The national economic burden of rare disease in the United States in 2019.

ABSTRACT:

Background

To provide a comprehensive assessment of the total economic burden of rare diseases (RD) in the United States (U.S.) in 2019. We followed a prevalence-based approach that combined the prevalence of 379 RDs with the per-person direct medical and indirect costs, to derive the national economic burden by patient age and type of RD. To estimate the prevalence and the direct medical cost of RD, we used claims data from three sources: Medicare 5% Standard Analytical File, Transformed Medicaid Statistical Information System, and Optum claims data for the privately insured. To estimate indirect and non-medical cost components, we worked with the rare disease community to design and implement a primary survey.

Results

There were an estimated 15.5 million U.S. children (N =

SUBMITTER: Yang G 

PROVIDER: S-EPMC9004040 | biostudies-literature | 2022 Apr

REPOSITORIES: biostudies-literature