Background BCG (Bacillus Calmette-Guerin), as reported by international guidelines, represents a first choice adjuvant immunotherapy for intracavitary treatment after transurethral resection of the bladder (TURB) for non muscle-invasive tumours, Ta-T1 and carcinoma in situ (CIS) at high risk of progression. When possible these patients should be treated for 6 weeks, followed by maintenance treatment for at least 1 year. If BCG is unavailable, the AIFA (Italian Medicines Agency), in several notes, although highlighting the risks and the possible disadvantages for patients, has recommended ensuring that all patients have induction treatment for 6 weeks, limiting the ongoing maintenance regime and starting a high surveillance of patients. Among the other therapeutic options to consider, it suggests to clinicians the use of radical cystectomy replacing the conservative treatment, with considerable discomforts for patients. The efficacy of different BCG strains (CONNAUGHTS, TICE, MOSCOW) seems to be comparable, according to EBM.

Purpose To evaluate the clinical and pharmacoeconomic impact on the treatment of patients with bladder cancer as result of an international lack of BCG, which lasted for several months.

Materials and methods A retrospective analysis was conducted on BCG patients treated during the drug shortage (Jan 2012-Sep 2013), evaluating their therapeutic courses in the following months (treatment suspension, final interruptions, reduced treatments, shift among different bacillus strains); furthermore the economic impact due to the drug shortage and to the following purchase abroad was evaluated.

Results Of 68 patients who were treated in the period under study only 52, who completed the treatment, are considered evaluable. These patients have undergone: 29 complete induction treatments and 3 complete maintenance treatments; 22 complete treatments with shift among different drug strains (2 induction, 20 maintenance); 12 treatments with treatment interruption (5 induction, 7 maintenance with an average of 4 administered cycles). 16 patients are still in treatment (3 induction, 13 maintenance). Of 31 patients who completed the induction, under close monitoring, 19 (61.3%) resumed the treatment after an average interruption of 3.4 months after recurrence or progression. The average cost of a bladder instillation increased from 61.8 €, before the shortage (CONNOUGHT strain) to 152.3 € (+146%) (TICE, MOSCOW strains), after importing from abroad.

Conclusions In high-risk patients, still considered suitable for conservative treatment or where that was not possible doing a radical cystectomy, induction and maintenance treatment should be the first choice, considering the high percentage of recurrence. Unfortunately the lack of drug has in many cases meant the interruption or temporary suspension of the treatment; this could cause long-lasting negative effects which will require further investigations to be confirmed.

No conflict of interest.