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Article: Sample size re-estimation in adaptive enrichment design
Title | Sample size re-estimation in adaptive enrichment design |
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Authors | |
Keywords | Conditional power Enrichment strategies Patient heterogeneity Phase III trial designs Sample size re-estimation |
Issue Date | 2021 |
Publisher | Elsevier Inc. The Journal's web site is located at http://www.elsevier.com/locate/conclintrial |
Citation | Contemporary Clinical Trials, 2021, v. 100, article no. 106216 How to Cite? |
Abstract | Clinical trial participants are often heterogeneous, which is a fundamental problem in the rapidly developing field of precision medicine. Participants heterogeneity causes considerable difficulty in the current phase III trial designs. Adaptive enrichment designs provide a flexible and intuitive solution. At the interim analysis, we enrich the subgroup of trial participants who have a higher likelihood to benefit from the new treatment. However, it is critical to control the level of the test size and maintain adequate power after enrichment of certain subgroup of participants. We develop two adaptive enrichment strategies with sample size re-estimation and verify their feasibility and practicability through extensive simulations and sensitivity analyses. The simulation studies show that the proposed methods can control the overall type I error rate and exhibit competitive improvement in terms of statistical power and expected sample size. The proposed designs are exemplified with a real trial application. |
Persistent Identifier | http://hdl.handle.net/10722/294866 |
ISSN | 2023 Impact Factor: 2.0 2023 SCImago Journal Rankings: 0.980 |
ISI Accession Number ID |
DC Field | Value | Language |
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dc.contributor.author | Lin, R | - |
dc.contributor.author | Yang, Z | - |
dc.contributor.author | Yuan, Y | - |
dc.contributor.author | Yin, G | - |
dc.date.accessioned | 2020-12-21T11:49:40Z | - |
dc.date.available | 2020-12-21T11:49:40Z | - |
dc.date.issued | 2021 | - |
dc.identifier.citation | Contemporary Clinical Trials, 2021, v. 100, article no. 106216 | - |
dc.identifier.issn | 1551-7144 | - |
dc.identifier.uri | http://hdl.handle.net/10722/294866 | - |
dc.description.abstract | Clinical trial participants are often heterogeneous, which is a fundamental problem in the rapidly developing field of precision medicine. Participants heterogeneity causes considerable difficulty in the current phase III trial designs. Adaptive enrichment designs provide a flexible and intuitive solution. At the interim analysis, we enrich the subgroup of trial participants who have a higher likelihood to benefit from the new treatment. However, it is critical to control the level of the test size and maintain adequate power after enrichment of certain subgroup of participants. We develop two adaptive enrichment strategies with sample size re-estimation and verify their feasibility and practicability through extensive simulations and sensitivity analyses. The simulation studies show that the proposed methods can control the overall type I error rate and exhibit competitive improvement in terms of statistical power and expected sample size. The proposed designs are exemplified with a real trial application. | - |
dc.language | eng | - |
dc.publisher | Elsevier Inc. The Journal's web site is located at http://www.elsevier.com/locate/conclintrial | - |
dc.relation.ispartof | Contemporary Clinical Trials | - |
dc.rights | This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License. | - |
dc.subject | Conditional power | - |
dc.subject | Enrichment strategies | - |
dc.subject | Patient heterogeneity | - |
dc.subject | Phase III trial designs | - |
dc.subject | Sample size re-estimation | - |
dc.title | Sample size re-estimation in adaptive enrichment design | - |
dc.type | Article | - |
dc.identifier.email | Yin, G: gyin@hku.hk | - |
dc.identifier.authority | Yin, G=rp00831 | - |
dc.description.nature | postprint | - |
dc.identifier.doi | 10.1016/j.cct.2020.106216 | - |
dc.identifier.scopus | eid_2-s2.0-85097746568 | - |
dc.identifier.hkuros | 320604 | - |
dc.identifier.volume | 100 | - |
dc.identifier.spage | 106216 | - |
dc.identifier.epage | 106216 | - |
dc.identifier.isi | WOS:000756199400005 | - |
dc.publisher.place | United States | - |
dc.identifier.issnl | 1551-7144 | - |