Objective: Turkish Ministry of Health defines “off-label” drug use as the use of licensed pharmaceutical products at doses outside the approved indication and the use of unlicensed medicinal products that are imported for the purpose of individual treatment. The aim of this study was to evaluate the use of off-label or unlicensed drugs in urological cancers in order to understand the perspective of Turkey within this area of healthcare provisions.

Methods: This study involved metastatic bladder cancer patients (n=105) receiving paclitaxel or other off-label drugs, metastatic renal cell cancer patients (n=194) receiving sorafenib, sunitinib or other off-label drugs, and metastatic testicular cancer patients (n=44) receiving paclitaxel, gemcitabine or other off-label drugs in Turkey. A computer search was performed using patient-based database of Turkish Medicines and Medical Devices Agency (TITCK).

Results: According to data from TITCK database, the number of applications for off-label drug use was 86 for metastatic bladder cancer, 136 for metastatic renal cell cancer and 44 for metastatic testicular cancer. The approval rates for off-label drugs were 90.47% for metastatic bladder cancer, 62.88% for metastatic renal cell cancer and 88.63% for metastatic testicular cancer. University hospitals made the majority of applications for metastatic bladder cancer (79.89%), metastatic renal cell cancer (64.76%) and metastatic testicular cancer (79.89%). The most preferred off-label drugs for bladder, renal and testicular cancers were paclitaxel (84.04%), sorafenib (68.42%) and paclitaxel (43.24%), respectively.

Conclusion: Off-label drug use in urological cancers is increasing in Turkey. If the use of off-label drugs increases in parallel to the use of off-label urology drugs, it is necessary to define new ways of evaluating applications.