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Serra, Alessandra ; Mozgunov, Pavel ; Jaki, Thomas ; Rigat, Fabio

What is the expected benefit of patient-centric clinical development in oncology?

Serra, Alessandra, Mozgunov, Pavel, Jaki, Thomas and Rigat, Fabio (2022) What is the expected benefit of patient-centric clinical development in oncology? Journal of Biopharmaceutical Statistics 32 (3), pp. 414-426.

Date of publication of this fulltext: 29 Sep 2025 11:38
Article
DOI to cite this document: 10.5283/epub.77853


Abstract

The identification and quantification of predictive biomarkers characterize personalized medicine approaches and patient-centric clinical development. In practice, the sponsor needs evaluating whether biomarker-informed clinical development strategies are more likely to benefit current and future patients. To this end, a simple metric is proposed and assessed here quantifying the expected ...

The identification and quantification of predictive biomarkers characterize personalized medicine approaches and patient-centric clinical development. In practice, the sponsor needs evaluating whether biomarker-informed clinical development strategies are more likely to benefit current and future patients. To this end, a simple metric is proposed and assessed here quantifying the expected clinical benefit (ECB) of clinical development programmes. Using simulation scenarios and endpoints relevant to oncology, the ECB of a simple biomarker-informed strategy is shown to be specific and sensitive. Also, the ECB difference is shown to increase in the biomarker-driven incremental efficacy and with the population prevalence of biomarker-positive study participants.



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Details

Item typeArticle
Journal or Publication TitleJournal of Biopharmaceutical Statistics
Publisher:Taylor & Francis Online
Open Access Type:CC-License
Volume:32
Number of Issue or Book Chapter:3
Page Range:pp. 414-426
Date18 July 2022
InstitutionsInformatics and Data Science > Department Machine Learning & Data Science > Lehrstuhl für Computational Statistics (Prof. Dr. Thomas Jaki)
Identification Number
ValueType
10.1080/10543406.2022.2065506DOI
KeywordsBiomarker, drug development, precision oncology, sequence of trials
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-778538
Item ID77853

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