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

Area of Science:

• Health Economics
• Neurology
• Pharmacoeconomics

Background:

• Amyotrophic lateral sclerosis (ALS) treatment cost-effectiveness requires updated analysis.
• Recent advances in disease staging and drug effect understanding necessitate reevaluation.
• The
• fine'til 9
• (FT9) staging method offers improved disease characterization.

Purpose of the Study:

• To reexamine the cost-effectiveness of riluzole for amyotrophic lateral sclerosis (ALS).
• To incorporate recent advances in disease staging (FT9) and stage-specific drug effects.
• To provide a framework for future health economic evaluations of ALS treatments.

Main Methods:

• Utilized the FT9 staging method for ALS.
• Estimated stage-specific health utilities (EQ-5D) from an institutional cohort.
• Employed Markov models to evaluate riluzole cost-effectiveness from healthcare and societal perspectives over 5- and 10-year horizons.
• Disaggregated costs into recurring costs (RCs) and transition/tollgate costs (TCs).

Main Results:

• Riluzole use yielded 0.182 quality-adjusted life years (QALYs) gained at a cost difference of $12,348 from the healthcare perspective over 5 years.
• The incremental cost-effectiveness ratio (ICER) was calculated at $67,658/QALY.
• ICER was sensitive to drug price and RCs, with higher TCs modestly reducing the ICER.

Conclusions:

• The study establishes a framework for health economic analyses of ALS treatments using FT9 staging.
• Prospective, stage-specific cost data are crucial for accurate future analyses.
• Separating transition costs (TCs) from recurring costs (RCs) is vital for mitigating the impact of background care costs on the ICER.