{"id":"https://openalex.org/W4408246296","doi":"https://doi.org/10.1002/cpt.3627","title":"Characterizing Treatment Effect Heterogeneity Using Real\u2010World Data","display_name":"Characterizing Treatment Effect Heterogeneity Using Real\u2010World Data","publication_year":2025,"publication_date":"2025-03-06","ids":{"openalex":"https://openalex.org/W4408246296","doi":"https://doi.org/10.1002/cpt.3627","pmid":"https://pubmed.ncbi.nlm.nih.gov/40047339","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/11993293"},"language":"en","primary_location":{"id":"doi:10.1002/cpt.3627","doi":"https://doi.org/10.1002/cpt.3627","is_oa":true,"landing_page_url":"https://doi.org/10.1002/cpt.3627","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":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Clinical Pharmacology &amp; Therapeutics","raw_type":"journal-article"},"volume":null,"type":"review","is_traditional":true,"indexed_in":["crossref","pubmed"],"open_access":{"is_oa":true,"oa_status":"hybrid","oa_url":"https://doi.org/10.1002/cpt.3627","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5087952855","display_name":"Haedi E Thelen","orcid":"https://orcid.org/0000-0001-8357-2255","observed_orcids":["https://orcid.org/0000-0001-8357-2255"]},"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":true,"raw_author_name":"Haedi Thelen","raw_affiliation_strings":["Department of Biostatistics, Epidemiology and Informatics Center for Real\u2010World Effectiveness and Safety of Therapeutics Perelman School of Medicine, University of Pennsylvania  Philadelphia Pennsylvania USA"],"raw_orcid":"https://orcid.org/0000-0001-8357-2255","affiliations":[{"raw_affiliation_string":"Department of Biostatistics, Epidemiology and Informatics Center for Real\u2010World Effectiveness and Safety of Therapeutics Perelman School of Medicine, University of Pennsylvania  Philadelphia Pennsylvania USA","institution_ids":["https://openalex.org/I79576946"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5012370449","display_name":"Sean Hennessy","orcid":"https://orcid.org/0000-0003-4726-9413","observed_orcids":["https://orcid.org/0000-0003-4726-9413"]},"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":"Sean Hennessy","raw_affiliation_strings":["Department of Biostatistics, Epidemiology and Informatics Center for Real\u2010World Effectiveness and Safety of Therapeutics Perelman School of Medicine, University of Pennsylvania  Philadelphia Pennsylvania USA"],"raw_orcid":"https://orcid.org/0000-0003-4726-9413","affiliations":[{"raw_affiliation_string":"Department of Biostatistics, Epidemiology and Informatics Center for Real\u2010World Effectiveness and Safety of Therapeutics Perelman School of Medicine, University of Pennsylvania  Philadelphia Pennsylvania USA","institution_ids":["https://openalex.org/I79576946"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":1,"corresponding_author_ids":["https://openalex.org/A5087952855"],"corresponding_institution_ids":["https://openalex.org/I79576946"],"apc_list":{"value":4410,"currency":"USD","value_usd":4410},"apc_paid":{"value":4410,"currency":"USD","value_usd":4410},"fwci":2.3645,"has_fulltext":false,"cited_by_count":20,"citation_normalized_percentile":{"value":0.91364903,"is_in_top_1_percent":false,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":98,"max":99},"biblio":{"volume":"117","issue":"5","first_page":"1209","last_page":"1216"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10845","display_name":"Advanced Causal Inference Techniques","score":0.9496999979019165,"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/T10845","display_name":"Advanced Causal Inference Techniques","score":0.9496999979019165,"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/T11235","display_name":"Statistical Methods in Clinical Trials","score":0.027799999341368675,"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/T14374","display_name":"Statistical Methods in