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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 type | Article | ||||
| Journal or Publication Title | Journal 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 | ||||
| Date | 18 July 2022 | ||||
| Institutions | Informatics and Data Science > Department Machine Learning & Data Science > Lehrstuhl für Computational Statistics (Prof. Dr. Thomas Jaki) | ||||
| Identification Number |
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| Keywords | Biomarker, drug development, precision oncology, sequence of trials | ||||
| Dewey Decimal Classification | 000 Computer science, information & general works > 004 Computer science | ||||
| Status | Published | ||||
| Refereed | Yes, this version has been refereed | ||||
| Created at the University of Regensburg | Partially | ||||
| URN of the UB Regensburg | urn:nbn:de:bvb:355-epub-778538 | ||||
| Item ID | 77853 |
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