{"id":"https://openalex.org/W4407586941","doi":"https://doi.org/10.1002/cpt.3576","title":"Use of Real\u2010World Data and Real\u2010World Evidence in Rare Disease Drug Development: A Statistical Perspective","display_name":"Use of Real\u2010World Data and Real\u2010World Evidence in Rare Disease Drug Development: A Statistical Perspective","publication_year":2025,"publication_date":"2025-02-14","ids":{"openalex":"https://openalex.org/W4407586941","doi":"https://doi.org/10.1002/cpt.3576","pmid":"https://pubmed.ncbi.nlm.nih.gov/39949314"},"language":"en","primary_location":{"id":"doi:10.1002/cpt.3576","doi":"https://doi.org/10.1002/cpt.3576","is_oa":false,"landing_page_url":"https://doi.org/10.1002/cpt.3576","pdf_url":null,"source":{"id":"https://openalex.org/S159852663","display_name":"Clinical Pharmacology & Therapeutics","issn_l":"0009-9236","issn":["0009-9236","1085-8733","1532-6535"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-a","doyens","jufo-3","ki-jl-2","medline","norway-2","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Clinical Pharmacology & Therapeutics","raw_type":"journal-article"},"volume":null,"type":"review","indexed_in":["arxiv","crossref","datacite","pubmed"],"open_access":{"is_oa":true,"oa_status":"green","oa_url":"https://arxiv.org/pdf/2410.06586","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5100332967","display_name":"Jie Chen","orcid":"https://orcid.org/0000-0002-8219-2370","observed_orcids":["https://orcid.org/0000-0002-8219-2370"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Jie Chen","raw_affiliation_strings":["ECR Global  Shanghai China"],"raw_orcid":"https://orcid.org/0000-0002-8219-2370","affiliations":[{"raw_affiliation_string":"ECR Global  Shanghai China","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5048956065","display_name":"Susan Gruber","orcid":"https://orcid.org/0000-0003-0127-0099","observed_orcids":["https://orcid.org/0000-0003-0127-0099"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Susan Gruber","raw_affiliation_strings":["TL Revolution  Cambridge Massachusetts USA"],"raw_orcid":"https://orcid.org/0000-0003-0127-0099","affiliations":[{"raw_affiliation_string":"TL Revolution  Cambridge Massachusetts USA","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5100559821","display_name":"Hana Lee","orcid":"https://orcid.org/0000-0002-8684-1040","observed_orcids":["https://orcid.org/0000-0002-8684-1040"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Hana Lee","raw_affiliation_strings":["TL Revolution  Cambridge Massachusetts USA"],"raw_orcid":"https://orcid.org/0000-0002-8684-1040","affiliations":[{"raw_affiliation_string":"TL Revolution  Cambridge Massachusetts USA","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5004584518","display_name":"Haitao Chu","orcid":"https://orcid.org/0000-0003-0932-598X","observed_orcids":["https://orcid.org/0000-0003-0932-598X"]},"institutions":[{"id":"https://openalex.org/I180857899","display_name":"Pfizer (United States)","ror":"https://ror.org/01xdqrp08","country_code":"US","type":"company","lineage":["https://openalex.org/I180857899"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Haitao Chu","raw_affiliation_strings":["Pfizer Inc  Groton Connecticut USA"],"raw_orcid":"https://orcid.org/0000-0003-0932-598X","affiliations":[{"raw_affiliation_string":"Pfizer Inc  Groton Connecticut USA","institution_ids":["https://openalex.org/I180857899"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5035424351","display_name":"Shiowjen Lee","orcid":"https://orcid.org/0000-0002-7924-5291","observed_orcids":["https://orcid.org/0000-0002-7924-5291"]},"institutions":[{"id":"https://openalex.org/I1320320070","display_name":"United States Food and Drug Administration","ror":"https://ror.org/034xvzb47","country_code":"US","type":"government","lineage":["https://openalex.org/I1299022934","https://openalex.org/I1320320070"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Shiowjen Lee","raw_affiliation_strings":["US Food and Drug Administration  Silver Spring Maryland USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"US Food and Drug Administration  Silver Spring Maryland USA","institution_ids":["https://openalex.org/I1320320070"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5100829713","display_name":"Haijun Tian","orcid":"https://orcid.org/0000-0003-1875-3169","observed_orcids":["https://orcid.org/0000-0003-1875-3169"]},"institutions":[{"id":"https://openalex.org/I168537998","display_name":"Eli Lilly (United States)","ror":"https://ror.org/01qat3289","country_code":"US","type":"company","lineage":["https://openalex.org/I168537998"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Haijun Tian","raw_affiliation_strings":["Eli Lilly & Co.  