Non-inferiority trials: the 'at least as good as' criterion with dichotomous data
The ‘at least as good as’ criterion, introduced by Laster and Johnson for a continuous response variate, is developed here for applications with dichotomous data. This approach is adaptive in nature, as the margin of non‐inferiority is not taken as a fixed difference; it varies as a function of the...
Gespeichert in:
| Veröffentlicht in: | Statistics in medicine 2006-04, Vol.25 (7), p.1115-1130 |
|---|---|
| Hauptverfasser: | , , |
| Format: | Artikel |
| Sprache: | eng |
| Schlagworte: | |
| Online-Zugang: | Volltext |
| Tags: |
Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!
|
| container_end_page | 1130 |
|---|---|
| container_issue | 7 |
| container_start_page | 1115 |
| container_title | Statistics in medicine |
| container_volume | 25 |
| creator | Laster, Larry L. Johnson, Mary F. Kotler, Mitchell L. |
| description | The ‘at least as good as’ criterion, introduced by Laster and Johnson for a continuous response variate, is developed here for applications with dichotomous data. This approach is adaptive in nature, as the margin of non‐inferiority is not taken as a fixed difference; it varies as a function of the positive control response. When the non‐inferiority margin is referenced as a high fraction of the positive control response, the procedure is seen to be uniformly more efficient than the fixed margin approach, yielding smaller sample sizes when sizing non‐inferiority trials under identically specified conditions. Extending this method to proportions is straightforward, but highlights special considerations in the design of non‐inferiority trials versus superiority trials, including potential trade‐offs in statistical efficiency and interpretability. Copyright © 2005 John Wiley & Sons, Ltd. |
| doi_str_mv | 10.1002/sim.2476 |
| format | Article |
| fullrecord | <record><control><sourceid>proquest_cross</sourceid><recordid>TN_cdi_proquest_miscellaneous_67757169</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>1019351821</sourcerecordid><originalsourceid>FETCH-LOGICAL-c3846-1cb8c3b6fb6c4636228cdf90f7b8b6556e9271da60fd0396273d81f9f5d173b3</originalsourceid><addsrcrecordid>eNp10F9rFDEUBfAgil1bwU8gwQfry7S5ySaZ6ZsUbSu1pVoUfAmZ_HFTZyY1yVL325tlBwXBp_vy43DuQegFkCMghB7nMB7RpRSP0AJIJxtCefsYLQiVshES-B56lvMdIQCcyqdoDwRrgUiyQDdXcWrC5F0KMYWywSUFPeQTXFYOH-qCB6dzwTrj7zHaeg-xqW7LJ_wQygrbYFaxxDGuM7a66AP0xNcE93y---j2_bvb0_Pm8vrs4vTtZWNYuxQNmL41rBe-F2YpmKC0NdZ3xMu-7QXnwnVUgtWCeEtYJ6hktgXfeW5Bsp7to9e72PsUf65dLmoM2bhh0JOrVZSQkksQXYWv_oF3cZ2mWk1RymDZdhIqerNDJsWck_PqPoVRp40CorYTqzqx2k5c6cs5b92Pzv6F86YVNDvwEAa3-W-Q-nzxcQ6cfcjF_frjdfpRn2CSq69XZ-rLh3P2jfNPCthv5KGS5A</addsrcrecordid><sourcetype>Aggregation Database</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>223148971</pqid></control><display><type>article</type><title>Non-inferiority trials: the 'at least as good as' criterion with dichotomous data</title><source>MEDLINE</source><source>Wiley Online Library All Journals</source><creator>Laster, Larry L. ; Johnson, Mary F. ; Kotler, Mitchell L.</creator><creatorcontrib>Laster, Larry L. ; Johnson, Mary F. ; Kotler, Mitchell L.</creatorcontrib><description>The ‘at least as good as’ criterion, introduced by Laster and Johnson for a continuous response variate, is developed here for applications with dichotomous data. This approach is adaptive in nature, as the margin of non‐inferiority is not taken as a fixed difference; it varies as a function of the positive control response. When the non‐inferiority margin is referenced as a high fraction of the positive control response, the procedure is seen to be uniformly more efficient than the fixed margin approach, yielding smaller sample sizes when sizing non‐inferiority trials under identically specified conditions. Extending this method to proportions is straightforward, but highlights special considerations in the design of non‐inferiority trials versus superiority trials, including potential trade‐offs in statistical efficiency and interpretability. Copyright © 2005 John Wiley & Sons, Ltd.