Clinical Drug Trial Definitions
Active Pharmaceutical Ingredient
Any substance or mixture of substances intended to be used in the manufacture of a drug (medicinal) product and that when used in the production of a drug becomes an active ingredient of the drug product. Such substances are intended to furnish pharmacological activity or other direct effect in the diagnosis, cure, mitigation, treatment or prevention of disease or to effect the structure and function of the body.
(Adverse Event.) An unwanted effect caused by the administration of drugs. Onset may be sudden or develop over time.
Acceptable range in which the product temperatures can reach within a set amount of time that does not compromise the integrity of the product.
Active Pharmaceutical Ingredient
In the U.S., the Food and Drug Administration (FDA) must approve a substance as a drug before it can be marketed. The approval process involves several steps including pre-clinical laboratory and animal studies, clinical trials for safety and efficacy, filing of a New Drug Application by the manufacturer of the drug, FDA review of the application, and FDA approval/rejection of application
A quality control process in which a representative sample of a batch of drug is analyzed using a variety of tests to ensure the quality of the drug. A certificate of analysis is usually given for each batch analysis.
When a point of view prevents impartial judgment on issues relating to the subject of that point of view. In clinical studies, bias is controlled by blinding and randomization
A randomized trial is "Blind" if the participant is not told which arm of the trial he is on. A clinical trial is "Blind" if participants are unaware on whether they are in the experimental or control arm of the study; also called masked. In a clinical trial, patients, investigators, and other personnel may be kept �blinded� to the medication prescribed in order to decrease biases and avoid placebo effect.
The process through which one or more parties to a clinical trial are unaware of the treatment assignments. In a single-blinded study, usually the subjects are unaware of the treatment assignments. In a double-blinded study, both the subjects and the investigators are unaware of the treatment assignments. Also, in a double-blinded study, the monitors and sometimes the data analysts are unaware. "Blinded" studies are conducted to prevent the unintentional biases that can affect subject data when treatment assignments are known.
Case Report Form (CRF)
A record of pertinent information collected on each subject during a clinical trial, as outlined in the study protocol.
Center for Drug Evaluation and Research
Current Good Manufacturing Practices
Pertaining to or founded on observation and treatment of participants, as distinguished from theoretical or basic science.
Clinical Drug Investigation
A scientifically designed and executed investigation of the effects of a drug (or vaccine) administered to human subjects. The goal is to define the safety, clinical efficacy, and pharmacological effects (including toxicity, side effects, incompatibilities, or interactions) of the drug. The U.S. government, through the Food and Drug Administration (FDA), requires strict testing of all new drugs and vaccines prior to their approval for use as therapeutic agents.
Clinical Drug Test
A research study designed to determine the safety and effectiveness of a new medicine in people. These studies begin only after extensive testing in a laboratory has shown promising results, and the research study has been reviewed for scientific merit by a panel of physicians and scientists on a local Institutional Review Board
Study of drug, biologic or device in human subjects with the intent to discover potential beneficial effects and/or determine its safety and efficacy. Also called clinical study and clinical investigation. Clinical Research is research conducted with human subjects including:
- Studies on the etiology and pathogenesis of human disease
- Therapeutic interventions
- Clinical trials
- Epidemiological studies
- Disease control research that investigates how scientific information on prevention, early detection and early diagnosis can be efficiently applied
- Operations and implementation research that uses analytical techniques such as epidemiological modeling and cost-effectiveness analysis to determine how to achieve better health outcomes and optimal service delivery
Clinical Research Associate
A Clinical Research Associate (CRA) assists in the design, preparation, planning, implementation and review of a clinical trial to ensure it adheres to regulatory and ethical standards. A clinical research associate ensures compliance with the clinical trial protocol, checks clinical site activities, makes on-site visits, reviews Case Report Forms (CRFs) and communicates with clinical research investigators. Clinical Research Associates also assist in communicating findings to the research community.
Clinical Research Coordinator (CRC)
Site administer for the clinical study. Duties are delegated by the investigator. Also called research, study or healthcare coordinator, and data manager, research nurse or protocol nurse.
