Updated estimates of eligibility for and response to genome-targeted oncology drugs among US cancer patients, 2006-2020

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. Materials and methods: 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. Results: 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% in 2020. Conclusions: 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 before 2018.

Drug mechanism enrichment analysis improves prioritization of therapeutics for repurposing

“…Molecularly targeted therapeutics have revolutionized cancer therapy. However, because as many as 85% of cancer patients are currently ineligible for targeted therapies (Haslam et al, 2021), there remains an urgent need to match patients with effective drugs.…”

Actionable Molecular Alterations Are Revealed in Majority of Advanced Non-Small Cell Lung Cancer Patients by Genomic Tumor Profiling at Progression after First Line Treatment

“…The responses to targeted therapy has increased from approximately 3% in 2006 to 7% in 2020 [ 4 ], which gives hope to the field of precision medicine. Precision medicine trials reports of only 8–27% actually receiving targeted treatment [ 9 , 10 , 11 , 12 , 13 , 14 , 15 , 16 , 17 , 18 , 19 ] and response rates of 0.8–3% [ 29 ].…”

“…Particularly in advanced Non-Small Cell Lung Cancer (NSCLC), treatment options have evolved with epidermal growth factor receptor ( EGFR ), anaplastic lymphoma kinase ( ALK )- and ROS proto-oncogene 1 ( ROS1 ) directed treatment [ 1 , 2 , 3 ]. Despite the increasing eligibility and response to genome-targeted therapy from 2006 (3%) to 2020 (7%) [ 4 ], the clinical benefit of precision medicine, beyond standard of care treatments, is still described as uncertain [ 5 ]. Real-time studies in advanced NSCLC have not yet demonstrated any improvements in survival by comprehensive molecular profiling [ 6 , 7 , 8 ], but this scenario might change due to an increasing numbers of promising targeted treatments.…”

Overall survival for oncology drugs approved for genomic indications

“…Approvals for genome-informed indications, which are often based on ORR, have been increasing in recent years. Currently, 13.6% of US cancer patients are eligible for genome targeted drugs and 7% of cancer patients may respond [3]. However, it is unknown what percentage of these drugs have proven OS gains for these indications and what the magnitude of those differences are.…”