Aim: PHEDRA (Platform for Haematology in EMEA: Data for Real World Analysis) is a unique, noninterventional project based on secondary data collection from real-world (RW) patient-level (health record) databases to understand treatment patterns in hematological malignancies. It compares ibrutinib's effectiveness with alternative treatments using RW data (RWD) and randomized clinical trials data.
Materials & methods: RWD are cleaned, validated, harmonized into a Common Data Model, and analyzed statistically alongside randomized clinical trial data. Treatment outcomes include overall and progression-free survival.
Results: To date, RWD (four databases) are available for 2840 patients in three indications, collected between 1990 and 2017.
Conclusion: PHEDRA is an innovative approach to generate evidence to inform optimal treatment decisions in RW settings.
Keywords: comparative effectiveness research; hematology; methodology.