File Download

There are no files associated with this item.

  Links for fulltext
     (May Require Subscription)
Supplementary

Article: Bayesian enhancement two‐stage design with error control for phase II clinical trials

TitleBayesian enhancement two‐stage design with error control for phase II clinical trials
Authors
KeywordsBET design
binary endpoint
cancer
phase II trial
posterior error rate
Issue Date2020
PublisherJohn Wiley & Sons Ltd. The Journal's web site is located at http://www.interscience.wiley.com/jpages/0277-6715/
Citation
Statistics in Medicine, 2020, v. 39 n. 29, p. 4452-4465 How to Cite?
AbstractPhase II clinical trials make a critical decision of go or no‐go to a subsequent phase III studies. A considerable proportion of promising drugs identified in phase II trials fail the confirmative efficacy test in phase III. Recognizing the low posterior probabilities of H1 when accepting the drug under Simon's two‐stage design, the Bayesian enhancement two‐stage (BET) design is proposed to strengthen the passing criterion. Under the BET design, the lengths of highest posterior density (HPD) intervals, posterior probabilities of H0 and H1 are computed to calibrate the design parameters, aiming to improve the stability of the trial characteristics and strengthen the evidence for proceeding the drug development forward. However, from a practical perspective, the HPD interval length lacks transparency and interpretability. To circumvent this problem, we propose the BET design with error control (BETEC) by replacing the HPD interval length with the posterior error rate. The BETEC design can achieve a balance between the posterior false positive rate and false negative rate and, more importantly, it has an intuitive and clear interpretation. We compare our method with the BET design and Simon's design through extensive simulation studies. As an illustration, we further apply BETEC to two recent clinical trials, and investigate its performance in comparison with other competitive designs. Being both efficient and intuitive, the BETEC design can serve as an alternative toolbox for implementing phase II single‐arm trials.
Persistent Identifierhttp://hdl.handle.net/10722/288175
ISSN
2019 Impact Factor: 1.783
2015 SCImago Journal Rankings: 1.811
ISI Accession Number ID

 

DC FieldValueLanguage
dc.contributor.authorJIN, H-
dc.contributor.authorYin, G-
dc.date.accessioned2020-10-05T12:08:58Z-
dc.date.available2020-10-05T12:08:58Z-
dc.date.issued2020-
dc.identifier.citationStatistics in Medicine, 2020, v. 39 n. 29, p. 4452-4465-
dc.identifier.issn0277-6715-
dc.identifier.urihttp://hdl.handle.net/10722/288175-
dc.description.abstractPhase II clinical trials make a critical decision of go or no‐go to a subsequent phase III studies. A considerable proportion of promising drugs identified in phase II trials fail the confirmative efficacy test in phase III. Recognizing the low posterior probabilities of H1 when accepting the drug under Simon's two‐stage design, the Bayesian enhancement two‐stage (BET) design is proposed to strengthen the passing criterion. Under the BET design, the lengths of highest posterior density (HPD) intervals, posterior probabilities of H0 and H1 are computed to calibrate the design parameters, aiming to improve the stability of the trial characteristics and strengthen the evidence for proceeding the drug development forward. However, from a practical perspective, the HPD interval length lacks transparency and interpretability. To circumvent this problem, we propose the BET design with error control (BETEC) by replacing the HPD interval length with the posterior error rate. The BETEC design can achieve a balance between the posterior false positive rate and false negative rate and, more importantly, it has an intuitive and clear interpretation. We compare our method with the BET design and Simon's design through extensive simulation studies. As an illustration, we further apply BETEC to two recent clinical trials, and investigate its performance in comparison with other competitive designs. Being both efficient and intuitive, the BETEC design can serve as an alternative toolbox for implementing phase II single‐arm trials.-
dc.languageeng-
dc.publisherJohn Wiley & Sons Ltd. The Journal's web site is located at http://www.interscience.wiley.com/jpages/0277-6715/-
dc.relation.ispartofStatistics in Medicine-
dc.rightsPreprint This is the pre-peer reviewed version of the following article: [FULL CITE], which has been published in final form at [Link to final article using the DOI]. This article may be used for non-commercial purposes in accordance with Wiley Terms and Conditions for Use of Self-Archived Versions. Postprint This is the peer reviewed version of the following article: [FULL CITE], which has been published in final form at [Link to final article using the DOI]. This article may be used for non-commercial purposes in accordance with Wiley Terms and Conditions for Use of Self-Archived Versions.-
dc.subjectBET design-
dc.subjectbinary endpoint-
dc.subjectcancer-
dc.subjectphase II trial-
dc.subjectposterior error rate-
dc.titleBayesian enhancement two‐stage design with error control for phase II clinical trials-
dc.typeArticle-
dc.identifier.emailYin, G: gyin@hku.hk-
dc.identifier.authorityYin, G=rp00831-
dc.description.naturelink_to_subscribed_fulltext-
dc.identifier.doi10.1002/sim.8734-
dc.identifier.pmid32854163-
dc.identifier.scopuseid_2-s2.0-85089857869-
dc.identifier.hkuros315635-
dc.identifier.volume39-
dc.identifier.issue29-
dc.identifier.spage4452-
dc.identifier.epage4465-
dc.identifier.eissn1097-0258-
dc.identifier.isiWOS:000562928500001-
dc.publisher.placeUnited Kingdom-
dc.identifier.issnl0277-6715-

Export via OAI-PMH Interface in XML Formats


OR


Export to Other Non-XML Formats