</description><identifier>ISSN: 0277-6715</identifier><identifier>EISSN: 1097-0258</identifier><identifier>DOI: 10.1002/sim.2476</identifier><identifier>PMID: 16381070</identifier><identifier>CODEN: SMEDDA</identifier><language>eng</language><publisher>Chichester, UK: John Wiley & Sons, Ltd</publisher><subject>adaptive versus fixed margins ; at least as good as ; Clinical trials ; Data Interpretation, Statistical ; Drug Evaluation - methods ; Humans ; Models, Statistical ; non-inferiority trials ; Randomized Controlled Trials as Topic - methods ; Research Design ; Sample Size ; Statistical analysis ; statistical efficiency ; Statistical methods</subject><ispartof>Statistics in medicine, 2006-04, Vol.25 (7), p.1115-1130</ispartof><rights>Copyright © 2005 John Wiley & Sons, Ltd.</rights><rights>Copyright 2005 John Wiley & Sons, Ltd.</rights><rights>Copyright John Wiley and Sons, Limited Apr 15, 2006</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c3846-1cb8c3b6fb6c4636228cdf90f7b8b6556e9271da60fd0396273d81f9f5d173b3</citedby><cites>FETCH-LOGICAL-c3846-1cb8c3b6fb6c4636228cdf90f7b8b6556e9271da60fd0396273d81f9f5d173b3</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://onlinelibrary.wiley.com/doi/pdf/10.1002%2Fsim.2476$$EPDF$$P50$$Gwiley$$H</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1002%2Fsim.2476$$EHTML$$P50$$Gwiley$$H</linktohtml><link.rule.ids>314,780,784,1416,27923,27924,45573,45574</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/16381070$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Laster, Larry L.</creatorcontrib><creatorcontrib>Johnson, Mary F.</creatorcontrib><creatorcontrib>Kotler, Mitchell L.</creatorcontrib><title>Non-inferiority trials: the 'at least as good as' criterion with dichotomous data</title><title>Statistics in medicine</title><addtitle>Statist. Med</addtitle><description>The ‘at least as good as’ criterion, introduced by Laster and Johnson for a continuous response variate, is developed here for applications with dichotomous data. This approach is adaptive in nature, as the margin of non‐inferiority is not taken as a fixed difference; it varies as a function of the positive control response. When the non‐inferiority margin is referenced as a high fraction of the positive control response, the procedure is seen to be uniformly more efficient than the fixed margin approach, yielding smaller sample sizes when sizing non‐inferiority trials under identically specified conditions. Extending this method to proportions is straightforward, but highlights special considerations in the design of non‐inferiority trials versus superiority trials, including potential trade‐offs in statistical efficiency and interpretability. Copyright © 2005 John Wiley & Sons, Ltd.</description><subject>adaptive versus fixed margins</subject><subject>at least as good as</subject><subject>Clinical trials</subject><subject>Data Interpretation, Statistical</subject><subject>Drug Evaluation - methods</subject><subject>Humans</subject><subject>Models, Statistical</subject><subject>non-inferiority trials</subject><subject>Randomized Controlled Trials as Topic - methods</subject><subject>Research Design</subject><subject>Sample Size</subject><subject>Statistical analysis</subject><subject>statistical efficiency</subject><subject>Statistical methods</subject><issn>0277-6715</issn><issn>1097-0258</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2006</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNp10F9rFDEUBfAgil1bwU8gwQfry7S5ySaZ6ZsUbSu1pVoUfAmZ_HFTZyY1yVL325tlBwXBp_vy43DuQegFkCMghB7nMB7RpRSP0AJIJxtCefsYLQiVshES-B56lvMdIQCcyqdoDwRrgUiyQDdXcWrC5F0KMYWywSUFPeQTXFYOH-qCB6dzwTrj7zHaeg-xqW7LJ_wQygrbYFaxxDGuM7a66AP0xNcE93y---j2_bvb0_Pm8vrs4vTtZWNYuxQNmL41rBe-F2YpmKC0NdZ3xMu-7QXnwnVUgtWCeEtYJ6hktgXfeW5Bsp7to9e72PsUf65dLmoM2bhh0JOrVZSQkksQXYWv_oF3cZ2mWk1RymDZdhIqerNDJsWck_PqPoVRp40CorYTqzqx2k5c6cs5b92Pzv6F86YVNDvwEAa3-W-Q-nzxcQ6cfcjF_frjdfpRn2CSq69XZ-rLh3P2jfNPCthv5KGS5A</recordid><startdate>20060415</startdate><enddate>20060415</enddate><creator>Laster, Larry L.</creator><creator>Johnson, Mary F.</creator><creator>Kotler, Mitchell L.</creator><general>John Wiley & Sons, Ltd</general><general>Wiley Subscription Services, Inc</general><scope>BSCLL</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>K9.