Indianapolis Indiana USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Eli Lilly & Co.  Indianapolis Indiana USA","institution_ids":["https://openalex.org/I168537998"]}]},{"author_position":"middle","author":{"id":null,"display_name":"Yan Wang","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1320320070","display_name":"United States Food and Drug Administration","ror":"https://ror.org/034xvzb47","country_code":"US","type":"government","lineage":["https://openalex.org/I1299022934","https://openalex.org/I1320320070"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Yan Wang","raw_affiliation_strings":["US Food and Drug Administration  Silver Spring Maryland USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"US Food and Drug Administration  Silver Spring Maryland USA","institution_ids":["https://openalex.org/I1320320070"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5110089658","display_name":"Weili He","orcid":"https://orcid.org/0009-0000-0989-7318","observed_orcids":["https://orcid.org/0009-0000-0989-7318"]},"institutions":[{"id":"https://openalex.org/I2948539688","display_name":"AbbVie (United States)","ror":"https://ror.org/02g5p4n58","country_code":"US","type":"company","lineage":["https://openalex.org/I2948539688"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Weili He","raw_affiliation_strings":["AbbVie  North Chicago Illinois USA"],"raw_orcid":"https://orcid.org/0009-0000-0989-7318","affiliations":[{"raw_affiliation_string":"AbbVie  North Chicago Illinois USA","institution_ids":["https://openalex.org/I2948539688"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5010460004","display_name":"Thomas Jemielita","orcid":"https://orcid.org/0000-0001-7788-1828","observed_orcids":["https://orcid.org/0000-0001-7788-1828"]},"institutions":[{"id":"https://openalex.org/I1285764155","display_name":"Merck & Co., Inc., Rahway, NJ, USA (United States)","ror":"https://ror.org/02891sr49","country_code":"US","type":"company","lineage":["https://openalex.org/I1285764155"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Thomas Jemielita","raw_affiliation_strings":["Merck & Co., Inc.  Rahway New Jersey USA"],"raw_orcid":"https://orcid.org/0000-0001-7788-1828","affiliations":[{"raw_affiliation_string":"Merck & Co., Inc.  Rahway New Jersey USA","institution_ids":["https://openalex.org/I1285764155"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5024734361","display_name":"Yang Song","orcid":"https://orcid.org/0000-0001-5040-2604","observed_orcids":["https://orcid.org/0000-0001-5040-2604"]},"institutions":[{"id":"https://openalex.org/I61970326","display_name":"Vertex Pharmaceuticals (United States)","ror":"https://ror.org/00anb1726","country_code":"US","type":"company","lineage":["https://openalex.org/I61970326"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Yang Song","raw_affiliation_strings":["Vertex Pharmaceuticals  Boston Massachusetts USA"],"raw_orcid":"https://orcid.org/0000-0001-5040-2604","affiliations":[{"raw_affiliation_string":"Vertex Pharmaceuticals  Boston Massachusetts USA","institution_ids":["https://openalex.org/I61970326"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5109148006","display_name":"Roy Tamura","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I2613432","display_name":"University of South Florida","ror":"https://ror.org/032db5x82","country_code":"US","type":"education","lineage":["https://openalex.org/I2613432"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Roy Tamura","raw_affiliation_strings":["University of South Florida  Tampa Florida USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"University of South Florida  Tampa Florida USA","institution_ids":["https://openalex.org/I2613432"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5016639693","display_name":"L\u00fc Tian","orcid":"https://orcid.org/0000-0002-5893-0169","observed_orcids":["https://orcid.org/0000-0002-5893-0169"]},"institutions":[{"id":"https://openalex.org/I97018004","display_name":"Stanford University","ror":"https://ror.org/00f54p054","country_code":"US","type":"education","lineage":["https://openalex.org/I97018004"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Lu Tian","raw_affiliation_strings":["Stanford University  Stanford California USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Stanford University  Stanford California USA","institution_ids":["https://openalex.org/I97018004"]}]},{"author_position":"middle","author":{"id":null,"display_name":"Yihua