{"id":"W4409665556","doi":"10.1111/cts.70215","title":"Unlocking the Mysteries of Rare Disease Drug Development: A Beginner's Guide for Clinical Pharmacologists","year":2025,"lang":"en","type":"review","venue":"Clinical and Translational Science","topic":"Chronic Lymphocytic Leukemia Research","field":"Medicine","cited_by":2,"is_retracted":false,"has_abstract":true,"ca_institutions":"AstraZeneca (Canada)","funders":"","keywords":"Drug development; Disease; Rare disease; Medicine; Data science; Intensive care medicine; Bioinformatics; Computer science; Drug; Pharmacology; Pathology; Biology","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":[],"consensus_categories":[],"category_scores_codex":[0.003892908,0.001292592,0.001736429,0.002694455,0.0007716289,0.003653188,0.001611212,0.004072522,0.007736969],"category_scores_gemma":[0.006083139,0.0007229911,0.001125456,0.001990069,0.002395319,0.008451759,0.002421898,0.01206798,0.006478573],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.001790553,"about_ca_system_score_gemma":0.003445184,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001413709,"about_ca_topic_score_gemma":0.003380235,"domain_scores_codex":[0.9989294,0.0003425187,0.0001426298,0.0001044533,0.0003980971,0.00008278051],"domain_scores_gemma":[0.9956191,0.002897838,0.0002487323,0.0001274672,0.0006832868,0.0004235021],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.000066889,0.0001258156,0.0001663463,0.005930258,0.00006788251,0.0003834294,0.00035389,0.0005877481,0.0009752482,0.03326953,0.4042401,0.5538328],"study_design_scores_gemma":[0.00001658552,0.00005912263,0.00009908091,0.002715938,0.00001699963,0.000645223,0.00009475289,0.00009883478,0.00008849975,0.014265,0.9818807,0.00001929782],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.0001288922,0.9639746,0.002872526,0.02468185,0.003370925,0.00004781732,0.00007914938,0.00009713904,0.004747125],"genre_scores_gemma":[0.001005874,0.9683405,0.006033643,0.01513672,0.00418493,0.00009255993,0.0001058757,0.00003578444,0.005064123],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.007736969,"threshold_uncertainty_score":0.02588278,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.225484600808778,"score_gpt":0.549651174667454,"score_spread":0.3241665738586759,"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."}}