{"id":"https://openalex.org/W4414879142","doi":"https://doi.org/10.1017/s0266462325100457","title":"Recommended methods for the collection of clinical expert judgment in rare diseases: Generating evidence to support reimbursement of orphan drugs","display_name":"Recommended methods for the collection of clinical expert judgment in rare diseases: Generating evidence to support reimbursement of orphan drugs","publication_year":2025,"publication_date":"2025-01-01","ids":{"openalex":"https://openalex.org/W4414879142","doi":"https://doi.org/10.1017/s0266462325100457","pmid":"https://pubmed.ncbi.nlm.nih.gov/41054189","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/12592965"},"language":"en","primary_location":{"id":"doi:10.1017/s0266462325100457","doi":"https://doi.org/10.1017/s0266462325100457","is_oa":true,"landing_page_url":"https://doi.org/10.1017/s0266462325100457","pdf_url":"https://www.cambridge.org/core/services/aop-cambridge-core/content/view/F5C99074BED98B74B0EA854AF094B060/S0266462325100457a.pdf/div-class-title-recommended-methods-for-the-collection-of-clinical-expert-judgment-in-rare-diseases-generating-evidence-to-support-reimbursement-of-orphan-drugs-div.pdf","source":{"id":"https://openalex.org/S83891556","display_name":"International Journal of Technology Assessment in Health Care","issn_l":"0266-4623","issn":["0266-4623","1471-6348"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["anvur-scientific","cwts-core","doaj","dongbi-c","doyens","fnege-4","jufo-1","ki-jl-1","medline","norway-1","poland-70","russia-white-list-2","vabb-shw"],"host_organization":"https://openalex.org/P4310311721","host_organization_name":"Cambridge University Press","host_organization_lineage":["https://openalex.org/P4310311721","https://openalex.org/P4310311702"],"host_organization_lineage_names":["Cambridge University Press","University of Cambridge"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"International Journal of Technology Assessment in Health Care","raw_type":"journal-article"},"volume":null,"type":"article","is_traditional":true,"indexed_in":["crossref","datacite","doaj","pubmed"],"open_access":{"is_oa":true,"oa_status":"gold","oa_url":"https://www.cambridge.org/core/services/aop-cambridge-core/content/view/F5C99074BED98B74B0EA854AF094B060/S0266462325100457a.pdf/div-class-title-recommended-methods-for-the-collection-of-clinical-expert-judgment-in-rare-diseases-generating-evidence-to-support-reimbursement-of-orphan-drugs-div.pdf","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5113943169","display_name":"Annabel Griffiths","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210157105","display_name":"Costello Medical Consulting (United Kingdom)","ror":"https://ror.org/04pe4vg07","country_code":"GB","type":"company","lineage":["https://openalex.org/I4210157105"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Annabel Griffiths","raw_affiliation_strings":["Costello Medical"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Costello Medical","institution_ids":["https://openalex.org/I4210157105"]}]},{"author_position":"middle","author":{"id":null,"display_name":"Lorna Dunning","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I332035737","display_name":"National Institute for Health and Care Excellence","ror":"https://ror.org/015ah0c92","country_code":"GB","type":"government","lineage":["https://openalex.org/I332035737"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Lorna Dunning","raw_affiliation_strings":["National Institute for Health and Care Excellence"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"National Institute for Health and Care Excellence","institution_ids":["https://openalex.org/I332035737"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5073230749","display_name":"Karen M. Facey","orcid":"https://orcid.org/0000-0001-6670-5271","observed_orcids":["https://orcid.org/0000-0001-6670-5271"]},"institutions":[{"id":"https://openalex.org/I98677209","display_name":"University of Edinburgh","ror":"https://ror.org/01nrxwf90","country_code":"GB","type":"education","lineage":["https://openalex.org/I98677209"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Karen Facey","raw_affiliation_strings":["University of Edinburgh"],"raw_orcid":"https://orcid.org/0000-0001-6670-5271","affiliations":[{"raw_affiliation_string":"University of Edinburgh","institution_ids":["https://openalex.org/I98677209"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5019501211","display_name":"Dina Jankovi\u0107","orcid":"https://orcid.org/0000-0002-9311-1409","observed_orcids":["https://orcid.org/0000-0002-9311-1409"]},"institutions":[{"id":"https://openalex.org/I52099693","display_name":"University