genome-targeted therapy
eligibility
response
Updated estimates of eligibility for and response to genome-targeted oncology drugs among US cancer patients, 2006-2020
Summary
Precision oncology relies upon genomic sequencing of a patient’s tumor to determine optimal treatment. Precision therapies typically target genetic aberrations within cancer, and this approach has widespread enthusiasm driven by high response rates. Often, genomically-targeted drugs gain Food and Drug Administration (FDA) approval in single-arm trials that lack a comparator group. As such, response rates, which measure the percentage of patients who have tumor shrinkage beyond the RECIST 1.1 cut-off of 30%, are often used as a study endpoint.
Prior studies have evaluated the percentage of US cancer patients with advanced or metastatic cancer who are eligible for and respond to this class of medications. Specifically, genome-targeted therapy was found to apply to 8.3% of US cancer patients as of 2018, and 4.9% might experience a partial or complete response. However, since that publication, the number of FDA approvals for drugs targeting genetic indications has grown rapidly. We therefore sought to update the estimates of both eligibility for and response to genome-targeted and genome-informed therapies for drugs that have been FDA-approved to reflect estimates as of 2020.
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genome-targeted therapy, eligibility, response
