Cost-Effectiveness of Acthar Gel versus Standard of Care for the Treatment of Advanced Symptomatic Sarcoidosis

References

• Statement on sarcoidosis. Joint Statement of the American Thoracic Society (ATS), the European Respiratory Society (ERS) and the World Association of Sarcoidosis and Other Granulomatous Disorders (WASOG) adopted by the ATS Board of Directors and by the ERS Executive Committee, February 1999. Am J Respir Crit Care Med. 1999;160(2):736–755. doi:10.1164/ajrccm.160.2.ats4-99
   PubMedGoogle Scholar
• Valeyre D, Prasse A, Nunes H, Uzunhan Y, Brillet PY, Muller-Quernheim J. Sarcoidosis. Lancet. 2014;383(9923):1155–1167. doi:10.1016/S0140-6736(13)60680-7
   PubMed Web of Science ®Google Scholar
• Soto-Gomez N, Peters JI, Nambiar AM. Diagnosis and management of sarcoidosis. Am Fam Physician. 2016;93(10):840–848.
   PubMed Web of Science ®Google Scholar
• Wu JJ, Schiff KR. Sarcoidosis. Am Fam Physician. 2004;70(2):312–322.
   PubMed Web of Science ®Google Scholar
• Baughman RP, Field S, Costabel U, et al. Sarcoidosis in America. Analysis based on health care use. Ann Am Thorac Soc. 2016;13(8):1244–1252. doi:10.1513/AnnalsATS.201511-760OC
   PubMed Web of Science ®Google Scholar
• Sharp M, Eakin MN, Drent M. Socioeconomic determinants and disparities in sarcoidosis. Curr Opin Pulm Med. 2020;26(5):568–573. doi:10.1097/MCP.0000000000000704
   PubMed Web of Science ®Google Scholar
• Patel N, Kalra R, Doshi R, et al. Hospitalization rates, prevalence of cardiovascular manifestations, and outcomes associated with sarcoidosis in the United States. J Am Heart Assoc. 2018;7(2). doi:10.1161/JAHA.117.007844
   Web of Science ®Google Scholar
• Mirsaeidi M, Baughman RP, Sahoo D, Tarau E. Results from a phase 4, multicenter, randomized, double-blind, placebo-controlled study of repository corticotropin injection for the treatment of pulmonary sarcoidosis. Pulm Ther. 2023;9:1–17. doi:10.1007/s41030-022-00213-9
   PubMedGoogle Scholar
• Rice JB, White AG, Johnson M, et al. Quantitative characterization of the relationship between levels of extended corticosteroid use and related adverse events in a US population. Curr Med Res Opin. 2018;34(8):1519–1527. doi:10.1080/03007995.2018.1474090
   PubMed Web of Science ®Google Scholar
• Rice JB, White AG, Johnson M, et al. Healthcare resource use and cost associated with varying dosages of extended corticosteroid exposure in a US population. J Med Econ. 2018;21(9):846–852. doi:10.1080/13696998.2018.1474750
   PubMed Web of Science ®Google Scholar
• Mallinckrodt Pharmaceuticals. Acthar® Gel prescribing information; 1952. https://www.acthar.com/pdf/Acthar-PI.pdf. Accessed September 27, 2023.
   Google Scholar
• Rahaghi FF, Baughman RP, Saketkoo LA, et al. Delphi consensus recommendations for a treatment algorithm in pulmonary sarcoidosis. Eur Respir Rev. 2020;29(155):190146. doi:10.1183/16000617.0146-2019
   PubMed Web of Science ®Google Scholar
• Baughman RP, Valeyre D, Korsten P, et al. ERS clinical practice guidelines on treatment of sarcoidosis. Eur Respir J. 2021;58:2004079. doi:10.1183/13993003.04079-2020
   PubMed Web of Science ®Google Scholar
• Baughman RP, Sweiss N, Keijsers R, et al. Repository corticotropin for chronic pulmonary sarcoidosis. Lung. 2017;195(3):313–322. doi:10.1007/s00408-017-9994-4
   PubMed Web of Science ®Google Scholar
• Baughman RP, Barney JB, O’Hare L, Lower EE. A retrospective pilot study examining the use of Acthar gel in sarcoidosis patients. Respir Med. 2016;110:66–72. doi:10.1016/j.rmed.2015.11.007
   PubMed Web of Science ®Google Scholar
• Chopra I, Qin Y, Kranyak J, et al. Repository corticotropin injection in patients with advanced symptomatic sarcoidosis: retrospective analysis of medical records. Ther Adv Respir Dis. 2019;13:1753466619888127. doi:10.1177/1753466619888127
   Web of Science ®Google Scholar
• Bindra J, Chopra I, Hayes K, Niewoehner J, Panaccio MP, Wan GJ. Understanding predictors of response to repository corticotropin injection treatment among patients with advanced symptomatic sarcoidosis. J Health Econ Outcomes Res. 2022;9(1):90–100. doi:10.36469/jheor.2022.33295
   PubMedGoogle Scholar
• Quanjer PH, Pretto JJ, Brazzale DJ, Boros PW. Grading the severity of airways obstruction: new wine in new bottles. Eur Respir J. 2014;43(2):505–512. doi:10.1183/09031936.00086313
   PubMed Web of Science ®Google Scholar
• Barreiro TJ, Perillo I. An approach to interpreting spirometry. Am Fam Physician. 2004;69(5):1107–1114.
