OBJECTIVES: Real-world data (RWD) sources can provide valuable insights into outcomes and costs of healthcare interventions. However, availability and accessibility to these sources vary across different countries and there is a critical unmet need for a comprehensive repository of RWD sources to access patient outcomes and tailor healthcare interventions more effectively, especially in areas of oncology and rare diseases. This study aimed to review the current state and potential of RWD sources in North America (USA, Canada and Mexico), with a focus on oncology and rare diseases.

METHODS: We conducted a targeted review of articles published in PubMed, using keyword-based screening and snowball methodology in accordance with Preferred Reporting Item for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Population, Intervention & Comparators, Outcomes and Study design (PICOS)-based criteria was applied to identify articles on relevant RWD sources for North American oncology and rare disease populations. Information on database characteristics and access to these sources was extracted and a repository of these identified databases was created using Microsoft PowerBI™.

RESULTS: The 95 identified RWE sources (USA=26, Canada=68 and Mexico=1) fall into various categories: administrative/claims (n=58); registries (n=25); and electronic medical records (n=12). Information available in these databases includes demographic and clinical patient characteristics, health care utilization (HCRU) as well as quality of life (QoL). While some databases are available for public access, others require an application process, fees, and licenses.

CONCLUSIONS: We identified 95 RWE databases for oncology and rare diseases, with varying accessibility. Quality of data in all identified RWD sources remained rich in clinical and economic variables. RWD is vital in oncology and rare disease research, where sample sizes are often inadequate to gather information on clinical outcomes, HCRU, QoL and other relevant patient data. Effective utilization of these RWD sources could provide relatable insights and further healthcare research in oncology and rare disease.