Not discussed are FDA approvals that expand the use of an anticancer therapeutic previously approved for a given cancer type to include treatment with that therapeutic at different timepoints during the course of clinical care or treatment of a different subtype of the same cancer. For example, the August 2022 FDA approval expanded the use of fam-trastuzumab-deruxtecan-nxki (Enhertu), for the treatment of patients with metastatic HER2-low breast cancer that is not removable by surgery. This expansion occurred nearly three years after the molecularly targeted therapeutic was first approved for treating metastatic HER2-positive breast cancer and was based on results from a phase III clinical trial. The study showed that patients with metastatic breast cancer who were treated with fam-trastuzumab-deruxtecan-nxki lived nearly twice as long without their cancer progressing and lived six months longer overall than those treated with standard chemotherapy (352). Fam-trastuzumab-deruxtecan-nxki is the first treatment approved for patients with HER2-low breast cancer subtype, a newly defined subset of HER2-negative breast cancer. New medical products used across the continuum of clinical cancer care transform lives by improving survival and quality of life. However, not all patients receive the standard of care recommended for the type of cancer with which they have been diagnosed and the stage of cancer at the time of diagnosis (see Sidebar 30, p. 78). Thus, it is imperative that all stakeholders committed to driving progress against cancer work together to address the challenge of disparities in cancer treatment because these can be associated with adverse differences in survival. Recent studies have shown that disparities in survival for prostate cancer or multiple myeloma between Black patients and White patients can be eliminated when both population groups have equivalent access to care and to standard treatments (13). Mastering Clinical Trial Design Recent advances in our understanding of cancer biology have led to novel ways of designing and conducting clinical trials. Notably, cancer clinical trials more frequently utilize novel designs compared to trials in other disease areas. For example, a recent study reported that 27 percent of cancer clinical trials worldwide used these mechanisms in 2022 compared to only 6 percent in all other disease areas (332). One of the new approaches is to use the master protocol to answer multiple questions within a single overall clinical trial. Basket trials are one type of master protocol clinical trial. In the basket trial depicted here, one drug is being tested against a particular genetic mutation (green dots) across liver, lung, colon, and stomach cancers. This approach allows the clinical testing of new anticancer therapeutics to be streamlined because the therapeutic is matched with the right patients at the start of the trial. This precision approach reduces the number of patients who need to be enrolled in the trial and decreases the length of time it takes for a new anticancer therapeutic to be tested and made available to patients if the trial shows it is safe and effective. FIGURE 14 Screen tumors for the mutation that matches the therapeutic being tested Patients without the mutation do not qualify for the study Patients with the mutation receive the matching therapeutic BASKET TRIALS Advancing the Frontiers of Cancer Science and Medicine AACR Cancer Progress Report 2023 73
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