   PubMed Web of Science ®Google Scholar
• Heij L, Dahan A, Hoitsma E. Sarcoidosis and pain caused by small-fiber neuropathy. Pain Res Treat. 2012;2012:256024. doi:10.1155/2012/256024
   PubMedGoogle Scholar
• Hinz A, Brahler E, Mode R, Wirtz H, Bosse-Henck A. Anxiety and depression in sarcoidosis: the influence of age, gender, affected organs, concomitant diseases and dyspnea. Sarcoidosis Vasc Diffuse Lung Dis. 2012;29(2):139–146.
   PubMed Web of Science ®Google Scholar
• IBM. IBM Micromedex RED BOOK. IBM; 2021.
   Google Scholar
• Mallinckrodt Pharmaceuticals. Data on File: Acthar Gel in Participants With Pulmonary Sarcoidosis (PULSAR); 2023.
   Google Scholar
• El Jammal T, Jamilloux Y, Gerfaud-Valentin M, Valeyre D, Seve P. Refractory Sarcoidosis: a Review. Ther Clin Risk Manag. 2020;16:323–345. doi:10.2147/TCRM.S192922
   PubMed Web of Science ®Google Scholar
• Drugs.com. Drugs & Medications A to Z; 2023. Available from: https://www.drugs.com/. Accessed January 21, 2023.
   Google Scholar
• Peel JK, Keshavjee S, Krahn M, Sander B. Economic evaluations and costing studies of lung transplantation: a scoping review. J Heart Lung Transplant. 2021;40(12):1625–1640. doi:10.1016/j.healun.2021.08.007
   PubMed Web of Science ®Google Scholar
• Tripathi R, Knusel KD, Ezaldein HH, Honaker JS, Bordeaux JS, Scott JF. Incremental health care expenditure of chronic cutaneous ulcers in the United States. JAMA Dermatol. 2019;155(6):694–699. doi:10.1001/jamadermatol.2018.5942
   PubMed Web of Science ®Google Scholar
• Foy AJ, Mandrola JM. Heavy heart: the economic burden of heart disease in the United States now and in the future. Prim Care. 2018;45(1):17–24. doi:10.1016/j.pop.2017.11.002
   PubMed Web of Science ®Google Scholar
• Rein DB, Zhang P, Wirth KE, et al. The economic burden of major adult visual disorders in the United States. Arch Ophthalmol. 2006;124(12):1754–1760. doi:10.1001/archopht.124.12.1754
   PubMedGoogle Scholar
• Williams SA, Chastek B, Sundquist K, et al. Economic burden of osteoporotic fractures in US managed care enrollees. Am J Manag Care. 2020;26(5):e142–e149.
   PubMed Web of Science ®Google Scholar
• Nguyen MH, Burak Ozbay A, Liou I, et al. Healthcare resource utilization and costs by disease severity in an insured national sample of US patients with chronic hepatitis B. J Hepatol. 2019;70(1):24–32. doi:10.1016/j.jhep.2018.09.021
   PubMed Web of Science ®Google Scholar
• Lalonde L, Choiniere M, Martin E, Berbiche D, Perreault S, Lussier D. Costs of moderate to severe chronic pain in primary care patients - a study of the ACCORD Program. J Pain Res. 2014;7:389–403. doi:10.2147/JPR.S55388
   PubMed Web of Science ®Google Scholar
• White AG, Birnbaum HG, Mareva MN, et al. Direct costs of opioid abuse in an insured population in the United States. J Manag Care Pharm. 2005;11(6):469–479. doi:10.18553/jmcp.2005.11.6.469
   PubMedGoogle Scholar
• Chow W, Doane MJ, Sheehan J, Alphs L, Le H. Economic burden among patients with major depressive disorder: an analysis of healthcare resource use, work productivity, and direct and indirect costs by depression severity. Am J Manag Care. 2019;16:e188–96.
