{"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":"codex-gemma-dda1882f352a","candidate_categories":["metaepi_narrow","scholarly_communication","insufficient_payload"],"consensus_categories":[],"category_scores_codex":[0.004175076,0.0006968326,0.002698711,0.0005756713,0.001045755,0.001096741,0.001717895,0.0001738718,0.0003289928],"category_scores_gemma":[0.007784947,0.0004828069,0.001295479,0.0002761571,0.0001189869,0.0008089286,0.0001458437,0.0006837449,0.001272048],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.001187624,"about_ca_system_score_gemma":0.0003696847,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00003406783,"about_ca_topic_score_gemma":0.00001128626,"domain_scores_codex":[0.9913005,0.001446567,0.005341392,0.0006860588,0.0006048275,0.0006206908],"domain_scores_gemma":[0.9861873,0.002130049,0.008985038,0.001596959,0.000254744,0.00084592],"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":[0.0004441166,0.0009854088,0.007527105,0.005035943,0.001404177,0.00003896221,0.0009480644,0.0002589777,1.773448e-8,0.0007738614,0.7990775,0.1835059],"study_design_scores_gemma":[0.0004467484,0.0001972768,0.001419077,0.01418106,0.0003003493,0.00000811486,0.0003280715,0.000001653774,6.425553e-8,0.0001963162,0.9824286,0.0004926433],"study_design_candidate":"not_applicable","study_design_consensus":"not_applicable","genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.000883421,0.9654896,0.00004565389,0.02364938,0.003464785,0.002361799,0.003856574,0.00002913902,0.0002196673],"genre_scores_gemma":[0.01049053,0.9512904,0.00003468772,0.03519264,0.001183698,0.0004830383,0.0003742901,0.0002274126,0.0007232854],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.1833511,"threshold_uncertainty_score":0.9999402,"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."}}