A clinical trial is a research study to answer specific questions about vaccines or new therapies or new ways of using known treatments. Clinical trials (also called medical research and research studies) are used to determine whether new drugs or treatments are both safe and effective. Clinical trials are intended to discover or verify the clinical, pharmacological and/or other pharmacodynamic effects of one or more investigational medicinal product(s), and/or to identify any adverse reactions to one or more investigational medicinal product(s) and/or to study absorption, distribution, metabolism and excretion of one or more investigational medicinal product(s) with the object of ascertaining its (their) safety and/or efficacy. Carefully conducted clinical trials are the fastest and safest way to find treatments that work in people. Trials are in four phases: Phase I tests a new drug or treatment in a small group; Phase II expands the study to a larger group of people; Phase III expands the study to an even larger group of people; and Phase IV takes place after the drug or treatment has been licensed and marketed
Clinical Trial Sponsor
a clinical trial sponsor is an individual, company, institution or organization that assumes responsibility for the initiation, management, or financing of a clinical trial.
In epidemiology, a group of individuals with some characteristics in common.
a temperature-controlled supply chain. An uninterrupted flow of a specific thermal profile throughout the manufacturing, packaging and distribution of a temperature-sensitive product. An unbroken cold chain is an uninterrupted series of storage and distribution activities which maintain a given temperature range.
Cold Chain Management
Management of all phases of the cold chain, including products in transit, in process, in storage, and in display.
Cold Chain Product
A product that is temperature sensitive.
A comparator drug is an agent that the investigational drug is being compared to in a clinical trial. A comparator drug may be the current standard of care for the disease state being studied.
a medication to which an experimental drug is being compared in a clinical drug trial.
Contract Research Organizations
an organization that offers clients a wide range of pharmaceutical research services. U.S. Food and Drug Administration regulations state that a CRO is "a person [i.e., a legal person, which may be a corporation] that assumes, as an independent contractor with the sponsor, one or more of the obligations of a sponsor, e.g., design of a protocol, selection or monitoring of investigations, evaluation of reports, and preparation of materials to be submitted to the Food and Drug Administration."
Services offered by CROs include: product development and formulation, clinical trial management (preclinical through phase IV), central laboratory services for processing trial samples, data management services for preparation of an FDA New Drug Application (NDA) or an Abbreviated New Drug Application (ANDA), and many other complementary services. CROs can offer their clients the experience of moving a new drug from its conception to FDA marketing approval without the drug sponsor having to maintain a staff for these services, which often have limited duration.
A specific circumstance when the use of certain treatments could be harmful.
a control is the nature of the intervention control.
a drug to which the drug being investigated in a clinical trail is compared. Control drugs are used in clinical research trials in which a new investigational drug is being compared to an established control drug with the aim to prove that the new investigational drug is either more effective than the control drug or has comparable efficacy.
A comparison group of study subjects who are not treated with the investigational agent. The subjects in this group may receive no therapy, a different therapy, or a placebo. The control group is the standard by which experimental observations are evaluated
Control is a standard against which experimental observations may be evaluated. In clinical trials, one group of participants is given an experimental drug, while another group (i.e., the control group) is given either a standard treatment for the disease or a placebo.
Clinical Research Associate
Case Report Forms
Contract Research Organizations
An in-transit temperature recorder that measures temperature during a specified time period (also called a digital data logger, data monitor, temperature data logger, in-transit temperature recorder).
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. A DSMB may recommend that a trial be stopped if there are safety concerns or if the trial objectives have been achieved.
Drug Enforcement Agency
An instrument, apparatus, implement, machine, contrivance, implant, in vitro reagent, or other similar or related article, including any component, part or accessory, which is intended for use in the diagnosis, cure, treatment or prevention of disease. A device does not achieve its intended purpose through chemical action in the body and is not dependent upon being metabolized to achieve its purpose.
Refers to 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.
A clinical trial in which two or more doses of an agent (such as a drug) are tested against each other to determine which dose works best and is least harmful.
The design of a study in which neither the investigator or the subject knows which medication (or placebo) the subject is receiving.
A clinical trial design in which neither the participating individuals nor the study staff knows which participants are receiving the experimental drug and which are receiving a placebo (or another therapy). Double-blind trials are thought to produce objective results, since the expectations of the doctor and the participant about the experimental drug do not affect the outcome; also called double-masked study. See Blinded Study, Single-Blind Study, and Placebo.
according to the Food, Drug, and Cosmetic Act (1): a substance recognized in an official pharmacopoeia or formulary (2): a substance intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease (3): a substance other than food intended to affect the structure or function of the body (4): a substance intended for use as a component of a medicine but not a device or a component, part, or accessory of a device
Drug or Device Accountability Records (DAR)
Required documentation for material accountability, quantity used and left over, and date of disposal.
the act of sourcing and gaining possession of drug products through special effort.
A finished dosage form (e.g. tablet, capsule, or solution) that contains the active drug ingredient usually combined with inactive ingredients.
