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 identification

• EU PAS number:
  EUPAS1000000954
• Study ID:
  1000000954
• Official title and acronym:
  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
• DARWIN EU® study:
  No
• Study countries:
  European Union

  Iceland

  Norway

  Switzerland

  United Kingdom

  United States
• Study description:
  Chimeric antigen receptor T-cell (CAR-T) cell therapy has emerged as an important cellular immunotherapy for cancer treatment, with six treatments approved by the EMA since 2018. Non-interventional studies play a key role for supporting regulatory decision-making, especially when randomised controlled trials are unfeasible or unethical, which is often the case in the CAR-T cell area, where evidence on effectiveness and safety evidence often relies on single arm trials.; ; This study aimed to identify, describe and assess the data quality of a set of selected data sources relevant to the conduct of CAR-T cell non-interventional studies. It identifies 43 data sources from Europe and the US and compiled detailed metadata in a structured repository and assessed up to 22 pre-selected data sources across five dimensions of data quality, including reliability, extensiveness, coherence, relevance, and timeliness. The findings will inform future feasibility assessments, support data source selection, and enhance the interpretation of ongoing or planned real-world studies in CAR-T therapy.;
• Study status:
  Ongoing

Research institutions and networks

Institutions

Evidence and Access/Analytica Laser, Certara

France

United Kingdom (Northern Ireland)

Study timelines

Date when funding contract was signed

Actual:08/12/2024

Study start date

Actual:15/01/2025

Date of final study report

Planned:15/07/2026

Sources of funding

EMA

Regulatory

• Was the study required by a regulatory body?:
  No
• Is the study required by a Risk Management Plan (RMP)?:
  Not applicable