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 In this section:

                Clinical Trials: What Patients Need to Know

Clinical Trials: What Patients Need to Know

                        What Patients Need to Know About Institutional Review Boards
          Glossary of Terms


                        Clinical Research Versus Medical Treatment
          Informed Consent for Clinical Trials
          What Are the Different Types of Clinical Research? Home
          For Patients
          Clinical Trials: What Patients Need to Know
          Glossary of TermsClinical Trials: What Patients Need to Know Glossary of Terms
  
  	
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A B C D E F G H I J K L M N O P Q R S T U V W X Y Z

A

**Adverse Event** - drug reaction is also known as a side effect, is any undesirable experience associated with the use of a medicine in a patient. Adverse events can range from mild to severe. Serious adverse events are those that can cause disability, are life-threatening, result in hospitalization or death, or are birth defects.

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B

**Boxed Warning** - This type of warning is also commonly referred to as a “black box warning.” It appears on a prescription drug’s label and is designed to call attention to serious or life-threatening risks.

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C

**Cardiovascular disease** - also called heart disease is a class of diseases that involve the heart, the blood vessels (arteries, capillaries, and veins) or both.

**CDRH** - (Center for Devices and Radiological Health) assure that patients and providers have timely and continued access to safe, effective, and high-quality medical devices and safe radiation-emitting products.

**ClinicalTrials.Gov** - is an online registry of clinical trials that are being conducted around the world.  ClinicalTrials.gov is operated by the National Library of Medicine at the National Institutes of Health and can be accessed by anyone who has access to the internet.

**Comparison** - To learn more, researchers compare results from patients in the experimental groups with results from patients in the control groups.

**Compassionate Use** - Expanded access, also called “compassionate use,” provides a pathway for patients to gain access to investigational drugs, biologics and medical devices for serious diseases or conditions. Investigational drugs and devices have not yet been approved by the FDA and they have not been proven to be safe and effective. Therefore, they may be effective in the treatment of a condition, or they may not. It is important to remember that the drug/biologic/medical device may have unexpected serious side effects and that patients need to consider all the possible risks when seeking access to an investigational medical product.
To gain access to an investigational medical product outside of a clinical trial, the sponsors must decide whether to make their experimental medical product available to patients via expanded access.  FDA regulations specify two groups of people eligible for expanded access:

those with life-threatening diseases or conditions for which “there is a reasonable likelihood that death will occur within a matter of months or in which premature death is likely without early treatment”
	those with serious diseases or conditions that have a “substantial impact on day-to-day functioning”In most cases, patients who seek compassionate use must have exhausted all approved therapies for their condition, and be unable toenroll in a clinical trial.

**Confidentiality regarding participants** - This refers to the practice of maintaining as private all information related to clinical trial participants, including their personal identity and all personal medical information. Results from the study will usually be presented in terms of trends or overall findings and will not mention any specific participants.

**Consumer Medication Information (CMI)** - Compared to a Medication Guide,  a Consumer Medication Information sheet  gives broader  information on how to use a medicine. CMI sheets are not developed or regulated by FDA. These information sheets are prepared by pharmacies and given out with prescription drugs. CMI sheets are not available on the FDA Web site.

**Control group** - The group of participants that receives standard treatment or a placebo. The control group may also be made up of healthy volunteers. Researchers compare results from the control group with results from the experimental group to find and learn from any differences.

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D

**DailyMed** - Developed with the National Library of Medicine, DailyMed is a Web site that gives physicians and patients electronic access to FDA-approved drug labels.

**Diabetes** - is a disease in which blood glucose levels are above normal. Most of the food we eat is turned into glucose, or sugar, for our bodies to use for energy.

**Double-blind research design** - A study in which neither the participant nor the researcher knows whether the participant is in the treatment or control group.

**Double-blind, randomized, controlled clinical trial** - This is a clinical trial in which the researchers evenly divide study participants into a group receiving the experimental intervention and a group receiving standard or no treatment. Neither group knows how it has been assigned. This practice reduces the chance for a “placebo effect,” in which a treatment with no active ingredient produces results expected from a treatment with an active ingredient.

**Drugs@FDA** - Drugs@FDA is a resource allows  you  to  search for information about  FDA approved brand  name  and  generic  drugs  and therapeutic  biological products . These are proteins derived from living material (such as cells or tissues) used to treat or cure disease. You can search in many ways, including by drug name and active ingredient.

**Drug Product Recalls** - FDA provides information on drug products that have been recalled due to manufacturing problems and/or safety concerns.  In addition to information released to the public by a manufacturer using the normal media channels.

**Drug Recall** - A drug recall is an action taken by a firm to remove a product from the market that FDA considers to be in violation of the law. Recalls are classified as Class I, Class II, or Class III. Class I recalls are the most serious and involve situations where there is a reasonable probability that the use of or exposure to a volatile product, will cause serious adverse health consequences or death. A drug may be recalled due to factors such as problems with packaging, manufacturing, or contamination.

