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Policy Issues in the Development of Personalized Medicine in Oncology: Workshop Summary Glossary Allele – any one of a series of two or more different genes that occupy the same position (locus) on a chromosome. Analytical validity – the accuracy of a test in detecting the specific entity that it was designed to detect. This accuracy does not imply any clinical significance, such as diagnosis. Clinical trial – a formal study carried out according to a prospectively defined protocol that is intended to discover or verify the safety and effectiveness of procedures or interventions in humans. Clinical utility – the clinical and psychological benefits and risks of positive and negative results of a given technique or test. Clinical validity – the accuracy of a test for a specific clinical purpose, such as diagnosing or predicting risk for a disorder. Companion Diagnostic Test – in this report, companion diagnostic tests include tests that are predictive of a therapeutic response that have gone through the FDA approval process.
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Policy Issues in the Development of Personalized Medicine in Oncology: Workshop Summary Diagnostic – the investigative tools and techniques used in biological studies or to identify or determine the presence of a disease or other condition. Epidermal growth factor receptor (EGFR) – a receptor that is overproduced in several solid tumors, including breast and lung cancers. Its overproduction is linked to a poorer prognosis because it enables cell proliferation, migration, and the development of blood vessels. Several new drugs recently approved by the Food and Drug Administration specifically target EGFR. Genomics – the study of all of the nucleotide sequences, including structural genes, regulatory sequences, and noncoding DNA segments, in the chromosomes of an organism or tissue sample. One example of the application of genomics in oncology is the use of microarray or other techniques to uncover the genetic “fingerprint” of a tissue sample. This genetic fingerprint is the pattern that stems from the variable expression of different genes in normal and cancer tissues. Herceptin – see Human epidermal growth factor receptor 2. Human epidermal growth factor receptor 2 (HER2) – a growth factor receptor that is used as a breast cancer biomarker for prognosis and treatment with the drug trastuzumab (Herceptin), which targets the HER2 protein. The HER2 protein is overexpressed in approximately 25 percent of breast cancer patients due to amplification of the gene. Laboratory-Developed Tests – in this report, laboratory-developed tests include tests that are predictive of a therapeutic response that have not gone through the FDA approval process. The laboratories that provide these tests are, however, subject to oversight by CMS under CLIA. Off-label use – using a drug that either has not been approved by the Food and Drug Administration or has not been approved for the purpose for which it is being used. Phase I trial – clinical trial in a small number of patients in which the toxicity and dosing of an intervention are assessed.
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Policy Issues in the Development of Personalized Medicine in Oncology: Workshop Summary Phase II trial – clinical trial in which the safety and preliminary efficacy of an intervention are assessed in patients. Phase III trial – large-scale clinical trial in which the safety and efficacy of an intervention are assessed in large numbers of patients. The Food and Drug Administration generally requires new drugs to be tested in Phase III trials before they can be put on the market. Predictive tests – in this report, tests that are predictive of a therapeutic response are referred to as “predictive tests.” Premarket approval – a Food and Drug Administration approval for a new test or device that enables it to be marketed for clinical use. To receive this approval, the manufacturer of the product must submit clinical data showing the product is safe and effective for its intended use. Premarket notification or 510(k) – a Food and Drug Administration review process that enables a new test or device to be marketed for clinical use. This review process requires manufacturers to submit data showing the accuracy and precision of their product, as well as, in some cases, its analytical sensitivity and specificity. Manufacturers also have to provide documentation supporting the claim that their product is substantially equivalent to one already on the market. This review does not typically consider the clinical safety and effectiveness of the product. Proficiency testing – laboratories performing non-waived tests must enroll laboratory personnel in tests specific to the subspecialty relevant to the tests they will be evaluating. The Clinical Laboratory Improvement Amendments require proficiency testing of personnel at least once every 2 years. Proteomics – the study of the structure, function, and interactions of the proteins produced by the genes of a particular cell, tissue, or organism. The application of proteomics in oncology may involve mass spectroscopy, two-dimensional polyacrylamide gel electrophoresis, protein chips, and other techniques to uncover the protein “fingerprint” of a tissue sample. This protein fingerprint is the pattern that stems from the various amounts and types of all the proteins in the sample.
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Policy Issues in the Development of Personalized Medicine in Oncology: Workshop Summary Qualification – the evidentiary process of linking an assay with biological and clinical endpoints that is dependent on the intended application. Ras gene – a gene encoding for a signal transduction protein that has been found to cause cancer when the gene is altered (mutated). Agents that block its activity may stop the growth of cancer. Trastuzumab – see Human epidermal growth factor receptor 2. Validation – the process of assessing the assay or measurement performance characteristics.