Clinical trial or investigation
FDA (U.S. Food and Drug Administration)
IRB (Institutional Review Board)
Protected health information (PHI)
Standard of care
Behavioral research: Research project designed to increase understanding of important social problems, learn about people's decision making and communication strategies and improve the quality of people's lives.
Blinded studies: "Single-blind" study means that the participant is not told which of the two or more possible study interventions (medicine A, B, or C, for instance) will be given, but the study team will know. "Double-blind" study means that the investigators and study coordinators also do not know what the participant is receiving either.
Control group: The control group are research participants who receive either standard of care for the illness or placebo (no medicine) as compared to participants who are receiving the study drug, medical device, procedure, or prevention.
Effectiveness/Efficacy: If researchers show that a drug has 'efficacy' (or is 'efficacious'), they show that it improves a disease or condition in a controlled environment, like a laboratory. Proving efficacy is a first step to show that the drug can work when it is taken in the right way. If researchers show that a drug is 'effective', they show that it improves a disease or condition outside of a controlled environment. For instance, if a drug lowers cholesterol in a study, it is efficacious. But if that drug has to be taken exactly 1 hour after eating, it might not be effective because it is too hard for people to remember to take it at exactly the right time.
FDA (U.S. Food and Drug Administration): The U.S. government agency that (1) enforces laws on the manufacturing, tests, and use of drugs and medical devices and (2) approves a new drug or device before it is made available to the public.
Gene therapy: Involves the insertion, alteration, or removal of genes within a person's cells and tissues to treat disease, correct defective genes that are responsible for disease development or to change a normal gene that allows viral infection.
Informed consent: Informed consent is the process of learning the key facts about a research study before deciding whether or not to participate. It is also a continuing process throughout the study to provide information for participants. To help someone decide whether or not to participate, the doctors involved in the trial explain the details of the study. Then the research team provides an informed consent document that includes details about the study, such as its purpose, duration, required procedures, and key contacts. Risks and potential benefits are explained in the informed consent document. The participant then decides whether or not to sign the document. Informed consent is not a contract, and the participant may withdraw from the trial at any time.
Investigation: See research.
Investigator: The person in charge of carrying out a research study. May also be referred to as Principal Investigator. A co-investigator is a researcher that helps with a study, but does not have full responsibility for the study.
Observational research: Observational research is where there is no intervention (drugs, devices, etc.) but an investigator will follow the course of a disease in participants. The person in the study may treat or not treat their disease as they wish and the investigator will follow their health, usually by reviewing their medical records. For instance, an observational research study may follow a group of smokers and a group of non-smokers to check their rates of lung infections and compare them to each other.
Out-patient: Studies or procedures that do not require an overnight stay are called outpatient. A person who enters a medical facility but does not stay overnight may be called an 'outpatient' as well.
Patient: A person seeking medical care. Participants in clinical studies are not called 'patients', because they may not be receiving medical care. For instance, they may be receiving a new drug that has not yet been proven to work, or they may be receiving a placebo.
Pharmacokinetics: The processes of absorption, distribution, metabolism, and excretion of a drug or vaccine. If your trial involves pharmacokinetics, it usually means there will be a few blood draws over a period of a few hours to see how the study drug or vaccine is absorbed into your body, distributed throughout your body, and excreted from your body over time.
Phase: Clinical trials are conducted in phases. The trials at each phase have a different purpose and help scientists answer different questions:
In Phase I trials, researchers test an experimental drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase II trials, the experimental study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase III trials, the experimental study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the experimental drug or treatment to be used safely.
In Phase IV trials, post marketing studies (after the drug is approved by the FDA) help us learn additional information including the drug's risks, benefits, and optimal use.
Placebo: A placebo looks like a drug, but is actually an inactive substance that is not expected to have any effect on your health. Sometimes a placebo is called a 'dummy pill' or 'sugar pill'. Old placebos were often true 'sugar pills', but modern placebos usually do not contain sugar. A research study may also use a placebo for a medical device or procedure. For instance, a placebo device could look like the real device, but not actually function.
Protocol: A protocol is a study plan on which all research studies are based. The plan is carefully designed to safeguard the health of the participants as well as answer specific 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. The protocol usually also gives the background and reason the study is being conducted.
Risks: There are risks to participating in research. Some of the risks for participating in clinical trials are that there may be unpleasant, serious or even life-threatening side effects to experimental treatment. The experimental treatment may not be effective for the participant and/or the protocol may require more of their time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements. In any study where you give your personal health information (PHI) there is the risk that your confidentiality could be breached, however, every research study has a plan to help prevent problems with confidentiality.
Side effects: Side effects are any actions or effects of the experimental drug or treatment that are not desired or expected. Experimental treatments must be evaluated for both immediate and long-term side effects.
Survey: See Questionnaire.