**Drug Withdrawal** - In rare cases, FDA may need to reassess and change its approval decision on a drug. A conclusion that a drug should no longer be marketed is based on the nature and frequency of the adverse events and how the drug’s benefit and risk balance compares with treatment alternatives. When FDA believes that a drug’s benefits no longer outweigh its risks, the agency will ask the manufacturer to withdraw the drug.

**Durable power of attorney** - The authority to act for another person in specified or all legal or financial matters.

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E

**Early Communication about an Ongoing Safety Review** - This type of communication is part of FDA’s effort to communicate early with the public when the agency is still evaluating data and has not reached a conclusion. FDA shares information in the interest of informing doctors and patients about the issues that are under review and when FDA experts anticipate completing their review.

**Experiment** - A study done to answer a question. Other words to describe an experiment are “research,” “study,” and “protocol.”

**Expanded Access** - also called “compassionate use,” provides a pathway for patients to gain access to investigational drugs, biologics and medical devices for serious diseases or conditions. Investigational drugs and devices have not yet been approved by the FDA and they have not been proven to be safe and effective. Therefore, they may be effective in the treatment of a condition, or they may not. It is important to remember that the drug/biologic/medical device may have unexpected serious side effects and that patients need to consider all the possible risks when seeking access to an investigational medical product.
To gain access to an investigational medical product outside of a clinical trial, the sponsors must decide whether to make their experimental medical product available to patients via expanded access.  FDA regulations specify two groups of people eligible for expanded access:

those with life-threatening diseases or conditions for which “there is a reasonable likelihood that death will occur within a matter of months or in which premature death is likely without early treatment”
	those with serious diseases or conditions that have a “substantial impact on day-to-day functioning”In most cases, patients who seek compassionate use must have exhausted all approved therapies for their condition, and be unable toenroll in a clinical trial.

**Experimental group** - The group of participants in a study that receive the experimental or study intervention (such as medication or psychotherapy).

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F

**FDA Adverse Event Reporting System (FAERS)** - is a computerized database containing reports of adverse events. It supports FDA’s post-market safety surveillance program for all approved drugs and therapeutic biologics. More information on FAERS

**FDASIA Section 907** - directed FDA to report on the extent to which demographic subgroups (sex, age, race and ethnicity) participate in clinical trials in marketing applications for drugs, biologics, and devices. This report provided an important opportunity to take a closer look at the inclusion and analysis of demographic subgroups.

**Federal Register** - abbreviated FR or sometimes Fed. Reg., is the official journal of the federal government of the United States that contains most routine publications and public notices of government agencies. It is a daily (except federal holidays) publication. The Federal Register is compiled by the Office of the Federal Register (within the National Archives and Records Administration) and is printed by the Government Printing Office. The final rules promulgated by a federal agency and published in the Federal Register are ultimately reorganized by topic or subject matter and codified in the Code of Federal Regulations (CFR), which is updated annually. There are no copyright restrictions on the Federal Register; as a work of the U.S. government, it is in the public domain.[1] Citations from the Federal Register are [volume] FR [page number] ([date]), e.g., 65 FR 741 (Jan. 6, 2000).

**Form FDA 3926** - Used by physicians when submitting requests for expanded access to investigational drugs, including emergency requests. This form is designed specifically for single patient requests only.

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H

**Healthy volunteer** - In a clinical study, a person who does not have the disorder or disease being studied. Results from healthy controls are compared to results from the group being studied.

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I

**In vitro** - In glass, as in a test tube. An in vitro test is one that is done in glass or plastic vessels in the laboratory. In vitro is the opposite of in vivo.

**In vivo** - In the living organism. For example, an experiment that is done in vivo is done in the body of a living organism. In vivo is the opposite of in vitro.

**Inclusion/exclusion criteria** - are the factors that allow someone to participate in a clinical trial. Exclusion criteria are the factors that prevent someone from participating in the trial. These factors may include a person’s illness, health history, past treatment, age, sex, or where he or she lives.

**Informed consent** - When a participant provides informed consent, it means that he or she has learned the key facts about a research study and agrees to take part in it. For more detailed information, see the “What is informed consent?” section in this guide.

**Inpatient** - A person who is hospitalized for at least one night to receive treatment or participate in a study.

**Institutional Review Board-**As defined in the Code of Federal Regulations Title 21 Part 56 means any board, committee, or other group formally designated by an institution to review, to approve the initiation of, and to conduct periodic review of, biomedical research involving human subjects. The primary purpose of such review is to assure the protection of the rights and welfare of the human subjects.