Epidemiology","score":0.0034000000450760126,"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"}}],"keywords":[{"id":"https://openalex.org/keywords/treatment-heterogeneity","display_name":"treatment effect heterogeneity","score":0.9309999942779541},{"id":"https://openalex.org/keywords/real-world-data","display_name":"real-world data","score":0.8920000195503235},{"id":"https://openalex.org/keywords/pharmacoepidemiology","display_name":"pharmacoepidemiology","score":0.9049999713897705},{"id":"https://openalex.org/keywords/subgroup-analysis","display_name":"subgroup analysis","score":0.9950000047683716},{"id":"https://openalex.org/keywords/disease-risk-score","display_name":"disease risk score","score":0.9359999895095825},{"id":"https://openalex.org/keywords/effect-modifiers","display_name":"effect modifiers","score":0.7160000205039978},{"id":"https://openalex.org/keywords/personalized-treatment","display_name":"personalized treatment","score":0.5849999785423279},{"id":"https://openalex.org/keywords/model-misspecification","display_name":"model misspecification","score":0.7149999737739563},{"id":"https://openalex.org/keywords/spurious-association","display_name":"spurious association","score":0.6299999952316284}],"concepts":[{"id":"https://openalex.org/C41008148","wikidata":"https://www.wikidata.org/wiki/Q21198","display_name":"Computer science","level":0,"score":0.6333681344985962},{"id":"https://openalex.org/C97256817","wikidata":"https://www.wikidata.org/wiki/Q1462316","display_name":"Spurious relationship","level":2,"score":0.5896111130714417},{"id":"https://openalex.org/C187960798","wikidata":"https://www.wikidata.org/wiki/Q7631152","display_name":"Subgroup analysis","level":3,"score":0.49188852310180664},{"id":"https://openalex.org/C2522767166","wikidata":"https://www.wikidata.org/wiki/Q2374463","display_name":"Data science","level":1,"score":0.4639180898666382},{"id":"https://openalex.org/C2780233690","wikidata":"https://www.wikidata.org/wiki/Q535347","display_name":"Transparency (behavior)","level":2,"score":0.42028310894966125},{"id":"https://openalex.org/C535046627","wikidata":"https://www.wikidata.org/wiki/Q30612","display_name":"Clinical trial","level":2,"score":0.4199081063270569},{"id":"https://openalex.org/C539667460","wikidata":"https://www.wikidata.org/wiki/Q2414942","display_name":"Management science","level":1,"score":0.4084760844707489},{"id":"https://openalex.org/C112930515","wikidata":"https://www.wikidata.org/wiki/Q4389547","display_name":"Risk analysis (engineering)","level":1,"score":0.38960370421409607},{"id":"https://openalex.org/C119857082","wikidata":"https://www.wikidata.org/wiki/Q2539","display_name":"Machine learning","level":1,"score":0.29632803797721863},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.2837986350059509},{"id":"https://openalex.org/C95190672","wikidata":"https://www.wikidata.org/wiki/Q815382","display_name":"Meta-analysis","level":2,"score":0.20967423915863037},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.0},{"id":"https://openalex.org/C142724271","wikidata":"https://www.wikidata.org/wiki/Q7208","display_name":"Pathology","level":1,"score":0.0},{"id":"https://openalex.org/C38652104","wikidata":"https://www.wikidata.org/wiki/Q3510521","display_name":"Computer security","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}],"mesh":[{"descriptor_ui":"D000096962","descriptor_name":"Treatment Effect Heterogeneity","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":"D012107","descriptor_name":"Research Design","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D016032","descriptor_name":"Randomized Controlled Trials as Topic","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D016896","descriptor_name":"Treatment