The length of time a drug retains its properties without loss of potency; usually referred to as shelf life.
a supply of drug products for a clinical trial
A modification of the effect of a drug when administered with another drug. The effect may be an increase or a decrease in the action of either substance, or it may be an adverse effect that is not normally associated with either drug.
A refrigerant that is solid carbon dioxide, which sublimates to a gas at very cold temperatures. Dry ice is used as a pharmaceutical and food services coolant in insulated/thermal packaging.
(Of a drug or treatment). The maximum ability of a drug or treatment to produce a result regardless of dosage. A drug passes efficacy trials if it is effective at the dose tested and against the illness for which it is prescribed. In the procedure mandated by the FDA, Phase II clinical trials gauge efficacy, and Phase III trials confirm it
Overall outcome that the protocol is designed to evaluate. Common endpoints are severe toxicity, disease progression, or death.
The branch of medical science that deals with the study of incidence and distribution and control of a disease in a population. EPS - Expanded polystyrene - a lightweight, cost effective, cushioning insulating material used in packaging for temperature-sensitive goods.
a clinical drug trial designed to evaluate whether an experimental drug is equivalent or similar to an established control drug or not.
Refers to any of the 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.
A drug that is not FDA licensed for use in humans, or as a treatment for a particular condition
the date that a drug should be used by. The date printed on the packaging of drug products that indicates the time after which they should not be used.
United States Food and Drug Administration
A generic drug is the chemical equivalent of a brand name drug that has an expired patent. When a brand name drug's patent expires, other pharmaceutical companies can produce the same active chemical compound and sell the drug under its generic name.
Good Laboratory Practices
Good Manufacturing Practices
Good Clinical Practices
an international quality standard that is provided by International Conference on Harmonisation (ICH), an international body that defines standards, which governments can transpose into regulations for clinical trials involving human subjects. Good Clinical Practice guidelines include protection of human rights as a subject in clinical trial. It also provides assurance of the safety and efficacy of the newly developed compounds. Good Clinical Practice Guidelines include standards on how clinical trials should be conducted, define the roles and responsibilities of clinical trial sponsors, clinical research investigators, and monitors. In the pharmaceutical industry monitors are often called Clinical Research Associates.
Good Manufacturing Practices
standard guidelines set out by the FDA to ensure drug development is carried out in safe and quality processes, to avoid contamination and ensure repeatability.
Investigational Medicinal Product
In Vitro Testing
Non-clinical testing conducted in an artificial environment such as a test tube or culture medium.
In Vivo Testing
Testing conducted in living animal and human systems.
Institutional Review Board (IRB)
An independent group of professionals designated to review and approve the clinical protocol, informed consent forms, study advertisements, and patient brochures, to ensure that the study is safe and effective for human participation. It is also the IRB's responsibility to ensure that the study adheres to the FDA's regulations.
An investigational drug is a chemical or biological drug that is used in a clinical investigation.
(1) An investigational drug can be:
(a) A new chemical compound, which has not been released by the Food and Drug Administration (FDA) for general use, or
(b) An approved drug that is being studied for an approved or unapproved use, dose, dosage form, administration schedule, or under an Investigational New Drug (IND) application, in a controlled, randomized, or blinded clinical trial.
(2) Concurrent medications, comparators, or rescue medications used in the investigational trial that are not the drug(s) being studied are not defined as investigational drugs unless they are not commercially approved or not available through commercial channels. Prescription drugs, over-the-counter drugs, nutritional supplements, herbal preparations, and legend items used for diagnosis or treatment and meeting the definition in subparagraph 2f(1), are considered investigational drugs.
Investigational Medicinal Product
a pharmaceutical form of an active substance or placebo being tested or used as a reference in a clinical trial, including products already with a marketing authorization but used or assembled (formulated or packaged) in a way different from the authorised form, or when used for an unauthorised indication, or when used to gain further information about the authorised form.
Investigational New Drug Application (IND)
The petition through which a drug sponsor requests the FDA to allow human testing of its drug product.
A medical professional, usually a physician but may also be a nurse, pharmacist or other health care professional, under whose direction an investigational drug is administered or dispensed.
International Pharmaceutical Industry Congress
a drug or a medicine
(a) Any substance or combination of substances presented as having properties for
treating or preventing disease in human beings; or
(b) Any substance or combination of substances which may be used in or administered to human beings either with a view to restoring, correcting or modifying physiological functions by exerting a pharmacological, immunological or metabolic action, or to making a medical diagnosis.
