Basic Clinical Trial Terminology for Subject Know How.

Clinical Trial is a part of Drug Development Process which is again a part of domain
known as “LIFE SCIENCES”.

“LIFE SCIENCES”. is different from “HEALTHCARE” domain which primary involves

insurance & medical sector i.e medical billing, insurance billing, insurance agent, hospital
staff etc.

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.

Compound (Later known as Investigational Product) – It is the product which is

developed in Laboratories (Drug discovery, millions are spent on labs annually).
Sometimes before testing this product on human i.e running a clinical trial;
sometimes as per FDA regulations some product need to go through Pre-Clinical
Process which means testing the compound/investigational product on animals first
for safety issues.

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.