Glossary

Accelerated Approval—the process by which the FDA rapidly approves experimental treatments for serious or life-threatening conditions, often based on data using surrogate endpoints.

Advance Care Planning—involves taking the time to learn and discuss end-of-life care options and services before a health crisis, then making choices based on a person’s priorities, beliefs and values and sharing his or her wishes in writing through an advance directive.

Anatomical Therapeutic Classification (ATC)—used for the classification of drugs, this system divides drugs into different groups according to the organ or system on which they act and/or their therapeutic and chemical characteristics.

Bevacizumab (Avastin)—a monoclonal antibody drug used to treat meta-static cancer of the colon and rectum, usually in combination with 5-fluorouracil-basd chemotherapy. Bevacizumab is also used in the treatment 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.

Biomarker—a biochemical substance found in blood, other body fluids, or tissues used as an indicator of a biologic state that is objectively measured and evaluated as an indicator of normal biologic processes, pathogenic processes, or a response to therapeutic intervention.



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Glossary Accelerated Approval—the process by which the FDA rapidly approves experimental treatments for serious or life-threatening conditions, often based on data using surrogate endpoints. Advance Care Planning—involves taking the time to learn and discuss end-of-life care options and services before a health crisis, then making choices based on a person’s priorities, beliefs and values and sharing his or her wishes in writing through an advance directive. Anatomical Therapeutic Classification (ATC)—used for the classification of drugs, this system divides drugs into different groups according to the organ or system on which they act and/or their therapeutic and chemical characteristics. Bevacizumab (Avastin)—a monoclonal antibody drug used to treat meta- static cancer of the colon and rectum, usually in combination with 5-fluorouracil-basd chemotherapy. Bevacizumab is also used in the treatment 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. Biomarker—a biochemical substance found in blood, other body fluids, or tissues used as an indicator of a biologic state that is objectively measured and evaluated as an indicator of normal biologic processes, pathogenic processes, or a response to therapeutic intervention. 

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0 ASSESSINg ANd ImPROVINg VAluE IN CANCER CARE Bortezomib (Velcade)—a drug approved in the United States for treating relapsed multiple myeloma and mantle cell lymphoma. NICE recom- mended against use of bortezomib in 2006. Cancer registry—a system that monitors cancer cases that have been diag- nosed or treated in one institution or a specific geographic area. 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. Clinical endpoint—a characteristic or variable that reflects how a patient feels, functions, or survives in response to a medical intervention. Clinical practice guidelines—systematically defined statements to assist practitioner and patient decisions about appropriate health care for specific clinical circumstances. 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. Cluster randomization—the randomization of groups (or clusters) of sub- jects. May be used as a randomization strategy in clinical trials. Cohort study—an observational study in which outcomes in a group of patients that received an intervention are compared with outcomes in a similar group, that is, the cohort, either contemporary or historical, of patients that did not receive the intervention. Coinsurance—the percentage of medical care costs covered by an insured individual beyond the deductible. In many cases, coinsurance is paid by the insured individual until a predefined limit is reached, after which all costs are covered by the health care plan. Coinsurance also is used to refer to supplemental insurance used to pay the fees not covered by the primary health care plan. Coinsurance is often synonymous with co-payment. Colectomy—excision of a portion of the colon or its entirety (open vs. laparoscopic-assisted). Co-payment or co-pay—the percentage of medical care costs covered by an insured individual beyond the deductible. Co-payment is often shortened to “co-pay,” and may be synonymous with coinsurance (see Coinsurance above). Cost-effectiveness—the degree to which a service or a medical treat- ment meets a specified goal at an acceptable cost and level of quality. Cost-effectiveness analysis is a comparison of alternative interven- tions in which costs are measured in monetary units and outcomes

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 glOSSARY are measured in non-monetary units, e.g., reduced mortality or morbidity. Cost sharing—a general set of financing arrangements via deductibles, co- pays and/or coinsurance in which a person covered by the health plan must pay some of the costs to receive care. Coverage with Evidence Development (CED)—a CMS program whereby prospective data collection on a product is required for national Medi- care coverage. A product that has an insufficient evidence base for CMS coverage determination could be evaluated through CED. Do-not-resuscitate (DNR) order—a document that informs medical per- sonnel not to attempt a resuscitation in the event of a patient’s cardiac or respiratory arrest. The order is written after a discussion with the patient, family, or designated surrogate decision maker. Also called a do-not-attempt resuscitation (DNAR) order. Effectiveness—how well a treatment works in practice. Efficacy—the capacity for producing a desired result or effect under ideal conditions, for example, in a laboratory setting or within the protocol of a carefully managed randomized controlled trial. End-of-life care—the care provided to a person in their final stages of life. Erlotinib (Tarceva)—a drug used to treat locally advanced or metastatic non-small cell lung cancer and other cancers. It targets epidermal growth factor receptor tyrosine kinase, and specific genetic mutations correlate to patients’ response to the drug. FDA premarket approval—FDA approval for a new drug 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. Functional status—a measure of an individual’s ability to perform nor- mal activities of life. Encompasses a wide variety of patient-focused outcomes including physical functioning, emotional well-being, and social functioning. Health technology assessment (HTA)—the systematic evaluation of properties, effects, and/or impacts of health care technology. It may address the direct, intended consequences of technologies as well as their indirect, unintended consequences. Its main purpose is to inform technology-related policy making in health care. HTA is conducted by interdisciplinary groups using explicit analytical frameworks drawing from a variety of methods.

