{"title":"遗传性血管性水肿(HAE)发作按需治疗的成本-效果模型","authors":"C. Tyson, A. Relan, P. Adams, A. Haynes, R. Magar","doi":"10.1080/21556660.2019.1658300","DOIUrl":null,"url":null,"abstract":"Abstract Background: Hereditary angioedema (HAE) is a rare C1-inhibitor (C1-INH) deficiency disease involving recurrent painful episodes of severe swelling that should be promptly treated. Aims: To determine cost and utility estimates for on-demand treatment of HAE attacks in order to better clarify and control expenses related to disease management. Methods: A decision-tree model included four comparators (ecallantide, icatibant, plasma-derived [pd] C1-INH, and recombinant human C1-INH [rhC1-INH]) and incorporated probabilities for self-administration vs healthcare provider administration, re-dosing, and hospitalization risk. Modeled costs comprised HAE therapies and healthcare system expenses. Effectiveness considered utility during attacks (0.51), no-attack baseline (0.83), and time to attack resolution. Overall drug cost and effectiveness per attack were used to estimate cost per quality-adjusted life year (QALY). Sensitivity analyses were performed to establish cost-effectiveness ranges. A budget impact model was developed for a health plan of 1 million (M) covered lives. Results: Costs and utility per attack were, respectively, $12,342 and 0.804 for rhC1-INH, $14,369 and 0.749 for icatibant, $13,993 and 0.759 for pdC1-INH, and $20,315 and 0.786 for ecallantide. At a mean annual attack rate of 26.9, cost per QALY was $402,769 for rhC1-INH, $475,942 for icatibant, $462,275 for pdC1-INH, and $666,153 for ecallantide. Re-dose rate was identified as a primary driver of cost-effectiveness variability. Estimated annual cost to the plan was $6.64 M for rhC1-INH, $7.73 M for icatibant, $7.53 M for pdC1-INH, and $10.93 M for ecallantide. A 5000-trial probabilistic sensitivity analysis (PSA) indicated that rhC1-INH was the most cost-effective in many scenarios, while ecallantide was the least cost-effective: mean costs (effectiveness) from PSA were $12,390 (0.786) for rhC1-INH, $14,132 (0.738) for icatibant, $13,050 (0.746) for pdC-1INH, and $20,286 (0.785) for ecallantide. Conclusions: This model demonstrated that rhC1-INH was the most cost-effective and ecallantide the least cost-effective on-demand HAE treatment and, overall, cost-effectiveness was substantially impacted by re-dosing rates. For icatibant, re-dosing rates of up to 44% to treat an HAE attack have been reported, and prescribing information allows up to three doses per 24-h period to treat a single attack. Driven by higher re-dosing rates, icatibant suffers from a higher per-attack drug cost and comparatively poor effectiveness.","PeriodicalId":15631,"journal":{"name":"Journal of Drug Assessment","volume":null,"pages":null},"PeriodicalIF":2.4000,"publicationDate":"2019-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/21556660.2019.1658300","citationCount":"0","resultStr":"{\"title\":\"Cost-effectiveness model for on-demand treatment of hereditary angioedema (HAE) attacks\",\"authors\":\"C. Tyson, A. Relan, P. Adams, A. Haynes, R. Magar\",\"doi\":\"10.1080/21556660.2019.1658300\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Abstract Background: Hereditary angioedema (HAE) is a rare C1-inhibitor (C1-INH) deficiency disease involving recurrent painful episodes of severe swelling that should be promptly treated. Aims: To determine cost and utility estimates for on-demand treatment of HAE attacks in order to better clarify and control expenses related to disease management. Methods: A decision-tree model included four comparators (ecallantide, icatibant, plasma-derived [pd] C1-INH, and recombinant human C1-INH [rhC1-INH]) and incorporated probabilities for self-administration vs healthcare provider administration, re-dosing, and hospitalization risk. Modeled costs comprised HAE therapies and healthcare system expenses. Effectiveness considered utility during attacks (0.51), no-attack baseline (0.83), and time to attack resolution. Overall drug cost and effectiveness per attack were used to estimate cost per quality-adjusted life year (QALY). Sensitivity analyses were performed to establish cost-effectiveness ranges. A budget impact model was developed for a health plan of 1 million (M) covered lives. Results: Costs and utility per attack were, respectively, $12,342 and 0.804 for rhC1-INH, $14,369 and 0.749 for icatibant, $13,993 and 0.759 for pdC1-INH, and $20,315 and 0.786 for