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Jaki, Thomas ; Burdon, Abigail ; Chen, Xijin ; Mozgunov, Pavel ; Zheng, Haiyan ; Baird, Richard

Early phase clinical trials in oncology: Realising the potential of seamless designs

Article

Jaki, Thomas , Burdon, Abigail , Chen, Xijin , Mozgunov, Pavel, Zheng, Haiyan and Baird, Richard (2023) Early phase clinical trials in oncology: Realising the potential of seamless designs. European Journal of Cancer 189, p. 112916.

DOI to cite this document: 10.5283/epub.75717


Abstract

Background: The pharmaceutical industry's productivity has been declining over the last two decades and high attrition rates and reduced regulatory approvals are being seen. The development of oncology drugs is particularly challenging with low rates of approval for novel treatments when compared with other therapeutic areas. Reliably establishing the potential of novel treatment and the ...

Background: The pharmaceutical industry's productivity has been declining over the last two decades and high attrition rates and reduced regulatory approvals are being seen. The development of oncology drugs is particularly challenging with low rates of approval for novel treatments when compared with other therapeutic areas. Reliably establishing the potential of novel treatment and the corresponding optimal dosage is a key component to ensure efficient overall development. A growing interest lies in terminating developments of poor treatments quickly while enabling accelerated development for highly promising interventions. Methods: One approach to reliably establish the optimal dosage and the potential of a novel treatment and thereby improve efficiency in the drug development pathway is the use of novel statistical designs that make efficient use of the data collected. Results: In this paper, we discuss different (seamless) strategies for early oncology development and illustrate their strengths and weaknesses through real trial examples. We provide some directions for good practices in early oncology development, discuss frequently seen missed opportunities for improved efficiency and some future opportunities that have yet to fully develop their potential in early oncology treatment development. Discussion: Modern methods for dose-finding have the potential to shorten and improve dose-finding and only small changes to current approaches are required to realise this potential. (c) 2023 The Author(s). Published by Elsevier Ltd. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).



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Details

Item typeArticle
Journal or Publication TitleEuropean Journal of Cancer
PublisherElsevier
Open Access TypeCC-License
Place of PublicationOXFORD
Volume189
Page Rangep. 112916
Date13 May 2023
Date of publication18 Mar 2025 10:04
InstitutionsInformatics and Data Science
Identification Number
ValueType
10.1016/j.ejca.2023.05.005DOI
KeywordsCONTINUAL REASSESSMENT METHOD; POSTMENOPAUSAL WOMEN; PRACTICAL DESIGN; FULVESTRANT; CANCER; DRUG; ANASTROZOLE; ATTRITION; EFFICACY; Efficient trials; Model-based designs; Dose-determination
Dewey Decimal Classification000 Computer science, information & general works > 004 Computer science
StatusPublished
RefereedYes, this version has been refereed
Created at the University of RegensburgPartially
URN of the UB Regensburgurn:nbn:de:bvb:355-epub-757172
Item ID75717

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