Promising zone two-stage design for a single-arm study with binary outcome
Adaptive designs are increasingly used in clinical trials to assess the effectiveness of new drugs. For a single-arm study with a binary outcome, several adaptive designs were developed by using numerical search algorithms and the conditional power approach. The design based on numerical search algo...
Saved in:
| Published in: | Statistical methods in medical research Vol. 32; no. 6; p. 1159 |
|---|---|
| Main Author: | |
| Format: | Journal Article |
| Language: | English |
| Published: |
England
01.06.2023
|
| Subjects: | |
| ISSN: | 1477-0334, 1477-0334 |
| Online Access: | Get more information |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| Abstract | Adaptive designs are increasingly used in clinical trials to assess the effectiveness of new drugs. For a single-arm study with a binary outcome, several adaptive designs were developed by using numerical search algorithms and the conditional power approach. The design based on numerical search algorithms is able to identify the global optimal design, but the computational intensity limits the usage of these designs. The conditional power approach searches for the optimal design without expensive computing time. In addition, promising zone strategy was proposed to move on drug development to the follow-up stages when the interim results are promising. We propose to develop two adaptive designs: One based on the conditional power approach, and the other based on the promising zone strategy. These two designs preserve types I and II error rates. It is preferable to satisfy the monotonic property for adaptive designs: The second stage sample size decreases as the first stage responses go up. We theoretically prove this important property for the two proposed designs. The proposed designs can be easily applied to real trials with limited computing resources. |
|---|---|
| AbstractList | Adaptive designs are increasingly used in clinical trials to assess the effectiveness of new drugs. For a single-arm study with a binary outcome, several adaptive designs were developed by using numerical search algorithms and the conditional power approach. The design based on numerical search algorithms is able to identify the global optimal design, but the computational intensity limits the usage of these designs. The conditional power approach searches for the optimal design without expensive computing time. In addition, promising zone strategy was proposed to move on drug development to the follow-up stages when the interim results are promising. We propose to develop two adaptive designs: One based on the conditional power approach, and the other based on the promising zone strategy. These two designs preserve types I and II error rates. It is preferable to satisfy the monotonic property for adaptive designs: The second stage sample size decreases as the first stage responses go up. We theoretically prove this important property for the two proposed designs. The proposed designs can be easily applied to real trials with limited computing resources.Adaptive designs are increasingly used in clinical trials to assess the effectiveness of new drugs. For a single-arm study with a binary outcome, several adaptive designs were developed by using numerical search algorithms and the conditional power approach. The design based on numerical search algorithms is able to identify the global optimal design, but the computational intensity limits the usage of these designs. The conditional power approach searches for the optimal design without expensive computing time. In addition, promising zone strategy was proposed to move on drug development to the follow-up stages when the interim results are promising. We propose to develop two adaptive designs: One based on the conditional power approach, and the other based on the promising zone strategy. These two designs preserve types I and II error rates. It is preferable to satisfy the monotonic property for adaptive designs: The second stage sample size decreases as the first stage responses go up. We theoretically prove this important property for the two proposed designs. The proposed designs can be easily applied to real trials with limited computing resources. Adaptive designs are increasingly used in clinical trials to assess the effectiveness of new drugs. For a single-arm study with a binary outcome, several adaptive designs were developed by using numerical search algorithms and the conditional power approach. The design based on numerical search algorithms is able to identify the global optimal design, but the computational intensity limits the usage of these designs. The conditional power approach searches for the optimal design without expensive computing time. In addition, promising zone strategy was proposed to move on drug development to the follow-up stages when the interim results are promising. We propose to develop two adaptive designs: One based on the conditional power approach, and the other based on the promising zone strategy. These two designs preserve types I and II error rates. It is preferable to satisfy the monotonic property for adaptive designs: The second stage sample size decreases as the first stage responses go up. We theoretically prove this important property for the two proposed designs. The proposed designs can be easily applied to real trials with limited computing resources. |
| Author | Shan, Guogen |
| Author_xml | – sequence: 1 givenname: Guogen orcidid: 0000-0001-8690-6599 surname: Shan fullname: Shan, Guogen organization: Department of Biostatistics, University of Florida, Gainesville, FL, USA |
| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/36998163$$D View this record in MEDLINE/PubMed |
| BookMark | eNpNj0tLxDAYRYOMOA_9AW4kSzfVfEmax1IGnwzoYvYlTZNaaZuxSRnGX2_FEVzduzgc7l2iWR96h9AlkBsAKW-JFpQqQikDEFwyeYIWwKXMCGN89q_P0TLGD0KIJFyfoTkTWisQbIFe3obQNbHpa_w1yXHahywmUztcudjUPfZhwAb_AK3LzNDhmMbqgPdNesdl05vhgMOYbOjcOTr1po3u4pgrtH24366fss3r4_P6bpNZLmTKuDbEUaNy4jgjYHjJQSgrpCyF5Zp5D6UH6XNvgTJdMuWJrbTNK-V4BXSFrn-1uyF8ji6mYtpvXdua3oUxFlRqplUuhZ7QqyM6lp2rit3QdNPg4u8-_QaOFV5b |
| CitedBy_id | crossref_primary_10_3389_fnagi_2025_1576373 crossref_primary_10_3233_JAD_240285 crossref_primary_10_1016_j_foodqual_2025_105523 crossref_primary_10_1080_10543406_2023_2234028 crossref_primary_10_1007_s00362_024_01606_5 crossref_primary_10_1177_17407745241309056 |
| ContentType | Journal Article |
