Budget Impact Analysis of Eliglustat for the Treatment of Gaucher Disease Type 1 in the United States

Abstract

BACKGROUND: Gaucher disease type 1 (GD1) is a rare, genetic, lysosomal storage disease with no cure. Current treatment options include intravenous (IV) enzyme replacement therapy ([ERT]; imiglucerase, velaglucerase alfa, or taliglucerase alfa) or oral substrate reduction therapy ([SRT]; eliglustat or miglustat). The cost to U.S. payers of an IV-administered drug can vary depending on the site of care (i.e., home, outpatient clinic, or hospital setting). Treatment with oral eliglustat may present an opportunity for cost savings. OBJECTIVE: To evaluate the budget impact from a U.S. payer perspective associated with transitioning patients receiving ERTs to the oral SRT eliglustat for the treatment of adults with GD1.METHODS: A budget impact model estimated the change in pharmaceutical and administration costs resulting from increasing the market share of eliglustat from 12% (current) to 44% (new). The market share for eliglustat was drawn equally from existing shares of imiglucerase (40%) and velaglucerase alfa (40%) and assumed to be static over the analysis period. ERT costs were adjusted to account for site of care-based markup and the proportion of patients receiving infusions in each site of care (home, infusion center, or hospital outpatient). Annual ERT costs were calculated assuming a biweekly dose of 47.4 U per kg, a 72-kg patient weight, and 24 infusions per year. The effect of key variables was tested in the sensitivity analyses. All costs are expressed in 2017 U.S. dollars. RESULTS:In a new plan with 5 million members and 25 GD1 treated patients, increased use of eliglustat resulted in an annual savings of $1,526,710 and a total savings of $4,580,130 (13.6%) over 3 years. The corresponding annual per member per month savings was $0.025. This is further illustrated in the sensitivity and scenario analyses where the use of eliglustat was cost saving in all cases. Shifting more patients receiving ERT in the hospital outpatient setting to eliglustat resulted in increased savings.CONCLUSIONS: Based on these analyses, increased use of eliglustat resulted in meaningful cost savings to a payer's overall budget. Cost savings are highest among patients switching from ERT administered in a hospital outpatient setting. The results suggest that cost savings are also likely achievable from initiating patients on oral eliglustat instead of infusion-based therapy from the outset of treatment.

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“…Current oral forms of SRT are marketed to patients with Gaucher disease at a cost of several hundred thousand dollars per patient per year. 8,24 Furthermore, participation requires repeated lumbar puncture, which some patients do not find trivial.…”

“…Eliglustat is contraindicated in patients with preexisting cardiac disease, long QT syndrome, and renal or hepatic impairment and in those taking may many drugs including digoxin, colchicine and metoprolol, and is often avoided in the elderly. Current oral forms of SRT are marketed to patients with Gaucher disease at a cost of several hundred thousand dollars per patient per year . Furthermore, participation requires repeated lumbar puncture, which some patients do not find trivial.…”

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“…Approved SRTs (miglustat and eliglustat) also require life-long administration, repeated dosing (three and two times per day, respectively) and, particularly for miglustat, significant side effects due to non-specific inhibition of other enzymes 46 . Both modalities are very costly with estimated annual cost of $300,000 to $450,000 (estimated life-time cost of$ 6 to $22 million dollars) limiting their availability worldwide 47,48 . In the past, allo-HSCT was used effectively and led to rapid improvement in the hematological and visceral parameters as well as regression of skeletal disease, but given its significant morbidity and mortality, its use has been reserved for individuals with neurologic or progressive disease unresponsive to ERT and SRT [49][50][51][52] .…”

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“…Although the disease is mainly diagnosed in childhood, its manifestation in adults is often missed or identified late due to the failure to recognize the heterogeneous clinical presentation. Current therapeutic options for GD1 are intravenous enzyme replacement therapy (ERT) and oral substrate reduction therapy (SRT) [15]; however, high inter-individual variability in the clinical response to ERT has been associated with a high risk of long-term complications. One-quarter of patients still have thrombocytopenia after four years of therapy [16].…”