What is the expected benefit of patient-centric clinical development in oncology?. Issue 3 (4th May 2022)

• Record Type: Journal Article
• Authors: Serra, Alessandra; Mozgunov, Pavel; Jaki, Thomas; Rigat, Fabio
• Is Part Of: Journal of biopharmaceutical statistics. Volume 32:Issue 3(2022)
• Page Start: 414
• Page End: 426
• Publication Date: 2022-05-04
• Subjects: Biomarker -- drug development -- precision oncology -- sequence of trials; Pharmacy -- Statistical methods -- Periodicals; Drugs -- Testing -- Statistical methods -- Periodicals; Biometry -- Periodicals; Biopharmaceutics -- Periodicals; Pharmacokinetics -- Periodicals; 615.19
• DOI: 10.1080/10543406.2022.2065506
• Languages: English
• ISSNs: 1054-3406

Abstract

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 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.