{"id":"https://openalex.org/W2125543555","doi":"https://doi.org/10.1016/s2213-2600(13)70085-7","title":"Rare lung disease and orphan drug development","display_name":"Rare lung disease and orphan drug development","publication_year":2013,"publication_date":"2013-06-25","ids":{"openalex":"https://openalex.org/W2125543555","doi":"https://doi.org/10.1016/s2213-2600(13)70085-7","mag":"2125543555","pmid":"https://pubmed.ncbi.nlm.nih.gov/24429246"},"language":"en","primary_location":{"id":"doi:10.1016/s2213-2600(13)70085-7","doi":"https://doi.org/10.1016/s2213-2600(13)70085-7","is_oa":false,"landing_page_url":"https://doi.org/10.1016/s2213-2600(13)70085-7","pdf_url":null,"source":{"id":"https://openalex.org/S4210192742","display_name":"The Lancet Respiratory Medicine","issn_l":"2213-2600","issn":["2213-2600","2213-2619"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["cwts-core","dongbi-a","doyens","jufo-3","ki-jl-3","medline","norway-2","poland-200","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"The Lancet Respiratory Medicine","raw_type":"journal-article"},"volume":null,"type":"review","is_traditional":true,"indexed_in":["crossref","pubmed"],"open_access":{"is_oa":true,"oa_status":"green","oa_url":"http://hdl.handle.net/11380/982575","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5043303194","display_name":"Paolo Spagnolo","orcid":"https://orcid.org/0000-0002-1096-0596","observed_orcids":["https://orcid.org/0000-0002-1096-0596"]},"institutions":[{"id":"https://openalex.org/I4210109058","display_name":"Azienda Ospedaliero-Universitaria di Modena","ror":"https://ror.org/01hmmsr16","country_code":"IT","type":"healthcare","lineage":["https://openalex.org/I4210109058"]}],"countries":["IT"],"is_corresponding":true,"raw_author_name":"Paolo Spagnolo","raw_affiliation_strings":["Centre for Rare Lung Diseases, Department of Medical and Surgical Sciences for Children and Adults, University Hospital of Modena, Modena, Italy. Electronic address: paolo.spagnolo@unimore.it","Centre for Rare Lung Diseases, Department of Medical and Surgical Sciences for Children and Adults, University Hospital of Modena, Modena, Italy"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Centre for Rare Lung Diseases, Department of Medical and Surgical Sciences for Children and Adults, University Hospital of Modena, Modena, Italy. Electronic address: paolo.spagnolo@unimore.it","institution_ids":["https://openalex.org/I4210109058"]},{"raw_affiliation_string":"Centre for Rare Lung Diseases, Department of Medical and Surgical Sciences for Children and Adults, University Hospital of Modena, Modena, Italy","institution_ids":["https://openalex.org/I4210109058"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5114002768","display_name":"Roland M. du Bois","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I47508984","display_name":"Imperial College London","ror":"https://ror.org/041kmwe10","country_code":"GB","type":"education","lineage":["https://openalex.org/I47508984"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Roland M du Bois","raw_affiliation_strings":["Imperial College, London, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Imperial College, London, UK","institution_ids":["https://openalex.org/I47508984"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5014703126","display_name":"Vincent Cottin","orcid":"https://orcid.org/0000-0002-5591-0955","observed_orcids":["https://orcid.org/0000-0002-5591-0955"]},"institutions":[{"id":"https://openalex.org/I4210100596","display_name":"Hospices Civils de Lyon","ror":"https://ror.org/01502ca60","country_code":"FR","type":"healthcare","lineage":["https://openalex.org/I4210100596"]},{"id":"https://openalex.org/I4210136143","display_name":"H\u00f4pital Louis Pradel","ror":"https://ror.org/0396v4y86","country_code":"FR","type":"healthcare","lineage":["https://openalex.org/I4210100596","https://openalex.org/I4210136143"]}],"countries":["FR"],"is_corresponding":false,"raw_author_name":"Vincent Cottin","raw_affiliation_strings":["National Reference Centre for Rare Pulmonary Diseases, Department of Respiratory Medicine, Louis Pradel Hospital, Hospices Civils de Lyon, University Claude Bernard