A Model-Based Economic Evaluation of Hypothetical Treatments for Amyotrophic Lateral Sclerosis in the UK: Implications for Pricing of New and Emerging Health Technologies

Hardiman O, Al-Chalabi A, Chio A, Corr EM, Logroscino G, Robberecht W, et al. Amyotrophic lateral sclerosis. Nat Rev Dis Primers. 2017;3:17071.

Article  PubMed  Google Scholar 

GBD 2016 Motor Neuron Disease Collaborators. Global, regional, and national burden of motor neuron diseases 1990–2016: a systematic analysis for the Global Burden of Disease Study 2016. Lancet Neurol. 2018;17(12):1083–97.

Article  Google Scholar 

Hardiman O, Al-Chalabi A, Brayne C, Beghi E, van den Berg LH, Chio A, et al. The changing picture of amyotrophic lateral sclerosis: lessons from European registers. J Neurol Neurosurg Psychiatry. 2017;88(7):557–63.

Article  PubMed  Google Scholar 

Rooney J, Byrne S, Heverin M, Tobin K, Dick A, Donaghy C, et al. A multidisciplinary clinic approach improves survival in ALS: a comparative study of ALS in Ireland and Northern Ireland. J Neurol Neurosurg Psychiatry. 2015;86(5):496–501.

Article  PubMed  Google Scholar 

Aridegbe T, Kandler R, Walters SJ, Walsh T, Shaw PJ, McDermott CJ. The natural history of motor neuron disease: assessing the impact of specialist care. Amyotroph Lateral Scler Frontotemporal Degener. 2013;14(1):13–9.

Article  PubMed  Google Scholar 

European Medicines Agency. Rilutek (riluzole) 50mg film-coated tablets. London: Summary of product characteristics; 1996.

Google Scholar 

U.S. Food and Drug Administration. Search orphan drug designations and approvals—Riluzole (rilutek) 1993.

Petrov D, Mansfield C, Moussy A. ALS clinical trials review: 20 years of failure. Are we any closer to registering a new treatment? Front Aging Neurosci. 2017. https://doi.org/10.3389/fnagi.2017.00068.

Article  PubMed  PubMed Central  Google Scholar 

Gao M, Zhu L, Chang J, Cao T, Song L. Safety and efficacy of edaravone in patients with amyotrophic lateral sclerosis: a systematic review and meta-analysis. Clin Drug Investig. 2023;43(1):1–11.

Article  CAS  PubMed  Google Scholar 

Miller TM, Cudkowicz ME, Genge A, Shaw PJ, Sobue G, Bucelli RC, et al. Trial of antisense oligonucleotide tofersen for SOD1 ALS. N Engl J Med. 2022;387(12):1099–110.

Article  CAS  PubMed  Google Scholar 

Mitsubishi Tanabe Pharma America. Company Press Release: Mitsubishi Tanabe Pharma America, Inc. (MTPA) Statement on Ferrer’s Phase 3 ADORE Clinical Trial of FNP122 (FAB122). 2024. https://www.mt-pharma-america.com/media/news/press-releases/2024/01/17/mitsubishi-tanabe-pharma-america-inc-mtpa-statement-on-ferrers-phase-3-adore-clinical-trial-of-fnp122-fab122. Accessed 26 Apr 2024.

Moore A, Young CA, Hughes DA. Economic studies in motor neurone disease: a systematic methodological review. Pharmacoeconomics. 2017;35(4):397–413.

Article  PubMed  Google Scholar 

Thakore NJ, Pioro EP, Udeh BL, Lapin BR, Katzan IL. A cost-effectiveness framework for amyotrophic lateral sclerosis, applied to riluzole. Value Health. 2020;23(12):1543–51.

Article  PubMed  Google Scholar 

clinicaltrials.gov. A programme for amyotrophic lateral sclerosis care in Europe (ALS-CarE). 2015. https://clinicaltrials.gov/study/NCT03081338?term=%22als-care%22&rank=1#publications. Accessed 06 Jan 2024.

Hardiman O, Van Den Berg L, Al-Chalabi A, McDermott C, Tappenden P, Chio A, et al. ALS-CARE: a programme for ALS care in Europe. Patient-level data. 2013.

Galvin M, Carney S, Corr B, Mays I, Pender N, Hardiman O. Needs of informal caregivers across the caregiving course in amyotrophic lateral sclerosis: a qualitative analysis. BMJ Open. 2018;27(8):e018721.

