Malgorzata Ciepielewska

ALS amyotrophic lateral sclerosis CNDR Canadian Neuromuscular Disease Registry CReATe Clinical Research in ALS and Related Disorders for Therapeutic Development EMA European Medicines Agency ENCALS European Network to Cure ALS EURALS European ALS Consortium FDA Food and Drug Administration GTAC Genomic Translation for ALS Care HEOR health economics and outcomes research ICMRA International Coalition of Medicines Regulatory Authorities ISPE International Society for Pharmacoepidemiology ISPOR...

Summary Background We aimed to evaluate overall survival in US patients with amyotrophic lateral sclerosis (ALS) treated with intravenous (IV) edaravone compared with those not treated with IV edaravone in a real-world setting. Methods This exploratory retrospective comparative effectiveness observational analysis included patients with ALS who were enrolled in an administrative claims database from 8 August 2017 to 31 March 2020.

ALS amyotrophic lateral sclerosis CDM Clinformatics Data Mart IV intravenous RCT randomized controlled trial US United States Amyotrophic lateral sclerosis (ALS) can be challenging to evaluate in randomized controlled trials (RCTs) due to numerous factors including, but not limited to, the rarity of its occurrence, clinical and pathological heterogeneity, inadequate animal models, methodologic study design challenges, lack of sensitive biomarkers, and delays in diagnosis [1, 2].