Zhao","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210153546","display_name":"Flatiron Health (United States)","ror":"https://ror.org/0508h6p74","country_code":"US","type":"company","lineage":["https://openalex.org/I4210153546"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Yihua Zhao","raw_affiliation_strings":["Flatiron Health  San Francisco California USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Flatiron Health  San Francisco California USA","institution_ids":["https://openalex.org/I4210153546"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5100454462","display_name":"Yong Chen","orcid":"https://orcid.org/0000-0003-3568-6850","observed_orcids":["https://orcid.org/0000-0003-3568-6850"]},"institutions":[{"id":"https://openalex.org/I79576946","display_name":"University of Pennsylvania","ror":"https://ror.org/00b30xv10","country_code":"US","type":"education","lineage":["https://openalex.org/I79576946"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Yong Chen","raw_affiliation_strings":["University of Pennsylvania  Philadelphia Pennsylvania USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"University of Pennsylvania  Philadelphia Pennsylvania USA","institution_ids":["https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5110130449","display_name":"Mark J. van der Laan","orcid":"https://orcid.org/0000-0003-1432-5511","observed_orcids":["https://orcid.org/0000-0003-1432-5511"]},"institutions":[{"id":"https://openalex.org/I95457486","display_name":"University of California, Berkeley","ror":"https://ror.org/01an7q238","country_code":"US","type":"education","lineage":["https://openalex.org/I95457486"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Mark van der Laan","raw_affiliation_strings":["University of California at Berkeley  Berkeley California USA"],"raw_orcid":"https://orcid.org/0000-0003-1432-5511","affiliations":[{"raw_affiliation_string":"University of California at Berkeley  Berkeley California USA","institution_ids":["https://openalex.org/I95457486"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5100642162","display_name":"Lei Nie","orcid":"https://orcid.org/0000-0002-7791-9395","observed_orcids":["https://orcid.org/0000-0002-7791-9395"]},"institutions":[{"id":"https://openalex.org/I1320320070","display_name":"United States Food and Drug Administration","ror":"https://ror.org/034xvzb47","country_code":"US","type":"government","lineage":["https://openalex.org/I1299022934","https://openalex.org/I1320320070"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Lei Nie","raw_affiliation_strings":["US Food and Drug Administration  Silver Spring Maryland USA"],"raw_orcid":"https://orcid.org/0000-0002-7791-9395","affiliations":[{"raw_affiliation_string":"US Food and Drug Administration  Silver Spring Maryland USA","institution_ids":["https://openalex.org/I1320320070"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":11,"corresponding_author_ids":["https://openalex.org/A5100642162"],"corresponding_institution_ids":["https://openalex.org/I1320320070"],"apc_list":{"value":4410,"currency":"USD","value_usd":4410},"apc_paid":null,"fwci":2.0341,"has_fulltext":true,"cited_by_count":13,"citation_normalized_percentile":{"value":0.87939698,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":98,"max":99},"biblio":{"volume":"117","issue":"4","first_page":"946","last_page":"960"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T11235","display_name":"Statistical Methods in Clinical Trials","score":0.9692000150680542,"subfield":{"id":"https://openalex.org/subfields/2613","display_name":"Statistics and Probability"},"field":{"id":"https://openalex.org/fields/26","display_name":"Mathematics"},"domain":{"id":"https://openalex.org/domains/3","display_name":"Physical Sciences"}},"topics":[{"id":"https://openalex.org/T11235","display_name":"Statistical Methods in Clinical Trials","score":0.9692000150680542,"subfield":{"id":"https://openalex.org/subfields/2613","display_name":"Statistics and Probability"},"field":{"id":"https://openalex.org/fields/26","display_name":"Mathematics"},"domain":{"id":"https://openalex.org/domains/3","display_name":"Physical Sciences"}},{"id":"https://openalex.org/T12547","display_name":"Pharmaceutical studies and practices","score":0.016300000250339508,"subfield":{"id":"https://openalex.org/subfields/2735","display_name":"Pediatrics, Perinatology and Child Health"},"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/T11792","display_name":"Pharmaceutical Economics and