OBJECTIVE: Since 1983, hemin has been FDA-approved for acute intermittent 
porphyria (AIP) attacks. In 2019, FDA approved givosiran for the treatment of 
adults with acute hepatic porphyria. The objective of this research was to 
estimate and compare the total cost of AIP-related healthcare for patients 
treated with hemin or givosiran.
METHODS: A microsimulation cost model was developed to estimate the annual 
economic impact of hemin versus givosiran treatment for patients with AIP from 
the U.S. healthcare payer perspective. Hemin treatment costs were calculated 
from the Hemin Shipment Data in which patients were defined as receiving acute 
attack treatment or prophylaxis treatment based on shipment patterns. Three 
separate hemin subpopulations were considered: one attack per year, multiple 
attacks per year, and hemin prophylaxis. Treatment costs for givosiran (with 
hemin for acute attacks) were simulated based on Phase III trial efficacy 
results applied to individual treatment histories in the Hemin Shipment Data. 
Other healthcare utilization was also considered. Outcomes were annualized and 
expenditures inflated to 2019.
RESULTS: For all patients with AIP, the average annual total cost of care with 
hemin was 78% lower (difference = $482,113; 95% CI=$373,638-$594,778) than the 
average annual total cost of care with givosiran. Average annual total cost of 
care with hemin was between 46% and 92% lower than givosiran for the three hemin 
subpopulations: one attack per year (difference = $545,219; 95% 
CI=$436,584-$657,239), multiple attacks per year (difference = $459,366; 95% 
CI=$350,291-$574,403), and hemin prophylaxis (difference = $311,950; 95% 
CI=$191,898-$435,893). Cost savings with hemin were robust to one-way and 
probabilistic sensitivity as well as scenario analyses.
CONCLUSIONS: Hemin is expected to provide cost savings compared to givosiran for 
all AIP patients and subpopulations. Lower annual total costs of care with hemin 
range from $311,950 to $545,219 less depending on whether the patient uses hemin 
prophylactically or for acute treatment attacks.