Lyon 1, Lyon, France"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"National Reference Centre for Rare Pulmonary Diseases, Department of Respiratory Medicine, Louis Pradel Hospital, Hospices Civils de Lyon, University Claude Bernard Lyon 1, Lyon, France","institution_ids":["https://openalex.org/I4210100596","https://openalex.org/I4210136143"]}]}],"institutions":[],"countries_distinct_count":3,"institutions_distinct_count":4,"corresponding_author_ids":["https://openalex.org/A5043303194"],"corresponding_institution_ids":["https://openalex.org/I4210109058"],"apc_list":{"value":7280,"currency":"USD","value_usd":7280},"apc_paid":null,"fwci":2.4082,"has_fulltext":false,"cited_by_count":50,"citation_normalized_percentile":{"value":0.89330025,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":87,"max":99},"biblio":{"volume":"1","issue":"6","first_page":"479","last_page":"487"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T11792","display_name":"Pharmaceutical Economics and Policy","score":0.5673999786376953,"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/T11792","display_name":"Pharmaceutical Economics and Policy","score":0.5673999786376953,"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/T10665","display_name":"Cystic Fibrosis Research Advances","score":0.054099999368190765,"subfield":{"id":"https://openalex.org/subfields/2740","display_name":"Pulmonary and Respiratory Medicine"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},{"id":"https://openalex.org/T10870","display_name":"Interstitial Lung Diseases and Idiopathic Pulmonary Fibrosis","score":0.04179999977350235,"subfield":{"id":"https://openalex.org/subfields/2740","display_name":"Pulmonary and Respiratory Medicine"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/rare-lung-disease","display_name":"rare lung disease","score":0.984000027179718},{"id":"https://openalex.org/keywords/orphan-drugs","display_name":"orphan drugs","score":0.9200000166893005},{"id":"https://openalex.org/keywords/orphan-drug-development","display_name":"orphan drug development","score":0.8489999771118164},{"id":"https://openalex.org/keywords/rare-diseases","display_name":"rare diseases","score":0.9150000214576721},{"id":"https://openalex.org/keywords/drug-development-incentives","display_name":"drug development incentives","score":0.7630000114440918},{"id":"https://openalex.org/keywords/pharmaceutical-industry-investment","display_name":"pharmaceutical industry investment","score":0.7749999761581421},{"id":"https://openalex.org/keywords/drug-development-challenges","display_name":"drug development challenges","score":0.3190000057220459}],"concepts":[{"id":"https://openalex.org/C75480439","wikidata":"https://www.wikidata.org/wiki/Q1367466","display_name":"Orphan drug","level":2,"score":0.9339770078659058},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.8691682815551758},{"id":"https://openalex.org/C2779701055","wikidata":"https://www.wikidata.org/wiki/Q929833","display_name":"Rare disease","level":3,"score":0.7002884745597839},{"id":"https://openalex.org/C29122968","wikidata":"https://www.wikidata.org/wiki/Q1414816","display_name":"Incentive","level":2,"score":0.6059868931770325},{"id":"https://openalex.org/C177713679","wikidata":"https://www.wikidata.org/wiki/Q679690","display_name":"Intensive care medicine","level":1,"score":0.5934054255485535},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.5705598592758179},{"id":"https://openalex.org/C64903051","wikidata":"https://www.wikidata.org/wiki/Q2198549","display_name":"Drug development","level":3,"score":0.49562013149261475},{"id":"https://openalex.org/C160735492","wikidata":"https://www.wikidata.org/wiki/Q31207","display_name":"Health care","level":2,"score":0.47493866086006165},{"id":"https://openalex.org/C2780035454","wikidata":"https://www.wikidata.org/wiki/Q8386","display_name":"Drug","level":2,"score":0.36245954036712646},{"id":"https://openalex.org/C50522688","wikidata":"https://www.wikidata.org/wiki/Q189833","display_name":"Economic