4

Local or systemic immunotherapy may be used to treat bladder cancer. The most common local immunotherapy for bladder cancer is Bacillus Calmette-Guérin (BCG).4 Systemic immunotherapies used to treat bladder cancer include avelumab, nivolumab, and pembrolizumab. With proper genomic testing to identify genetic factors and biomarkers in a patient’s tumor, a healthcare provider may find that targeted therapy is the most appropriate course of care.4

Erdafitinib, enfortumab vedotin-ejfv, and sacituzumab govitecan are targeted therapies for specific types and stages of UC.4 For example, the first targeted therapy, erdafitinib (BALVERSA®), was approved by the U.S. Food & Drug Administration (FDA) in 2019 to treat adult patients with locally advanced or metastatic UC with particular FGFR3 or FGFR2 genetic changes and has a specific FDA-approved companion test to identify appropriate patients.5

Recent Approvals

Enfortumab vedotin-ejfv (PADCEV®) + Pembrolizumab (KEYTRUDA®)

The FDA granted accelerated approval to enfortumab vedotin-ejfv (PADCEV®) with pembrolizumab (KEYTRUDA®) in April 2023 for patients with locally advanced or metastatic UC who are ineligible for cisplatin-containing chemotherapy.6 A multi-cohort study (dose escalation cohort, Cohort A, Cohort K), EV-103/KEYNOTE-869 evaluated efficacy.7 Single-arm cohorts — dose escalation cohort