@article{3bb4b2fa6d9949d695989d2c8bf0ef0a,
title = "A Cost-Effectiveness Framework for Amyotrophic Lateral Sclerosis, Applied to Riluzole",
abstract = "Objectives: Reexamine cost-effectiveness of riluzole in the treatment of amyotrophic lateral sclerosis (ALS) in light of recent advances in disease staging and understanding of stage-specific drug effect. Methods: ALS was staged according to the “fine'til 9” (FT9) staging method. Stage-specific health utilities (EQ-5D, US valuation) were estimated from an institutional cohort, whereas literature informed costs and transition probabilities. Costs at 2018 prices were disaggregated into recurring costs (RCs) and “one-off” transition/“tollgate” costs (TCs). Five- and 10-year horizons starting in stage 1 disease were examined from healthcare sector and societal perspectives using Markov models to evaluate riluzole use, at a threshold of $100 000/quality-adjusted life year (QALY). Probabilistic and deterministic sensitivity analyses were conducted. Results: Mean EQ-5D utilities for stages 0 to 4 were 0.79, 0.74, 0.63, 0.54, and 0.46, respectively. From the healthcare sector perspective at the 5-year horizon, riluzole use contributed to 0.182 QALY gained at the cost difference of $12 348 ($5403 riluzole cost, $8870 RC and −$1925 TC differences), translating to an incremental cost-effectiveness ratio (ICER) of $67 658/QALY. Transition probability variation contributed considerably to ICER uncertainty (−30.2% to +90.0%). ICER was sensitive to drug price and RCs, whereas higher TCs modestly reduced ICER due to delayed tollgates. Conclusion: This study provides a framework for health economic studies of ALS treatments using FT9 staging. Prospective stage-specific and disaggregated cost measurement is warranted for accurate future cost-effectiveness analyses. Appropriate separation of TCs from RCs substantially mitigates the high burden of background cost of care on the ICER.",
keywords = "EQ-5D, FT9, amyotrophic lateral sclerosis, fixed costs, riluzole, staging",
author = "Thakore, {Nimish J.} and Pioro, {Erik P.} and Udeh, {Belinda L.} and Lapin, {Brittany R.} and Katzan, {Irene L.}",
note = "Funding Information: Conflict of Interest Disclosures: Dr Thakore reports grants from Novartis Pharmaceuticals , outside the submitted work. Dr Pioro reports grants from National Institutes of Health and Center for Disease Control during the conduct of the study; additional grant support outside the submitted work is from the ALS Association and CDC / NIH , as well as personal fees from Avanir Pharmaceuticals, Inc, Biohaven Pharmaceuticals, Cytokinetics, Inc, ITF Pharma, Inc, MT Pharma America, Inc, NeuroTherapia, Inc, and Otsuka America, Inc. Dr Udeh has nothing to disclose. Dr Lapin has nothing to disclose. Dr Katzan reports grants from TEVA Pharmaceuticals and grants from Novartis Pharmaceuticals , outside the submitted work. Funding Information: Author Contributions: Concept and design: Thakore, Pioro, Udeh, Acquisition of data: Pioro, Analysis and interpretation of data: Thakore, Lapin, Drafting of the manuscript: Thakore, Lapin, Critical revision of the paper for important intellectual content: Pioro, Udeh, Lapin, Katzan, Statistical analysis: Thakore, Lapin, Provision of study materials or patients: Pioro, Administrative, technical, or logistic support: Udeh, Katzan, Supervision: Katzan, Conflict of Interest Disclosures: Dr Thakore reported receiving grants from Novartis Pharmaceuticals outside the submitted work. Dr Pioro reported receiving grants from the National Institutes of Health, the ALS Association, the Centers for Disease Control and Prevention/National Institute of Justice outside the submitted work; personal fees from Avanir Pharmaceuticals, Inc, Biohaven Pharmaceuticals, Inc, Cytokinetics, Inc, ITF Pharma, Inc, MT Pharma America, Inc, NeuroTherapia, Inc, and Otsuka America, Inc outside the submitted work; and clinical trial support from Biohaven Pharmaceuticals, Inc outside the submitted work. Dr Katzan reported receiving grants from TEVA Pharmaceuticals and Novartis Pharmaceuticals outside the submitted work. No other disclosures were reported. Funding/Support: This work was supported by an internal NICORE (Neurological Institute Center for Outcomes Research and Evaluation, Cleveland Clinic) grant, but not by any external funding. Role of the Funder/Sponsor: The funder had no role in the design and conduct of the study; collection, management, analysis, and interpretation of the data; preparation, review, or approval of the manuscript; and decision to submit the manuscript for publication. Acknowledgment: The authors thank NICORE, Cleveland Clinic, for supporting the effort. Mark Votruba, PhD, Eve Wittenberg, PhD, and anonymous reviewers provided invaluable insight and suggestions. Publisher Copyright: {\textcopyright} 2020 ISPOR–The Professional Society for Health Economics and Outcomes Research",
year = "2020",
month = dec,
doi = "10.1016/j.jval.2020.06.012",
language = "English (US)",
volume = "23",
pages = "1543--1551",
journal = "Value in Health",
issn = "1098-3015",
publisher = "Elsevier Limited",
number = "12",
}
