Importance: To date, the benefit of genome-driven cancer therapy has not been quantified.
Objective: We sought to estimate the annual percentage of patients in the United States with advanced or metastatic cancer who could be eligible for and benefit from US Food and Drug Administration (FDA)-approved genome-driven therapy from 2006 to 2018.
Design, setting, and participants: Retrospective cross-sectional study using publically available data of (1) demographic characteristics of patients with advanced or metastatic cancer; (2) FDA data on cancer drugs approved from January 2006 through January 2018; (3) measures of response and duration of response from drug labels; and (4) published reports estimating the frequency of various genomic aberrations used to estimate what percentage of patients would have been eligible for and would have benefited from genome-driven therapy during the studied period.
Main outcomes and measures: Estimated percentage of US patients with cancer eligible for and benefiting from genome-targeted and genome-informed therapy by year, response rate of genome-informed indications, and duration of response.
Results: A total of 31 drugs with 38 FDA-approved indications met our inclusion criteria for genome-targeted or genome-informed therapy from January 1, 2006, through January 31, 2018. The estimated number of patients eligible for genome-targeted therapy in 2006 was 28 729 of a total 564 830 patients with metastatic cancer, or 5.09% (95% CI, 5.03%-5.14%). By 2018, this number had increased to 50 811 of 609 640, or 8.33% (95% CI, 8.26%-8.40%). For genome-informed therapy in 2006, the eligible number of patients was 59 301 of 564 830, or 10.50% (95% CI, 10.42%-10.58%). In 2018, genome-informed treatment could be offered to 94 157 of 609 640, or 15.44% (95% CI, 15.35%-15.53%) of patients with metastatic cancer. The percentage of patients with cancer estimated to benefit from genome-targeted therapy in 2006 was 0.70% (95% CI, 0.68%-0.72%), and in 2018, it had increased to 4.90% (95% CI, 4.85%-4.95%). For genome-informed treatment in 2006, the percentage estimated to benefit was 1.31% (95% CI, 1.28%-1.34%), and in 2018, it had increased to 6.62% (95% CI, 6.56%-6.68%). The median overall response rate for all genome-informed drugs through January 2018 was 54%, and the median duration of response was 29.5 months.
Conclusions and relevance: Although the number of patients eligible for genome-driven treatment has increased over time, these drugs have helped a minority of patients with advanced cancer. To accelerate progress in precision oncology, novel trial designs of genomic therapies should be developed, and broad portfolios of drug development, including immunotherapeutic and cytotoxic approaches, should be pursued.