{"id":"W4390791175","doi":"10.1186/s13023-023-02974-1","title":"Applying the win ratio method in clinical trials of orphan drugs: an analysis of data from the COMET trial of avalglucosidase alfa in patients with late-onset Pompe disease","year":2024,"lang":"en","type":"article","venue":"Orphanet Journal of Rare Diseases","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":8,"is_retracted":false,"has_abstract":true,"ca_institutions":"McGill University","funders":"Sanofi","keywords":"Context (archaeology); Clinical endpoint; Clinical trial; Medicine; Orphan drug; Confidence interval; Internal medicine; Physical therapy; Bioinformatics","routes":{"ca_aff":true,"ca_fund":false,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":false},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":["metaresearch"],"consensus_categories":[],"category_scores_codex":[0.2085501,0.00216862,0.005324639,0.004134164,0.000846175,0.00207397,0.001841267,0.002210725,0.004140903],"category_scores_gemma":[0.2354003,0.0006584395,0.01028701,0.002505578,0.001843558,0.001832258,0.002872515,0.003577814,0.0005130033],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0009927219,"about_ca_system_score_gemma":0.001833055,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000636776,"about_ca_topic_score_gemma":0.0008578901,"domain_scores_codex":[0.6682709,0.2994962,0.009842721,0.008173829,0.01269011,0.001526285],"domain_scores_gemma":[0.5959899,0.3608414,0.01785837,0.01725845,0.006634416,0.00141746],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"observational","study_design_scores_codex":[0.2279583,0.004739953,0.2577046,0.00942552,0.1248628,0.003452234,0.00263486,0.05239348,0.01068666,0.01192104,0.01262759,0.281593],"study_design_scores_gemma":[0.04958415,0.1036932,0.274914,0.001287555,0.05254522,0.004634123,0.00158756,0.4264256,0.0103363,0.02793757,0.04606163,0.0009931218],"study_design_candidate":"observational","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.7185906,0.009677653,0.2458863,0.002227608,0.0008168834,0.01186401,0.005361303,0.0009883008,0.004587318],"genre_scores_gemma":[0.8774969,0.0005631298,0.1083868,0.001158288,0.0002468237,0.008346843,0.002559108,0.000238714,0.001003232],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.2085501,"threshold_uncertainty_score":0.9759987,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.1007479342262677,"score_gpt":0.4411992037223181,"score_spread":0.3404512694960504,"validation_status":"score_only:v0-immature-baseline","note":"Baseline scores from an immature model (maturity gate not passed). Scores rank; they never assert a category."}}