ecallantide. At a mean annual attack rate of 26.9, cost per QALY was $402,769 for rhC1-INH, $475,942 for icatibant, $462,275 for pdC1-INH, and $666,153 for ecallantide. Re-dose rate was identified as a primary driver of cost-effectiveness variability. Estimated annual cost to the plan was $6.64 M for rhC1-INH, $7.73 M for icatibant, $7.53 M for pdC1-INH, and $10.93 M for ecallantide. A 5000-trial probabilistic sensitivity analysis (PSA) indicated that rhC1-INH was the most cost-effective in many scenarios, while ecallantide was the least cost-effective: mean costs (effectiveness) from PSA were $12,390 (0.786) for rhC1-INH, $14,132 (0.738) for icatibant, $13,050 (0.746) for pdC-1INH, and $20,286 (0.785) for ecallantide. Conclusions: This model demonstrated that rhC1-INH was the most cost-effective and ecallantide the least cost-effective on-demand HAE treatment and, overall, cost-effectiveness was substantially impacted by re-dosing rates. For icatibant, re-dosing rates of up to 44% to treat an HAE attack have been reported, and prescribing information allows up to three doses per 24-h period to treat a single attack. Driven by higher re-dosing rates, icatibant suffers from a higher per-attack drug cost and comparatively poor effectiveness.\",\"PeriodicalId\":15631,\"journal\":{\"name\":\"Journal of Drug Assessment\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":2.4000,\"publicationDate\":\"2019-09-03\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://sci-hub-pdf.com/10.1080/21556660.2019.1658300\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of Drug Assessment\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1080/21556660.2019.1658300\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Drug Assessment","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1080/21556660.2019.1658300","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Cost-effectiveness model for on-demand treatment of hereditary angioedema (HAE) attacks
Abstract Background: Hereditary angioedema (HAE) is a rare C1-inhibitor (C1-INH) deficiency disease involving recurrent painful episodes of severe swelling that should be promptly treated. Aims: To determine cost and utility estimates for on-demand treatment of HAE attacks in order to better clarify and control expenses related to disease management. Methods: A decision-tree model included four comparators (ecallantide, icatibant, plasma-derived [pd] C1-INH, and recombinant human C1-INH [rhC1-INH]) and incorporated probabilities for self-administration vs healthcare provider administration, re-dosing, and hospitalization risk. Modeled costs comprised HAE therapies and healthcare system expenses. Effectiveness considered utility during attacks (0.51), no-attack baseline (0.83), and time to attack resolution. Overall drug cost and effectiveness per attack were used to estimate cost per quality-adjusted life year (QALY). Sensitivity analyses were performed to establish cost-effectiveness ranges. A budget impact model was developed for a health plan of 1 million (M) covered lives. Results: Costs and utility per attack were, respectively, $12,342 and 0.804 for rhC1-INH, $14,369 and 0.749 for icatibant, $13,993 and 0.759 for pdC1-INH, and $20,315 and 0.786 for ecallantide. At a mean annual attack rate of 26.9, cost per QALY was $402,769 for rhC1-INH, $475,942 for icatibant, $462,275 for pdC1-INH, and $666,153 for ecallantide. Re-dose rate was identified as a primary driver of cost-effectiveness variability. Estimated annual cost to the plan was $6.64 M for rhC1-INH, $7.73 M for icatibant, $7.53 M for pdC1-INH, and $10.93 M for ecallantide. A 5000-trial probabilistic sensitivity analysis (PSA) indicated that rhC1-INH was the most cost-effective in many scenarios, while ecallantide was the least cost-effective: mean costs (effectiveness) from PSA were $12,390 (0.786) for rhC1-INH, $14,132 (0.738) for icatibant, $13,050 (0.746) for pdC-1INH, and $20,286 (0.785) for ecallantide. Conclusions: This model demonstrated that rhC1-INH was the most cost-effective and ecallantide the least cost-effective on-demand HAE treatment and, overall, cost-effectiveness was substantially impacted by re-dosing rates. For icatibant, re-dosing rates of up to 44% to treat an HAE attack have been reported, and prescribing information allows up to three doses per 24-h period to treat a single attack. Driven by higher re-dosing rates, icatibant suffers from a higher per-attack drug cost and comparatively poor effectiveness.