of York","ror":"https://ror.org/04m01e293","country_code":"GB","type":"education","lineage":["https://openalex.org/I52099693"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Dina Jankovic","raw_affiliation_strings":["University of York"],"raw_orcid":"https://orcid.org/0000-0002-9311-1409","affiliations":[{"raw_affiliation_string":"University of York","institution_ids":["https://openalex.org/I52099693"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5057088758","display_name":"Carlos Gonz\u00e1lez Malla","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I24354313","display_name":"Universidad de Buenos Aires","ror":"https://ror.org/0081fs513","country_code":"AR","type":"education","lineage":["https://openalex.org/I24354313"]}],"countries":["AR"],"is_corresponding":false,"raw_author_name":"Carlos Gonz\u00e1lez Malla","raw_affiliation_strings":["University of Buenos Aires"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"University of Buenos Aires","institution_ids":["https://openalex.org/I24354313"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5102733706","display_name":"Eric Low","orcid":"https://orcid.org/0000-0001-8600-3357","observed_orcids":["https://orcid.org/0000-0001-8600-3357"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Eric Low","raw_affiliation_strings":["Eric Low Consulting"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Eric Low Consulting","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5038451583","display_name":"Michela Meregaglia","orcid":"https://orcid.org/0000-0003-0092-5970","observed_orcids":["https://orcid.org/0000-0003-0092-5970"]},"institutions":[{"id":"https://openalex.org/I71209653","display_name":"Bocconi University","ror":"https://ror.org/05crjpb27","country_code":"IT","type":"education","lineage":["https://openalex.org/I71209653"]}],"countries":["IT"],"is_corresponding":false,"raw_author_name":"Michela Meregaglia","raw_affiliation_strings":["SDA Bocconi School of Management"],"raw_orcid":"https://orcid.org/0000-0003-0092-5970","affiliations":[{"raw_affiliation_string":"SDA Bocconi School of Management","institution_ids":["https://openalex.org/I71209653"]}]},{"author_position":"middle","author":{"id":null,"display_name":"Fabian Schmidt","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4405268425","display_name":"Recordati Rare Diseases, Inc. (United States)","ror":"https://ror.org/00x68eg25","country_code":"US","type":"company","lineage":["https://openalex.org/I4405268425"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Fabian Schmidt","raw_affiliation_strings":["Recordati Rare Diseases"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Recordati Rare Diseases","institution_ids":["https://openalex.org/I4405268425"]}]},{"author_position":"middle","author":{"id":null,"display_name":"Kevin Wilson","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I84884186","display_name":"Newcastle University","ror":"https://ror.org/01kj2bm70","country_code":"GB","type":"education","lineage":["https://openalex.org/I84884186"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Kevin Wilson","raw_affiliation_strings":["Newcastle University"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Newcastle University","institution_ids":["https://openalex.org/I84884186"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5024080502","display_name":"Sheela Upadhyaya","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Sheela Upadhyaya","raw_affiliation_strings":["Life Sciences Consultant"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Life Sciences Consultant","institution_ids":[]}]},{"author_position":"middle","author":{"id":null,"display_name":"Noa Chapman","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210157105","display_name":"Costello Medical Consulting (United Kingdom)","ror":"https://ror.org/04pe4vg07","country_code":"GB","type":"company","lineage":["https://openalex.org/I4210157105"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Noa Chapman","raw_affiliation_strings":["Costello Medical"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Costello Medical","institution_ids":["https://openalex.org/I4210157105"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5119871416","display_name":"Chloe Zentai","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210157105","display_name":"Costello Medical Consulting (United Kingdom)","ror":"https://ror.org/04pe4vg07","country_code":"GB","type":"company","lineage":["https://openalex.org/I4210157105"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Chloe