Outcome","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D017891","descriptor_name":"Pharmacoepidemiology","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":true}],"locations_count":3,"locations":[{"id":"doi:10.1002/cpt.3627","doi":"https://doi.org/10.1002/cpt.3627","is_oa":true,"landing_page_url":"https://doi.org/10.1002/cpt.3627","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":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Clinical Pharmacology &amp; Therapeutics","raw_type":"journal-article"},{"id":"pmid:40047339","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/40047339","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:pubmedcentral.nih.gov:11993293","doi":null,"is_oa":true,"landing_page_url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC11993293/","pdf_url":null,"source":{"id":"https://openalex.org/S2764455111","display_name":"PubMed Central","issn_l":null,"issn":null,"is_oa":true,"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":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"acceptedVersion","is_accepted":true,"is_published":false,"raw_source_name":"Clin Pharmacol Ther","raw_type":"Text"}],"best_oa_location":{"id":"doi:10.1002/cpt.3627","doi":"https://doi.org/10.1002/cpt.3627","is_oa":true,"landing_page_url":"https://doi.org/10.1002/cpt.3627","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":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Clinical Pharmacology &amp; Therapeutics","raw_type":"journal-article"},"sustainable_development_goals":[{"score":0.8371999859809875,"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3"}],"study_designs":[],"awards":[{"id":"https://openalex.org/G4089655671","display_name":"Acute Kidney Injury with Immune Checkpoint Inhibitors and Beta-Lactam Antibiotics","funder_award_id":"F32DK141217","funder_id":"https://openalex.org/F4320337357","funder_display_name":"National Institute of Diabetes and Digestive and Kidney Diseases"},{"id":"https://openalex.org/G9000885189","display_name":"Clinical Pharmacoepidemiology Training Grant","funder_award_id":"T32GM075766","funder_id":"https://openalex.org/F4320337354","funder_display_name":"National Institute of General Medical Sciences"}],"funders":[{"id":"https://openalex.org/F4320337354","display_name":"National Institute of General Medical Sciences","ror":"https://ror.org/04q48ey07"},{"id":"https://openalex.org/F4320337357","display_name":"National Institute of Diabetes and Digestive and Kidney Diseases","ror":"https://ror.org/00adh9b73"}],"has_content":{"pdf":false,"grobid_xml":false},"content_urls":null,"referenced_works_count":59,"referenced_works":["https://openalex.org/W1573555636","https://openalex.org/W1604649242","https://openalex.org/W1971918017","https://openalex.org/W1999130958","https://openalex.org/W2004722883","https://openalex.org/W2006290997","https://openalex.org/W2028659545","https://openalex.org/W2035655846","https://openalex.org/W2098931866","https://openalex.org/W2105963543","https://openalex.org/W2108233388","https://openalex.org/W2109587213","https://openalex.org/W2114647004","https://openalex.org/W2115709314","https://openalex.org/W2126436234","https://openalex.org/W2128780239","https://openalex.org/W2142541082","https://openalex.org/W2162586165","https://openalex.org/W2297716127","https://openalex.org/W2473310816","https://openalex.org/W2515858651","https://openalex.org/W2554140069","https://openalex.org/W2591897172","https://openalex.org/W2593111166","https://openalex.org/W2624816748","https://openalex.org/W2756079951","https://openalex.org/W2767884503","https://openalex.org/W2793811202","https://openalex.org/W2809949684","https://openalex.org/W2810227481","https://openalex.org/W2898559532","https://openalex.org/W2900742318","https://openalex.org/W2904040183","https://openalex.org/W2952356497","https://openalex.org/W2963941908","https://openalex.org/W2970529599","https://openalex.org/W2984947744","https://openalex.org/W2986844826","https://openalex.org/W3012167797","https://openalex.org/W3012413426","https://openalex.org/W3047950129","https://openalex.org/W3093678378","https://openalex.org/W3119523053","https://openalex.org/W3181187190