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. 2023 Feb;29(2):10.18553/jmcp.2023.29.2.216. doi: 10.18553/jmcp.2023.29.2.216

TABLE 3.

Votes on Other Benefits and Contextual Considerations for AMX0035 and Oral Edaravone

When making judgments of overall long-term value for money, what is the relative priority that should be given to any effective treatment for ALS, on the basis of the following contextual considerations?
Contextual consideration Very low priority Low priority Average priority High priority Very high priority
Acuity of need for treatment of individual patients based on short-term risk of death or progression to permanent disability 0 0 2 3 10
Magnitude of the lifetime impact on individual patients of the condition being treated 0 2 3 4 6
What are the relative effects of AMX0035 plus standard of care vs standard of care alone on the following outcomes that inform judgment of the overall long-term value for money of AMX0035?
Potential other benefit or disadvantage Major negative effect Minor negative effect No difference Minor positive effect Major positive effect
Patients’ ability to achieve major life goals related to education, work, or family life 0 0 2 10 3
Caregivers’ quality of life and/or ability to achieve major life goals related to education, work, or family life 0 0 3 11 1
What are the relative effects of oral edaravone plus standard of care vs standard of care alone on the following outcomes that inform judgment of the overall long-term value for money of oral edaravone?
Potential other benefit or disadvantage Major negative effect Minor negative effect No difference Minor positive effect Major positive effect
Patients’ ability to achieve major life goals related to education, work, or family life 0 0 4 10 1
Caregivers’ quality of life and/or ability to achieve major life goals related to education, work, or family life 0 0 5 10 0
Patients’ ability to manage and sustain treatment given the complexity of regimen compared with intravenous edaravone 0 0 0 2 13

ALS = amyotrophic lateral sclerosis.