Incorporating patient preferences and burden-of-disease in evaluating ALS drug candidate AMX0035: a Bayesian decision analysis perspective.
Amyotrophic lateral sclerosis & frontotemporal degeneration（2023）
摘要
BDA provides a useful framework to incorporate subjective perspectives of ALS patients and objective burden-of-disease metrics to evaluate the therapeutic effects of AMX0035 in its phase 2 trial.
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关键词
Amyotrophic lateral sclerosis,Bayesian decision analysis,clinical trial development,patient value,pharmaceutical regulation,survival analysis
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