OBJECTIVES:

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that leads to muscle atrophy and death. Two recently Food and Drug Administration (FDA) approved drugs, sodium phenylbutyrate/taurursodiol (PB/TURSO) and the oral formulation of edaravone, provide additional treatment options for patients with ALS. Our objective was to evaluate the cost-effectiveness of these two products in addition to their respective standards of care (SOC) versus SOC alone from both the US payer perspective and modified societal perspective.

METHODS:

We developed a Markov cohort model with monthly cycles based on the King’s ALS staging system over a lifetime time horizon. The model incorporated data from pivotal clinical trials (NCT03127514 for PB/TURSO, NCT01492686 for oral edaravone). Model inputs included disease progression, health state utilities, direct and indirect health state costs, and drug costs. Annual drug costs of $158,313 and $167,244 were used for PB/TURSO and oral edaravone, respectively. We performed one-way sensitivity and probabilistic sensitivity analyses to evaluate uncertainty. Both costs and outcomes were discounted at a rate of 3% annually. The SOC in the assessment of PB/TURSO included riluzole and intravenous edaravone, while the SOC for oral edaravone only included riluzole.

RESULTS:

In the base-case analysis, PB/TURSO added to SOC produced 0.14 additional quality-adjusted life-years (QALYs) with increased costs of $289,036 resulting in an incremental cost-effectiveness ratio of $2.067 million per QALY gained compared to SOC alone. Treatment with oral edaravone added to its SOC produced an additional 0.04 QALYs and an incremental cost of $423,567 resulting in an incremental cost-effectiveness ratio exceeding $11 million per QALY gained compared to SOC alone.

CONCLUSIONS:

Treatments for ALS are limited and the recent FDA approvals of PB/TURSO and oral edaravone provide additional options to patients that provide clinical benefit. However, these treatments are not considered cost-effective at their current prices based on commonly used cost-effectiveness thresholds.