Ruxolitinib in CMML: A Case Study of Innovative Trial Design in a Rare Cancer.

Shastri, Aditi; Adrianzen-Herrera, Diego A · Clin Cancer Res · 2021

editorial · Level V

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Abstract

Alternative statistical designs cannot fully mitigate the limitations of traditional clinical trials in rare cancers. Creative study designs that integrate early clinical data and correlative outcomes from concomitant translational and laboratory models to evaluate the efficacy of druggable targets can potentially expedite access to novel therapies for these patients.<i>See related article by Hunter et al., p. 6095</i>.

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