Policy","score":0.007600000128149986,"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/real-world-data","display_name":"real-world data","score":0.9739999771118164},{"id":"https://openalex.org/keywords/real-world-evidence","display_name":"real-world evidence","score":1.0},{"id":"https://openalex.org/keywords/rare-diseases","display_name":"rare diseases","score":0.9269999861717224},{"id":"https://openalex.org/keywords/drug-development","display_name":"drug development","score":0.7960000038146973},{"id":"https://openalex.org/keywords/clinical-trial-design","display_name":"clinical trial design","score":0.7099999785423279},{"id":"https://openalex.org/keywords/natural-history-research","display_name":"natural history research","score":0.9810000061988831},{"id":"https://openalex.org/keywords/regulatory-decision-making","display_name":"regulatory decision-making","score":0.9309999942779541},{"id":"https://openalex.org/keywords/marketing-applications","display_name":"marketing applications","score":0.6169999837875366},{"id":"https://openalex.org/keywords/regulatory-guidance","display_name":"regulatory guidelines","score":0.5170000195503235}],"concepts":[{"id":"https://openalex.org/C535046627","wikidata":"https://www.wikidata.org/wiki/Q30612","display_name":"Clinical trial","level":2,"score":0.6928730010986328},{"id":"https://openalex.org/C64903051","wikidata":"https://www.wikidata.org/wiki/Q2198549","display_name":"Drug development","level":3,"score":0.6396843791007996},{"id":"https://openalex.org/C2781047461","wikidata":"https://www.wikidata.org/wiki/Q679692","display_name":"Biopharmaceutical","level":2,"score":0.6386686563491821},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.5071227550506592},{"id":"https://openalex.org/C2779701055","wikidata":"https://www.wikidata.org/wiki/Q929833","display_name":"Rare disease","level":3,"score":0.4842205047607422},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.4825519919395447},{"id":"https://openalex.org/C19351080","wikidata":"https://www.wikidata.org/wiki/Q1395034","display_name":"New product development","level":2,"score":0.47236353158950806},{"id":"https://openalex.org/C163276114","wikidata":"https://www.wikidata.org/wiki/Q484591","display_name":"Natural history","level":2,"score":0.4134133458137512},{"id":"https://openalex.org/C177713679","wikidata":"https://www.wikidata.org/wiki/Q679690","display_name":"Intensive care medicine","level":1,"score":0.3886728584766388},{"id":"https://openalex.org/C2522767166","wikidata":"https://www.wikidata.org/wiki/Q2374463","display_name":"Data science","level":1,"score":0.37079793214797974},{"id":"https://openalex.org/C2780035454","wikidata":"https://www.wikidata.org/wiki/Q8386","display_name":"Drug","level":2,"score":0.32974088191986084},{"id":"https://openalex.org/C144133560","wikidata":"https://www.wikidata.org/wiki/Q4830453","display_name":"Business","level":0,"score":0.32208502292633057},{"id":"https://openalex.org/C41008148","wikidata":"https://www.wikidata.org/wiki/Q21198","display_name":"Computer science","level":0,"score":0.29926598072052},{"id":"https://openalex.org/C98274493","wikidata":"https://www.wikidata.org/wiki/Q128406","display_name":"Pharmacology","level":1,"score":0.2774553894996643},{"id":"https://openalex.org/C150903083","wikidata":"https://www.wikidata.org/wiki/Q7108","display_name":"Biotechnology","level":1,"score":0.19432613253593445},{"id":"https://openalex.org/C162853370","wikidata":"https://www.wikidata.org/wiki/Q39809","display_name":"Marketing","level":1,"score":0.16078847646713257},{"id":"https://openalex.org/C142724271","wikidata":"https://www.wikidata.org/wiki/Q7208","display_name":"Pathology","level":1,"score":0.15889352560043335},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.10037198662757874},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.0}],"mesh":[{"descriptor_ui":"D000076722","descriptor_name":"Drug Development","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":true},{"descriptor_ui":"D000076722","descriptor_name":"Drug Development","qualifier_ui":"Q000706","qualifier_name":"statistics & numerical data","is_major_topic":true},{"descriptor_ui":"D002986","descriptor_name":"Clinical Trials as Topic","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":false},{"descriptor_ui":"D002986","descriptor_name":"Clinical Trials as Topic","qualifier_ui":"Q000706","qualifier_name":"statistics & numerical data","is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D009965","descriptor_name":"Orphan Drug Production","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":true},{"descriptor_ui":"D009965","descriptor_name":"Orphan