growth","level":1,"score":0.21154338121414185},{"id":"https://openalex.org/C142724271","wikidata":"https://www.wikidata.org/wiki/Q7208","display_name":"Pathology","level":1,"score":0.19907811284065247},{"id":"https://openalex.org/C60644358","wikidata":"https://www.wikidata.org/wiki/Q128570","display_name":"Bioinformatics","level":1,"score":0.19729307293891907},{"id":"https://openalex.org/C118552586","wikidata":"https://www.wikidata.org/wiki/Q7867","display_name":"Psychiatry","level":1,"score":0.1840595006942749},{"id":"https://openalex.org/C175444787","wikidata":"https://www.wikidata.org/wiki/Q39072","display_name":"Microeconomics","level":1,"score":0.0},{"id":"https://openalex.org/C162324750","wikidata":"https://www.wikidata.org/wiki/Q8134","display_name":"Economics","level":0,"score":0.0},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.0}],"mesh":[{"descriptor_ui":"D000328","descriptor_name":"Adult","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002986","descriptor_name":"Clinical Trials as Topic","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D004345","descriptor_name":"Drug Industry","qualifier_ui":"Q000191","qualifier_name":"economics","is_major_topic":false},{"descriptor_ui":"D005260","descriptor_name":"Female","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005544","descriptor_name":"Forecasting","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006291","descriptor_name":"Health Policy","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":"D008171","descriptor_name":"Lung Diseases","qualifier_ui":"Q000175","qualifier_name":"diagnosis","is_major_topic":false},{"descriptor_ui":"D008171","descriptor_name":"Lung Diseases","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":true},{"descriptor_ui":"D008297","descriptor_name":"Male","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D009626","descriptor_name":"Terminology as Topic","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":false},{"descriptor_ui":"D009965","descriptor_name":"Orphan Drug Production","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":true},{"descriptor_ui":"D012657","descriptor_name":"Self-Help Groups","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D017281","descriptor_name":"Cost of Illness","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D019564","descriptor_name":"Unnecessary Procedures","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D035583","descriptor_name":"Rare Diseases","qualifier_ui":"Q000175","qualifier_name":"diagnosis","is_major_topic":false},{"descriptor_ui":"D035583","descriptor_name":"Rare Diseases","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":true},{"descriptor_ui":"D055808","descriptor_name":"Drug Discovery","qualifier_ui":"Q000191","qualifier_name":"economics","is_major_topic":false},{"descriptor_ui":"D055808","descriptor_name":"Drug Discovery","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":false},{"descriptor_ui":"D057210","descriptor_name":"Delayed Diagnosis","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false}],"locations_count":3,"locations":[{"id":"doi:10.1016/s2213-2600(13)70085-7","doi":"https://doi.org/10.1016/s2213-2600(13)70085-7","is_oa":false,"landing_page_url":"https://doi.org/10.1016/s2213-2600(13)70085-7","pdf_url":null,"source":{"id":"https://openalex.org/S4210192742","display_name":"The Lancet Respiratory Medicine","issn_l":"2213-2600","issn":["2213-2600","2213-2619"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["cwts-core","dongbi-a","doyens","jufo-3","ki-jl-3","medline","norway-2","poland-200","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"The Lancet Respiratory Medicine","raw_type":"journal-article"},{"id":"pmid:24429246","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/24429246","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":"The Lancet. Respiratory medicine","raw_type":"Journal Article"},{"id":"pmh:oai:iris.unimore.it:11380/982575","doi":null,"is_oa":true,"landing_page_url":"http://hdl.handle.net/11380/982575","pdf_url":null,"source":{"id":"https://openalex.org/S4306400718","display_name":"IRIS UNIMORE (University of Modena and Reggio Emilia)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I122346577","host_organization_name":"University of Modena and Reggio