A Cost-Effectiveness Framework for Amyotrophic Lateral Sclerosis, Applied to Riluzole

Nimish J. Thakore*, Erik P. Pioro, Belinda L. Udeh, Brittany R. Lapin, Irene L. Katzan

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

6 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)1543-1551
Number of pages9
JournalValue in Health
Issue number12
StatePublished - Dec 2020


  • EQ-5D
  • FT9
  • amyotrophic lateral sclerosis
  • fixed costs
  • riluzole
  • staging

ASJC Scopus subject areas

  • Public Health, Environmental and Occupational Health
  • Health Policy


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