Zentai","raw_affiliation_strings":["Costello Medical"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Costello Medical","institution_ids":["https://openalex.org/I4210157105"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5085734783","display_name":"Sari D Wright","orcid":"https://orcid.org/0009-0006-8130-2503","observed_orcids":["https://orcid.org/0009-0006-8130-2503"]},"institutions":[{"id":"https://openalex.org/I4210157105","display_name":"Costello Medical Consulting (United Kingdom)","ror":"https://ror.org/04pe4vg07","country_code":"GB","type":"company","lineage":["https://openalex.org/I4210157105"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Sari Wright","raw_affiliation_strings":["Costello Medical"],"raw_orcid":"https://orcid.org/0009-0006-8130-2503","affiliations":[{"raw_affiliation_string":"Costello Medical","institution_ids":["https://openalex.org/I4210157105"]}]},{"author_position":"last","author":{"id":null,"display_name":"Isabelle Newell","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210157105","display_name":"Costello Medical Consulting (United Kingdom)","ror":"https://ror.org/04pe4vg07","country_code":"GB","type":"company","lineage":["https://openalex.org/I4210157105"]}],"countries":["GB"],"is_corresponding":true,"raw_author_name":"Isabelle Newell","raw_affiliation_strings":["Costello Medical"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Costello Medical","institution_ids":["https://openalex.org/I4210157105"]}]}],"institutions":[],"countries_distinct_count":4,"institutions_distinct_count":8,"corresponding_author_ids":[],"corresponding_institution_ids":["https://openalex.org/I4210157105"],"apc_list":{"value":3550,"currency":"USD","value_usd":3550},"apc_paid":{"value":3550,"currency":"USD","value_usd":3550},"fwci":0.7446,"has_fulltext":true,"cited_by_count":1,"citation_normalized_percentile":{"value":0.76346193,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":92,"max":95},"biblio":{"volume":"41","issue":"1","first_page":"e76","last_page":"e76"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10804","display_name":"Health Systems, Economic Evaluations, Quality of Life","score":0.5746999979019165,"subfield":{"id":"https://openalex.org/subfields/2002","display_name":"Economics and Econometrics"},"field":{"id":"https://openalex.org/fields/20","display_name":"Economics, Econometrics and Finance"},"domain":{"id":"https://openalex.org/domains/2","display_name":"Social Sciences"}},"topics":[{"id":"https://openalex.org/T10804","display_name":"Health Systems, Economic Evaluations, Quality of Life","score":0.5746999979019165,"subfield":{"id":"https://openalex.org/subfields/2002","display_name":"Economics and Econometrics"},"field":{"id":"https://openalex.org/fields/20","display_name":"Economics, Econometrics and Finance"},"domain":{"id":"https://openalex.org/domains/2","display_name":"Social Sciences"}},{"id":"https://openalex.org/T12443","display_name":"Delphi Technique in Research","score":0.3391000032424927,"subfield":{"id":"https://openalex.org/subfields/3312","display_name":"Sociology and Political Science"},"field":{"id":"https://openalex.org/fields/33","display_name":"Social Sciences"},"domain":{"id":"https://openalex.org/domains/2","display_name":"Social Sciences"}},{"id":"https://openalex.org/T11792","display_name":"Pharmaceutical Economics and Policy","score":0.031099999323487282,"subfield":{"id":"https://openalex.org/subfields/2002","display_name":"Economics and Econometrics"},"field":{"id":"https://openalex.org/fields/20","display_name":"Economics, Econometrics and Finance"},"domain":{"id":"https://openalex.org/domains/2","display_name":"Social Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/rare-diseases","display_name":"rare diseases","score":0.8370000123977661},{"id":"https://openalex.org/keywords/orphan-drugs","display_name":"orphan drugs","score":0.9449999928474426},{"id":"https://openalex.org/keywords/drug-reimbursement","display_name":"drug reimbursement","score":0.8629999756813049},{"id":"https://openalex.org/keywords/value-of-evidence","display_name":"value of evidence","score":0.44600000977516174},{"id":"https://openalex.org/keywords/consensus-building","display_name":"consensus building","score":0.6809999942779541},{"id":"https://openalex.org/keywords/stakeholder-engagement","display_name":"stakeholder engagement","score":0.9520000219345093},{"id":"https://openalex.org/keywords/conflict-of-interest","display_name":"conflicts of interest","score":0.8669999837875366},{"id":"https://openalex.org/keywords/reporting-recommendations","display_name":"reporting