Statement of Need

About 500,000 people in the United States are currently affected by bladder cancer, making the disease the fourth most common cancer among men and the eleventh most common among women.

About 70% of bladder cancer patients present with superficial, nonmuscle invasive disease. Intravesical administration of bacillus Calmette-Guérin (BCG) following resection of the nonmuscle invasive tumor is the current standard management strategy. Patients with carcinoma in situ (CIS) treated with intravesical BCG have a 60% to 70% chance of a complete and durable response. About 20% of patients discontinue BCG therapy due to local and systemic toxicity, however, and 30% to 40% show evidence of recurrence following treatment. Patients who cannot tolerate BCG therapy or have a tumor recurrence after one or more inductions need careful assessment and consideration of salvage therapies. The therapeutic options include a repeat course of BCG alone, BCG plus interferon, and/or intravesical chemotherapy. For some patients, radical cystectomy may be the best option for long-term disease-free survival. Unfortunately, because of comorbid conditions, the patient may not be clinically suitable for this major surgical procedure or may not be accepting of the surgery’s physical and psychological consequences.

Several chemotherapeutic agents are used for intravesical therapy. Gemcitabine has shown efficacy when used systemically against advanced bladder cancers; this has prompted the examination of its use intravesically. Although this form of administration has acceptable safety, its usefulness in the treatment of BCG-refractory CIS is unclear. For patients with BCG-refractory CIS, valrubicin is the only FDA-approved agent for salvage therapy, but response rates are only approximately 20% at 6 months. If a patient does not have a complete response or if CIS recurs, cystectomy must be reconsidered.

In BCG-refractory patients, there is a lack of agreement and clarity among urologists regarding when valrubicin should be offered, or when other clinical options should be considered prior to radical cystectomy. Practicing urologists need to be aware of the full range of treatment options available for this high-risk patient population, who receive little benefit from additional courses of BCG and are either unwilling or inappropriate surgical candidates.

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