Article  Google Scholar 

Galvin M, Gavin T, Mays I, Heverin M, Hardiman O. Individual quality of life in spousal ALS patient-caregiver dyads. Health Qual Life Outcomes. 2020. https://doi.org/10.1186/s12955-020-01551-5.

Article  PubMed  PubMed Central  Google Scholar 

Thurn T, Borasio GD, Chiò A, Galvin M, McDermott CJ, Mora G, et al. Physicians’ attitudes toward end-of-life decisions in amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2019;20(1–2):74–81.

Article  PubMed  Google Scholar 

National Institute for Health and Care Excellence. NICE health technology evaluations: The manual. London. 2022.

Makam AN, Nikitin D, Richardson M, Mohammed R, McKenna A, Pearson SD, et al. AMX0035 and oral edaravone for amyotrophic lateral sclerosis. Final report published by the Institute for Clinical and Economic Review (ICER). 2022.

CADTH Common Drug Review. Pharmacoeconomic review report: Edaravone Ontario 2019.

Fang T, Al Khleifat A, Stahl DR, Lazo La Torre C, Uk-Mnd Lical S, et al. Comparison of the King’s and MiToS staging systems for ALS. Amyotroph Lateral Scler Frontotemporal Degener. 2017;18(3–4):227–32.

Article  PubMed  PubMed Central  Google Scholar 

Putter H, Fiocco M, Geskus RB. Tutorial in biostatistics: competing risks and multi-state models. Stat Med. 2007;26(11):2389–430.

Article  CAS  PubMed  Google Scholar 

Jackson CH. Multistate modelling with R: The msm package. Version 1.7. J Stat Softw. 2011. https://doi.org/10.18637/jss.v038.i08.

Article  Google Scholar 

Office for National Statistics. National life tables: England 2018–2020. London. 2021.

van Hout B, Janssen MF, Feng YS, Kohlmann T, Busschbach J, Golicki D, et al. Interim scoring for the EQ-5D-5L: mapping the EQ-5D-5L to EQ-5D-3L value sets. Value Health. 2012;15(5):708–15.

Article  PubMed  Google Scholar 

Hernández Alava M, Pudney S, Wailoo A. Estimating EQ-5D by age and sex for the UK. Sheffield, UK. 2022.

Song JI, Shin DW, Choi JY, Kang J, Baek YJ, Mo HN, et al. Quality of life and mental health in the bereaved family members of patients with terminal cancer. Psychooncology. 2012;21(11):1158–66.

Article  PubMed  Google Scholar 

Jones KC, Burns A. Unit costs of health and social care 2021. Kent, UK. 2021.

NHS England. National schedule of NHS costs 2020/21. London, UK. 2022.

Joint Formulary Committee. British National Formulary (online). London, UK. 2022.

NHS Business Services Authority. What are the current costs of NHS treatment in England? 2023. https://faq.nhsbsa.nhs.uk/knowledgebase/article/KA-03997/en-us. Accessed 05 Jan 2023.

Weisbrod BA. The valuation of human capital. J Polit Econ. 1961;69(5):425–36.

Article  Google Scholar 

McNamara S, Schneider PP, Love-Koh J, Doran T, Gutacker N. Quality-adjusted life expectancy norms for the English population. Value Health. 2022;38:901.

Google Scholar 

Moore A, Young CA, Hughes DA. Health utilities and costs for motor neurone disease. Value Health. 2019;22(11):1257–65.

Article  PubMed  Google Scholar 

Atassi N, Berry J, Shui A, Zach N, Sherman A, Sinani E, et al. The PRO-ACT database: design, initial analyses, and predictive features. Neurology. 2014;83(19):1719–25.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Tavakoli M. Disease progression in amyotrophic lateral sclerosis. Identifying the cost-utility of riluzole by disease stage. Eur J Health Econ. 2002;3(3):156–65.

Article  CAS  PubMed  Google Scholar 

Davis S. Assessing technologies that are not cost-effective at a zero price. Sheffield, UK. 2014.

Mladsi D, Barnett CL, Mader G, Russell-Smith TA, Unuigbe A, Bell T. The zero-price conundrum: exploration of scenarios where a clinically effective new drug might not be cost-effective at zero price. Value Health. 2023;26(3):384–91.

Article  PubMed  Google Scholar 

McFarlane R, Galvin M, Heverin M, Mac Domhnaill É, Murray D, Meldrum D, et al. PRECISION ALS-an integrated pan European patient data platform for ALS. Amyotroph Lateral Scler Frontotemporal Degener. 2023;24(5–6):389–93.

Article  CAS  PubMed  Google Scholar 

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