New clinical trial design in precision medicine: discovery, development and direction
Abstract In the era of precision medicine, it has been increasingly recognized that individuals with a certain disease are complex and different from each other. Due to the underestimation of the significant heterogeneity across participants in traditional “one-size-fits-all” trials, patient-centere...
| Main Authors: | , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Nature Publishing Group
2024-03-01
|
| Series: | Signal Transduction and Targeted Therapy |
| Online Access: | https://doi.org/10.1038/s41392-024-01760-0 |
| _version_ | 1797273133458128896 |
|---|---|
| author | Xiao-Peng Duan Bao-Dong Qin Xiao-Dong Jiao Ke Liu Zhan Wang Yuan-Sheng Zang |
| author_facet | Xiao-Peng Duan Bao-Dong Qin Xiao-Dong Jiao Ke Liu Zhan Wang Yuan-Sheng Zang |
| author_sort | Xiao-Peng Duan |
| collection | DOAJ |
| description | Abstract In the era of precision medicine, it has been increasingly recognized that individuals with a certain disease are complex and different from each other. Due to the underestimation of the significant heterogeneity across participants in traditional “one-size-fits-all” trials, patient-centered trials that could provide optimal therapy customization to individuals with specific biomarkers were developed including the basket, umbrella, and platform trial designs under the master protocol framework. In recent years, the successive FDA approval of indications based on biomarker-guided master protocol designs has demonstrated that these new clinical trials are ushering in tremendous opportunities. Despite the rapid increase in the number of basket, umbrella, and platform trials, the current clinical and research understanding of these new trial designs, as compared with traditional trial designs, remains limited. The majority of the research focuses on methodologies, and there is a lack of in-depth insight concerning the underlying biological logic of these new clinical trial designs. Therefore, we provide this comprehensive review of the discovery and development of basket, umbrella, and platform trials and their underlying logic from the perspective of precision medicine. Meanwhile, we discuss future directions on the potential development of these new clinical design in view of the “Precision Pro”, “Dynamic Precision”, and “Intelligent Precision”. This review would assist trial-related researchers to enhance the innovation and feasibility of clinical trial designs by expounding the underlying logic, which be essential to accelerate the progression of precision medicine. |
| first_indexed | 2024-03-07T14:39:14Z |
| format | Article |
| id | doaj.art-e2005b1451ad4f5486d51ae864d4a4c8 |
| institution | Directory Open Access Journal |
| issn | 2059-3635 |
| language | English |
| last_indexed | 2024-03-07T14:39:14Z |
| publishDate | 2024-03-01 |
| publisher | Nature Publishing Group |
| record_format | Article |
| series | Signal Transduction and Targeted Therapy |
| spelling | doaj.art-e2005b1451ad4f5486d51ae864d4a4c82024-03-05T20:29:35ZengNature Publishing GroupSignal Transduction and Targeted Therapy2059-36352024-03-019112910.1038/s41392-024-01760-0New clinical trial design in precision medicine: discovery, development and directionXiao-Peng Duan0Bao-Dong Qin1Xiao-Dong Jiao2Ke Liu3Zhan Wang4Yuan-Sheng Zang5Department of Medical Oncology, Changzheng Hospital, Naval Medical UniversityDepartment of Medical Oncology, Changzheng Hospital, Naval Medical UniversityDepartment of Medical Oncology, Changzheng Hospital, Naval Medical UniversityDepartment of Medical Oncology, Changzheng Hospital, Naval Medical UniversityDepartment of Medical Oncology, Changzheng Hospital, Naval Medical UniversityDepartment of Medical Oncology, Changzheng Hospital, Naval Medical UniversityAbstract In the era of precision medicine, it has been increasingly recognized that individuals with a certain disease are complex and different from each other. Due to the underestimation of the significant heterogeneity across participants in traditional “one-size-fits-all” trials, patient-centered trials that could provide optimal therapy customization to individuals with specific biomarkers were developed including the basket, umbrella, and platform trial designs under the master protocol framework. In recent years, the successive FDA approval of indications based on biomarker-guided master protocol designs has demonstrated that these new clinical trials are ushering in tremendous opportunities. Despite the rapid increase in the number of basket, umbrella, and platform trials, the current clinical and research understanding of these new trial designs, as compared with traditional trial designs, remains limited. The majority of the research focuses on methodologies, and there is a lack of in-depth insight concerning the underlying biological logic of these new clinical trial designs. Therefore, we provide this comprehensive review of the discovery and development of basket, umbrella, and platform trials and their underlying logic from the perspective of precision medicine. Meanwhile, we discuss future directions on the potential development of these new clinical design in view of the “Precision Pro”, “Dynamic Precision”, and “Intelligent Precision”. This review would assist trial-related researchers to enhance the innovation and feasibility of clinical trial designs by expounding the underlying logic, which be essential to accelerate the progression of precision medicine.https://doi.org/10.1038/s41392-024-01760-0 |
| spellingShingle | Xiao-Peng Duan Bao-Dong Qin Xiao-Dong Jiao Ke Liu Zhan Wang Yuan-Sheng Zang New clinical trial design in precision medicine: discovery, development and direction Signal Transduction and Targeted Therapy |
| title | New clinical trial design in precision medicine: discovery, development and direction |
| title_full | New clinical trial design in precision medicine: discovery, development and direction |
| title_fullStr | New clinical trial design in precision medicine: discovery, development and direction |
| title_full_unstemmed | New clinical trial design in precision medicine: discovery, development and direction |
| title_short | New clinical trial design in precision medicine: discovery, development and direction |
| title_sort | new clinical trial design in precision medicine discovery development and direction |
| url | https://doi.org/10.1038/s41392-024-01760-0 |
| work_keys_str_mv | AT xiaopengduan newclinicaltrialdesigninprecisionmedicinediscoverydevelopmentanddirection AT baodongqin newclinicaltrialdesigninprecisionmedicinediscoverydevelopmentanddirection AT xiaodongjiao newclinicaltrialdesigninprecisionmedicinediscoverydevelopmentanddirection AT keliu newclinicaltrialdesigninprecisionmedicinediscoverydevelopmentanddirection AT zhanwang newclinicaltrialdesigninprecisionmedicinediscoverydevelopmentanddirection AT yuanshengzang newclinicaltrialdesigninprecisionmedicinediscoverydevelopmentanddirection |