Drug Production","qualifier_ui":"Q000706","qualifier_name":"statistics & numerical data","is_major_topic":true},{"descriptor_ui":"D012107","descriptor_name":"Research Design","qualifier_ui":"Q000706","qualifier_name":"statistics & numerical data","is_major_topic":false},{"descriptor_ui":"D035583","descriptor_name":"Rare Diseases","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":true},{"descriptor_ui":"D035583","descriptor_name":"Rare Diseases","qualifier_ui":"Q000453","qualifier_name":"epidemiology","is_major_topic":true}],"locations_count":4,"locations":[{"id":"doi:10.1002/cpt.3576","doi":"https://doi.org/10.1002/cpt.3576","is_oa":false,"landing_page_url":"https://doi.org/10.1002/cpt.3576","pdf_url":null,"source":{"id":"https://openalex.org/S159852663","display_name":"Clinical Pharmacology & Therapeutics","issn_l":"0009-9236","issn":["0009-9236","1085-8733","1532-6535"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-a","doyens","jufo-3","ki-jl-2","medline","norway-2","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Clinical Pharmacology & Therapeutics","raw_type":"journal-article"},{"id":"pmid:39949314","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/39949314","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":"Clinical pharmacology and therapeutics","raw_type":"Journal Article"},{"id":"pmh:oai:arXiv.org:2410.06586","doi":null,"is_oa":true,"landing_page_url":"http://arxiv.org/abs/2410.06586","pdf_url":"https://arxiv.org/pdf/2410.06586","source":{"id":"https://openalex.org/S4306400194","display_name":"arXiv (Cornell University)","issn_l":"2331-8422","issn":["2331-8422"],"is_oa":true,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I205783295","host_organization_name":"Cornell University","host_organization_lineage":["https://openalex.org/I205783295"],"host_organization_lineage_names":[],"type":"repository"},"license":"public-domain","license_id":"https://openalex.org/licenses/public-domain","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":null,"raw_type":"text"},{"id":"doi:10.48550/arxiv.2410.06586","doi":"https://doi.org/10.48550/arxiv.2410.06586","is_oa":true,"landing_page_url":"https://doi.org/10.48550/arxiv.2410.06586","pdf_url":null,"source":{"id":"https://openalex.org/S4306400194","display_name":"arXiv (Cornell University)","issn_l":"2331-8422","issn":["2331-8422"],"is_oa":true,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I205783295","host_organization_name":"Cornell University","host_organization_lineage":["https://openalex.org/I205783295"],"host_organization_lineage_names":[],"type":"repository"},"license":"public-domain","license_id":"https://openalex.org/licenses/public-domain","version":null,"is_accepted":false,"is_published":false,"raw_source_name":null,"raw_type":"Preprint"}],"best_oa_location":{"id":"pmh:oai:arXiv.org:2410.06586","doi":null,"is_oa":true,"landing_page_url":"http://arxiv.org/abs/2410.06586","pdf_url":"https://arxiv.org/pdf/2410.06586","source":{"id":"https://openalex.org/S4306400194","display_name":"arXiv (Cornell University)","issn_l":"2331-8422","issn":["2331-8422"],"is_oa":true,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I205783295","host_organization_name":"Cornell University","host_organization_lineage":["https://openalex.org/I205783295"],"host_organization_lineage_names":[],"type":"repository"},"license":"public-domain","license_id":"https://openalex.org/licenses/public-domain","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":null,"raw_type":"text"},"sustainable_development_goals":[{"score":0.5683000087738037,"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being"}],"study_designs":[],"awards":[],"funders":[],"has_content":{"grobid_xml":true,"pdf":true},"content_urls":{"pdf":"https://content.openalex.org/works/W4407586941.pdf","grobid_xml":"https://content.openalex.org/works/W4407586941.grobid-xml"},"referenced_works_count":89,"referenced_works":["https://openalex.org/W1479881385","https://openalex.org/W1971722198","https://openalex.org/W1973521570","https://openalex.org/W1974616955","https://openalex.org/W1980766464","https://openalex.org/W2007919546","https://openalex.org/W2020946357","https://openalex.org/W2025926373","https://openalex.org/W2026888665","https://openalex.org/W2060020282","https://openalex.org/W2071066672","https://openalex.org/W2082299845","https://openalex.org/W2096998032","https://openalex.org/W2098724822","https://openalex.org/W2100950823","https://openalex.org/W2116739230","https://openalex.org/W2143891888","https://openalex.org/W2144409155","https://openalex.org/W2155169076","https://openalex.org/W2181278642","https://openalex.org/W2291276487","https://openalex.org/W2298338128","https://openalex.org/W2337357996","https://openalex.org/W2411505396","https://openalex.org/W2475026798","https://openalex.org/W2478459783","https://openalex.org/W2735337841","https://openalex.org/W2762307552","https://openalex.org/W2769583258","https://openalex.org/W2886144609","https://openalex.org/W2894412626","https://openalex.org/W2910555254","https://openalex.org/W2950774550","https://openalex.org/W2969147770","https://openalex.org/W2976369321","https://openalex.org/W2981549610","https://openalex.org/W2982183961","https://openalex.org/W3003751458","https://openalex.org/W3005073090","https://openalex.org/W3006306979","https://openalex.org/W3008601291","https://openalex.org/W3009081729","https://openalex.org/W3012403290","https://openalex.org/W3021380060","https://openalex.org/W3084470313","https://openalex.org/W3087108556","https://openalex.org/W3092673148","https://openalex.org/W3110766467","https://openalex.org/W3126374640","https://openalex.org/W3127624760","https://openalex.org/W3128104372","https://openalex.org/W3154284446","https://openalex.org/W3156947925","https://openalex.org/W3174000992","https://openalex.org/W3202096440","https://openalex.org/W3208408748","https://openalex.org/W3215111505","https://openalex.org/W4200580425","https://openalex.org/W4206544979","https://openalex.org/W4214918165","https://openalex.org/W4224120920","https://openalex.org/W4224433810","https://openalex.org/W4239728164","https://openalex.org/W4248240383","https://openalex.org/W4280532499","https://openalex.org/W4292731390","https://openalex.org/W4293739968","https://openalex.org/W4294811771","https://openalex.org/W4297262770","https://openalex.org/W4311204752","https://openalex.org/W4311209537","https://openalex.org/W4323921070","https://openalex.org/W4376485284","https://openalex.org/W4376485408","https://openalex.org/W4382181481","https://openalex.org/W4386593345","https://openalex.org/W4386820537","https://openalex.org/W4386961764","https://openalex.org/W4388096388","https://openalex.org/W4390059511","https://openalex.org/W4391878128","https://openalex.org/W4392922489","https://openalex.org/W4394741204","https://openalex.org/W4396583389","https://openalex.org/W4396895021","https://openalex.org/W4401545832","https://openalex.org/W4402482302","https://openalex.org/W4408403083","https://openalex.org/W7075661866"],"related_works":["https://openalex.org/W4408403083","https://openalex.org/W4311209537","https://openalex.org/W3003751458","https://openalex.org/W4313893320","https://openalex.org/W4404528439","https://openalex.org/W4380150998","https://openalex.org/W4394741204","https://openalex.org/W3214015392","https://openalex.org/W4318542558","https://openalex.org/W4410096285"],"abstract_inverted_index":{"Real-world":[0],"data":[1],"(RWD)":[2],"and":[3,15,26,61,77,83,92,98,102,128,136],"real-world":[4],"evidence":[5],"(RWE)":[6,45],"have":[7],"been":[8],"increasingly":[9],"used":[10],"in":[11,33,63,94,139],"medical":[12],"product":[13],"development":[14,37],"regulatory":[16,81],"decision-making,":[17],"especially":[18],"for":[19,66,113],"rare":[20,34,69,105,114],"diseases.":[21,70],"After":[22],"outlining":[23],"the":[24,31,39,42,50,57,84,88,99,124],"challenges":[25,32],"possible":[27],"strategies":[28],"to":[29,130],"address":[30],"disease":[35,106,115],"drug":[36],"(see":[38],"accompanying":[40],"paper),":[41],"Real-World":[43],"Evidence":[44],"Scientific":[46],"Working":[47],"Group":[48],"of":[49,59,90,104,126],"American":[51],"Statistical":[52],"Association":[53],"Biopharmaceutical":[54],"Section":[55],"reviews":[56],"roles":[58],"RWD":[60,91,127],"RWE":[62,93,129],"clinical":[64,107],"trials":[65,116],"drugs":[67],"treating":[68],"This":[71],"paper":[72],"summarizes":[73],"relevant":[74],"guidance":[75],"documents":[76],"frameworks":[78],"by":[79,120],"selected":[80],"agencies":[82],"current":[85],"practice":[86],"on":[87,123],"use":[89,125],"natural":[95,133],"history":[96,134],"studies":[97,122],"design,":[100],"conduct,":[101],"analysis":[103],"trials.":[108],"A":[109],"targeted":[110],"learning":[111],"roadmap":[112],"is":[117],"described,":[118],"followed":[119],"case":[121],"support":[131],"a":[132],"study":[135],"marketing":[137],"applications":[138],"various":[140],"settings.":[141]},"counts_by_year":[{"year":2026,"cited_by_count":6},{"year":2025,"cited_by_count":7}],"updated_date":"2026-10-31T12:09:11.096373","created_date":"2025-02-15T00:00:00"}