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Published on: April 11, 2016
Quantitation of Targetable Somatic Mutations Among Patients Evaluated by a Personalized Medicine Clinical Service:
Cory M Vela1, Todd C Knepper1, Nancy K Gillis1
1Division of Population Science, DeBartolo Family Personalized Medicine Institute, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.
Introduction:
Moffitt Cancer Center's Personalized Medicine Clinical Service (PMCS) reviews somatic next-generation sequencing (NGS) assay results, provides interpretations, and identifies potential therapeutic options. The number of individuals reviewed by our clinical service who are eligible for on-label or off-label drug therapy based on genetic test results has previously not been quantitated. We determined the number of patients harboring an actionable mutation that would qualify a patient for an on-label drug or consideration for off-label drug treatment.
Methods:
The Food and Drug Administration (FDA) Table of Pharmacogenomic Biomarkers in Drug Labeling was utilized to identify anticancer agents containing genomic markers in the Indications and Usage section of the drug label. A database containing discrete NGS patient data was queried retrospectively for those drugs and associated genomic mutations included in this study. On-label was defined as those patients who were eligible for a drug based on harboring a targetable mutation in the FDA-approved cancer type. Off-label was defined as those patients who may be considered for a drug based on harboring a targetable mutation in a non-FDA-approved cancer type.
Results:
A total of 1072 patients and 1131 NGS results were eligible for study inclusion. Fifty-two patients (4.9%) had results for more than one NGS assay. Seventeen drugs targeting ALK, BRAF, BRCA1/BRCA2, EGFR, or ERBB2 mutations met the study inclusion criteria. Of the entire patient population, 92 (8.6%) unique patients were eligible for at least one on-label drug; off-label use of at least one drug could be considered in 103 (9.6%) unique patients.
Conclusion:
Combining both on-label and off-label opportunities, 175 (16.3%) unique patients had actionable mutations in six genes. Because most patients reviewed by our PMCS have previously treated advanced disease with limited treatment options, identifying additional lines of therapy is of clinical utility.
Insights
Moffitt Cancer Center identified actionable mutations in 16.3% of patients through next-generation sequencing (NGS). This finding offers new therapeutic options for advanced cancer patients with limited treatment choices.
Area of Science:
- Oncology
- Genomics
- Personalized Medicine
Background:
- Moffitt Cancer Center's Personalized Medicine Clinical Service (PMCS) analyzes next-generation sequencing (NGS) results.
- The clinical utility of identifying actionable mutations for targeted therapies has not been previously quantified.
Purpose of the Study:
- To quantify the number of patients with actionable mutations eligible for on-label or off-label drug therapy.
- To determine the frequency of actionable mutations in specific genes relevant to cancer treatment.
Main Methods:
- Utilized the FDA Table of Pharmacogenomic Biomarkers to identify relevant anticancer agents and genomic markers.
- Retrospectively queried a database of NGS patient data to identify patients with targetable mutations.
- Defined 'on-label' as eligibility for FDA-approved drug use in the indicated cancer type and 'off-label' as potential consideration in non-FDA-approved types.
Main Results:
- 1072 patients and 1131 NGS results were analyzed.
- 17 drugs targeting ALK, BRAF, BRCA1/2, EGFR, or ERBB2 mutations met inclusion criteria.
- 92 patients (8.6%) were eligible for on-label drugs, and 103 patients (9.6%) for off-label drug consideration.
Conclusions:
- 175 unique patients (16.3%) harbored actionable mutations in six key genes.
- Identifying these mutations provides crucial additional therapeutic options for patients with advanced, previously treated disease.
- Personalized medicine approaches, guided by NGS, are vital for optimizing cancer care.
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