| DBID | CGR CUY CVF ECM EIF NPM 7X8 |
| DOI | 10.1177/09622802231164737 |
| DatabaseName | Medline MEDLINE MEDLINE (Ovid) MEDLINE MEDLINE PubMed MEDLINE - Academic |
| DatabaseTitle | MEDLINE Medline Complete MEDLINE with Full Text PubMed MEDLINE (Ovid) MEDLINE - Academic |
| DatabaseTitleList | MEDLINE - Academic MEDLINE |
| Database_xml | – sequence: 1 dbid: NPM name: PubMed url: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=PubMed sourceTypes: Index Database – sequence: 2 dbid: 7X8 name: MEDLINE - Academic url: https://search.proquest.com/medline sourceTypes: Aggregation Database |
| DeliveryMethod | no_fulltext_linktorsrc |
| Discipline | Medicine Statistics Mathematics |
| EISSN | 1477-0334 |
| ExternalDocumentID | 36998163 |
| Genre | Journal Article |
| GrantInformation_xml | – fundername: NCI NIH HHS grantid: R03 CA248006 – fundername: NIA NIH HHS grantid: R01 AG070849 |
| GroupedDBID | --- -TM .2G .2J .2N 0-V 01A 0R~ 123 1~K 29Q 31S 31U 31X 31Y 31Z 36B 4.4 53G 54M 5RE 5VS 6PF 7X7 88E 88I 8C1 8FE 8FG 8FI 8FJ 8R4 8R5 AABMB AABOD AACKU AACMV AACTG AADUE AAEWN AAGGD AAJIQ AAJOX AAJPV AANSI AAPEO AAQDB AAQXH AAQXI AARDL AARIX AATAA AATBZ AAWTL AAYTG ABAWP ABCCA ABCJG ABDLQ ABDWY ABEIX ABFWQ ABHKI ABHQH ABJCF ABJIS ABKRH ABLUO ABPGX ABPNF ABQKF ABQXT ABRHV ABTDE ABUJY ABUWG ABVFX ABVVC ABYTW ACARO ACDSZ ACDXX ACFEJ ACFMA ACGBL ACGFS ACGOD ACGZU ACIWK ACJER ACLHI ACLZU ACOFE ACOXC ACROE ACRPL ACSIQ ACUAV ACUIR ACXKE ACXMB ADBBV ADDLC ADEIA ADNMO ADNON ADRRZ ADTBJ ADUKL ADVBO ADYCS AECGH AECVZ AEDTQ AENEX AEPTA AEQLS AERKM AESZF AEUHG AEWDL AEWHI AEXNY AFEET AFKBI AFKRA AFKRG AFMOU AFQAA AFUIA AFWMB AGKLV AGNHF AGWFA AGWNL AHDMH AHHFK AHMBA AJEFB AJMMQ AJUZI AJXAJ ALIPV ALKWR ALMA_UNASSIGNED_HOLDINGS ALSLI AMCVQ ANDLU ARALO ARTOV ASOEW ASPBG AUTPY AUVAJ AVWKF AYAKG AZFZN AZQEC B8O B8R B8Z B93 B94 BBRGL BDDNI BENPR BGLVJ BKIIM BPACV BPHCQ BSEHC BVXVI BYIEH C45 CAG CBRKF CCPQU CFDXU CGR COF CORYS CQQTX CS3 CUY CVF DC- DD- DD0 DE- DF0 DO- DOPDO DU5 DV7 DWQXO D~Y EBS ECM EIF EJD EMOBN F5P FEDTE FHBDP FYUFA GNUQQ GROUPED_SAGE_PREMIER_JOURNAL_COLLECTION H13 HCIFZ HEHIP HF~ HMCUK HVGLF HZ~ J8X K.F K.J L6V M1P M2P M2S M4V M7S N9A NPM O9- OVD P.B P2P PHGZT PQQKQ PROAC PSQYO PTHSS Q1R Q2X Q7K Q7L Q7X Q82 Q83 RIG ROL S01 SAUOL SCNPE SDB SFB SFC SFK SFN SFT SGA SGP SGR SGV SGX SGZ SHG SNB SPJ SPV SQCSI STM TEORI TN5 UKHRP YHZ ZONMY ZPPRI ZRKOI 7X8 AAPII ABIDT ADEBD AJGYC AJHME AJVBE AMVHM SASJQ |
| ID | FETCH-LOGICAL-c467t-49a0e2a850e4301a4b4168c677b6c493ff1bf17f5fc1239b38f0cd9c5d8e4d12 |
| IEDL.DBID | 7X8 |
| ISICitedReferencesCount | 5 |
| ISICitedReferencesURI | http://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=Summon&SrcAuth=ProQuest&DestLinkType=CitingArticles&DestApp=WOS_CPL&KeyUT=000960754400001&url=https%3A%2F%2Fcvtisr.summon.serialssolutions.com%2F%23%21%2Fsearch%3Fho%3Df%26include.ft.matches%3Dt%26l%3Dnull%26q%3D |
| ISSN | 1477-0334 |
| IngestDate | Thu Oct 02 10:59:04 EDT 2025 Thu Apr 03 07:02:05 EDT 2025 |
| IsDoiOpenAccess | false |
| IsOpenAccess | true |
| IsPeerReviewed | true |
| IsScholarly | true |
| Issue | 6 |
| Keywords | optimal two-stage design single-arm study Adaptive design promising zone design conditional power binary response |
| Language | English |