Material Safety Data Sheet
New Drug Application (NDA)
An application submitted by the manufacturer of a drug to the FDA - after clinical trials have been completed - for a license to market the drug for a specified indication. A new drug application is the compilation of all non-clinical, clinical, pharmacological, pharmacokinetic and stability information required about a drug by the FDA in order to approve the drug for marketing in the U.S.
A clinical trial designed to show that a new treatment is equivalent to standard treatment (i.e. not inferior).
a study where the medicinal product(s) is (are) prescribed in the usual manner in accordance with the terms of the marketing authorisation. The assignment of the patient to a particular therapeutic strategy is not decided in advance by a trial protocol but falls within current practice and the prescription of the medicine is clearly separated from the decision to include the patient in the study. No additional diagnostic or monitoring procedures shall be applied to the patients and epidemiological methods shall be used for the analysis of collected data.
A drug prescribed for conditions other than those approved by the FDA.
Office of Human Research Protections
A clinical trial in which doctors and participants know which drug or vaccine is being administered.
An FDA category that refers to medications used to treat diseases and conditions that occur rarely. There is little financial incentive for the pharmaceutical industry to develop medications for these diseases or conditions. Orphan drug status, however, gives a manufacturer specific financial incentives to develop and provide such medications.
1. pertaining to pharmacy or drugs.
2. a medicinal drug.
Pharmaceutical Clinical Research
a clinical research trial designed to evaluate the effectiveness of a pharmaceutical drug product.
a company that specializes in distributing the prescription drug products of the pharmaceutical manufacturers. The pharmaceutical distributor has certain rights, responsibilities and restrictions related to marketing and selling the pharmaceutical companies products to the ultimate end-users.
Pharmaceutical Distributors Association
a trade association of state-licensed wholesale distributors of prescription drugs.
the manufacturers of pharmaceuticals. Also one of the biggest research contributors to human and veterinary medicine. This includes basic research into chemotherapy but includes also the otherwise neglected fields of formulations, packaging, dosing regimens and dispensing information. The industry also has access to the biggest database relating to chemicals and their uses.
medicinal drug products
Anyone or any organization that buys or offers to buy pharmaceuticals for resale and distribution to corporations, individuals, or entities other than the consumer.
The processes (in a living organism) of absorption, distribution, metabolism, and excretion of a drug or vaccine.
The science and activities relating to the detection, assessment, understanding and prevention of adverse effects or any other drug-related problem. Pharmacovigilance is the same as �drug monitoring�.
Phase I Study
The first of four phases of clinical trials, Phase I studies are designed to establish the effects of a new drug in humans. These studies are usually conducted on small populations of healthy humans to specifically determine a drug's toxicity, absorption, distribution and metabolism.
Phase II Study
After the successful completion of phase I trials, a drug is then tested for safety and efficacy in a slightly larger population of individuals who are afflicted with the disease or condition for which the drug was developed.
Phase III Study
The third and last pre-approval round of testing of a drug is conducted on large populations of afflicted patients. Phase III studies usually test the new drug in comparison with the standard therapy currently being used for the disease in question. The results of these trials usually provide the information that is included in the package insert and labeling.
Phase IV Study
After a drug has been approved by the FDA, phase IV studies are conducted to compare the drug to a competitor, explore additional patient populations, or to further study any adverse events.
Phase I Trials
Initial studies to determine the metabolism and pharmacologic actions of drugs in humans, the side effects associated with increasing doses, and to gain early evidence of effectiveness; may include healthy participants and/or patients.
Phase II Trials
Controlled clinical studies conducted to evaluate the effectiveness of the drug for a particular indication or indications in patients with the disease or condition nder study and to determine the common short-term side effects and risks.
Phase III Trials
Expanded controlled and uncontrolled trials after preliminary evidence suggesting effectiveness of the drug has been obtained, and are intended to gather additional information to evaluate the overall benefit-risk relationship of the drug and provide and adequate basis for physician labeling.
Phase IV Trials
Post-marketing studies to delineate additional information including the drug's risks, benefits, and optimal use.
A placebo is an inactive pill, liquid, or powder that has no treatment value. It is an inactive substance in the same form as the active drug. In clinical trials, experimental treatments are often compared with placebos to assess the treatment's effectiveness. For example, in a controlled clinical trial, one group may be given a real medication while another group is given a placebo that looks just like it in order to learn if the differences observed are due to the medication or to the power of suggestion. Placebos are widely used in drug trials.
Placebo Controlled Study
A method of investigation of drugs in which an inactive substance (the placebo) is given to one group of participants, while the drug being tested is given to another group. The results obtained in the two groups are then compared to see if the investigational treatment is more effective in treating the condition.
