Medicaid Service Utilization by Beneficiaries with Rare Diseases

This national analysis examines Medicaid enrollees with diagnosed rare diseases (RDs) using 2020–2023 Transformed Medicaid Statistical Information System (T-MSIS) claims data, with primary emphasis on 2023 population characteristics, service utilization, eligibility pathways, and geographic distribution. The study identified more than 6 million Medicaid beneficiaries with an RD, including substantial proportions of children, young adults, and rural residents. Compared with other Medicaid enrollees, beneficiaries with RDs had markedly higher use of inpatient, emergency department, long-term care, outpatient, primary care, and prescription drug services. Nearly one-third were dually eligible for Medicare and Medicaid, and 44.7% qualified through home- and community-based services (HCBS) waivers, underscoring the importance of these pathways for access to care. These findings highlight Medicaid’s central role in financing and delivering care for people with rare diseases and establish an important baseline for evaluating future policy changes affecting benefits, eligibility, rural access, and long-term services and supports.