{"id":"W4408930014","doi":"10.1371/journal.pone.0304540","title":"Application of observational research methods to real-world studies for rare disease drugs: A scoping review protocol","year":2025,"lang":"en","type":"review","venue":"PLoS ONE","topic":"Genomics and Rare Diseases","field":"Biochemistry, Genetics and Molecular Biology","cited_by":1,"is_retracted":false,"has_abstract":true,"ca_institutions":"SickKids Foundation; Sunnybrook Health Science Centre; Public Health Ontario; University of Toronto","funders":"","keywords":"Observational study; Randomized controlled trial; Medicine; Systematic review; Rare disease; Protocol (science); Disease; Intensive care medicine; Clinical trial; MEDLINE; Alternative medicine; 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":["metaresearch"],"consensus_categories":[],"category_scores_codex":[0.1838341,0.004918833,0.01181335,0.01890731,0.005244493,0.009418705,0.007133367,0.01096728,0.07133165],"category_scores_gemma":[0.1463363,0.006191373,0.01314532,0.01457504,0.006071787,0.009113456,0.009448898,0.008865269,0.01980486],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.01471278,"about_ca_system_score_gemma":0.09635242,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00319995,"about_ca_topic_score_gemma":0.007244651,"domain_scores_codex":[0.9016875,0.04506502,0.03645113,0.005779352,0.008384735,0.002632281],"domain_scores_gemma":[0.8395968,0.06707719,0.0223041,0.0148803,0.05036369,0.005777911],"domain_codex":null,"domain_gemma":"methods","domain_candidate":"methods","domain_consensus":null,"study_design_codex":"systematic_review","study_design_gemma":"systematic_review","study_design_scores_codex":[0.007086359,0.00103436,0.001813935,0.7244263,0.002083653,0.00132343,0.00721099,0.002364368,0.002708183,0.02222111,0.08253567,0.1451916],"study_design_scores_gemma":[0.01605892,0.00161997,0.00523216,0.5497673,0.003707344,0.0008917146,0.004696875,0.002068383,0.002641389,0.02156054,0.3911794,0.0005759921],"study_design_candidate":"systematic_review","study_design_consensus":"systematic_review","genre_codex":"protocol","genre_gemma":"protocol","genre_scores_codex":[0.000263069,0.001051755,0.003255058,0.0006004276,0.0002848884,0.990676,0.002887796,0.00008020717,0.0009007377],"genre_scores_gemma":[0.0003072064,0.0008067628,0.008283881,0.0002143757,0.00003263551,0.9895834,0.0004861186,0.000008787927,0.0002768793],"genre_candidate":"protocol","genre_consensus":"protocol","teacher_disagreement_score":0.8161659,"threshold_uncertainty_score":0.9722195,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.3775274304357784,"score_gpt":0.5708686487372452,"score_spread":0.1933412183014668,"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."}}