   Google Scholar
• Foundation for Sarcoidosis Research. Commonly Prescribed Sarcoidosis Medications; 2019. Available from: https://www.stopsarcoidosis.org/commonly-prescribed-sarcoidosis-medications/. Accessed January 21, 2023.
   Google Scholar
• Meyer KC. Lung transplantation for pulmonary sarcoidosis. Sarcoidosis Vasc Diffuse Lung Dis. 2019;36(2):92–107. doi:10.36141/svdld.v36i2.7163
   PubMed Web of Science ®Google Scholar
• Rao DA, Dellaripa PF. Extrapulmonary manifestations of sarcoidosis. Rheum Dis Clin North Am. 2013;39(2):277–297. doi:10.1016/j.rdc.2013.02.007
   PubMed Web of Science ®Google Scholar
• Foundation for Sarcoidosis Research. Opioid epidemic impacts patients suffering from orphan disease of sarcoidosis; 2017. Available from: https://www.stopsarcoidosis.org/press-release-opioid-epidemic/. Accessed January 21, 2023.
   Google Scholar
• Harned M, Sloan P. Safety concerns with long-term opioid use. Expert Opin Drug Saf. 2016;15(7):955–962. doi:10.1080/14740338.2016.1177509
   PubMed Web of Science ®Google Scholar
• Khan NA, Donatelli CV, Tonelli AR, et al. Toxicity risk from glucocorticoids in sarcoidosis patients. Respir Med. 2017;132:9–14. doi:10.1016/j.rmed.2017.09.003
   PubMed Web of Science ®Google Scholar
• Entrop JP, Kullberg S, Grunewald J, Eklund A, Brismar K, Arkema EV. Type 2 diabetes risk in sarcoidosis patients untreated and treated with corticosteroids. ERJ Open Res. 2021;7(2). doi:10.1183/23120541.00028-2021
   PubMedGoogle Scholar
• Rice JB, White A, Lopez A, et al. Economic burden of sarcoidosis in a commercially-insured population in the United States. J Med Econ. 2017;20(10):1048–1055. doi:10.1080/13696998.2017.1351371
   PubMed Web of Science ®Google Scholar
• Birnbaum HG, Ivanova JI, Samuels S, et al. Economic impact of multiple sclerosis disease-modifying drugs in an employed population: direct and indirect costs. Curr Med Res Opin. 2009;25(4):869–877. doi:10.1185/03007990902743869
   PubMed Web of Science ®Google Scholar
• Luo F, Li M, Florence C. State-level economic costs of opioid use disorder and fatal opioid overdose - United States, 2017. MMWR Morb Mortal Wkly Rep. 2021;70(15):541–546. doi:10.15585/mmwr.mm7015a1
   PubMed Web of Science ®Google Scholar
• Patel AS, Siegert RJ, Creamer D, et al. The development and validation of the king’s sarcoidosis questionnaire for the assessment of health status. Thorax. 2013;68(1):57–65. doi:10.1136/thoraxjnl-2012-201962
   PubMed Web of Science ®Google Scholar
• Atkins CP, Jones AP, Wilson AM. A comparison of measurements of health-related quality of life in patients with sarcoidosis. Thorax. 2018;73(Supple 4):A189.
