Using Healthcare Claims Data to Identify Health Disparities for Individuals with Diagnosed and Undiagnosed Familial Hypercholesterolemia

Background Individuals with familial hypercholesterolemia (FH) require intensive lipid-lowering therapy, starting with high-intensity statins and adding ezetimibe and PCSK9 inhibitors (PCSK9i) as needed to reach target LDL-C levels. There are limited data on disparities in the use of these therapies among individuals with FH in the US. Methods: We queried a large US healthcare claims repository consisting of 324 million individuals, focusing on prescriptions for high-intensity statins, ezetimibe, and PCSK9i in two patient groups: those diagnosed with FH (ICD-10 E.78.01) and those not diagnosed with FH but identified as having probable FH (PFH) via the FIND-FH(R) machine learning algorithm. We used multivariable regression models to examine correlations with demographic/socioeconomic variables. Results In the FH cohort (n = 85,457), 45.9% were female, 79.4% identified as White, 12.2% Black, and 8.4% as Hispanic. In the PFH cohort (n = 287,580), 42.2% were female, 78.2% White, 13.7% as Black, and 8.1% as Hispanic. Males were more likely to be prescribed high-intensity statins than females: odds ratio (OR) [95% confidence interval (CI)] = 2.05 [1.97, 2.13] and 1.60 [1.56,1.63] in the FH and the PFH cohorts, respectively. In both cohorts, White individuals were more likely to get ezetimibe, PCSK9i, or combination therapy compared to Black individuals (ORs: 1.12-1.40). Higher income was associated with increased odds of receiving these treatments (OR: 1.17-1.59 for incomes >$50,000). Higher education was linked to a higher likelihood of combination therapy (ORs [95% CI] = 1.49 [1.33, 1.68] and 1.18 [1.10, 1.27] in the FH and PFH cohorts, respectively). Conclusions: Real-world data indicate that more aggressive lipid-lowering therapy (ezetimibe and PCSK9i) is more often prescribed to White individuals, individuals with higher income, or those with advanced education, highlighting the need to improve equity in cardiovascular risk reduction for all individuals with FH.