An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER)

References

• ZOLGENSMA (Onasemnogene abeparvovec) PI. In: Administration UFaD, ed2019.
   Google Scholar
• Mercuri E, Finkel RS, Muntoni F, et al. Diagnosis and management of spinal muscular atrophy: part 1: recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. Neuromuscul Disord. 2018;28(2):103–12.
   PubMed Web of Science ®Google Scholar
• Lally C, Jones C, Farwell W, et al. Indirect estimation of the prevalence of spinal muscular atrophy Type I, II, and III in the USA. Orphanet J Rare Dis. 2017;12(1):175.
   PubMedGoogle Scholar
• De Sanctis R, Pane M, Coratti G, et al. Clinical phenotypes and trajectories of disease progression in type 1 spinal muscular atrophy. Neuromuscul Disord. 2018;28(1):24–28.
   Web of Science ®Google Scholar
• Finkel RS, McDermott MP, Kaufmann P, et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology. 2014;83(9):810–817.
   PubMed Web of Science ®Google Scholar
• Services. DoHaH. Recommended Uniform Screening Panel (2018, July). In: Services. DoHaH, ed2018.
   Google Scholar
• Al-Zaidy SA, Mendell JR. From clinical trials to clinical practice: practical considerations for gene replacement therapy in SMA type 1. Pediatr Neurol. 2019;100:3–11.
   Web of Science ®Google Scholar
• Mendell JR, Al-Zaidy S, Shell R, et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N Engl J Med. 2017;377(18):1713–1722.
   PubMed Web of Science ®Google Scholar
• Al-Zaidy S, Pickard AS, Kotha K, et al. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019;54(2):179–185.
   PubMed Web of Science ®Google Scholar
• Malone DC, Dean R, Arjunji R, et al. Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients. J Mark Access Health Policy. 2019;7(1):1601484.
   PubMedGoogle Scholar
• Zerres K, Rudnik-Schöneborn S, Forrest E, et al. A collaborative study on the natural history of childhood and juvenile onset proximal spinal muscular atrophy (type II and III SMA): 569 patients. J Neurol Sci. 1997;146(1):67–72.
   PubMed Web of Science ®Google Scholar
• Shieh PB, Gu T, Chen E, et al. Treatment patterns and cost of care among patients with Spinal Muscular Atrophy. Cure SMA Annual Meeting; June 29-July 1, 2017, 2017; Orlando, FL.
   Google Scholar
• Gregoretti C, Ottonello G, Chiarini Testa MB, et al. Survival of patients with spinal muscular atrophy type 1. Pediatrics. 2013;131(5):e1509–1514.
   PubMed Web of Science ®Google Scholar
• (ICER) IfCaER. Spinraza and Zolgensma for Spinal Muscular Atrophy: Effectiveness and Value. Final Evidence Report. April 3, 2019 (Updated May 24, 2019) 2019.
   Google Scholar
• Review IIfCaE. Modifications to the ICER value assessment framework for treatments for ultra-rare diseases. November 2017 2017.
   Google Scholar
• Castro D, Farrar M, Finkel R, et al. Longer-term assessment of the safety and efficacy of nusinersen for the treatment of infantile-onset Spinal Muscular Atrophy (SMA): an interim analysis of the SHINE study. American academy of neurology. Los Angeles, CA;2018. April 21–27.
   Google Scholar
• Finkel RS, Mercuri E, Darras BT, et al. Nusinersen versus Sham control in infantile-onset spinal muscular atrophy. N Engl J Med. 2017;377(18):1723–1732.
   PubMed Web of Science ®Google Scholar
• Kolb SJ, Coffey CS, Yankey JW, et al. Natural history of infantile-onset spinal muscular atrophy. Ann Neurol. 2017;82(6):883–891.
   PubMed Web of Science ®Google Scholar
• Thompson R, Vaidya S, Teynor M The utility of different approaches to developing health utilities data in childhood rare diseases – a case study in Spinal Muscular Atrophy (SMA). ISPOR 20TH Annual European Congress 4–8 November 2017; 2017; Glasgow, Scotland.
   Google Scholar
• Tappenden P, Hamilton J, Kaltenthaler E, et al. Nusinersen for treating spinal muscular atrophy: a single technology appraisal. School of Health and Related Research (ScHARR); 2018.
   Google Scholar
• Noyes J, Godfrey C, Beecham J. Resource use and service costs for ventilator-dependent children and young people in the UK. Health Soc Care Community. 2006;14(6):508–522.
   Web of Science ®Google Scholar
• Lowes LP, Alfano LN, Arnold WD, et al. Impact of age and motor function in a phase 1/2A study of infants with SMA type 1 receiving single-dose gene replacement therapy. Pediatr Neurol. 2019;98:39–45.
   Web of Science ®Google Scholar
• Van Den Broek BTA, Page K, Paviglianiti A, et al. Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies. Blood Adv. 2018;2(1):49–60.
   Web of Science ®Google Scholar
• Finkel RS, Mercuri E, Meyer OH, et al. Diagnosis and management of spinal muscular atrophy: part 2: pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics. Neuromuscul Disord. 2018;28(3):197–207.
   PubMed Web of Science ®Google Scholar
• Bach JR, Gupta K, Reyna M, et al. Spinal muscular atrophy type 1: prolongation of survival by noninvasive respiratory aids. Pediatr Asthma Allergy Immunol. 2009;22(4):151–161.
   Google Scholar
• Dabbous O, Maru B, Jansen JP, et al. Survival, motor function, and motor milestones: comparison of AVXS-101 relative to nusinersen for the treatment of infants with spinal muscular atrophy type 1. Adv Ther. 2019;36(5):1164–1176.
   PubMed Web of Science ®Google Scholar
• Pearson SD, Ollendorf DA, Chapman R. New Cost-Effectiveness methods to determine Value-Based Prices for potential cures: what are the options? Value Health. 2019;22(6):656–660.
   PubMed Web of Science ®Google Scholar
• Mendell JR, Al-Zaidy S, Shell R, et al. AVXS-101 Phase 1 gene replacement therapy clinical trial in SMA type 1: continued event-free survival and achievement of developmental milestones. 22nd Annual SMA Researcher Meeting; June 14–16, 2018, 2018; Dallas, TX.
   Google Scholar
• Database USM. Human mortality database. 2017; https://usa.mortality.org/
   Google Scholar
• The Lewis Group Inc. Cost of amyotrophic lateral sclerosis, muscular dystrophy, and spinal muscular atrophy in the USA. 2012; https://www.mda.org/sites/default/files/Cost_Illness_Report_0.pdf
   Google Scholar