Economic burden of propionic acidemia in the United States: a claims-based study

TL;DR

This study finds that propionic acidemia in the US leads to significantly higher healthcare costs and resource use compared to controls, especially in young children.

Contribution

The first study to quantify the economic burden of propionic acidemia in the US using claims data, stratified by age.

Findings

Patients with propionic acidemia had significantly higher inpatient admissions and emergency room visits compared to controls.

Annualized healthcare costs were highest in children aged 0–2 years with propionic acidemia, reaching $205,883.

Hospitalizations accounted for the majority of propionic acidemia-related costs, with inpatient admissions costing $33,575 on average.

Abstract

There is currently no prior literature evaluating the economic burden of propionic acidemia (PA) in the US. This study evaluated the healthcare resource utilization (HRU) and expenditures associated with PA, overall and stratified by age.

The IQVIA PharMetrics® Plus Claims database was used to identify patients with PA and matched (1:1) non-PA control individuals, who were stratified into 0–2, 3–6, 7–12, 13–17, and 18 + years age strata. All-cause HRU and costs were compared between the 2 cohorts by age stratum; PA-related HRU/costs were described for patients with PA.

Among 230 paired observations across age strata, patients with PA had significantly higher all-cause HRU per-person-year (PPY) than control individuals. Patients with PA had 0.47–2.31 inpatient admissions PPY compared to 0.00-0.17 for control individuals (rate ratio: 10.36–78.55, all p < 0.001). Patients with PA had 2.23–4.46 times more emergency room visits and 1.89–8.21 times more outpatient visits than control individuals as assessed by rate ratios. Patients with PA incurred significantly higher annualized all-cause total healthcare costs than control individuals, with the highest difference in the 0–2 year old ($205,883) and the lowest in the 7–12 year old age stratum ($20,168; both p < 0.001). Among patients with PA, annualized mean PA-related total medical costs were $38,724 overall; inpatient admissions accounted for most costs ($33,575). Patients with PA who experienced metabolic decompensation events (MDEs) had higher HRU and costs than those without MDEs.

Patients with PA, with or without MDEs, had significantly increased HRU and costs than matched controls without PA. Economic burden, largely driven by hospitalizations, was significantly higher among patients with PA than control individuals across all pediatric and adult age strata.

The online version contains supplementary material available at 10.1186/s13023-025-03836-8.

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