OBJECTIVES: Multiple myeloma (MM) is a rare cancer that typically manifests later in life. With an increasingly aging population across the globe, understanding real-world management and burden of disease for MM is critical. A rapid literature review was conducted to identify published real-world evidence (RWE) in MM to inform future research.

METHODS: A search strategy was executed in PubMed to identify relevant studies published in English from 2012-2017. Eligible studies included adult populations (aged ≥18 years) diagnosed with MM and reported retrospective analyses of patient data collected in real-world settings. Studies were qualitatively compared based on data source, methods of analysis, and outcomes reported.

RESULTS: The review identified 43 retrospective RWE studies with an MM population. The majority were performed in the United States (n=20); 9 studies were conducted in Canada. The mean age reported was between 51-76 years with 41-71% males. Study sample sizes ranged from n=27 (chart review) to n=61,731 (administrative data). Patients were identified using ICD codes or physician diagnosis. Most studies utilized electronic medical records/patient charts (n=24), administrative claims data (n=8), or registry/specialty databases (n=6); while only 5 studies obtained patient information from multiple data sources. The most commonly reported outcomes were overall survival (n=31), complete response rate (n=12), and progression-free survival (n=11), whereas costs (n=6), healthcare utilization (n=2), and other clinical outcomes (e.g. treatment patterns) were rarely reported. Few studies examined clinically relevant subgroups, tested for competing events in survival outcomes, or accounted for missing data.

CONCLUSIONS: This review identified an unmet need for linking multiple data sources to examine treatment patterns and outcomes, such as healthcare utilization and costs, in real‑world settings among patients with MM. Future studies should also account for common issues in observational data, such as clinically relevant subgroups, competing events, and missing data, to improve rigor and interpretation of findings.