Identification, Characterisation, and Data Quality Assessment of Data Sources for Real-World Safety and Effectiveness Studies of Chimeric antigen receptor T-cell (CAR-T cell) therapies

Study type

• Study topic:
  Human medicinal product
• Study type:
  Not applicable
• Scope of the study:
  Feasibility analysis

  Scoping review (including literature review)
• If ‘Not applicable’, further details on the study type:
  This is a description, characterisation and data quality assessment of real-world data sources for CAR-T cell therapy research.
• Data collection methods:
  No individual level data collected for the purpose of the study

Study drug and medical condition

• Medicinal product name:
  ABECMA

  BREYANZI

  CARVYKTI

  KYMRIAH

  TECARTUS

  YESCARTA
• Anatomical Therapeutic Chemical (ATC) code:
  (L01XL07) idecabtagene vicleucel

  idecabtagene vicleucel

  (L01XL08) lisocabtagene maraleucel

  lisocabtagene maraleucel

  (L01XL05) ciltacabtagene autoleucel

  ciltacabtagene autoleucel

  (L01XL04) tisagenlecleucel

  tisagenlecleucel

  (L01XL06) brexucabtagene autoleucel

  brexucabtagene autoleucel

  (L01XL03) axicabtagene ciloleucel

  axicabtagene ciloleucel

Population studied

• Short description of the study population:
  This study included European and US real‑world data sources that capture data relating to CAR-T cell therapy recipients.

Study design details

• Study design:
  This study characterised 43 identified CAR-T cell therapy data sources and a data quality assessment was conducted for up to 22 pre-selected data sources.
• Main study objective:
  To identify, describe, and assess the quality of data sources relevant for conducting CAR T cell non interventional studies.
• Setting:
  The study comprised data sources, such as registries, claims databases, and electronic health records from Europe and the US involving CAR-T cell recipients, identified using bibliographic databases (PubMed, Embase), online searches, and the HMA EMA Catalogue of real world data sources.
• Interventions:
  This project did not involve any patient‑level intervention and is neither a clinical trial nor a non‑interventional study; rather, it was a non‑interventional methodological assessment conducted solely to characterise and evaluate the data quality of selected real‑world data sources for potential future regulatory studies on CAR‑T cell therapies.
• Outcomes:
  A searchable Excel‑based data repository was developed using data obtained from published sources, featuring a user‑friendly interface and enabling filtering of data sources by key characteristics such as disease area or geography. It includes foundational data quality and descriptive metrics for 43 data sources and metrics. The data quality assessment was conducted for 22 data sources, with capture of metrics of data quality collected via published sources and direct contact with data owners.