recommendations","score":0.38600000739097595},{"id":"https://openalex.org/keywords/health-technology-assessment","display_name":"health technology assessment","score":0.5299999713897705}],"concepts":[{"id":"https://openalex.org/C2779703844","wikidata":"https://www.wikidata.org/wiki/Q760125","display_name":"Reimbursement","level":3,"score":0.8776999711990356},{"id":"https://openalex.org/C155911762","wikidata":"https://www.wikidata.org/wiki/Q422321","display_name":"Blueprint","level":2,"score":0.8610000014305115},{"id":"https://openalex.org/C75480439","wikidata":"https://www.wikidata.org/wiki/Q1367466","display_name":"Orphan drug","level":2,"score":0.6958000063896179},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.5206999778747559},{"id":"https://openalex.org/C3020452639","wikidata":"https://www.wikidata.org/wiki/Q454812","display_name":"Clinical judgment","level":2,"score":0.4648999869823456},{"id":"https://openalex.org/C2779473830","wikidata":"https://www.wikidata.org/wiki/Q1540899","display_name":"MEDLINE","level":2,"score":0.4260999858379364},{"id":"https://openalex.org/C26517878","wikidata":"https://www.wikidata.org/wiki/Q228039","display_name":"Key (lock)","level":2,"score":0.38769999146461487},{"id":"https://openalex.org/C19648533","wikidata":"https://www.wikidata.org/wiki/Q691640","display_name":"Evidence-based medicine","level":3,"score":0.37720000743865967},{"id":"https://openalex.org/C512399662","wikidata":"https://www.wikidata.org/wiki/Q3505712","display_name":"Family medicine","level":1,"score":0.3659999966621399},{"id":"https://openalex.org/C166052673","wikidata":"https://www.wikidata.org/wiki/Q83021","display_name":"Empirical evidence","level":2,"score":0.3400000035762787},{"id":"https://openalex.org/C204787440","wikidata":"https://www.wikidata.org/wiki/Q188504","display_name":"Alternative medicine","level":2,"score":0.328000009059906},{"id":"https://openalex.org/C177713679","wikidata":"https://www.wikidata.org/wiki/Q679690","display_name":"Intensive care medicine","level":1,"score":0.3181999921798706},{"id":"https://openalex.org/C58328972","wikidata":"https://www.wikidata.org/wiki/Q184609","display_name":"Expert system","level":2,"score":0.28200000524520874},{"id":"https://openalex.org/C2992293740","wikidata":"https://www.wikidata.org/wiki/Q4781752","display_name":"Drug approval","level":3,"score":0.2766000032424927},{"id":"https://openalex.org/C162118730","wikidata":"https://www.wikidata.org/wiki/Q1128453","display_name":"Actuarial science","level":1,"score":0.27390000224113464},{"id":"https://openalex.org/C2779701055","wikidata":"https://www.wikidata.org/wiki/Q929833","display_name":"Rare disease","level":3,"score":0.272599995136261},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.2632000148296356},{"id":"https://openalex.org/C19527891","wikidata":"https://www.wikidata.org/wiki/Q1120908","display_name":"Medical physics","level":1,"score":0.2619999945163727}],"mesh":[{"descriptor_ui":"D003657","descriptor_name":"Decision Making","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007349","descriptor_name":"Insurance, Health, Reimbursement","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D007600","descriptor_name":"Judgment","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D009965","descriptor_name":"Orphan Drug Production","qualifier_ui":"Q000191","qualifier_name":"economics","is_major_topic":true},{"descriptor_ui":"D012051","descriptor_name":"Reimbursement Mechanisms","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D013673","descriptor_name":"Technology Assessment, Biomedical","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":true},{"descriptor_ui":"D013673","descriptor_name":"Technology Assessment, Biomedical","qualifier_ui":"Q000458","qualifier_name":"organization & administration","is_major_topic":true},{"descriptor_ui":"D035501","descriptor_name":"Uncertainty","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D035583","descriptor_name":"Rare Diseases","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":true}],"locations_count":7,"locations":[{"id":"doi:10.1017/s0266462325100457","doi":"https://doi.org/10.1017/s0266462325100457","is_oa":true,"landing_page_url":"https://doi.org/10.1017/s0266462325100457","pdf_url":"https://www.cambridge.org/core/services/aop-cambridge-core/content/view/F5C99074BED98B74B0EA854AF094B060/S0266462325100457a.pdf/div-class-title-recommended-methods-for-the-collection-of-clinical-expert-judgment-in-rare-diseases-generating-evidence-to-support-reimbursement-of-orphan-drugs-div.pdf","source":{"id":"https://openalex.org/S83891556","display_name":"International Journal of Technology Assessment in Health