Multi-Center Phase III Clinical Trials and NCI Cooperative Groups: Workshop Summary (2009)

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Glossary Accrual—the enrollment of qualified patients into clinical trials. Accrue—to enroll qualified patients into clinical trials. Adjuvant therapy—medical treatment given in addition to a primary treatment. In the case of cancer, this can be chemotherapy, radiation, or hormone therapy given in addition to surgical removal of a tumor, for example. Adjuvant therapies are used to enhance the effect of primary treatment, and would not necessarily be expected to have therapeutic effect in the absence of the primary treatment. Adverse event—any negative or unwanted effect from a drug, device, or medical test. Bevacizumab (Avastin)—a monoclonal antibody drug used to treat metastatic cancer of the colon or rectum, usually in combination with 5-fluorouracil–based chemotherapy. Bevacizumab is also used in the treat- ment of advanced, recurrent, or metastatic non-squamous non-small cell lung cancer, in combination with carboplatin and paclitaxel, or other cancer drugs, and metastatic HER2-negative breast cancer, in combination with paclitaxel. Biospecimen bank—a facility that collects, catalogs, and stores samples of biological materials (such as urine, blood, tissue, cells, DNA, RNA, and protein) used for laboratory research. 105 

106 MULTI-CENTER PHASE III CLINICAL TRIALS Biostatistics—the use of statistics to analyze biological or health science data. Cetuximab (Erbitux)—a monoclonal antibody drug used to treat advanced or metastatic cancer of the colon and rectum, usually in combination with chemotherapy or irinotecan, another cancer drug. It is currently being used in research trials for treatment of head and neck cancers. Contract research organization—an organization that offers a range of clinical trial–related services, including development of protocols, patient recruitment, collection and analysis of data, and preparation of regulatory documents. Cooperative agreement—an administrative and funding instrument uti- lized by federal agencies to provide assistance to award recipients. Unlike grants, cooperative agreements are utilized when substantial governmen- tal involvement is expected. Cooperative group—the collection of researchers, cancer centers, aca- demic medical centers, community hospitals, private research institutions, and community physicians who organize to design and implement clini- cal trials to study new cancer treatments, methods of cancer prevention and early detection, and quality of life issues. The cooperative groups are administered by the NCI, and are organized around specific diseases, treatment modalities, or geography. Employer self-insured plan—a health plan in which the employer assumes the financial risk of providing health care benefits to its employees. Erlotinib (Tarceva)—a drug used to treat locally advanced or metastatic non-small cell lung cancer and other cancers. Like gefitinib, it targets epidermal growth factor receptor tyrosine kinase, and specific genetic mutations correlate to patients’ response to the drug. Gefitinib (Iressa)—a drug used to treat locally advanced or non-small cell lung cancer and other cancers. Like erlotinib, it also targets epidermal growth factor receptor tyrosine kinase, and specific genetic mutations cor- relate to patients’ response to the drug. Grade 1 toxicities—mild adverse events. Grade 2 toxicities—moderate adverse events. 

GLOSSARY 107 Grade 3 toxicities—severe adverse events. Grade 4 toxicities—life-threatening or disabling adverse events. HCPCS—The HCPCS (Healthcare Common Procedure Coding System) is a standardized coding system that identifies products, supplies, and services in order to bill payors (such as CMS or insurance companies). HCPCS modifier—An HCPCS code descriptor utilized to provide addi- tional information regarding the service or item identified by the HCPCS code, including specific circumstances that may apply to the service or item. Investigational New Drug (IND)—A new molecular, antibiotic, or bio- logical drug that is used in a clinical investigation. It also includes a biological product used in vitro for diagnostic purposes. J-Code—An HCPCS code used to bill payors (such as CMS or insurance companies) for drugs. A J-code, as opposed to another letter code (i.e., A-code or B-code), generally signifies an injectable drug that cannot be self-administered. Lean process—a process improvement strategy designed to optimize workflow, reduce waste, and streamline business processes. Medical informatics—an integrative discipline concerned with the acquisition, storage, and use of information in the health and biomedical domain. New Drug Application (NDA)—FDA process to approve new pharma- ceuticals for sale and marketing in the United States based on efficacy and safety. Pathology quality assurance—a system of quality control activities that promote consistency and accuracy across collection, analysis, and clas- sification procedures in pathology. Phase I trial—a clinical trial in a small number of patients in which the toxicity and dosing of an intervention are assessed. Phase II trial—a clinical trial in which the safety and preliminary efficacy of an intervention are assessed. 

108 MULTI-CENTER PHASE III CLINICAL TRIALS Phase III trial—a large-scale clinical trial in which the safety and efficacy of an intervention are assessed in a large number 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. Phase IV trial—a large-scale trial undertaken after FDA approval for safety surveillance to detect rare or long-term adverse events. Also known as a post-marketing surveillance trial. Process map—a visual representation of a workflow comprising a stream of activities that transforms a well-defined input or set of inputs into a pre-defined set of outputs. Protocol—a study plan on which a clinical trial is based. The plan is designed to safeguard the health of participants as well as answer spe- cific research questions. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. 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. Serious adverse event (SAE)—an untoward medical occurrence that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability or incapacity, or is a congenital anomaly or birth defect. Special Protocol Assessment—An industry-initiated 45-day review pro- cess in which the FDA evaluates a clinical trial protocol before the trial is begun. The purpose of the special protocol assessment is to determine whether the clinical trial protocol will sufficiently address scientific and regulatory requirements for the planned new drug application (NDA) or biologic license application (BLA). Tamoxifen—a drug used to treat certain types of breast cancer, and to prevent breast cancer in women who are at high risk of developing breast cancer. Tamoxifen is an antiestrogen, blocking the effects of the estrogen hormone. Targeted therapy—a type of treatment that uses drugs or other substances (such as monoclonal antibodies) to identify and attack cancer cells with- 

GLOSSARY 109 out harming normal cells. Targeted therapy may be less harmful to nor- mal cells than other types of cancer treatments. Translational research—the translation of novel findings obtained from scientific medical research into testable hypotheses for evaluation in clini- cal trials in human subjects. Value-stream mapping—a lean process visualization technique used to analyze the flow of materials and information through a system. The goal of value-stream mapping is to understand and streamline the work pro- cesses by reducing waste, or activities that do not add value.