{"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":"codex-gemma-dda1882f352a","candidate_categories":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.0001762301,0.0005878651,0.0006515242,0.00007505879,0.0006805987,0.0001029866,0.0005930755,0.00016088,0.0002810458],"category_scores_gemma":[0.0004522313,0.0004043656,0.0001957041,0.0002661587,0.0004034996,0.0004033482,0.0003670156,0.0001915887,0.00003756092],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001354372,"about_ca_system_score_gemma":0.0009316581,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00003163475,"about_ca_topic_score_gemma":0.0001141135,"domain_scores_codex":[0.9962987,0.0007465133,0.00114542,0.000923208,0.0002673501,0.0006187629],"domain_scores_gemma":[0.9976082,0.0003955964,0.0008934579,0.0004828734,0.0003926872,0.0002271637],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"observational","study_design_scores_codex":[0.006323868,0.003365498,0.1471619,0.003364052,0.001706707,0.00001289767,0.2131583,0.0002074384,0.1824242,0.007584608,0.003682772,0.4310077],"study_design_scores_gemma":[0.0156428,0.01271803,0.5614565,0.01219224,0.001663202,0.00003406075,0.0285488,0.0007441994,0.2994186,0.007366964,0.05520293,0.005011697],"study_design_candidate":"observational","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9905957,0.004740937,0.0007843459,0.0001781694,0.0004825781,0.002114808,0.0007303781,0.00009397791,0.0002790828],"genre_scores_gemma":[0.9977979,0.00007449994,0.0007246634,0.0001156286,0.00003523178,0.0008541041,0.0002578996,0.00002583609,0.0001142175],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.425996,"threshold_uncertainty_score":0.9998408,"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."}}