{"id":"W3014562531","doi":"10.2147/ppa.s233830","title":"&lt;p&gt;Assessing Preferences for Rare Disease Treatment: Qualitative Development of the Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire (PNH-PPQ©)&lt;/p&gt;","year":2020,"lang":"en","type":"article","venue":"Patient Preference and Adherence","topic":"Complement system in diseases","field":"Immunology and Microbiology","cited_by":16,"is_retracted":false,"has_abstract":true,"ca_institutions":"Health Sciences Centre; Sunnybrook Health Science Centre","funders":"Alexion Pharmaceuticals","keywords":"Medicine; Patient-reported outcome; Paroxysmal nocturnal hemoglobinuria; Eculizumab; Patient experience; Family medicine; Debriefing; Disease; Pediatrics; Quality of life (healthcare); Health care; Internal medicine; Nursing; Immunology","routes":{"ca_aff":true,"ca_fund":false,"ca_venue":false,"about_ca":true,"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":[0.01751745,0.0003471181,0.0002818405,0.0008160488,0.001545995,0.001076028,0.0006925403,0.0005698981,0.003611317],"category_scores_gemma":[0.01687737,0.0002874651,0.0004192039,0.0006966323,0.002494084,0.001189371,0.002167696,0.001033122,0.0004601989],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.002495331,"about_ca_system_score_gemma":0.004401747,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.002017052,"about_ca_topic_score_gemma":0.003801617,"domain_scores_codex":[0.9920226,0.006589164,0.0002509196,0.0002490246,0.0004474377,0.0004406759],"domain_scores_gemma":[0.9841893,0.01252527,0.0009110847,0.0004063668,0.001275263,0.0006925483],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"qualitative","study_design_gemma":"qualitative","study_design_scores_codex":[0.0002804836,0.0002544872,0.01218681,0.003037171,0.00001874522,0.001525486,0.8206002,0.0006152989,0.01129281,0.005764375,0.01380924,0.1306149],"study_design_scores_gemma":[0.0001688315,0.0006371026,0.02009638,0.001432789,0.00002518932,0.001030137,0.8536535,0.001821528,0.008200912,0.006844336,0.1059502,0.0001391044],"study_design_candidate":"qualitative","study_design_consensus":"qualitative","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9204887,0.0005248711,0.04303896,0.00493296,0.0001584303,0.01413861,0.002796071,0.0001274841,0.01379402],"genre_scores_gemma":[0.8874025,0.0006433243,0.07708454,0.001993663,0.00004166885,0.02618824,0.0006205986,0.00008694959,0.005938394],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.01751745,"threshold_uncertainty_score":0.09264225,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.09551028481856084,"score_gpt":0.30504505465421,"score_spread":0.2095347698356492,"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."}}