","https://openalex.org/W3196122178","https://openalex.org/W3197648598","https://openalex.org/W4232249876","https://openalex.org/W4322496502","https://openalex.org/W4327908860","https://openalex.org/W4328048934","https://openalex.org/W4328049154","https://openalex.org/W4361283651","https://openalex.org/W4388555386","https://openalex.org/W4388656403","https://openalex.org/W4392243479","https://openalex.org/W4392777592","https://openalex.org/W4392962309","https://openalex.org/W4392962322","https://openalex.org/W4401037694"],"related_works":["https://openalex.org/W4327908860","https://openalex.org/W7162250425","https://openalex.org/W2326698866","https://openalex.org/W4401037694","https://openalex.org/W4416755919","https://openalex.org/W1975301241","https://openalex.org/W7127266129","https://openalex.org/W7098387907","https://openalex.org/W4401613484","https://openalex.org/W3011522221"],"abstract_inverted_index":{"Characterizing":[0],"heterogeneity":[1],"of":[2,10,97,117,129,241],"treatment":[3,144,228],"effects":[4],"(HTE)":[5],"is":[6,171,224],"a":[7,126],"fundamental":[8],"goal":[9],"pharmacoepidemiology,":[11],"addressing":[12],"why":[13],"medications":[14],"work":[15],"differently":[16],"across":[17],"patient":[18,40,123,232],"populations.":[19],"This":[20],"paper":[21,48],"reviews":[22],"state-of-the-art":[23],"methods":[24,114,140,165,189],"for":[25,103,148,183,214,226],"studying":[26,63],"HTE":[27,51,150,203,216,223],"using":[28,245],"real-world":[29],"data":[30],"(RWD),":[31],"which":[32,93],"offer":[33,78],"larger":[34],"study":[35,248],"sizes":[36],"and":[37,52,72,81,158,179,195,239],"more":[38],"diverse":[39],"populations":[41],"compared":[42],"to":[43,62,155,176,193,230,247],"randomized":[44],"clinical":[45,104],"trials.":[46],"The":[47,164],"first":[49],"defines":[50],"discusses":[53],"its":[54],"measurement.":[55],"It":[56],"then":[57],"examines":[58],"three":[59],"leading":[60],"approaches":[61,210],"HTE:":[64],"subgroup":[65,94],"analysis,":[66],"disease":[67],"risk":[68,131],"score":[69,128],"(DRS)":[70],"methods,":[71],"effect":[73,109],"modeling":[74,139,209],"methods.":[75],"Subgroup":[76,169],"analyses":[77],"simplicity,":[79],"transparency,":[80],"provide":[82,161,205],"insights":[83,135],"into":[84,125,136],"drug":[85],"mechanisms.":[86,137],"However,":[87],"they":[88],"face":[89],"difficulties":[90],"in":[91],"resolving":[92],"or":[95,204],"combination":[96],"characteristics":[98,124,185],"should":[99],"be":[100],"the":[101,237],"basis":[102],"decision":[105],"making":[106],"when":[107,244],"multiple":[108,122,184],"modifiers":[110],"are":[111,153,190,218],"present.":[112],"DRS":[113,188],"address":[115],"some":[116],"these":[118],"limitations":[119,240],"by":[120],"incorporating":[121],"summary":[127],"outcome":[130],"but":[132,152,173,198,217],"may":[133,159,199],"obscure":[134],"Effect":[138,208],"directly":[141],"predict":[142],"individual":[143],"effects,":[145],"offering":[146],"potential":[147,213],"precise":[149],"characterization,":[151],"prone":[154],"model":[156],"misspecification":[157],"not":[160,181,200],"mechanistic":[162,206],"insights.":[163],"each":[166,242],"have":[167,211],"tradeoffs.":[168],"analysis":[170],"straightforward":[172],"can":[174],"lead":[175],"spurious":[177],"associations":[178],"does":[180],"account":[182],"at":[186],"once.":[187],"relatively":[191],"simple":[192],"implement":[194],"clinically":[196],"useful,":[197],"completely":[201],"describe":[202],"insight.":[207],"great":[212],"characterizing":[215],"still":[219],"being":[220],"developed.":[221],"Understanding":[222],"essential":[225],"personalizing":[227],"strategies":[229],"improve":[231],"outcomes.":[233],"Researchers":[234],"must":[235],"weigh":[236],"strengths":[238],"approach":[243],"RWD":[246],"HTE.":[249]},"counts_by_year":[{"year":2026,"cited_by_count":9},{"year":2025,"cited_by_count":11}],"updated_date":"2026-11-11T12:16:26.678958","created_date":"2025-03-08T00:00:00"}
