Economic burden of propionic acidemia in the United States: a claims-based study
Abstract Background 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. Methods The IQVIA PharMetrics® Plus Claim...
Saved in:
| Main Authors: | , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
BMC
2025-06-01
|
| Series: | Orphanet Journal of Rare Diseases |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s13023-025-03836-8 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1850102497746616320 |
|---|---|
| author | Sue Perera Geetanjoli Banerjee Erin E. Cook Fan Mu Mu Cheng Adina Zhang Jessie Jie Lan Lin Zou Vanja Sikirica |
| author_facet | Sue Perera Geetanjoli Banerjee Erin E. Cook Fan Mu Mu Cheng Adina Zhang Jessie Jie Lan Lin Zou Vanja Sikirica |
| author_sort | Sue Perera |
| collection | DOAJ |
| description | Abstract Background 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. Methods 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. Results 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. Conclusions 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. |
| format | Article |
| id | doaj-art-4c81ff8a03ef4c559f0a7d8bab5619bd |
| institution | DOAJ |
| issn | 1750-1172 |
| language | English |
| publishDate | 2025-06-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj-art-4c81ff8a03ef4c559f0a7d8bab5619bd2025-08-20T02:39:44ZengBMCOrphanet Journal of Rare Diseases1750-11722025-06-0120111210.1186/s13023-025-03836-8Economic burden of propionic acidemia in the United States: a claims-based studySue Perera0Geetanjoli Banerjee1Erin E. Cook2Fan Mu3Mu Cheng4Adina Zhang5Jessie Jie Lan6Lin Zou7Vanja Sikirica8Moderna Therapeutics, IncModerna Therapeutics, IncAnalysis Group, Inc.Analysis Group, Inc.Analysis Group, Inc.Analysis Group, Inc.Analysis Group, Inc.Analysis Group, Inc.Moderna Therapeutics, IncAbstract Background 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. Methods 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. Results 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. Conclusions 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.https://doi.org/10.1186/s13023-025-03836-8AgeEconomic burdenHealthcare costsHealthcare resource utilizationMetabolic decompensation eventsPropionic academia |
| spellingShingle | Sue Perera Geetanjoli Banerjee Erin E. Cook Fan Mu Mu Cheng Adina Zhang Jessie Jie Lan Lin Zou Vanja Sikirica Economic burden of propionic acidemia in the United States: a claims-based study Orphanet Journal of Rare Diseases Age Economic burden Healthcare costs Healthcare resource utilization Metabolic decompensation events Propionic academia |
| title | Economic burden of propionic acidemia in the United States: a claims-based study |
| title_full | Economic burden of propionic acidemia in the United States: a claims-based study |
| title_fullStr | Economic burden of propionic acidemia in the United States: a claims-based study |
| title_full_unstemmed | Economic burden of propionic acidemia in the United States: a claims-based study |
| title_short | Economic burden of propionic acidemia in the United States: a claims-based study |
| title_sort | economic burden of propionic acidemia in the united states a claims based study |
| topic | Age Economic burden Healthcare costs Healthcare resource utilization Metabolic decompensation events Propionic academia |
| url | https://doi.org/10.1186/s13023-025-03836-8 |
| work_keys_str_mv | AT sueperera economicburdenofpropionicacidemiaintheunitedstatesaclaimsbasedstudy AT geetanjolibanerjee economicburdenofpropionicacidemiaintheunitedstatesaclaimsbasedstudy AT erinecook economicburdenofpropionicacidemiaintheunitedstatesaclaimsbasedstudy AT fanmu economicburdenofpropionicacidemiaintheunitedstatesaclaimsbasedstudy AT mucheng economicburdenofpropionicacidemiaintheunitedstatesaclaimsbasedstudy AT adinazhang economicburdenofpropionicacidemiaintheunitedstatesaclaimsbasedstudy AT jessiejielan economicburdenofpropionicacidemiaintheunitedstatesaclaimsbasedstudy AT linzou economicburdenofpropionicacidemiaintheunitedstatesaclaimsbasedstudy AT vanjasikirica economicburdenofpropionicacidemiaintheunitedstatesaclaimsbasedstudy |