{"id":"https://openalex.org/W3198313617","doi":"https://doi.org/10.1080/19466315.2021.1961854","title":"Comparative Effectiveness Research using Bayesian Adaptive Designs for Rare Diseases: Response Adaptive Randomization Reusing Participants","display_name":"Comparative Effectiveness Research using Bayesian Adaptive Designs for Rare Diseases: Response Adaptive Randomization Reusing Participants","publication_year":2021,"publication_date":"2021-08-31","ids":{"openalex":"https://openalex.org/W3198313617","doi":"https://doi.org/10.1080/19466315.2021.1961854","mag":"3198313617","pmid":"https://pubmed.ncbi.nlm.nih.gov/36875290","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/9979780"},"language":"en","primary_location":{"id":"doi:10.1080/19466315.2021.1961854","doi":"https://doi.org/10.1080/19466315.2021.1961854","is_oa":false,"landing_page_url":"https://doi.org/10.1080/19466315.2021.1961854","pdf_url":null,"source":{"id":"https://openalex.org/S104563881","display_name":"Statistics in Biopharmaceutical Research","issn_l":"1946-6315","issn":["1946-6315"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["cwts-core","jufo-1","ki-jl-1","norway-1","poland-100","russia-white-list-2","vabb-shw"],"host_organization":"https://openalex.org/P4310320547","host_organization_name":"Taylor & Francis","host_organization_lineage":["https://openalex.org/P4310320547","https://openalex.org/P4310320449"],"host_organization_lineage_names":["Taylor & Francis","Informa"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Statistics in Biopharmaceutical Research","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref","pubmed"],"open_access":{"is_oa":true,"oa_status":"green","oa_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/9979780","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5102666462","display_name":"Fengming Tang","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1339091601","display_name":"Saint Luke's Health System","ror":"https://ror.org/0127qs140","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1339091601"]},{"id":"https://openalex.org/I4210128618","display_name":"University of Kansas Medical Center","ror":"https://ror.org/036c9yv20","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210128618"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Fengming Tang","raw_affiliation_strings":["Department of Biostatistics & Data Science, University of Kansas Medical Center, Kansas City, KS","Saint Luke\u2019s Health System, Kansas City, MO"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Biostatistics & Data Science, University of Kansas Medical Center, Kansas City, KS","institution_ids":["https://openalex.org/I4210128618"]},{"raw_affiliation_string":"Saint Luke\u2019s Health System, Kansas City, MO","institution_ids":["https://openalex.org/I1339091601"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5004067964","display_name":"Byron J. Gajewski","orcid":"https://orcid.org/0000-0001-7589-8247","observed_orcids":["https://orcid.org/0000-0001-7589-8247"]},"institutions":[{"id":"https://openalex.org/I4210128618","display_name":"University of Kansas Medical Center","ror":"https://ror.org/036c9yv20","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210128618"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Byron J. Gajewski","raw_affiliation_strings":["Department of Biostatistics & Data Science, University of Kansas Medical Center, Kansas City, KS"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Biostatistics & Data Science, University of Kansas Medical Center, Kansas City, KS","institution_ids":["https://openalex.org/I4210128618"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":2,"corresponding_author_ids":["https://openalex.org/A5102666462"],"corresponding_institution_ids":["https://openalex.org/I1339091601","https://openalex.org/I4210128618"],"apc_list":null,"apc_paid":null,"fwci":0.0,"has_fulltext":false,"cited_by_count":3,"citation_normalized_percentile":{"value":0.16455879,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":89,"max":96},"biblio":{"volume":"15","issue":"1","first_page":"154","last_page":"163"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T11235","display_name":"Statistical Methods in Clinical Trials","score":1.0,"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":1.0,"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/T11798","display_name":"Optimal Experimental Design Methods","score":0.0,"subfield":{"id":"https://openalex.org/subfields/1803","display_name":"Management Science and Operations Research"},"field":{"id":"https://openalex.org/fields/18","display_name":"Decision Sciences"},"domain":{"id":"https://openalex.org/domains/2","display_name":"Social