A physical or emotional change, occurring after a substance is taken or administered, that is not the result of any special property of the substance. The change may be beneficial, reflecting the expectations of the participant and, often, the expectations of the person giving the substance.
Polyurethane Insulated Panel
A flat piece of polyurethane designed to fit as a panel in a temperature-sensitive container (also called rigid thermal protection, rigid foam board insulation, rigid polyurethane foam sheet, rigid urethane foam, foam insulation panel, panel sheet insulated containers, expanded packaging polystyrene).
Refers to the testing of experimental drugs in the test tube or in animals - the testing that occurs before trials in humans may be carried out.
Research using animals to find out if a drug, procedure, or treatment is likely to be useful. The aim is to collect data in support of safety. Preclinical studies take place before any testing in humans is done.
Refers to trials to find better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vaccines, vitamins, minerals, or lifestyle changes.
The time and temperature for each step of the transit process (e.g., packaging, warehouse, transit, etc.)
A study plan on which all clinical trials are based. The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions. A protocol is a detailed set of instructions and requirements used in the execution of a test and/or procedure. 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. While in a clinical trial, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment
Testing that provides reasonable assurance that the qualified product or process will produce similar results under stated conditions.
Systems and procedures designed to ensure that a study is being performed in compliance with Good Clinical Practice (GCP) guidelines and that the data being generated is accurate.
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.
A method based on chance by which study participants are assigned to a treatment group. Randomization minimizes the differences among groups by equally distributing people with particular characteristics among all the trial arms. The researchers do not know which treatment is better. From what is known at the time, any one of the treatments chosen could be of benefit to the participant (See Arm).
the process of changing the labelling on a drug product
the process of removing a drug product from its original manufacturer�s package or container and placing the drug product in different packaging. Repackaging may also include simple alterations to the original manufacturer�s packaging in some way. During repackaging the drug product must not be altered in any way and the new packaging is required to contain the same substantive labeling declarations (e.g. identity, strength, and directions for use) as the original manufacturer�s packaging.
Repass or Retest Date
A repass or retest date is the date assigned by the manufacturer after which the drug substances need to be examined to ensure that they are within suitable specifications for use in the manufacture of a drug product.
the process of supplying a new batch or lot of drug products for a clinical trial already in progress.
Request for Proposal
Request for Quotation
22�C or 72�F.
Refers to trials which test the best way to detect certain diseases or health conditions.
the length of time a material may be stored without deterioration; the length of time it remains usable.
The process of transporting a package from one location to another.
Any undesired actions or effects of a drug or treatment. Negative or adverse effects may include headache, nausea, hair loss, skin irritation, or other physical problems. Experimental drugs must be evaluated for both immediate and long-term side effects
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.
Standard Operating Procedures
is an individual, company, institution or organization that assumes responsibility for the initiation, management, or financing of a clinical trial.
Often refers to the ability of a product to remain efficacious under varying temperature/time exposures.
A treatment currently in wide use and approved by the FDA, considered to be effective in the treatment of a specific disease or condition.
Standards of Care
Treatment regimen or medical management based on state of the art participant care.
The probability that an event or difference occurred by chance alone. In clinical trials, the level of statistical significance depends on the number of participants studied and the observations made, as well as the magnitude of differences observed.
The temperature at which a product is stored.
a study drug is an investigational drug being studied during a clinical trial.
a study medication is a commercially available drug which is being studied during a clinical trial.
a clinical drug trial in which an experimental drug is compared to a drug, which is the established standard of care, with the aim of proving the experimental drug superior to the standard of care drug product.
A product for which stability is impacted by temperatures outside a prescribed range (e.g., vaccine). Test Drug � a test drug is an investigational drug being studied during a clinical trial.
Test Drug Supplies
the available supply of the investigational drug being studied during a clinical trial
a test medication is a commercially available medication which is being studied during a clinical trial.
a drug or medication being investigated during a clinical trial
The acceptable range for a specified measurement.
refers to the completeness of the information about every step in a process chain. Traceability is the ability to verify the history, location, or application of an item by means of recorded identification.
IND stands for Investigational New Drug application, which is part of the process to get approval from the FDA for marketing a new prescription drug in the U.S. It makes promising new drugs available to desperately ill participants as early in the drug development process as possible. Treatment INDs are made available to participants before general marketing begins, typically during Phase III studies. To be considered for a treatment IND a participant cannot be eligible to be in the definitive clinical trial.
Refers to trials which test new treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.
A study that provides documented evidence that assures that a specific process or system will consistently meet predetermined specifications.