{"id":"W4401648132","doi":"10.1002/cpt.3407","title":"Getting the Dose Right in Drug Development for Rare Diseases: Barriers and Enablers","year":2024,"lang":"en","type":"article","venue":"Clinical Pharmacology & Therapeutics","topic":"Genomics and Rare Diseases","field":"Biochemistry, Genetics and Molecular Biology","cited_by":12,"is_retracted":false,"has_abstract":true,"ca_institutions":"AstraZeneca (Canada)","funders":"","keywords":"Modalities; Medicine; Intensive care medicine; Drug development; Clinical trial; Disease; Population; Orphan drug; Drug; Bioinformatics; 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":[0.1131457,0.001081824,0.002575322,0.001216369,0.001864328,0.01201785,0.003212755,0.007524082,0.008134621],"category_scores_gemma":[0.09482071,0.0009671269,0.001608464,0.001300997,0.01258802,0.02002188,0.01018842,0.01548663,0.003119482],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.005368432,"about_ca_system_score_gemma":0.01156523,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001771771,"about_ca_topic_score_gemma":0.002218816,"domain_scores_codex":[0.9565551,0.02210777,0.004452396,0.004057352,0.01133827,0.00148917],"domain_scores_gemma":[0.8831905,0.09110691,0.006673935,0.007437137,0.008142835,0.00344867],"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":[0.0007524038,0.0004291571,0.002501434,0.004723025,0.0001968339,0.0004986839,0.00173863,0.00269523,0.004515669,0.2802281,0.04918164,0.6525391],"study_design_scores_gemma":[0.0003916001,0.002163014,0.002165182,0.005969716,0.0001697245,0.001856799,0.001799819,0.001782537,0.006220021,0.2887438,0.6885031,0.0002346398],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"commentary","genre_gemma":"other","genre_scores_codex":[0.01199946,0.3230542,0.1117192,0.5051302,0.006690186,0.0007170741,0.0004587874,0.0006647308,0.03956607],"genre_scores_gemma":[0.224257,0.327744,0.2388394,0.1755287,0.01501495,0.00256942,0.0005217462,0.001262264,0.0142626],"genre_candidate":"other","genre_consensus":null,"teacher_disagreement_score":0.1131457,"threshold_uncertainty_score":0.5983788,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02623634451511147,"score_gpt":0.3615465485684599,"score_spread":0.3353102040533484,"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."}}