FDA clinical trial Glossary
- Adverse Reaction: (Adverse event.) An unwanted effect caused by the administration of drugs. Adverse reactions may occur suddenly or develop over time.
- Approved Drugs: In the United States, the US Food and Drug Administration (FDA) must approve a drug before it can be marketed. The approval process involves several steps including preclinical laboratory and animal studies, clinical trials for safety and efficacy, filing of a new drug application by the manufacturer of the drug, and FDA approval or rejection of the application.
- Arm: A treatment group in a randomized trial. Most randomized trials have 2 "arms, " but some have 3 or more.
- Baseline: Information gathered at the beginning of a study and used as the comparison for variations found in the study. A drug's safety and efficacy are often determined by measuring changes from the baseline.
- Blind: A procedure in which one or more parties to the clinical trial (the subject/patient, investigator, and/or sponsor medical personnel) are kept unaware of which participants are receiving the treatment being tested. This helps remove study bias.
- Clinical Investigator: A medical researcher in charge of carrying out a clinical trial.
- Clinical Trial: A research study to determine whether new drugs or treatments are both safe and effective. Trials are in 4 phases: Phase 1 tests a new drug or treatment in a small group; phase 2 expands the study to a larger group of people; phase 3 expands the study to an even larger group of people; and phase 4 takes place after the drug or treatment has been licensed and marketed.
- Cohort: A group of individuals with some characteristics in common.
- Community-Based Clinical Trial (CBCT): A clinical trial conducted primarily through primary-care physicians rather than academic research facilities.
- Comparative Study: A study in which a participant is randomly assigned to a treatment group in order to compare the results of the separate treatment groups.
- Compassionate Use: A way to provide experimental, not-yet-approved treatment to patients who are critically ill and are not responsive to other treatments. Physicians may need to get case-by-case approval from the FDA for "compassionate use" of a drug or therapy.
- Confidentiality: Trial participants' personal identity and all personal medical information must be held confidential. Investigators should obtain the trial participants' consent to the use of records for data verification purposes prior to the trial.
- Control Group: The group that does not receive the experimental treatment. Often, one group of patients will receive an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.
- Core Registration Studies: The primary safety and efficacy studies (usually phase 3) that government health agencies use to decide whether to approve the investigational drug or new use of a marketed drug.
- Data Safety and Monitoring Board (DSMB): An independent committee, composed of community representatives and clinical research experts, that reviews data while a clinical trial is in progress to ensure that participants are not exposed to undue risk.
- Diagnostic Trials: Trials that are conducted to find better tests or procedures for diagnosing a particular disease or condition. Diagnostic trials usually include people who have signs or symptoms of the disease or condition being studied.
- Documentation: All records describing or recording the methods, conduct, and/or results of a trial, the factors affecting a trial, and the actions taken or omitted.
- Dose-Ranging Study: A clinical trial that tests and compares 2 or more doses of a drug against each other to determine which dose is most effective and least harmful.
- Double-Blind Study: A clinical trial in which neither the participants nor the study staff knows which participants are receiving the experimental drug and which are receiving a placebo (or another therapy).
- Efficacy: A drug is judged to be effective if it is effective at the dose tested and against the illness it targets. Phase 2 clinical trials judge efficacy, and phase 3 trials confirm it.
- Eligibility Criteria: The criteria for participant selection, which covers inclusion and exclusion criteria.
- Enrolling: The act of signing up participants into a study. The participants must meet the eligibility criteria and must go through the informed consent process.
- Expanded Access: These are FDA procedures, such as compassionate use, parallel track, and treatment IND, that distribute experimental drugs to participants who are failing on currently available treatments for their condition and also are unable to participate in ongoing clinical trials.
- Experimental Drug: A drug not yet approved by the FDA as a treatment.
- Good Clinical Practice (GCP): A standard for the design, implementation, and reporting of clinical trials. These GCPs assure that the test results are accurate and that the subjects' rights are protected.
- Inclusion/Exclusion Criteria: These determine whether an individual may take part in a clinical trial. Criteria are based on age, gender, the type and stage of a disease, previous treatment history, and other medical conditions.
- Informed Consent: The process of informing potential trial participants of all key facts about a clinical trial, including its purpose, the procedures involved, and the potential risks and benefits, and then voluntarily agreeing to participate in the clinical trial.
- Institutional Review Board (IRB): A committee of physicians, researchers, community advocates, and others who ensure that a clinical trial is ethical and that the rights of study participants are protected. All clinical trials in the United States must be approved by an IRB before they begin. The IRB will also periodically review the research in order to make sure physicians' rights are protected.