</scope><scope>7X8</scope></search><sort><creationdate>20060415</creationdate><title>Non-inferiority trials: the 'at least as good as' criterion with dichotomous data</title><author>Laster, Larry L. ; Johnson, Mary F. ; Kotler, Mitchell L.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c3846-1cb8c3b6fb6c4636228cdf90f7b8b6556e9271da60fd0396273d81f9f5d173b3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2006</creationdate><topic>adaptive versus fixed margins</topic><topic>at least as good as</topic><topic>Clinical trials</topic><topic>Data Interpretation, Statistical</topic><topic>Drug Evaluation - methods</topic><topic>Humans</topic><topic>Models, Statistical</topic><topic>non-inferiority trials</topic><topic>Randomized Controlled Trials as Topic - methods</topic><topic>Research Design</topic><topic>Sample Size</topic><topic>Statistical analysis</topic><topic>statistical efficiency</topic><topic>Statistical methods</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Laster, Larry L.</creatorcontrib><creatorcontrib>Johnson, Mary F.</creatorcontrib><creatorcontrib>Kotler, Mitchell L.</creatorcontrib><collection>Istex</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>MEDLINE - Academic</collection><jtitle>Statistics in medicine</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Laster, Larry L.</au><au>Johnson, Mary F.</au><au>Kotler, Mitchell L.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Non-inferiority trials: the 'at least as good as' criterion with dichotomous data</atitle><jtitle>Statistics in medicine</jtitle><addtitle>Statist. Med</addtitle><date>2006-04-15</date><risdate>2006</risdate><volume>25</volume><issue>7</issue><spage>1115</spage><epage>1130</epage><pages>1115-1130</pages><issn>0277-6715</issn><eissn>1097-0258</eissn><coden>SMEDDA</coden><abstract>The ‘at least as good as’ criterion, introduced by Laster and Johnson for a continuous response variate, is developed here for applications with dichotomous data. This approach is adaptive in nature, as the margin of non‐inferiority is not taken as a fixed difference; it varies as a function of the positive control response. When the non‐inferiority margin is referenced as a high fraction of the positive control response, the procedure is seen to be uniformly more efficient than the fixed margin approach, yielding smaller sample sizes when sizing non‐inferiority trials under identically specified conditions. Extending this method to proportions is straightforward, but highlights special considerations in the design of non‐inferiority trials versus superiority trials, including potential trade‐offs in statistical efficiency and interpretability. Copyright © 2005 John Wiley & Sons, Ltd.</abstract><cop>Chichester, UK</cop><pub>John Wiley & Sons, Ltd</pub><pmid>16381070</pmid><doi>10.1002/sim.2476</doi><tpages>16</tpages><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0277-6715 |
| ispartof | Statistics in medicine, 2006-04, Vol.25 (7), p.1115-1130 |
| issn | 0277-6715 1097-0258 |
| language | eng |
| recordid | cdi_proquest_miscellaneous_67757169 |
| source | MEDLINE; Wiley Online Library All Journals |
| subjects | adaptive versus fixed margins at least as good as Clinical trials Data Interpretation, Statistical Drug Evaluation - methods Humans Models, Statistical non-inferiority trials Randomized Controlled Trials as Topic - methods Research Design Sample Size Statistical analysis statistical efficiency Statistical methods |
| title | Non-inferiority trials: the 'at least as good as' criterion with dichotomous data |
| url | https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2025-01-11T22%3A27%3A57IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-proquest_cross&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=Non-inferiority%20trials:%20the%20'at%20least%20as%20good%20as'%20criterion%20with%20dichotomous%20data&rft.jtitle=Statistics%20in%20medicine&rft.au=Laster,%20Larry%20L.&rft.date=2006-04-15&rft.volume=25&rft.issue=7&rft.spage=1115&rft.epage=1130&rft.pages=1115-1130&rft.issn=0277-6715&rft.eissn=1097-0258&rft.coden=SMEDDA&rft_id=info:doi/10.1002/sim.2476&rft_dat=%3Cproquest_cross%3E1019351821%3C/proquest_cross%3E%3Curl%3E%3C/url%3E&disable_directlink=true&sfx.directlink=off&sfx.report_link=0&rft_id=info:oai/&rft_pqid=223148971&rft_id=info:pmid/16381070&rfr_iscdi=true |