Recent innovations in adaptive trial designs: A review of design opportunities in translational research
Clinical trials are constantly evolving in the context of increasingly complex research questions and potentially limited resources. In this review article, we discuss the emergence of “adaptive” clinical trials that allow for the preplanned modification of an ongoing clinical trial based on the acc...
| Main Authors: | , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Cambridge University Press
2023-01-01
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| Series: | Journal of Clinical and Translational Science |
| Subjects: | |
| Online Access: | https://www.cambridge.org/core/product/identifier/S205986612300537X/type/journal_article |
| _version_ | 1797810245776441344 |
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| author | Alexander M. Kaizer Hayley M. Belli Zhongyang Ma Andrew G. Nicklawsky Samantha C. Roberts Jessica Wild Adane F. Wogu Mengli Xiao Roy T. Sabo |
| author_facet | Alexander M. Kaizer Hayley M. Belli Zhongyang Ma Andrew G. Nicklawsky Samantha C. Roberts Jessica Wild Adane F. Wogu Mengli Xiao Roy T. Sabo |
| author_sort | Alexander M. Kaizer |
| collection | DOAJ |
| description | Clinical trials are constantly evolving in the context of increasingly complex research questions and potentially limited resources. In this review article, we discuss the emergence of “adaptive” clinical trials that allow for the preplanned modification of an ongoing clinical trial based on the accumulating evidence with application across translational research. These modifications may include terminating a trial before completion due to futility or efficacy, re-estimating the needed sample size to ensure adequate power, enriching the target population enrolled in the study, selecting across multiple treatment arms, revising allocation ratios used for randomization, or selecting the most appropriate endpoint. Emerging topics related to borrowing information from historic or supplemental data sources, sequential multiple assignment randomized trials (SMART), master protocol and seamless designs, and phase I dose-finding studies are also presented. Each design element includes a brief overview with an accompanying case study to illustrate the design method in practice. We close with brief discussions relating to the statistical considerations for these contemporary designs. |
| first_indexed | 2024-03-13T07:06:23Z |
| format | Article |
| id | doaj.art-42a107b8420a476abc7cc829ec215812 |
| institution | Directory Open Access Journal |
| issn | 2059-8661 |
| language | English |
| last_indexed | 2024-03-13T07:06:23Z |
| publishDate | 2023-01-01 |
| publisher | Cambridge University Press |
| record_format | Article |
| series | Journal of Clinical and Translational Science |
| spelling | doaj.art-42a107b8420a476abc7cc829ec2158122023-06-06T10:23:52ZengCambridge University PressJournal of Clinical and Translational Science2059-86612023-01-01710.1017/cts.2023.537Recent innovations in adaptive trial designs: A review of design opportunities in translational researchAlexander M. Kaizer0https://orcid.org/0000-0003-2334-5514Hayley M. Belli1Zhongyang Ma2Andrew G. Nicklawsky3Samantha C. Roberts4Jessica Wild5Adane F. Wogu6https://orcid.org/0000-0002-6592-6512Mengli Xiao7Roy T. Sabo8Department of Biostatistics and Informatics, University of Colorado Anschutz Medical Campus, Aurora, CO, USADepartment of Population Health, New York University Grossman School of Medicine, New York, NY, USADepartment of Population Health, New York University Grossman School of Medicine, New York, NY, USADepartment of Pediatrics, University of Colorado Anschutz Medical Campus, Aurora, CO, USADepartment of Biostatistics and Informatics, University of Colorado Anschutz Medical Campus, Aurora, CO, USADepartment of Biostatistics and Informatics, University of Colorado Anschutz Medical Campus, Aurora, CO, USADepartment of Biostatistics and Informatics, University of Colorado Anschutz Medical Campus, Aurora, CO, USADepartment of Biostatistics and Informatics, University of Colorado Anschutz Medical Campus, Aurora, CO, USADepartment of Biostatistics, Virginia Commonwealth University, Richmond, VA, USAClinical trials are constantly evolving in the context of increasingly complex research questions and potentially limited resources. In this review article, we discuss the emergence of “adaptive” clinical trials that allow for the preplanned modification of an ongoing clinical trial based on the accumulating evidence with application across translational research. These modifications may include terminating a trial before completion due to futility or efficacy, re-estimating the needed sample size to ensure adequate power, enriching the target population enrolled in the study, selecting across multiple treatment arms, revising allocation ratios used for randomization, or selecting the most appropriate endpoint. Emerging topics related to borrowing information from historic or supplemental data sources, sequential multiple assignment randomized trials (SMART), master protocol and seamless designs, and phase I dose-finding studies are also presented. Each design element includes a brief overview with an accompanying case study to illustrate the design method in practice. We close with brief discussions relating to the statistical considerations for these contemporary designs.https://www.cambridge.org/core/product/identifier/S205986612300537X/type/journal_articleAdaptive trialssample size re-estimationtreatment arm selectionsmart designsmaster protocols |
| spellingShingle | Alexander M. Kaizer Hayley M. Belli Zhongyang Ma Andrew G. Nicklawsky Samantha C. Roberts Jessica Wild Adane F. Wogu Mengli Xiao Roy T. Sabo Recent innovations in adaptive trial designs: A review of design opportunities in translational research Journal of Clinical and Translational Science Adaptive trials sample size re-estimation treatment arm selection smart designs master protocols |
| title | Recent innovations in adaptive trial designs: A review of design opportunities in translational research |
| title_full | Recent innovations in adaptive trial designs: A review of design opportunities in translational research |
| title_fullStr | Recent innovations in adaptive trial designs: A review of design opportunities in translational research |
| title_full_unstemmed | Recent innovations in adaptive trial designs: A review of design opportunities in translational research |
| title_short | Recent innovations in adaptive trial designs: A review of design opportunities in translational research |
| title_sort | recent innovations in adaptive trial designs a review of design opportunities in translational research |
| topic | Adaptive trials sample size re-estimation treatment arm selection smart designs master protocols |
| url | https://www.cambridge.org/core/product/identifier/S205986612300537X/type/journal_article |
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