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 ASSESSINg ANd ImPROVINg VAluE IN CANCER CARE Hospice—a discrete site of care in the form of an inpatient hospital or nurs- ing home unit or a free-standing facility; an organization or program that provides, arranges, and advises on a wide range of medical and supportive services for dying patients and their families and friends; an approach to care for dying patients based on clinical, social, and metaphysical or spiritual principles. Hospitalist—a physician specializing in hospital inpatient care. 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 protein. Implantable cardiac defibrillator (ICD)—a surgically implanted elec- tronic device capable of sending an electric shock to the heart to stop an extremely rapid, irregular heartbeat and restore the normal heart rhythm. Intention to treat analysis—a type of analysis of clinical trial data in which all patients are included in the analysis based on their original assign- ment to intervention or control groups, regardless of whether patients failed to fully participate in the trial for any reason, including whether they actually received their allocated treatment, dropped out of the trial, or crossed over to another group. Laparoscopy or laparoscopic-assisted surgery—a type of surgical proce- dure in which a small incision is made through which a viewing tube (laparoscope) is inserted. The laparoscope contains a small camera on the eyepiece, allowing the surgeon to examine the abdominal and pelvic organs on a video monitor. Other small incisions can be made to insert instruments to perform procedures. Laparoscopy is less invasive than regular, open abdominal surgery. Large, simple trial—prospective, randomized controlled trials that use large numbers of patients, broad patient inclusion criteria, multiple study sites, minimal data requirements, and electronic registries; their purposes include detecting small and moderate treatment effects, gain- ing effectiveness data, and improving external validity. Medicare—a public health care program for individuals aged 65 and older, those who have permanent kidney failure, and people with certain disabilities. It includes Part A for coverage of hospitalization-related expenses, Part B for coverage of medical care, and Part D for prescrip- tion drug coverage. Part C, sometimes called a Medicare Advantage

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 glOSSARY Plan, is a combination of Part A and Part B but the coverage is provided through private insurance companies approved by Medicare. Metastasis—spread of cancer from its original anatomical site to one or more additional body sites. Moral hazard—the prospect that a person insulated from risk may behave differently than he or she would behave if fully exposed to the risk. Off-label use—use of a drug that either has not been approved by the FDA or has not been approved for the purpose for which it is being used. Palliative care—treatment of symptoms associated with the effects of can- cer and its treatment, with a focus on reducing pain and suffering and improving quality of life. Panitumumab (Vectibix)—a monoclonal antibody drug used to treat met- astatic colon cancer expressing the epidermal growth factor receptor. Pharmacy benefit manager (PBM)—an administrator of prescription drug programs, primarily responsible for processing and paying prescription drug claims. A PBM is also responsible for developing and maintaining the formulary, contracting with pharmacies, and negotiating discounts and rebates with drug manufacturers. Phase I trial—clinical trial in a small number of patients in which the toxicity and optimal dosing of an intervention are assessed. 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 a large number of patients (sometimes divided into Phase IIIa trials conducted before regulatory submission and Phase IIIb trials conducted after regulatory submission but before approval). Phase IV trial—postmarketing study to monitor long-term effects and provide additional information on safety and efficacy, including for different regimens and patient groups. Price elasticity—measures how much the quantity or supply of a good, or demand for it, changes if its price changes. If the percentage change in quantity is more than the percentage change in price, the good is price elastic; if it is less, the good is inelastic. Price sensitivity—the extent to which price is an important criterion in the customer’s decision-making process. Quality-adjusted life-year (QALy)—a unit of health care outcomes that adjusts gains (or losses) in years of life subsequent to a health care

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 ASSESSINg ANd ImPROVINg VAluE IN CANCER CARE intervention by the quality of life during those years. QALYs can pro- vide a common unit for comparing cost-effectiveness across different interventions and health problems. Analogous units include disability- adjusted life-years (DALYs) and healthy-years equivalents (HYEs). Randomized controlled trial (RCT)—a true prospective experiment in which investigators randomly assign an eligible sample of patients to one or more treatment groups and a control group and follow patients’ outcomes (also known as a randomized clinical trial). Relative value unit (RVU)—a comparable service measure used to permit comparison of the amounts of resources required to perform various health care services. It is determined by assigning weight to such fac- tors as personnel time, level of skill, and sophistication of equipment required to render patient services. Response rate—the percentage of patients in whom treatment results in a significant change in the clinical endpoint of interest. Standardized patient—an individual who is trained to act as a real patient in order to simulate a set of symptoms or problems. Statistical power—the probability of detecting a treatment effect of a given magnitude when a treatment effect of at least that magnitude truly exists. Surrogate endpoint—a biomarker that is intended to substitute for a clini- cal endpoint in a therapeutic clinical trial and is expected to predict clinical benefit (or lack thereof ) based on epidemiologic, therapeutic, pathophysiologic, or other scientific evidence. Time to tumor progression (progression-free survival)—the time inter- val from the start of treatment to cancer progression. It is a measure of the clinical benefit from therapy. Trastuzumab (Herceptin)—see HER2. Value-based insurance design—a system of patient coinsurance based on the value—not simply the price—of health care services.