Abstract
Prior studies have evaluated the percentage of cancer patients with advanced or metastatic cancer who are eligible for and respond to genome-targeted therapy, but since that publication, the number of Food and Drug Administration (FDA) approvals for drugs targeting genetic indications has grown rapidly. We sought to update the estimates of both eligibility for and response to genome-targeted and genome-informed therapies in US cancer patients for FDA-approved drugs to reflect estimates as of 2020. We used mortality data from the American Cancer Society to estimate eligibility for these drugs, based on prevalence statistics from the published literature. We then multiplied eligibility by the response rate in the FDA label to generate an estimate for the percentage of US cancer patients who respond. For genome-targeted therapy, we estimate that the eligibility increased from 5.13% in 2006 to 13.60% in 2020. For genome-targeted therapy, we estimate that the response increased from 2.73% in 2006 to 7.04% in2020. The percentage of US cancer patients who are eligible for and respond to genome-targeted therapy has increased over time. Most of the increase in eligibility for genome-targeted therapies was seen after 2018, whereas most of the increase in response was seen before2018.
Highlights
Precision oncology relies upon genomic sequencing of a patient’s tumor to determine optimal treatment.[1]
For genome-informed therapy, we estimate that the eligibility was 10.70% in 2006, Eligibility of genomically targeted therapy
For genome-targeted therapy, we estimate that the response was 2.73% in 2006, increased to 5.48% by the end of 2018, and has since increased to 7.04% in 2020 (Figure 3)
Summary
Precision oncology relies upon genomic sequencing of a patient’s tumor to determine optimal treatment.[1]. Genomically-targeted drugs gain Food and Drug Administration (FDA) approval in single-arm trials that lack a comparator group.[2] 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.[3]. Prior studies have evaluated the percentage of cancer patients with advanced or metastatic cancer who are eligible for and respond to genome-targeted therapy, but since that publication, the number of Food and Drug Administration (FDA) approvals for drugs targeting genetic indications has grown rapidly. We sought to update the estimates of both eligibility for and response to genome-targeted and genome-informed therapies in US cancer patients for FDA-approved drugs to reflect estimates as of 2020. Conclusions: The percentage of US cancer patients who are eligible for and respond to genome-targeted therapy has increased over time.
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