Application of sample size re-estimation in clinical trials: A systematic review
Background: Sample size re-estimation (SSR) is a method used to recalculate sample size during clinical trial conduct to address a lack of adequate information and can have a significant impact on study size, duration, resources, and cost. Few studies to date have summarized the conditions and circu...
| Main Authors: | , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2023-12-01
|
| Series: | Contemporary Clinical Trials Communications |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2451865423001564 |
| _version_ | 1797389387536793600 |
|---|---|
| author | Hirotaka Mano Yuji Tanaka Shunichiro Orihara Junji Moriya |
| author_facet | Hirotaka Mano Yuji Tanaka Shunichiro Orihara Junji Moriya |
| author_sort | Hirotaka Mano |
| collection | DOAJ |
| description | Background: Sample size re-estimation (SSR) is a method used to recalculate sample size during clinical trial conduct to address a lack of adequate information and can have a significant impact on study size, duration, resources, and cost. Few studies to date have summarized the conditions and circumstances under which SSR is applied. We therefore performed a systematic review of the literature related to SSR to better understand its application in clinical trial settings. Methods: PubMed was used as the primary search source, supplemented with information from ClinicalTrials.gov where necessary details were lacking from PubMed. A systematic review was performed according to a pre-specified search strategy to identify clinical trials using SSR. Features of SSR, such as study phase and study start year, were summarized. Results: In total, 253 publications met the pre-specified search criteria and 27 clinical trials were subsequently determined as relevant in SSR usage. Among trials where the study phase was provided, 2 (7.4%) trials were Phase I, 5 (18.5%) trials were Phase II, 11 (40.7%) trials were Phase III, and 2 (7.4%) trials were Phase IV. Conclusion: Our results showed that SSR is also used in Phase I and II, which involve earlier decision making. We expect that SSR will continue to be used in early-phase trials where sufficient prior information may not be available. Furthermore, no major trends were observed in relation to therapy area or type of SSR, meaning that SSR may become a feasible and widely applied method in the future. |
| first_indexed | 2024-03-08T22:56:13Z |
| format | Article |
| id | doaj.art-11afed4e414240c2bceea79f31b68429 |
| institution | Directory Open Access Journal |
| issn | 2451-8654 |
| language | English |
| last_indexed | 2024-03-08T22:56:13Z |
| publishDate | 2023-12-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Contemporary Clinical Trials Communications |
| spelling | doaj.art-11afed4e414240c2bceea79f31b684292023-12-16T06:08:34ZengElsevierContemporary Clinical Trials Communications2451-86542023-12-0136101210Application of sample size re-estimation in clinical trials: A systematic reviewHirotaka Mano0Yuji Tanaka1Shunichiro Orihara2Junji Moriya3Biostatistics Group, Biometrics Department, Development Unit, R&D Division, Kyowa Kirin Co., Ltd., Otemachi Financial City Grand Cube, 1-9-2 Otemachi, Chiyoda-ku, Tokyo, JapanBiostatistics Group, Biometrics Department, Development Unit, R&D Division, Kyowa Kirin Co., Ltd., Otemachi Financial City Grand Cube, 1-9-2 Otemachi, Chiyoda-ku, Tokyo, JapanBiostatistics Group, Biometrics Department, Development Unit, R&D Division, Kyowa Kirin Co., Ltd., Otemachi Financial City Grand Cube, 1-9-2 Otemachi, Chiyoda-ku, Tokyo, JapanCorresponding author.; Biostatistics Group, Biometrics Department, Development Unit, R&D Division, Kyowa Kirin Co., Ltd., Otemachi Financial City Grand Cube, 1-9-2 Otemachi, Chiyoda-ku, Tokyo, JapanBackground: Sample size re-estimation (SSR) is a method used to recalculate sample size during clinical trial conduct to address a lack of adequate information and can have a significant impact on study size, duration, resources, and cost. Few studies to date have summarized the conditions and circumstances under which SSR is applied. We therefore performed a systematic review of the literature related to SSR to better understand its application in clinical trial settings. Methods: PubMed was used as the primary search source, supplemented with information from ClinicalTrials.gov where necessary details were lacking from PubMed. A systematic review was performed according to a pre-specified search strategy to identify clinical trials using SSR. Features of SSR, such as study phase and study start year, were summarized. Results: In total, 253 publications met the pre-specified search criteria and 27 clinical trials were subsequently determined as relevant in SSR usage. Among trials where the study phase was provided, 2 (7.4%) trials were Phase I, 5 (18.5%) trials were Phase II, 11 (40.7%) trials were Phase III, and 2 (7.4%) trials were Phase IV. Conclusion: Our results showed that SSR is also used in Phase I and II, which involve earlier decision making. We expect that SSR will continue to be used in early-phase trials where sufficient prior information may not be available. Furthermore, no major trends were observed in relation to therapy area or type of SSR, meaning that SSR may become a feasible and widely applied method in the future.http://www.sciencedirect.com/science/article/pii/S2451865423001564Adaptive designClinical trialSample size re-estimationSystematic review |
| spellingShingle | Hirotaka Mano Yuji Tanaka Shunichiro Orihara Junji Moriya Application of sample size re-estimation in clinical trials: A systematic review Contemporary Clinical Trials Communications Adaptive design Clinical trial Sample size re-estimation Systematic review |
| title | Application of sample size re-estimation in clinical trials: A systematic review |
| title_full | Application of sample size re-estimation in clinical trials: A systematic review |
| title_fullStr | Application of sample size re-estimation in clinical trials: A systematic review |
| title_full_unstemmed | Application of sample size re-estimation in clinical trials: A systematic review |
| title_short | Application of sample size re-estimation in clinical trials: A systematic review |
| title_sort | application of sample size re estimation in clinical trials a systematic review |
| topic | Adaptive design Clinical trial Sample size re-estimation Systematic review |
| url | http://www.sciencedirect.com/science/article/pii/S2451865423001564 |
| work_keys_str_mv | AT hirotakamano applicationofsamplesizereestimationinclinicaltrialsasystematicreview AT yujitanaka applicationofsamplesizereestimationinclinicaltrialsasystematicreview AT shunichiroorihara applicationofsamplesizereestimationinclinicaltrialsasystematicreview AT junjimoriya applicationofsamplesizereestimationinclinicaltrialsasystematicreview |