2019P - Disparities in urothelial carcinoma (UC) drug approval: Contrasting North America and Europe

Background

The global landscape of drug approvals for UC is uncertain. We analysed the UC drug approvals characteristics across countries from North America and Europe.

Methods

We searched for drugs approvals for UC in the United States (US)[BL1], Canada (CAN), France (FRA), United Kingdom (UK), Spain (SPA), Italy (ITA), and Portugal (POR) from December 2005 to March 2024. Labels and reports from each country’s drug regulatory agency were reviewed. We collected data on date of approval, trial characteristics, efficacy, toxicity, and quality of life (QoL). Substantial clinical benefit (SCB) was based on the ESMO-MCBS criteria.

Results

Fifteen indications were approved since February 2017 (Table). Most supporting trials were single-arm (57%), open-label (93%), and phase 3 (60%). Overall survival (OS) was the primary endpoint in 46%. QoL was a secondary or exploratory endpoint in 20% and 26%, respectively. OS benefit was reported only in 40%. None of them achieved QoL benefit. SCB was found in only 46% of them. All countries approved antiPD-1/PDL1 therapy in second-line (2L) and maintenance avelumab (AVE) in first-line (1L-M). Only POR did not approve adjuvant (adj) nivolumab (NIVO). Immunotherapy for platinum-ineligible (P-unfit) patients was approved only in the US, CAN, and the UK. In third-line (3L), erdafitinib (ERDA) and enfortumab vedotin (EV) were approved only in 3 and 4 countries, respectively. Only the US approved therapies for non-muscle invasive bladder cancer (NMIBC). Drugs with SCB were predominantly approved by the US (100%), followed by CAN (71%) and FRA (71%). Table: 2019P

*Had SCB criteria. Abbreviations: ATEZO: atezolizumab, CGP: cisplatin-based plus PEM, EVP: efortumab-vedotin plus PEM, NFV: nadofaragene firadenovec, SG: Sacituzumab-govitecan, and Y: yes.

Conclusions

The US led in drugs approvals for UC, followed by CAN and FRA. Significant disparities were observed across Europe, even for drugs with SCB. Additionally, most of indications lacked evidence on OS, QoL, or SCB.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

The authors.

Funding

Has not received any funding.

Disclosure

J.C. Tapia: Other, Conference registration fee, travel, accommodations, and expenses Merck, Pfizer, Roche, MSD, Eusa Pharma UK. J. Gavira: Financial Interests, Personal, Speaker’s Bureau: Astellas, LEO Pharma; Non-Financial Interests, Personal, Training: Ipsen, BMS, Novartis, Roche. D. Matthews: Financial Interests, Personal, Speaker’s Bureau: Astellas, Janssen. M. Santoni: Financial Interests, Personal and Institutional, Advisory Board, research support and honoraria: Jansen, BMS, Ipsen, Astellas, A.A.A., Bayer. M.N. Young: Financial Interests, Personal, Invited Speaker, Invited speaker to “First Thoughts in renal cell carcinoma” conference in June 2023: Eisai. R. Flippot: Financial Interests, Institutional, Invited Speaker: Ipsen, Pfizer; Financial Interests, Institutional, Advisory Board: MSD (Merck Sharp Dohme), Eisai, Astellas, Bayer, Johnson & Johnson, Merck Serono; Financial Interests, Personal, Other, Travel Expenses: Bristol Myers Squibb; Financial Interests, Institutional, Coordinating PI: Bayer. R. Frazer: Financial Interests, Personal, Speaker, Consultant, Advisor: BMS, Eisai, Ewopharma, Ipsen, Merck, MSD, Novartis, Pfizer, Pierre Fabre, Recordati, Roche, Sanofi, Sevier. G. Anguera Palacios: Financial Interests, Personal, Speaker’s Bureau: Astellas Pharma, BMS, Ipsen, Merck, Bayer, Pfizer, Jansen. J.P. Maroto Rey: Financial Interests, Personal, Speaker, Consultant, Advisor: Astellas Pharma, Ipsen, BMS, Merck, Bayer, Janssen, Pfizer. All other authors have declared no conflicts of interest.