Cost savings with hemin versus givosiran for the treatment of patients with acute intermittent porphyria (AIP)

Abstract

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

Prophylactic Heme Arginate Infusion for Acute Intermittent Porphyria

“…The financial burden of treatment for patients with AIP has been the focus of several recent studies (Gouya et al, 2018;Blaylock et al, 2020;Massachi et al, 2020). While FDAapproved givosiran has been shown to reduce the severity and frequency of porphyric attacks in acute hepatic porphyria (Balwani et al, 2020), hemin was found to be the less costly option compared to givosiran for AIP patients due accessibility and current cost for use (Massachi et al, 2020).…”

“…The financial burden of treatment for patients with AIP has been the focus of several recent studies (Gouya et al, 2018;Blaylock et al, 2020;Massachi et al, 2020). While FDAapproved givosiran has been shown to reduce the severity and frequency of porphyric attacks in acute hepatic porphyria (Balwani et al, 2020), hemin was found to be the less costly option compared to givosiran for AIP patients due accessibility and current cost for use (Massachi et al, 2020). Based on the published study by Massachi et al, 2020, the total cost of care with hemin for patients with single/multiple attacks per year, and hemin prophylaxis was between 46 and 92% lower compared to givosiran treatment in (Massachi et al, 2020).…”

“…Recently, an exciting RNA interference (RNAi) therapy, givosiran, was FDA-approved for reducing the severity and frequency of porphyric attacks in acute hepatic porphyria (Balwani et al, 2020;Honor et al, 2021;Thapar et al, 2021). but its accessibility and pricing may remain limiting issues for use (Massachi et al, 2020;Thapar et al, 2021). Other prevention strategies for AIP attacks include hormone-suppression therapy (Anderson et al, 1990;Herrick et al, 1990;Kauppinen and Mustajoki, 1992), prophylactic heme therapy (Kauppinen, 2005;Yarra et al, 2019), and in extreme cases, liver transplantation (Soonawalla et al, 2004;Dowman et al, 2012;Balwani et al, 2017;Stein et al, 2017).…”

Spotlight on Givosiran as a Treatment Option for Adults with Acute Hepatic Porphyria: Design, Development, and Place in Therapy

“…A recent analysis of total costs for givosiran vs IV heme, either for treatment of acute attacks or for prophylaxis, assuming once weekly IV heme vs once monthly givosiran, showed that IV heme is much less expensive. 88 Specifically, for all patients with AIP, the average annual total cost of care for IV heme therapy was $482,113 [78%] lower than for givosiran.…”

Therapy Follows Diagnosis: Old and New Approaches for the Treatment of Acute Porphyrias, What We Know and What We Should Know

“…Massachi et al [ 117 ] compared the costs for givosiran with those for hemin in the US and arrived at the conclusion of lower annual total costs of care with hemin treatment. The novel individualized dosing approach (see above) had, however, not been available at the time of this analysis.…”