   Google Scholar
• Dempsey TM, Thao V, Moriarty JP, Borah BJ, Limper AH. Cost-effectiveness of the anti-fibrotics for the treatment of idiopathic pulmonary fibrosis in the United States. BMC Pulm Med. 2022;22(1):18. doi:10.1186/s12890-021-01811-0
   PubMed Web of Science ®Google Scholar
• Anyanwu AC, McGuire A, Rogers CA, Murday AJ. Assessment of quality of life in lung transplantation using a simple generic tool. Thorax. 2001;56(3):218–222. doi:10.1136/thorax.56.3.218
   PubMed Web of Science ®Google Scholar
• Chen SC, Bayoumi AM, Soon SL, et al. A catalog of dermatology utilities: a measure of the burden of skin diseases. J Investig Dermatol Symp Proc. 2004;9(2):160–168. doi:10.1046/j.1087-0024.2003.09112.x
   PubMedGoogle Scholar
• Di Tanna GL, Urbich M, Wirtz HS, et al. Health state utilities of patients with heart failure: a systematic literature review. Pharmacoeconomics. 2021;39(2):211–229. doi:10.1007/s40273-020-00984-6
   PubMed Web of Science ®Google Scholar
• Brown MM, Brown GC, Brown HC, Peet J, Roth Z. Value-based medicine, comparative effectiveness, and cost-effectiveness analysis of topical cyclosporine for the treatment of dry eye syndrome. Arch Ophthalmol. 2009;127(2):146–152. doi:10.1001/archophthalmol.2008.608
   PubMedGoogle Scholar
• Tormalehto S, Mononen ME, Aarnio E, Arokoski JPA, Korhonen RK, Martikainen J. Health-related quality of life in relation to symptomatic and radiographic definitions of knee osteoarthritis: data from Osteoarthritis Initiative (OAI) 4-year follow-up study. Health Qual Life Outcomes. 2018;16(1):154. doi:10.1186/s12955-018-0979-7
   PubMedGoogle Scholar
• Dan AA, Kallman JB, Srivastava R, Younoszai Z, Kim A, Younossi ZM. Impact of chronic liver disease and cirrhosis on health utilities using SF-6D and the health utility index. Liver Transpl. 2008;14(3):321–326. doi:10.1002/lt.21376
   PubMed Web of Science ®Google Scholar
• Institute for Clinical and Economic Review. Biologic therapies for treatment of asthma associated with type 2 inflammation: effectiveness, value, and value-based price benchmarks; 2018. Available from: https://icer.org/wp-content/uploads/2020/10/ICER_Asthma-Final-Report_Unredacted_08122020.pdf#page=75. Accessed January 05, 2023.
   Google Scholar
• Institute for Clinical and Economic Review. Extended-release opioid agonists and antagonist medications for addiction treatment (MAT) in patients with opioid use disorder: effectiveness and value; 2018. Available from: https://icer.org/wp-content/uploads/2020/10/ICER_OUD_Final_Evidence_Report_120318.pdf. Accessed January 05, 2023.
   Google Scholar
• Wetherington S, Delong L, Kini S, et al. Pain quality of life as measured by utilities. Pain Med. 2014;15(5):865–870. doi:10.1111/pme.12405
   PubMed Web of Science ®Google Scholar
• Kolovos S, Bosmans JE, van Dongen JM, et al. Utility scores for different health states related to depression: individual participant data analysis. Qual Life Res. 2017;26(7):1649–1658. doi:10.1007/s11136-017-1536-2
   PubMed Web of Science ®Google Scholar
• Dubois RW. Cost-effectiveness thresholds in the USA: are they coming? Are they already here? J Comp Eff Res. 2016;5(1):9–11. doi:10.2217/cer.15.50
   PubMed Web of Science ®Google Scholar
• Institute for Clinical and Economic Review. 2020-2023 value assessment framework; 2020.
   Google Scholar
• Thokala P, Ochalek J, Leech AA, Tong T. Cost-effectiveness thresholds: the past, the present and the future. Pharmacoeconomics. 2018;36(5):509–522. doi:10.1007/s40273-017-0606-1
   PubMed Web of Science ®Google Scholar
• Tzanetakos C, Gourzoulidis G. Does a standard cost-effectiveness threshold exist? The Case of Greece. Value Health Reg Issues. 2023;36:18–26. doi:10.1016/j.vhri.2023.02.006
   PubMedGoogle Scholar
• United States Bureau of Labor Statistics. Consumer price index - medical care; 2022. Available from: https://www.bls.gov/cpi/data.htm. Accessed September 15, 2022.
   Google Scholar
• Gerke AK, Judson MA, Cozier YC, Culver DA, Koth LL. Disease burden and variability in sarcoidosis. Ann Am Thorac Soc. 2017;14(Supplement_6):S421–S428. doi:10.1513/AnnalsATS.201707-564OT
   PubMedGoogle Scholar