**Investigational Drug** - can also be called an experimental drug and is being studied to see if your disease or medical condition improves while taking it. Scientists are trying to prove in clinical trials:

If the drug is safe and effective.
	How the drug might be used in that disease.
	How much of the drug is needed.
	Information about the potential benefits and risks of taking the drug.back to top

L

**Letters to Health Care Professionals** - often referred to as “Dear Doctor” letters—that are developed by drug companies often with input from FDA. The letters educate healthcare professionals about new and important drug information.

M

**Medication Guides (Med Guide)** - are paper hand-outs/pamphlets that are required to be given to patients with certain medications by the pharmacist. Medication Guides communicate risk information that is specific to particular drugs and drug classes, and they contain FDA-approved information that can help patients avoid serious adverse events.

**MedWatch** - MedWatch is FDA’s safety information and adverse event reporting program. It provides important and timely medical product information to healthcare professionals, including information on prescription and over- the-counter drugs, biologics, medical devices, and special nutritional products. Healthcare professionals and consumers can also report serious problems they suspect are related to certain FDA-regulated products.

**Minimal Risk** - means that the probability and magnitude of harm or discomfort anticipated in the research are not greater in and of themselves than those ordinarily encountered in daily life or during the performance of routine physical or psychological examinations or tests.

**Molecular compound** - may be a solid, liquid or gas that has electrically neutral atoms held together by covalent chemical bonds.

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N

**National Cancer Institute** - (NCI) is part of the National Institutes of Health (NIH), which is one of 11 agencies that compose the Department of Health and Human Services (HHS). The NCI coordinates the National Cancer Program, which conducts and supports research, training, health information dissemination, and other programs with respect to the cause, diagnosis, prevention, and treatment of cancer, rehabilitation from cancer, and the continuing care of cancer patients and the families of cancer patients.

**National Institutes of Health (NIH)** - Part of the U.S. Department of Health and Human Services, NIH is the primary Federal agency for conducting and supporting medical research. NIH scientists investigate ways to prevent disease as well as the causes, treatments, and even cures for common and rare diseases. Composed of 27 Institutes and Centers, NIH provides leadership and financial support to researchers in every state and throughout the world.

**National Library of Medicine** - The world’s largest biomedical library, the National Library of Medicine (NLM) maintains and makes available a vast print collection and produces electronic information resources on a wide range of topics that are searched billions of times each year by millions of people around the globe. It also supports and conducts research, development, and training in biomedical informatics and health information technology. In addition, the Library coordinates a 6,000-member National Network of Libraries of Medicine that promotes and provides access to health information in communities across the United States.

**New Drug Approval Process** - After the animal testing stage, FDA decides whether it is reasonably safe for the company to move forward with clinical trials—studies that evaluate the safety and effectiveness of a drug in healthy people and in patients. The drug company submits the results of such studies to FDA for review. The agency conducts a thorough review of the safety and effectiveness data, and considers how the benefits compare to the risks when making a decision of whether or not to approve a drug.

**Nonprescription Drug Label (“Drug Facts”)** - For an over-the-counter (OTC), or nonprescription medicine, information printed on the medication bottle or package under the heading Drug Facts is important for taking care of yourself and your family. The Drug Facts tell you what a medicine is supposed to do, who should or should not take it, and how to use it. Safety information and instructions for use are displayed in a uniform and easy- to-read format.

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O

**"Off Label Use"** - also called unapproved use of an approved product, is when your healthcare provider uses an FDA-approved medical product for a use that has not been studied yet.

**Office of Minority Health (FDA)** - was established in 2010. OMH serves as the principal advisor to the Commissioner on minority health and health disparities. The Office provides leadership and direction in identifying agency actions that can help reduce health disparities, including the coordination of efforts across the Agency.

**Outpatient** - A person who receives treatment or participates in a study but is not hospitalized overnight.

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P

**Patient Network News** - This bi-weekly newsletter provided by the Office of Health and Constituent Affairs is intended to inform you of current FDA-related information on medical product: *approvals *labeling changes *safety warnings *ways to participate on upcoming public meetings *ways to comment on proposed regulatory guidances *other information of interest to patients and patient advocates

**Placebo** - An inactive pill. This is sometimes called a “sugar pill.” In some studies, participants may be assigned to take a placebo rather than the study medication. Ask the researcher if this is a possibility for the study that interests you.

**Placebo effect** - Sometimes people taking a study medication receive benefits that are not from the chemicals in the medicine. This is called a “placebo effect.” For example, if a participant feels hopeful about a treatment, he or she may be more likely to notice positive changes than negatives ones. A researcher’s hope may also sway a participant’s response. Double-blind research design helps minimize the placebo effect.

