{"id":"W2981176489","doi":"10.12927/hcpol.2019.25937","title":"Orphan Drug Pricing and Costs: A Case Study of Kalydeco and Orkambi","year":2019,"lang":"en","type":"article","venue":"Healthcare policy","topic":"Health Systems, Economic Evaluations, Quality of Life","field":"Economics, Econometrics and Finance","cited_by":17,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Calgary","funders":"Canadian Institutes of Health Research","keywords":"Ivacaftor; Orphan drug; Revenue; Cystic fibrosis; Drug pricing; Narrative review; Medicine; Business; Actuarial science; Intensive care medicine; Internal medicine; Cystic fibrosis transmembrane conductance regulator; Bioinformatics; Finance","routes":{"ca_aff":true,"ca_fund":true,"ca_venue":true,"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":[0.0009656312,0.0004146287,0.0005087912,0.00149536,0.006576574,0.002466212,0.001038041,0.002601055,0.006522063],"category_scores_gemma":[0.003331707,0.0003483315,0.0005652145,0.002042472,0.00188545,0.001336997,0.002308021,0.002572273,0.0003291147],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.007209791,"about_ca_system_score_gemma":0.003866688,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.1228581,"about_ca_topic_score_gemma":0.2112678,"domain_scores_codex":[0.9990465,0.000344828,0.0000285502,0.00004114398,0.00009601084,0.0004429714],"domain_scores_gemma":[0.9979662,0.0009783943,0.0003347388,0.0000510922,0.0001324521,0.0005371491],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"case_report","study_design_scores_codex":[0.001698562,0.00573634,0.434073,0.001460988,0.0003728049,0.2896941,0.08354876,0.008203722,0.002630395,0.0812123,0.02026971,0.07109933],"study_design_scores_gemma":[0.0005170107,0.001429842,0.3638307,0.001632836,0.0003180818,0.05772408,0.4701863,0.01379796,0.001365826,0.009451932,0.07951277,0.0002327436],"study_design_candidate":"case_report","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9839484,0.0009290106,0.0003798702,0.002900767,0.00001994879,0.000131142,0.0001795075,0.000006302588,0.01150506],"genre_scores_gemma":[0.9939039,0.001428388,0.0006837711,0.0004493424,0.00001844163,0.00007661945,0.0000957209,0.00001001051,0.003333729],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.1228581,"threshold_uncertainty_score":0.2442859,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.2408333544792701,"score_gpt":0.463508640434325,"score_spread":0.222675285955055,"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."}}