| LinkModel | DirectLink |
| MergedId | FETCHMERGED-LOGICAL-c467t-49a0e2a850e4301a4b4168c677b6c493ff1bf17f5fc1239b38f0cd9c5d8e4d12 |
| Notes | ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23 |
| ORCID | 0000-0001-8690-6599 |
| OpenAccessLink | https://pmc.ncbi.nlm.nih.gov/articles/PMC10641844/pdf/nihms-1943513.pdf |
| PMID | 36998163 |
| PQID | 2793985769 |
| PQPubID | 23479 |
| ParticipantIDs | proquest_miscellaneous_2793985769 pubmed_primary_36998163 |
| PublicationCentury | 2000 |
| PublicationDate | 2023-06-01 |
| PublicationDateYYYYMMDD | 2023-06-01 |
| PublicationDate_xml | – month: 06 year: 2023 text: 2023-06-01 day: 01 |
| PublicationDecade | 2020 |
| PublicationPlace | England |
| PublicationPlace_xml | – name: England |
| PublicationTitle | Statistical methods in medical research |
| PublicationTitleAlternate | Stat Methods Med Res |
| PublicationYear | 2023 |
| SSID | ssj0007049 |
| Score | 2.38318 |
| Snippet | Adaptive designs are increasingly used in clinical trials to assess the effectiveness of new drugs. For a single-arm study with a binary outcome, several... |
| SourceID | proquest pubmed |
| SourceType | Aggregation Database Index Database |
| StartPage | 1159 |
| SubjectTerms | Algorithms Research Design Sample Size |
| Title | Promising zone two-stage design for a single-arm study with binary outcome |
| URI | https://www.ncbi.nlm.nih.gov/pubmed/36998163 https://www.proquest.com/docview/2793985769 |
| Volume | 32 |
| WOSCitedRecordID | wos000960754400001&url=https%3A%2F%2Fcvtisr.summon.serialssolutions.com%2F%23%21%2Fsearch%3Fho%3Df%26include.ft.matches%3Dt%26l%3Dnull%26q%3D |
| hasFullText | |
| inHoldings | 1 |
| isFullTextHit | |
| isPrint | |
| link | http://cvtisr.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwpZ1LS8NAEMcHtSL14KO-6osVvC5mu5vs7klELCK29FCkt5Lsw4smtQ8FP72zSaonQfCSUwKTfc0vM5P_AFyKxDnHjaZOJYYKYWKaaampUg7hXBijytDF06Ps99VopAd1wG1Wl1Uuz8TyoLaFCTHyqw4uJK2QjvX15I2GrlEhu1q30FiFBkeUCSVdcvSjFi4r_GVCShpxLuqsZim4pJMgBIPOkQVFLS5_J8zS03S3_2vjDmzVjEluqkWxCysub8Fm71ugddaCjV6dU29BM_BmJde8Bw-DaYEzj_6MfBa5I_OPgiI_Pjtiy1IPgoxLUhJueHE0nb6SUp-WhHAuycp_e0mxmKO5bh-G3bvh7T2t2y1Qg6flnAqdRq6TqjhyArd9KjKENWUSKbPECM29Z5ln0sfeoLvTGVc-Mlab2ConLOscwFqOlh0B0cxbqxIkC6YF86n2PmZGSxvEfVIRteFiOX5jfKeQokhzVyxm458RbMNhNQnjSSW7MeYJfhoiPh7_4ekTaIa-8FVN1yk0PO5ldwbr5h0HdHpeLhO89ge9L6aoxhg |
| linkProvider | ProQuest |
| openUrl | ctx_ver=Z39.88-2004&ctx_enc=info%3Aofi%2Fenc%3AUTF-8&rfr_id=info%3Asid%2Fsummon.serialssolutions.com&rft_val_fmt=info%3Aofi%2Ffmt%3Akev%3Amtx%3Ajournal&rft.genre=article&rft.atitle=Promising+zone+two-stage+design+for+a+single-arm+study+with+binary+outcome&rft.jtitle=Statistical+methods+in+medical+research&rft.au=Shan%2C+Guogen&rft.date=2023-06-01&rft.eissn=1477-0334&rft.volume=32&rft.issue=6&rft.spage=1159&rft_id=info:doi/10.1177%2F09622802231164737&rft_id=info%3Apmid%2F36998163&rft_id=info%3Apmid%2F36998163&rft.externalDocID=36998163 |
| thumbnail_l | http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/lc.gif&issn=1477-0334&client=summon |
| thumbnail_m | http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/mc.gif&issn=1477-0334&client=summon |
| thumbnail_s | http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/sc.gif&issn=1477-0334&client=summon |