Real-life Enfortumab Vedotin Outcomes as First-line Urothelial Carcinoma Treatment in the Non-interventional Observational and Nationwide French Study

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  KEYTRUDA
• Study drug International non-proprietary name (INN) or common name:
  PEMBROLIZUMAB
• Anatomical Therapeutic Chemical (ATC) code:
  (L01FF02) pembrolizumab

  pembrolizumab
• Medical condition to be studied:
  Chemotherapy urothelial toxicity attenuation

Population studied

• Short description of the study population:
  Urothelial cancer is the most common type of bladder cancer. Enfortumab vedotin and pembrolizumab are approved medicines being used together to treat cancer in the bladder lining (urothelial cancer) that is unresectable or metastatic. Unresectable means the cancer cannot be removed by surgery. Metastatic means the cancer has spread to other parts of the body
• Age groups:
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)
• Special population of interest:
  Frail population

  Renal impaired
• Estimated number of subjects:
  500

Study design details

• Study design:
  In Part 1 of the study, baseline data from the French EV+P EAP will be re-used and completed with secondary data collection from patients’ charts.; In Part 2 of the study, patient baseline clinical characteristics will be re-used from the previously completed EV+P EAP baseline CRF.
• Main study objective:
  In Part 1 the main aim is to learn how long people receive treatment with enfortumab vedotin and pembrolizumab. Information about people stopping treatment or starting a new treatment will be collected.; In Part 2 the main aim is to learn if enfortumab vedotin and pembrolizumab can extend the lives of people taking part in the program. Information about people’s overall health outcomes will be collected.
• Outcomes:
  Part 1: Time to permanent discontinuation of EV and P will be defined as the time from the first dose of EV+P (index date) to, whichever occurs first:; ● The date of death, or; ● The date of initiation of any subsequent therapy, or; ● The date of permanent discontinuation of both EV and P; Part 2: To describe OS for EV+P
• Data analysis plan:
  The purpose of this secondary data use study is to leverage data available in the French EV+P EAP and medical charts of these patients to understand the use of EV+P (Part 1), and to leverage data in French national claims databases to understand the long-term effectiveness of EV+P (Part 2).; Methodology (Part 1); Collected data will be recorded from the EV+P early access program (EAP) baseline CRF and completed with data extracted from medical charts previously recorded during routine clinical visits. ; Eligible patients will include: Male or female patient ≥ 18 years of age with unresectable or metastatic urothelial cancer at the time EV+P treatment was initiated. Patient treated with at least 1 dose of EV and P between Q4 2024 and Q1 2025 as part of the French EV+P 1L EAP. Inclusion criteria and data collection are designed to ensure at least 1 year of follow-up for each patient.; Methodology (Part 2); All patients who participated in the French EV+P 1L EAP will be included. As such, patients in Part 1 represent a subgroup of patients from Part 2. Baseline characteristics at inclusion in the EAP will be documented from the EAP CRF database .; Additional baseline and follow-up information will be extracted from the French medico-administrative database [SNDS]. ; Sample Size; For Part 1, up to 50 sites will be selected to reach a sample size of at least 500 patients. ; For Part 2, the actual number of patients is unknown. Inclusions will be interrupted at the end of the French EV+P EAP, once EV and P are available for this indication through standard reimbursement.; Statistical analyses; The primary and secondary effectiveness endpoints will be analyzed by subgroups where sample sizes allow.