{"id":"W4327679283","doi":"10.1177/17407745231158906","title":"Dynamic use of historical controls in clinical trials for rare disease research: A re-evaluation of the MILES trial","year":2023,"lang":"en","type":"article","venue":"Clinical Trials","topic":"Tuberous Sclerosis Complex Research","field":"Medicine","cited_by":4,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"National Center for Advancing Translational Sciences; National Institute for Occupational Safety and Health; National Center for Research Resources; Pfizer; National Heart, Lung, and Blood Institute; Canadian Institutes of Health Research; National Institute of Neurological Disorders and Stroke; Pfizer Pharmaceuticals; Food and Drug Administration; U.S. Food and Drug Administration; University of South Florida; National Institutes of Health; U.S. Department of Health and Human Services; Cincinnati Children's Hospital Medical Center","keywords":"Randomization; Medicine; Clinical trial; Placebo; Propensity score matching; Randomized controlled trial; Selection bias; Comparability; Physical therapy; Internal medicine; Alternative medicine","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"codex-gemma-dda1882f352a","candidate_categories":["metaresearch"],"consensus_categories":["metaresearch"],"category_scores_codex":[0.4617188,0.000239135,0.004743658,0.0005377306,0.0001127091,0.00003937565,0.0006191925,0.0005609739,0.0002268336],"category_scores_gemma":[0.8829031,0.0001506209,0.003199812,0.001369592,0.0008632105,0.0001013937,0.0003444999,0.001181464,0.00003154258],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000543181,"about_ca_system_score_gemma":0.002815336,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00009720744,"about_ca_topic_score_gemma":0.0001017271,"domain_scores_codex":[0.8513215,0.1245962,0.01695232,0.001330825,0.00478096,0.001018258],"domain_scores_gemma":[0.3550931,0.6305912,0.004561801,0.003731558,0.004784806,0.001237618],"domain_codex":null,"domain_gemma":"methods","domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"observational","study_design_scores_codex":[0.6716157,0.006667882,0.0419412,0.0005515293,0.0009765677,0.00002159377,0.0001783476,0.0001840356,0.003561158,0.0009335901,0.1440829,0.1292855],"study_design_scores_gemma":[0.4404484,0.006512889,0.481774,0.001454866,0.002203459,9.081436e-7,0.0001833173,0.03876694,0.0001354106,0.01448788,0.01370961,0.0003223068],"study_design_candidate":"randomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9605178,0.0005748604,0.00007471121,0.01639521,0.002592782,0.01934967,0.0004092386,0.00004927721,0.00003640182],"genre_scores_gemma":[0.9949,0.0005790503,0.0003730837,0.0002006585,0.001555392,0.00139318,0.00009073791,0.00005936599,0.0008485609],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.5059949,"threshold_uncertainty_score":0.6142141,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.9597579830287777,"score_gpt":0.7068922928152247,"score_spread":0.252865690213553,"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."}}