- Investigational New Drug Application (IND): After preclinical testing, a company files an IND with the FDA to begin to test the drug in humans. The IND becomes effective if the FDA does not disapprove it within 30 days. The IND describes prior experiments, the new study protocol, the composition and manufacture of the investigational drug, and any toxic effects found in the animal studies.
- Investigator: The medical professional in charge of the clinical trial at a trial site, who coordinates with the study sponsor and the IRB. If several people are conducting the trial, the leader is the principal investigator.
- Multicenter Trial: A clinical trial being carried out at more than one site, using the same protocol and with the same goals.
- New Drug Application (NDA): An application submitted to the FDA by a drug's manufacturer after all 3 phases of clinical trial development. NDAs can be extremely long and can require a year or more to be reviewed by the FDA.
- Open-Label Trial: A clinical trial in which doctors and participants know which drug or vaccine participants are receiving.
- Orphan Drugs: Medications for diseases and conditions that occur rarely. Pharmaceutical companies have little financial incentive to develop drugs for these diseases. Orphan drug status, conferred by the FDA, gives a manufacturer specific financial incentives to develop such medications.
- Peer Review: Experts chosen by the study sponsor review the trials for scientific merit, participant safety, and ethical considerations.
- Phase (of Clinical Trial): Drug development is divided into 4 phases.
- Phase 1: A phase 1 study mainly evaluates the safety of a drug or device and usually includes a small number of healthy volunteers (20 to 100). The study looks at the drug's effects and how it works. This phase also notes and examines side effects that develop with increasing dosages. (Phase 1 trials with anticancer drugs are usually carried out in patients who already have the disease.)
- Phase 2: Phase 2 studies test the efficacy of a drug or device. Phase 2 studies are often randomized trials in which one group of patients receives the experimental drug and a second "control" group receives a standard treatment or placebo. Often these studies are "blinded, " which means that neither the patients nor the researchers know who has received the experimental drug. Trials in this phase may also test different doses of the drug.
- Phase 3: A drug that shows positive results in small groups of patients may be studied in a larger phase 3 trial to confirm its efficacy and note adverse events that occur with long-term use. Hundreds or thousands of patients may be involved.
- Phase 4: Phase 4 clinical trials take place after a drug or device receives approval for sale. These trials compare a drug with other drugs already being sold, monitor its long-term effectiveness, and determine the cost-effectiveness of a drug therapy relative to other traditional and new therapies. These are sometimes called "post-marketing" trials.
- Preclinical: The testing of experimental drugs in the test tube or in animals. This occurs before any of the phases of human trials can begin.
- Protocol: The clinical trials study plan, which describes what types of people may participate; the schedule of tests, procedures, medications, and dosages; and the length of the study.
- Quality-of-Life Trials (or Supportive Care Trials): Refers to trials that explore ways to improve comfort and quality of life for individuals with a chronic illness.
- Randomization: A method based on chance by which study participants are assigned to a treatment group. This minimizes the differences among groups by equally distributing people with particular characteristics among all the trial arms.
- Recruiting: The period prior to beginning a trial in which the investigators identify and enroll participants.
- Side Effects: Any undesired actions or effects of a drug or treatment. Experimental drugs must be evaluated for both immediate and long-term side effects.
- Serious Adverse Event: An adverse event that at any dose of the drug either: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; or results in persistent or significant disability/incapacity.
- Single-Blind Study: A study in which one party, either the investigator or participant, is unaware of what medication the participant is taking; also called single-masked study.
- Treatment IND: The Investigational New Drug application (IND) is part of the process to gain FDA approval for selling a new prescription drug in the United States. It makes promising new drugs available to desperately ill participants early in the drug development process. Treatment INDs are typically made available to participants during phase 3 studies. A treatment IND participant cannot be eligible to be in the definitive clinical trial.
- Washout: A period of time during a clinical study when a participant stops taking either a current medication that is not allowed during the study or the study drug.
BMC Medical Research Methodology at the 35th Annual Conference of the .. — BMC Pediatrics
The conference will focus on issues such as design and analysis of clinical trials, methods in biostatistics and development of clinical prediction models.
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Does the European clinical trials directive really improve clinical trial approval time?
To facilitate and improve clinical research within Europe, the European Union (EU) adopted in 2001 the Clinical Trials Directive (EUCTD). The aim of this study was to compare duration between submission of a clinical drug trial application and approval by regulatory authorities in EU countries regulated by EUCTD vs. EU countries regulated by local legislation and, second, to compare the duration of regulatory approval in Europe vs. the USA and Australia.