Sciences"}},{"id":"https://openalex.org/T10243","display_name":"Statistical Methods and Bayesian Inference","score":0.0,"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/response-adaptive-randomization","display_name":"response-adaptive randomization","score":0.9340000152587891},{"id":"https://openalex.org/keywords/rare-diseases","display_name":"rare diseases","score":0.8009999990463257},{"id":"https://openalex.org/keywords/comparative-effectiveness-research","display_name":"comparative effectiveness research","score":0.8550000190734863},{"id":"https://openalex.org/keywords/bayesian-adaptive-design","display_name":"Bayesian adaptive design","score":0.8240000009536743},{"id":"https://openalex.org/keywords/treatment-switching","display_name":"treatment switching","score":0.6349999904632568},{"id":"https://openalex.org/keywords/statistical-power","display_name":"statistical power","score":0.7770000100135803},{"id":"https://openalex.org/keywords/sample-size-reduction","display_name":"sample size reduction","score":0.8650000095367432},{"id":"https://openalex.org/keywords/trial-duration","display_name":"trial duration","score":0.625}],"concepts":[{"id":"https://openalex.org/C204243189","wikidata":"https://www.wikidata.org/wiki/Q1363085","display_name":"Randomization","level":3,"score":0.7256748676300049},{"id":"https://openalex.org/C107673813","wikidata":"https://www.wikidata.org/wiki/Q812534","display_name":"Bayesian probability","level":2,"score":0.6015322804450989},{"id":"https://openalex.org/C2982777018","wikidata":"https://www.wikidata.org/wiki/Q99268086","display_name":"Adaptive design","level":3,"score":0.516890287399292},{"id":"https://openalex.org/C2779318504","wikidata":"https://www.wikidata.org/wiki/Q1438035","display_name":"Research design","level":2,"score":0.4371985197067261},{"id":"https://openalex.org/C41008148","wikidata":"https://www.wikidata.org/wiki/Q21198","display_name":"Computer science","level":0,"score":0.43682026863098145},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.42932188510894775},{"id":"https://openalex.org/C105795698","wikidata":"https://www.wikidata.org/wiki/Q12483","display_name":"Statistics","level":1,"score":0.4229589104652405},{"id":"https://openalex.org/C149782125","wikidata":"https://www.wikidata.org/wiki/Q160039","display_name":"Econometrics","level":1,"score":0.3696245849132538},{"id":"https://openalex.org/C119857082","wikidata":"https://www.wikidata.org/wiki/Q2539","display_name":"Machine learning","level":1,"score":0.3499597907066345},{"id":"https://openalex.org/C33923547","wikidata":"https://www.wikidata.org/wiki/Q395","display_name":"Mathematics","level":0,"score":0.3366919755935669},{"id":"https://openalex.org/C168563851","wikidata":"https://www.wikidata.org/wiki/Q1436668","display_name":"Randomized controlled trial","level":2,"score":0.33445483446121216},{"id":"https://openalex.org/C535046627","wikidata":"https://www.wikidata.org/wiki/Q30612","display_name":"Clinical trial","level":2,"score":0.290545254945755},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.13751086592674255}],"mesh":[],"locations_count":3,"locations":[{"id":"doi:10.1080/19466315.2021.1961854","doi":"https://doi.org/10.1080/19466315.2021.1961854","is_oa":false,"landing_page_url":"https://doi.org/10.1080/19466315.2021.1961854","pdf_url":null,"source":{"id":"https://openalex.org/S104563881","display_name":"Statistics in Biopharmaceutical Research","issn_l":"1946-6315","issn":["1946-6315"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["cwts-core","jufo-1","ki-jl-1","norway-1","poland-100","russia-white-list-2","vabb-shw"],"host_organization":"https://openalex.org/P4310320547","host_organization_name":"Taylor & Francis","host_organization_lineage":["https://openalex.org/P4310320547","https://openalex.org/P4310320449"],"host_organization_lineage_names":["Taylor & Francis","Informa"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Statistics in Biopharmaceutical Research","raw_type":"journal-article"},{"id":"pmid:36875290","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/36875290","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":"Statistics in biopharmaceutical research","raw_type":"Journal Article"},{"id":"pmh:oai:pubmedcentral.nih.gov:9979780","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/9979780","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":null,"license_id":null,"version":"acceptedVersion","is_accepted":true,"is_published":false,"raw_source_name":"Stat Biopharm Res","raw_type":"Text"}],"best_oa_location":{"id":"pmh:oai:pubmedcentral.nih.gov:9979780","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/9979780","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":null,"license_id":null,"version":"acceptedVersion","is_accepted":true,"is_published":false,"raw_source_name":"Stat