Final published version
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Research output: Contribution to Journal/Magazine › Journal article › peer-review
Research output: Contribution to Journal/Magazine › Journal article › peer-review
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TY - JOUR
T1 - What is the expected benefit of patient-centric clinical development in oncology?
AU - Serra, Alessandra
AU - Mozgunov, Pavel
AU - Jaki, Thomas
AU - Rigat, Fabio
PY - 2022/7/31
Y1 - 2022/7/31
N2 - 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.
AB - 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.
KW - Biomarker
KW - drug development
KW - precision oncology
KW - sequence of trials
U2 - 10.1080/10543406.2022.2065506
DO - 10.1080/10543406.2022.2065506
M3 - Journal article
VL - 32
SP - 414
EP - 426
JO - Journal of Biopharmaceutical Statistics
JF - Journal of Biopharmaceutical Statistics
SN - 1054-3406
IS - 3
ER -