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

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. DISCLOSURES: This study was sponsored by Sanofi Genzyme. Evidera received funding from Sanofi Genzyme to conduct this study and prepare the manuscript. The sponsor collaborated on the study design, analysis, interpretation of results, and writing of the manuscript. Nalysnyk is an employee of and shareholder in Sanofi Genzyme. Ward, Cele, and Uyei are employees of Evidera, which provides consulting and other research services to biopharmaceutical companies. Sugarman was also an Evidera employee when the study was being conducted and the manuscript written. This study was presented as a poster at the Academy of Managed Care Pharmacy Nexus 2016, October 3-6, 2016; National City, MD, and at the International Society for Pharmacoeconomics and Outcomes Research, 22nd Annual International Meeting; May 20-24, 2017; Boston, MA.

Intravitreal Aflibercept Versus Ranibizumab for Wet Age-Related Macular Degeneration: A Cost-Effectiveness Analysis.

BACKGROUND: Age-related macular degeneration (AMD) is the leading cause of vision loss in the United States. The most severe vision loss occurs in patients with neovascular AMD, known as wet AMD (wAMD). The most commonly used antivascular endothelial growth factor (VEGF) therapies approved by the FDA to treat patients with wAMD are ranibizumab, 0.5 mg administered by intravitreal injection once a month (approximately every 28 days), and intravitreal aflibercept injection (IAI), 2 mg every 4 weeks (monthly) for the first 12 weeks (3 months), followed by IAI 2 mg once every 8 weeks (2 months). Given the similar efficacy and safety profiles between IAI and ranibizumab, their associated costs and comparative cost-effectiveness are key factors in determining which one represents a more rational investment of scarce health care resources to help address the increasing cost of prescription drugs in the United States, a source of concern for patients, prescribers, payers, and policymakers. OBJECTIVE: To assess the cost-effectiveness of intravitreal aflibercept injection 2 mg every 8 weeks after 3 initial monthly doses (IAI 2q8) versus ranibizumab 0.5 mg monthly (Rq4) and pro re nata (PRN) in the treatment of patients with wAMD from a U.S. payer perspective. METHODS: A Markov cohort model was developed to estimate the lifetime quality-adjusted life-years (QALYs) and costs of treating patients with wAMD with IAI 2q8, Rq4, and ranibizumab PRN. The model considered changes in best-corrected visual acuity in the affected and fellow eyes over time, and the effect of blindness on mortality. Efficacy for IAI 2q8 and Rq4 was from VIEW 1 and VIEW 2 studies and from the Comparison of AMD Treatments Trials for ranibizumab PRN. Utilities and costs (in 2016 U.S. dollars) were from published literature. Health outcomes and costs were discounted at an annual rate of 3%. RESULTS: Over a lifetime, IAI 2q8 provided equal health benefits with Rq4 (5.44 QALYs) at a lower total cost ($33,745 vs. $48,031) as a result of fewer injections. IAI 2q8 yielded slightly greater QALYs versus ranibizumab PRN (5.44 vs. 5.40) at a slightly higher cost ($33,745 vs. $33,652), with an incremental cost per QALY gained of $2,583. Results were sensitive to variations in drug acquisition costs and number of injections of both drugs and the baseline age of the cohort. CONCLUSIONS: IAI 2q8 can be cost saving and cost-effective compared with Rq4 and ranibizumab PRN for the treatment of wAMD in the United States. DISCLOSURES: This study was funded by Regeneron Pharmaceuticals, the manufacturer of aflibercept. Hernandez, Lanitis, Cele, and Toro-Diaz are employed by Evidera, which received funding from Regeneron Pharmaceuticals to conduct this study. Gibson and Kuznik are employed by and own stock in Regeneron Pharmaceuticals.

Intravitreal Aflibercept Versus Ranibizumab for Wet Age-Related Macular Degeneration: A Cost-effectiveness Analysis.

BACKGROUND: Age-related macular degeneration (AMD) is the leading cause of vision loss in the United States. The most severe vision loss occurs in patients with neovascular AMD, known as wet AMD (wAMD). The most commonly used antivascular endothelial growth factor (VEGF) therapies approved by the FDA to treat patients with wAMD are ranibizumab, 0.5 mg administered by intravitreal injection once a month (approximately every 28 days), and intravitreal aflibercept injection (IAI), 2 mg every 4 weeks (monthly) for the first 12 weeks (3 months), followed by IAI 2 mg once every 8 weeks (2 months). Given the similar efficacy and safety profiles between IAI and ranibizumab, their associated costs and comparative cost-effectiveness are key factors in determining which one represents a more rational investment of scarce health care resources to help address the increasing cost of prescription drugs in the United States, a source of concern for patients, prescribers, payers, and policymakers. OBJECTIVE: To assess the cost-effectiveness of intravitreal aflibercept injection 2 mg every 8 weeks after 3 initial monthly doses (IAI 2q8) versus ranibizumab 0.5 mg monthly (Rq4) and pro re nata (PRN) in the treatment of patients with wAMD from a U.S. payer perspective. METHODS: A Markov cohort model was developed to estimate the lifetime quality-adjusted life-years (QALYs) and costs of treating patients with wAMD with IAI 2q8, Rq4, and ranibizumab PRN. The model considered changes in best-corrected visual acuity in the affected and fellow eyes over time, and the effect of blindness on mortality. Efficacy for IAI 2q8 and Rq4 was from VIEW 1 and VIEW 2 studies and from the Comparison of AMD Treatments Trials for ranibizumab PRN. Utilities and costs (in 2016 U.S. dollars) were from published literature. Health outcomes and costs were discounted at an annual rate of 3%. RESULTS: Over a lifetime, IAI 2q8 provided equal health benefits with Rq4 (5.44 QALYs) at a lower total cost ($33,745 vs. $48,031) as a result of fewer injections. IAI 2q8 yielded slightly greater QALYs versus ranibizumab PRN (5.44 vs. 5.40) at a slightly higher cost ($33,745 vs. $33,652), with an incremental cost per QALY gained of $2,583. Results were sensitive to variations in drug acquisition costs and number of injections of both drugs and the baseline age of the cohort. CONCLUSIONS: IAI 2q8 can be cost saving and cost-effective compared with Rq4 and ranibizumab PRN for the treatment of wAMD in the United States. DISCLOSURES: This study was funded by Regeneron Pharmaceuticals, the manufacturer of aflibercept. Hernandez, Lanitis, Cele, and Toro-Diaz are employed by Evidera, which received funding from Regeneron Pharmaceuticals to conduct this study. Gibson and Kuznik are employed by and own stock in Regeneron Pharmaceuticals. Study concept and design were contributed by Hernandez, Lanitis, Kuznik, and Toro-Diaz. Cele, Toro-Diaz, and Lanitis took the lead in data collection, with assistance from the other authors. Data interpretation was performed by Cele, Toro-Diaz, Hernandez, Lanitis, and Kuznik. The manuscript was written by Hernandez, Lanitis, Gibson, Kuznik, and Cele and revised by Hernandez, Gibson, Kuznik, and Lanitis.