OBJECTIVES:

To quantitatively assess treatment attributes that are most important to people living with ALS (pALS) in the United States.

METHODS:

A 30-minute IRB approved web-enabled questionnaire was administered to pALS and caregivers responding as patient proxies (recruitment ongoing), to capture information on symptomology, treatment experiences, and treatment attribute preferences for ALS. The study included a Discrete Choice Experiment (DCE) to quantify preferences of pALS. Treatment attributes captured in the DCE included whether the therapy targeted a specific genetic variation associated with ALS, route of administration, efficacy, safety, requirement for genetic testing, and clinical monitoring. Descriptive statistics were calculated as means, standard deviations, frequencies, and percentages. The DCE was analyzed using a hierarchical Bayesian model.

RESULTS:

Based on interim results from 52 respondents (48 pALS, 4 caregivers), 69.2% have been prescribed an ALS treatment during their disease history of which 61.1% reported discontinuing at least one ALS treatment. The top reasons for discontinuation included failure to reduce ALS symptoms (44.1%), not slowing ALS progression enough (41.2%), and undesirable side effects (29.4%). The most important attributes pALS seek in a new treatment were effect on survival (relative importance 19.8) (65.0% decreased risk of death or permanent ventilation preferred to 40.0% decreased risk and no impact), treatment-related adverse events (18.5) (no serious adverse events (SAEs) preferred to SAEs reported in 7.0% and 15.0% of participants on treatment), and effect on ALSFRS-R (17.3) (6.0 difference in ALSFRS-R preferred to a 3.5 and 2.5 difference).

CONCLUSIONS:

Approximately two-thirds of respondents who have been prescribed an ALS treatment have discontinued at least one, largely due to lack of efficacy and treatment side effects, demonstrating a high unmet need for more effective and tolerable therapies. The most important attributes for new ALS treatments were effect on survival, safety, and effect on ALSFRS-R.