How Real Is Your Real-World Evidence (RWE)? Demonstration of the Need for Comprehensive Real-World Data (RWD) to Examine Patient Burden and Disease Journey in Patients With Sickle Cell Disease (SCD) in the United States (US)

OBJECTIVES: While the US has many RWD sources with which to generate RWE, oftentimes they provide incomplete answers to questions concerning disease burden/journey due to limited payer coverage or provider capture, and missing/incomplete/inadequate information on reimbursement, clinical data, and/or social determinants of health. We describe benefits of use of holistic RWD to address these concerns, using SCD as an example.

METHODS: The TriNetX Linked database comprises 22 healthcare organizations across the US that collectively provide de-identified electronic health record data combined with closed claims data for over 14 million persons; data are ingested and harmonized quarterly. We identified all persons in the database diagnosed with SCD since 2010 with insurance enrollment in 2023, and stratified them into payer-based cohorts (Commercial, Medicare, Medicaid, Dual-Eligible (Medicare+Medicaid), Multiple). We examined their demographics, clinical characteristics, and patterns of healthcare resource utilization (HCRU), during 2023.

RESULTS: A total of 8,494 patients met selection criteria, of whom 5,366 (63% of the population) were Medicaid enrollees, 522 (6%) Medicare Advantage, 2,144 (25%) Commercial, 93 (1%) Dual-Eligible, and 369 (4%) had multiple insurers. Mean age ranged from 29.3 years (Medicaid enrollees) to 56.7 years (Medicare enrollees). A total of 79%, 63%, and 55% of those in the Medicaid, Medicare, and Commercial cohorts, respectively, were Black; 5%, 4%, 5% were of Hispanic ethnicity. Medicare enrollees were more likely than Medicaid or commercially insured patients to be seen in emergency departments and/or hospitalized during 2023; Medicaid, Medicare, and commercially insured patients averaged 7.3, 13.5, and 4.3 vaso-occlusive crisis events in 2023, respectively.

CONCLUSIONS: Profiles of SCD patients in the US differ in potentially important ways in the US based at least partially by insurance coverage, as do their patterns of HCRU. It is important to account for this phenomenon when selected RWD to examine disease burden and/or journey.