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Abstract

Background and aims: Patients with acute intermittent porphyria (AIP) may suffer from acute non-specific attacks that often result in hospitalizations or emergency room (ER) visits. Prior to the recent approval of givosiran (November 2019), hemin was the only FDA-approved therapy for AIP attacks in the US. Our aim was to estimate the annual healthcare utilization and expenditures for AIP patients treated with hemin using real-world data.

Methods: Patients with ≥1 hemin claim and confirmed AIP diagnosis – 1 inpatient claim or 2 outpatient claims ≥30 d apart for AIP (2015–2017) or acute porphyria (prior to 2015) – were identified in MarketScan administrative claims dataset between 2007 and 2017. Continuous enrolment for ≥6 months from confirmed diagnosis was required. A secondary analysis (“active disease population”) limited the sample to adult patients with ≥3 attacks or 10 months of prophylactic use of hemin within a 12-month pre-index period. AIP-related care was defined by hemin use during an attack (daily glucose and/or hemin use) or prophylaxis (non-attack hemin use). Outcomes were annualized and expenditures were inflated to 2017.

Results: Across 10 years, patients with a confirmed AIP diagnosis (N?=?8,877) and ≥1 hemin claim (N?=?164) were restricted by ≥6 months continuous follow-up (N?=?139). AIP patients were mostly female (N?=?112; 81%), had median age of 40 and 3 years average follow-up. Annualized average total expenditures for AIP-related care were $113,477. Annualized average all-cause (any diagnosis) hospitalizations were statistically significantly lower for patients treated with hemin prophylaxis vs. acute treatment (1.0 vs. 2.1; p?<?.001). In the secondary analysis (N?=?27), annualized average total expenditures for AIP-related care were higher ($187,480).

Conclusions: For AIP patients treated with hemin, patients treated for acute attacks may use a greater number of resources compared to patients treated prophylactically.  相似文献   
2.
Passenger Facility Charge (PFC) and the Airport Improvement Program (AIP) are two major sources to finance U.S. airports. This paper develops a novel dynamic network DEA framework to investigate the substitutability between PFC and AIP funds. We find that the studied U.S. airports can substitute PFC for 8–35% of the current AIP funds and contribute significantly to the proposed plan of the US congress to cut AIP funding. In addition, the amount of PFC-for-AIP funds substitution negatively correlates with the productive efficiency of airports. The findings send an important message for future policy reforms on U.S. airport financing.  相似文献   
3.
This study investigates the effect on airport productive efficiency of two major funding sources used by US airports, namely the Airport Improvement Program (AIP) grants and the Passenger Facility Charges (PFC). A two-stage Data Envelopment Analysis (DEA) modeling approach is employed for this purpose. In the first stage, we estimate airport productive efficiency using a variable returns-to-scale DEA model with both desirable and undesirable outputs. In the second stage, random effects regression models are estimated with airport efficiency scores from the first stage as the dependent variable and PFC and a proxy for AIP grants as two of the explanatory variables. By applying the two-stage DEA model to 42 primary US airports, it is found that PFC use has a positive impact on airport productive efficiency, whereas the impact of AIP grants is negative. Multiple counterfactual scenarios are examined by altering the mix of the two types of funding sources. The results show that simultaneously raising the PFC ceiling and decreasing AIP grants could lead to greater airport productive efficiency. The US federal aviation authority would also benefit from realizing these scenarios, especially given the budgetary constraints it faces.  相似文献   
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