"Conducting Clinical Trials A-Z"

What exactly are clinical trials and the clinical trial process?

Clinicaltrials.gov provides a thorough introduction to clinical trials in a question and answer format.  The following glossaries are helpful resources to bookmark as well. 

Glossary of Clinical Trials Terms

CenterWatch Clinical Research Glossary

Basic Clinical Trial Terminology:

Clinical Research (per NIH) – research that is patient oriented; includes epidemiology and behavioral studies and outcome and health service research

Human Subject Research – Research which involves individuals, tissue, specimens, medical records, genetic material, behavioral and/or biomedical assessment or treatment.

Clinical Trial (per NIH) - prospective biomedical or behavioral studies designed to answer questions about intervention and determine safety and efficacy

Clinical Trial (per ICH GCP) – any investigation in human subjects intended to discover or verify the clinical, pharmacological and/or pharmacodynamic effects of an investigational product with the object of determining safety and/or efficacy.

Clinical Investigation (per FDA reg 312.3) – any experiment in which a drug is administered or dispensed to or used involving one or more human subjects.  For the purpose of this part, an experiment is any use of a drug except for the use of a marketed drug in the course of medical practice.

Clinical Trial Volunteer – Individuals and their private health information that are participating in a clinical research study and do not “belong” to an investigator.

Clinical Trials & Drug Development Process                           

Clinical trials are sponsored by government agencies, educational institutions, private non-profit organizations, industry and/or a combination of the above who partner to co-sponsored clinical research. The common mission is to develop, produce and evaluate the effectiveness of new therapies, treatments or devices for many different diseases. In a clinical trial, study sponsor develops the treatments and chooses qualified investigators to work on the clinical trials. Most clinical trials take place in four steps or phases.

   Investigational Product – a pharmaceutical form of an active ingredient, or placebo, device, biologic or therapeutic agent being tested in a clinical trial, including a product assembled in a way different from the approved form, or used for an unapproved indication or to gain further information about an approved use.

   Phase I Clinical Trials – A small number of people (usually between 15 to 30) take part in Phase I trials. Phase I trials are performed to determine how the drug affects the human body, how much medicine is safe and to determine how the drug should be administered. Phase I trials may involve patients with the disease under study or healthy human volunteers.

   Phase II Clinical Trials – A small number of people (approximately 100 to 200) take part in Phase II trials. These trials provide information about how well a drug works, and generate more information on the safety and benefit of the drug.

   Phase III Clinical Trials These trials usually involve large numbers of people nationwide or worldwide, approximately (1,000 to 3,000). A Phase III trial is held to see if the current practice is the best way to treat a patient, or if a new drug treatment or device provides better treatment. Phase III trials compare a new drug, combination of drugs, device, or procedure with a current standard of treatment.

   IND Application (Investigational New Drug) application through which a drug sponsor requests the FDA to allow human testing of the drug product.  Must submit detailed protocol, preclinical data and investigator credentials prior to initiating phase I trial.

   NDA Application (New Drug Application) The study sponsor gathers all data known about the drug and applies for Federal Drug Administration (FDA) approval, thereby enabling the sponsor to market the product.

   IDE  (Investigational Device Exemption) Application to FDA to allow human testing of unapproved devices.

   NDE (New Device Exemption) – The study sponsor gathers all data known about a device and applies for FDA approval, thereby enabling the sponsor to market the product.

   Phase IV Clinical Trials – The drug has received FDA approval and Phase IV trials are used to continue evaluating the drug.