Overview
Progression-free survival (PFS), a common endpoint used in multiple myeloma (MM) trials, is defined as the time to the earliest occurrence of disease progression or death. Disease progression in MM is determined by the International Myeloma Working Group (IMWG) Uniform Response Criteria based on imaging and biomarkers from blood, urine, and bone marrow biopsy testing; however, in routine care, these tests are often performed and recorded differently than in clinical trials.
This study was led by J&J Innovative Medicine in collaboration with academic partners and Flatiron Health, and funded in part by an FDA U01 grant. The work aimed to create and evaluate algorithmic approaches to determining progression-free survival (PFS) based on IMWG criteria using real-world data (RWD) from Flatiron Health. The results present a comprehensive assessment of important design choices for a real-world progression algorithm and the potential of using RWD to assess PFS in MM.
Why this matters
The growing availability of real-world data is enabling new tools and technology to improve the way people with cancer receive their treatment. This research showcases the potential of a novel real-world progression algorithm, however, the inclusion and assessment of performance for additional relevant data sources is important to further validate the algorithm. A validated progression algorithm would enable more reliable comparisons between PFS in RWD and clinical trials.