Emilia","host_organization_lineage":["https://openalex.org/I122346577"],"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":null,"raw_type":"info:eu-repo/semantics/article"}],"best_oa_location":{"id":"pmh:oai:iris.unimore.it:11380/982575","doi":null,"is_oa":true,"landing_page_url":"http://hdl.handle.net/11380/982575","pdf_url":null,"source":{"id":"https://openalex.org/S4306400718","display_name":"IRIS UNIMORE (University of Modena and Reggio Emilia)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I122346577","host_organization_name":"University of Modena and Reggio Emilia","host_organization_lineage":["https://openalex.org/I122346577"],"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":null,"raw_type":"info:eu-repo/semantics/article"},"sustainable_development_goals":[{"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3","score":0.9476000070571899}],"study_designs":[],"awards":[],"funders":[],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":68,"referenced_works":["https://openalex.org/W4693461","https://openalex.org/W47218526","https://openalex.org/W149560841","https://openalex.org/W157365614","https://openalex.org/W1515908253","https://openalex.org/W1916630549","https://openalex.org/W1954728404","https://openalex.org/W1963501849","https://openalex.org/W1966456594","https://openalex.org/W1971102493","https://openalex.org/W1980553424","https://openalex.org/W1981858341","https://openalex.org/W1983965561","https://openalex.org/W1984693219","https://openalex.org/W1988130713","https://openalex.org/W1992237498","https://openalex.org/W1993559550","https://openalex.org/W1994219663","https://openalex.org/W2016305001","https://openalex.org/W2017725542","https://openalex.org/W2019375534","https://openalex.org/W2021176556","https://openalex.org/W2025464337","https://openalex.org/W2031570078","https://openalex.org/W2035505404","https://openalex.org/W2036132972","https://openalex.org/W2044109368","https://openalex.org/W2053927466","https://openalex.org/W2065771939","https://openalex.org/W2067514811","https://openalex.org/W2081169091","https://openalex.org/W2083132229","https://openalex.org/W2092559291","https://openalex.org/W2093369894","https://openalex.org/W2096223780","https://openalex.org/W2096828977","https://openalex.org/W2099927259","https://openalex.org/W2106866809","https://openalex.org/W2110000830","https://openalex.org/W2111388691","https://openalex.org/W2114863766","https://openalex.org/W2116260471","https://openalex.org/W2116801544","https://openalex.org/W2123407823","https://openalex.org/W2125013258","https://openalex.org/W2132265655","https://openalex.org/W2132798299","https://openalex.org/W2137127902","https://openalex.org/W2141262339","https://openalex.org/W2143812614","https://openalex.org/W2144822715","https://openalex.org/W2147028601","https://openalex.org/W2148670040","https://openalex.org/W2150580148","https://openalex.org/W2153328864","https://openalex.org/W2154963490","https://openalex.org/W2162877219","https://openalex.org/W2167383172","https://openalex.org/W2171772467","https://openalex.org/W2312386011","https://openalex.org/W2319983832","https://openalex.org/W2330542938","https://openalex.org/W2334019448","https://openalex.org/W2965872185","https://openalex.org/W3140907331","https://openalex.org/W3146537718","https://openalex.org/W4285719527","https://openalex.org/W6817022203"],"related_works":["https://openalex.org/W2978030796","https://openalex.org/W2409069026","https://openalex.org/W2150774326","https://openalex.org/W2154963490","https://openalex.org/W2343920196","https://openalex.org/W1938685502","https://openalex.org/W2292423027","https://openalex.org/W2525628612","https://openalex.org/W4407026128","https://openalex.org/W4299358833"],"abstract_inverted_index":null,"counts_by_year":[{"year":2026,"cited_by_count":1},{"year":2024,"cited_by_count":1},{"year":2023,"cited_by_count":7},{"year":2022,"cited_by_count":2},{"year":2021,"cited_by_count":1},{"year":2020,"cited_by_count":4},{"year":2019,"cited_by_count":1},{"year":2018,"cited_by_count":1},{"year":2017,"cited_by_count":5},{"year":2016,"cited_by_count":5},{"year":2015,"cited_by_count":11},{"year":2014,"cited_by_count":6},{"year":2013,"cited_by_count":3}],"updated_date":"2026-11-04T10:54:05.112534","created_date":"2016-06-24T00:00:00"}