Care","issn_l":"0266-4623","issn":["0266-4623","1471-6348"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["anvur-scientific","cwts-core","doaj","dongbi-c","doyens","fnege-4","jufo-1","ki-jl-1","medline","norway-1","poland-70","russia-white-list-2","vabb-shw"],"host_organization":"https://openalex.org/P4310311721","host_organization_name":"Cambridge University Press","host_organization_lineage":["https://openalex.org/P4310311721","https://openalex.org/P4310311702"],"host_organization_lineage_names":["Cambridge University Press","University of Cambridge"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"International Journal of Technology Assessment in Health Care","raw_type":"journal-article"},{"id":"pmh:oai:europepmc.org:11402072","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/12592965","pdf_url":null,"source":{"id":"https://openalex.org/S4306400806","display_name":"Europe PMC (PubMed Central)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1303153112","host_organization_name":"European Bioinformatics Institute","host_organization_lineage":["https://openalex.org/I1303153112"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":null,"raw_type":"Text"},{"id":"pmid:41054189","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/41054189","pdf_url":null,"source":{"id":"https://openalex.org/S4306525036","display_name":"PubMed","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"International journal of technology assessment in health care","raw_type":"Journal Article"},{"id":"pmh:oai:eprints.whiterose.ac.uk:246193","doi":null,"is_oa":true,"landing_page_url":"https://eprints.whiterose.ac.uk/id/eprint/246193/1/THC_2510045_1.pdf","pdf_url":"https://eprints.whiterose.ac.uk/id/eprint/246193/1/THC_2510045_1.pdf","source":{"id":"https://openalex.org/S4306400854","display_name":"White Rose Research Online (University of Leeds, The University of Sheffield, University of York)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I2800616092","host_organization_name":"White Rose University Consortium","host_organization_lineage":["https://openalex.org/I2800616092"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"acceptedVersion","is_accepted":true,"is_published":false,"raw_source_name":null,"raw_type":"Article"},{"id":"pmh:oai:eprints.ncl.ac.uk:307349","doi":null,"is_oa":true,"landing_page_url":"https://eprints.ncl.ac.uk/fulltext.aspx?url=307349/55705D4C-E21A-4AC9-9FB1-D24BFEDB984D.pdf&pub_id=307349","pdf_url":"https://eprints.ncl.ac.uk/fulltext.aspx?url=307349/55705D4C-E21A-4AC9-9FB1-D24BFEDB984D.pdf&pub_id=307349","source":{"id":"https://openalex.org/S4306402485","display_name":"Newcastle University ePrints (Newcastle Univesity)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I84884186","host_organization_name":"Newcastle University","host_organization_lineage":["https://openalex.org/I84884186"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"International Journal of Technology Assessment in Health Care,","raw_type":"Article"},{"id":"doi:10.25405/ncl.34532298","doi":"https://doi.org/10.25405/ncl.34532298","is_oa":true,"landing_page_url":"https://doi.org/10.25405/ncl.34532298","pdf_url":null,"source":{"id":"https://openalex.org/S4306402485","display_name":"Newcastle University ePrints (Newcastle Univesity)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I84884186","host_organization_name":"Newcastle University","host_organization_lineage":["https://openalex.org/I84884186"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":null,"is_accepted":false,"is_published":false,"raw_source_name":null,"raw_type":"JournalArticle"},{"id":"doi:10.25405/ncl.34532298.v1","doi":"https://doi.org/10.25405/ncl.34532298.v1","is_oa":true,"landing_page_url":"https://doi.org/10.25405/ncl.34532298.v1","pdf_url":null,"source":{"id":"https://openalex.org/S4306402485","display_name":"Newcastle University ePrints (Newcastle Univesity)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I84884186","host_organization_name":"Newcastle University","host_organization_lineage":["https://openalex.org/I84884186"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":null,"is_accepted":false,"is_published":false,"raw_source_name":null,"raw_type":"JournalArticle"}],"best_oa_location":{"id":"doi:10.1017/s0266462325100457","doi":"https://doi.org/10.1017/s0266462325100457","is_oa":true,"landing_page_url":"https://doi.org/10.1017/s0266462325100457","pdf_url":"https://www.cambridge.org/core/services/aop-cambridge-core/content/view/F5C99074BED98B74B0EA854AF094B060/S0266462325100457a.pdf/div-class-title-recommended-methods-for-the-collection-of-clinical-expert-judgment-in-rare-diseases-generating-evidence-to-support-reimbursement-of-orphan-drugs-div.pdf","source":{"id":"https://openalex.org/S83891556","display_name":"International