BACKGROUND: Real-world evidence (RWE) plays a key role in regulatory and healthcare decision-making, but a lack of reproducibility and the potentially fragmentated nature of generated evidence may limit its utility for clinical decision-making. Heterogeneity in RWE resulting from inconsistent application of methodologies across data sources should be minimized through harmonization. By combining evidence-generation approaches and harmonizing methodologies, the FOUNTAIN platform aims to address 2 main objectives in patients with chronic kidney disease (CKD) and type 2 diabetes (T2D): (1) to describe utilization of different treatment options and event rates of clinical and laboratory outcomes, and (2) to assess the real-world effectiveness and safety of finerenone.
METHODS: Guided by an executive advisory committee of multidisciplinary experts and patient representatives, the FOUNTAIN platform aims to harmonize RWE generation across a portfolio of research projects, including the FINEGUST and FIRST research programs and a common data model (CDM)–based program for rapid RWE generation. The platform employs 3 strategies toward this aim: (1) establish long-term collaborations with pharmacoepidemiology research partners (RPs) and multidisciplinary experts; (2) combine different approaches for RWE generation, including research collaborations wherein RPs lead the execution of a global protocol in their local data source, as well as execution of a common protocol in multiple data sources through federated data networks mapped to the same CDM; and (3) ensure harmonization of medical definitions, overall methodology, and curation of reproducible artifacts across all studies conducted within FOUNTAIN.
RESULTS: Currently, the FOUNTAIN platform includes 9 RP collaborations and 5 CDM-mapped data sources from 7 countries (US, UK, CN, JP, NL, ES, DK). These databases and RPs were selected after a feasibility evaluation based on a fit-for-purpose framework. Six multicountry, multidatabase, cohort studies are ongoing to describe patient populations, current standard of care, comorbidity profiles, healthcare resource use, and treatment effectiveness and safety in different patient populations with CKD and T2D.
CONCLUSION: The FOUNTAIN platform aims to harmonize RWE generation across data sources and build research partnerships, ultimately producing RWE that can support decision-making among health authorities and inform clinical practice to improve care for patients with CKD and T2D.
