{"id":"W2766356879","doi":"10.1186/s13023-017-0718-x","title":"Measuring what matters to rare disease patients – reflections on the work by the IRDiRC taskforce on patient-centered outcome measures","year":2017,"lang":"en","type":"review","venue":"Orphanet Journal of Rare Diseases","topic":"Health Systems, Economic Evaluations, Quality of Life","field":"Economics, Econometrics and Finance","cited_by":206,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"National Institute of Neurological Disorders and Stroke; Universitaire Ziekenhuizen Leuven, KU Leuven; Instituto de Salud Carlos III; European Organisation for Rare Diseases; Patient-Centered Outcomes Research Institute; KU Leuven; National Center for Advancing Translational Sciences; Leids Universitair Medisch Centrum; Universiteit Leiden; Institut National de la Santé et de la Recherche Médicale; National Institutes of Health; University of Birmingham; Medical Center, University of Rochester; Newcastle University; University of Washington; Children's National Hospital; Duchenne Parent Project; Chiesi Farmaceutici; University of Rochester; Pfizer; Northwestern University; Istituto Superiore di Sanità; McMaster University; King's College London; Vrije Universiteit Amsterdam; Parent Project Muscular Dystrophy; National Institute for Health and Care Research","keywords":"Patient-reported outcome; Stakeholder; Outcome (game theory); Disease; Value (mathematics); Medicine; Rare disease; Psychology; Medical education; Nursing; Computer science; Quality of life (healthcare); Public relations; Pathology","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":["metaresearch"],"consensus_categories":[],"category_scores_codex":[0.6198406,0.00145017,0.003927426,0.005138599,0.006833644,0.01789353,0.01183077,0.01983756,0.003085224],"category_scores_gemma":[0.4775788,0.001328687,0.004650159,0.0056549,0.04513565,0.03233362,0.01955551,0.07579718,0.001695983],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.03062863,"about_ca_system_score_gemma":0.08219244,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.06569606,"about_ca_topic_score_gemma":0.05189745,"domain_scores_codex":[0.5981249,0.2952031,0.03266484,0.01236654,0.05382623,0.007814454],"domain_scores_gemma":[0.1662681,0.7134172,0.01246511,0.01535006,0.07741969,0.01507991],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"not_applicable","study_design_gemma":"not_applicable","study_design_scores_codex":[0.0002290328,0.0002969784,0.0038987,0.003620503,0.0001215814,0.0001817711,0.02261003,0.0008346368,0.0003192236,0.1447409,0.5302716,0.2928751],"study_design_scores_gemma":[0.0001967093,0.0004063267,0.006138779,0.03877278,0.0001096267,0.0005444688,0.0232638,0.001466754,0.000645404,0.09606346,0.8317782,0.0006136344],"study_design_candidate":"not_applicable","study_design_consensus":"not_applicable","genre_codex":"commentary","genre_gemma":"review","genre_scores_codex":[0.0004298686,0.02548083,0.001355154,0.9680517,0.003762324,0.00003609349,0.00006360399,0.00001655157,0.000803985],"genre_scores_gemma":[0.03452165,0.08421638,0.03472892,0.8259486,0.01681184,0.001401969,0.0004263993,0.0003391213,0.001605146],"genre_candidate":"review","genre_consensus":null,"teacher_disagreement_score":0.6198406,"threshold_uncertainty_score":0.4688042,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.5492244863313144,"score_gpt":0.4533849642184486,"score_spread":0.09583952211286578,"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."}}