**Post-Market Surveillance** - is the process by which a drug’s safety is monitored on an ongoing basis after a drug is approved by FDA. Post-market surveillance looks to identify problems that were not observed or recognized before approval and any problems that may arise because a drug may not be used as described in the drug labeling, or because a drug is being manufactured incorrectly.

**Pre-Clinical Data** - Before a drug can be tested in people in the United States; sponsors (drug manufacturers, research institutions, and other organizations that develop drugs) must show FDA results of testing they have done in laboratory animals and what they propose to do for human testing.

**Prediabetes** - means the amount of glucose, also called sugar, in your blood is higher than normal but not high enough to be called diabetes. Glucose is a form of sugar your body uses for energy. Too much glucose in your blood can damage your body over time. If you have prediabetes, also called impaired fasting glucose (IFG) or impaired glucose tolerance (IGT), you are more likely to develop type 2 diabetes, heart disease, and stroke.

**Privacy Policy** - We have a strict privacy policy. FDA does not collect personally identifiable information other than your e-mail address which is needed in order to provide the service. FDA will not use or share your e-mail address for any other purpose. The GovDelivery service FDA employs to provide this e-mail subscription service is not a government entity. Information you provide may be made available to GovDelivery and other non-governmental parties.

**Protocol** - A study done to answer a question. Other words to describe a protocol are “research,” “study,” and “experiment.” “Protocol” also refers to the plan that details what researchers will do during the study.

**Public Health Advisories** - These public health advisories provide important drug safety information and recommendations of actions that can be taken by patients or caregivers to avoid or minimize harm from a drug. They are issued when FDA has information that would help doctors and patients make better treatment choices.

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R

**Randomization/random assignment** - This is the process in which researchers evenly assign study participants into a group receiving the experimental treatment being studied, and others into a group receiving standard or no treatment. Participants are assigned to a group based on chance, not choice. You have the same chance to be placed in any of the test groups.

**Regulatory Agency** - is a public authority or government agency responsible for exercising autonomous authority over some area of human activity in a regulatory or supervisory capacity. An independent regulatory agency is a regulatory agency that is independent from other branches or arms of the government. Regulatory agencies deal in the area of administrative law—regulation or rulemaking (codifying and enforcing rules and regulations and imposing supervision or oversight for the benefit of the public at large).

**Research** - A study done to answer a question. Scientists do research when they’re not sure what will work best to help people with an illness. Other words to describe clinical research are “clinical trial,” “protocol,” “study,” and “experiment.”

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S

**Single-blind research design** - A study in which one party, either the investigator or participant, is unaware of what medication or intervention the participant is taking; also called single-masked study.

**Sodium** - an element that the body needs to work properly. Salt contains sodium. The body uses sodium to control blood pressure and blood volume. Sodium is also needed for your muscles and nerves to work properly.

**Sponsors** - Clinical trials are sponsored or funded by various organizations or individuals, including physicians, foundations, medical institutions, voluntary groups, and pharmaceutical companies, as well as Federal agencies such as NIH, FDA, the Department of Defense, and the Department of Veterans Affairs.

**Standard treatment** - The treatment that medical professionals consider at the time of the study to be the most prevalent and best available treatment.

**Standardized procedures** - These are study rules that researchers must follow exactly for every participant, regardless of what each participant is used to. For example, if you normally take a medicine by injection but the experiment is testing the same medicine in pill form, the researcher must prescribe pills to you. The researcher cannot use a different method for you.

**Study** - Conducted by a principal investigator who is often a doctor. Members of the research team regularly monitor the participant’s health to determine the study’s safety and effectiveness. Other words to describe a study are “clinical trial,” “protocol,” “experiment,” and “research.”

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T

**Type 1 Diabetes** - Diabetes mellitus type 1 (also known as type 1 diabetes, or T1DM; formerly insulin dependent diabetes or juvenile diabetes) is a form of diabetes mellitus that results from the autoimmune destruction of the insulin-producing beta cells in the pancreas.The subsequent lack of insulin leads to increased blood and urine glucose.

**Type 2 Diabetes** - Type 2 diabetes, once called non-insulin-dependent diabetes, is the most common form of diabetes, affecting 90% to 95% of the 26 million Americans with diabetes. Unlike people with type 1 diabetes, the bodies of people with type 2 diabetes make insulin. But either their pancreas does not make enough insulin or the body cannot use the insulin well enough. This is called insulin resistance. When there isn't enough insulin or the insulin is not used as it should be, glucose (sugar) can't get into the body's cells. When glucose builds up in the blood instead of going into cells, the body's cells are not able to function properly.

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                                                                                  Content current as of:
      04/19/2019Clinical Trials: What Patients Need to Know

          
                        What Patients Need to Know About Institutional Review Boards
          Glossary of Terms


                        Clinical Research Versus Medical Treatment
          Informed Consent for Clinical Trials
          What Are the Different Types of Clinical Research?
 
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