Biopharm Res","raw_type":"Text"},"sustainable_development_goals":[{"display_name":"Good health and well-being","score":0.5683000087738037,"id":"https://metadata.un.org/sdg/3"}],"study_designs":[],"awards":[{"id":"https://openalex.org/G9000122834","display_name":"Frontiers Clinical and Translational Science Institute at the University of Kansas","funder_award_id":"UL1TR002366","funder_id":"https://openalex.org/F4320332161","funder_display_name":"National Institutes of Health"},{"id":"https://openalex.org/G9000911857","display_name":"The University of Kansas Cancer Center","funder_award_id":"P30CA168524","funder_id":"https://openalex.org/F4320337351","funder_display_name":"National Cancer Institute"}],"funders":[{"id":"https://openalex.org/F4320332161","display_name":"National Institutes of Health","ror":"https://ror.org/01cwqze88"},{"id":"https://openalex.org/F4320332445","display_name":"University of Kansas Medical Center","ror":"https://ror.org/036c9yv20"},{"id":"https://openalex.org/F4320337351","display_name":"National Cancer Institute","ror":"https://ror.org/040gcmg81"},{"id":"https://openalex.org/F4320337472","display_name":"National Center for Advancing Translational Sciences","ror":"https://ror.org/04pw6fb54"}],"has_content":{"pdf":false,"grobid_xml":false},"content_urls":null,"referenced_works_count":17,"referenced_works":["https://openalex.org/W1937410663","https://openalex.org/W1953260577","https://openalex.org/W1963902713","https://openalex.org/W1998394614","https://openalex.org/W2007419664","https://openalex.org/W2010379916","https://openalex.org/W2022264347","https://openalex.org/W2032505587","https://openalex.org/W2045656233","https://openalex.org/W2125281699","https://openalex.org/W2442829077","https://openalex.org/W2492928450","https://openalex.org/W2511878324","https://openalex.org/W2582743722","https://openalex.org/W2594200836","https://openalex.org/W2752399231","https://openalex.org/W3049075137"],"related_works":["https://openalex.org/W7036299499","https://openalex.org/W4410602017","https://openalex.org/W2949741455","https://openalex.org/W7005278537","https://openalex.org/W1988140909","https://openalex.org/W2943797642","https://openalex.org/W4411436626","https://openalex.org/W2178838231","https://openalex.org/W4309668382","https://openalex.org/W6887943918"],"abstract_inverted_index":{"Slow":[0],"accrual":[1,182,192],"rate":[2,183,193],"is":[3,14,28,80,110,184],"a":[4,170,175],"major":[5],"challenge":[6,27],"in":[7,30,50,53],"clinical":[8,22,46,69],"trials":[9,23,152],"for":[10,21,114],"rare":[11],"diseases":[12],"and":[13,107,122,137,147,174],"identified":[15],"as":[16,190],"the":[17,41,67,131,159,181,191],"most":[18],"frequent":[19],"reason":[20],"to":[24,39,74,94,112,118,126,130],"fail.":[25],"This":[26],"amplified":[29],"comparative":[31],"effectiveness":[32],"research":[33],"where":[34,153],"multiple":[35],"treatments":[36,76,96],"are":[37,49],"compared":[38,150],"identify":[40],"best":[42],"treatment.":[43],"Novel":[44],"efficient":[45,138],"trial":[47,64,177],"designs":[48],"urgent":[51],"need":[52],"these":[54],"areas.":[55],"Our":[56],"proposed":[57,84,160],"response":[58],"adaptive":[59],"randomization":[60],"(RAR)":[61],"reusing":[62,162],"participants":[63,93,129,161],"design":[65,85,164],"mimics":[66],"real-world":[68],"practice":[70],"that":[71,98,135],"allows":[72],"patients":[73],"switch":[75,95],"when":[77,180],"desired":[78],"outcome":[79],"not":[81],"achieved.":[82,142],"The":[83,186],"increases":[86],"efficiency":[87,187],"by":[88],"two":[89],"strategies:":[90],"1)":[91],"Allowing":[92],"so":[97],"each":[99,154],"participant":[100,115,155],"can":[101,165],"have":[102],"more":[103,128],"than":[104],"one":[105,157],"observation":[106],"hence":[108],"it":[109],"possible":[111],"control":[113],"specific":[116],"variability":[117],"increase":[119],"statistical":[120],"power;":[121],"2)":[123],"Utilizing":[124],"RAR":[125,163],"allocate":[127],"promising":[132],"arms":[133],"such":[134],"ethical":[136],"studies":[139],"will":[140],"be":[141],"Extensive":[143],"simulations":[144],"were":[145],"conducted":[146],"showed":[148],"that,":[149],"with":[151,169],"receives":[156],"treatment,":[158],"achieve":[166],"comparable":[167],"power":[168],"smaller":[171],"sample":[172],"size":[173],"shorter":[176],"duration,":[178],"especially":[179],"low.":[185],"gain":[188],"decreases":[189],"increases.":[194]},"counts_by_year":[{"year":2026,"cited_by_count":1},{"year":2025,"cited_by_count":1},{"year":2017,"cited_by_count":1}],"updated_date":"2026-10-30T12:39:25.321706","created_date":"2021-09-13T00:00:00"}