Journal of Technology Assessment in Health Care","issn_l":"0266-4623","issn":["0266-4623","1471-6348"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["anvur-scientific","cwts-core","doaj","dongbi-c","doyens","fnege-4","jufo-1","ki-jl-1","medline","norway-1","poland-70","russia-white-list-2","vabb-shw"],"host_organization":"https://openalex.org/P4310311721","host_organization_name":"Cambridge University Press","host_organization_lineage":["https://openalex.org/P4310311721","https://openalex.org/P4310311702"],"host_organization_lineage_names":["Cambridge University Press","University of Cambridge"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"International Journal of Technology Assessment in Health Care","raw_type":"journal-article"},"sustainable_development_goals":[],"study_designs":[],"awards":[],"funders":[],"has_content":{"grobid_xml":true,"pdf":true},"content_urls":{"pdf":"https://content.openalex.org/works/W4414879142.pdf","grobid_xml":"https://content.openalex.org/works/W4414879142.grobid-xml"},"referenced_works_count":0,"referenced_works":[],"related_works":["https://openalex.org/W3177431421","https://openalex.org/W4416010559","https://openalex.org/W4415363366","https://openalex.org/W3028815474","https://openalex.org/W2942641342","https://openalex.org/W7095211706","https://openalex.org/W2539499788","https://openalex.org/W2161078130","https://openalex.org/W4229485020","https://openalex.org/W4414066571"],"abstract_inverted_index":{"BACKGROUND:":[0],"Developing":[1],"therapies":[2,224],"for":[3,57,99,107,219],"rare":[4,49,67,83,108,176,222],"diseases":[5,84],"is":[6,251],"challenging":[7],"due":[8],"to":[9,54,92,103,148,194,225,239],"limited":[10],"evidence":[11,135,238,250],"and":[12,30,60,76,97,117,153,159,167,199,211],"high":[13,180],"degrees":[14],"of":[15,20,33,62,73,169,175,208,221,243],"uncertainty":[16,34,244],"regarding":[17],"the":[18,45,58,143,173],"value":[19],"new":[21],"treatments.":[22],"Clinical":[23],"expert":[24,64,127,229],"judgment":[25,65,128,139,230],"can":[26,129,234],"inform":[27,240],"modeling":[28],"assumptions":[29],"address":[31,44],"areas":[32,242],"in":[35,48,66,82,86,96,131,245],"reimbursement":[36,105,246],"submissions.":[37],"As":[38],"current":[39],"protocols":[40],"do":[41],"not":[42],"adequately":[43],"challenges":[46,95],"faced":[47],"diseases,":[50],"this":[51],"research":[52],"aimed":[53,91],"generate":[55],"recommendations":[56,215],"collection":[59],"reporting":[61],"clinical":[63,101,197,228],"diseases.":[68],"METHODS:":[69],"An":[70],"international":[71],"group":[72,118],"industry,":[74],"payer,":[75],"patient":[77],"experts":[78],"with":[79,133,145],"a":[80,87,179,185,217],"background":[81],"participated":[85],"roundtable":[88],"meeting,":[89],"which":[90],"identify":[93,124],"practical":[94],"solutions":[98],"gathering":[100],"insights":[102],"aid":[104],"decisions":[106],"disease":[109,177,223],"therapies.":[110],"Recommendations":[111],"were":[112],"cocreated":[113],"through":[114],"iterative":[115],"discussions":[116],"agreement.":[119],"RESULTS:":[120],"Developers":[121,188],"should":[122,163,189],"proactively":[123],"uncertainties":[125],"that":[126],"address,":[130],"parallel":[132],"early":[134],"generation":[136],"planning.":[137],"Expert":[138],"method(s)":[140],"depend":[141],"on":[142,184,202],"uncertainties,":[144],"those":[146],"key":[147,241],"decision-making":[149],"requiring":[150],"more":[151,236],"robust":[152,237],"time-intensive":[154],"methods.":[155],"For":[156],"highly":[157],"complex":[158],"uncertain":[160],"topics,":[161],"methods":[162],"facilitate":[164],"consensus":[165],"building":[166],"expression":[168],"diverse":[170,191],"views.":[171],"Given":[172],"scarcity":[174],"experts,":[178],"time":[181],"burden":[182],"falls":[183],"few":[186],"experts.":[187],"engage":[190],"stakeholder":[192],"groups":[193],"integrate":[195],"broader":[196],"perspectives":[198],"reduce":[200],"reliance":[201],"specific":[203],"individuals":[204],"while":[205],"approaching":[206],"conflicts":[207],"interest":[209],"pragmatically":[210],"transparently.":[212],"CONCLUSIONS:":[213],"These":[214],"create":[216],"blueprint":[218],"developers":[220,233],"conduct":[226],"high-quality":[227],"studies.":[231],"Hence,":[232],"present":[235],"decisions,":[247],"where":[248],"empirical":[249],"unavailable.":[252]},"counts_by_year":[{"year":2026,"cited_by_count":1}],"updated_date":"2026-11-04T10:54:05.112534","created_date":"2025-10-10T00:00:00"}
