A clinical trial is any investigation in human subjects/patients intended: to discover or verify the clinical, pharmacological and/or other pharmacodynamic effects of an investigational product(s), and/or to identify any adverse reactions to an investigational product(s), and/or to study absorption, distribution, metabolism, and or excretion of an investigational product(s) with the object of ascertaining its safety and/or efficacy. Defined in ICH Guideline for Good Clinical Practice (GCP), May 1996. All clinical studies are performed in compliance with the regulator-approved study designs (protocol) and to strict international standards for GCP.
What is a protocol?
A clinical study protocol is a document that describes the background, rationale, objective(s), design, methodology, statistical considerations, Data Management and organisation of a Clinical Study. All clinical study protocols are approved by the Regulatory Authority in which the study is being conducted, thus each study should be conducted in full compliance with the protocol.
What is an investigational product?
An investigational product is a pharmaceutical form of an active ingredient or placebo, being tested or used as a reference in a Clinical Study. Investigational products can be either a non-marketed product or a marketing authorised product that is used or assembled (formulated or packaged) in a way different from what has already been approved by a regulatory authority. The term investigational product would also be used when a product authorised for marketing is being studied for an unapproved indication, or when used to gain further information about an approved use.
What are the different phases of Clinical Trials?
Clinical trials are organised into different phases of research, in order to build up a greater understanding of the new treatment, before it is approved or recommended for routine use.
What is called “Phase I” can also be referred to as Principle Testing. These are the earliest types of studies that are carried out in humans using an investigational product. They are typically conducted using very small numbers of well-informed, healthy volunteers in order to investigate the pharmacodynamics, pharmacokinetics and toxicity of a potential pharmaceutical therapy.
Phase II is also known as Concept Testing. Phase II studies are conducted in patients with the targeted condition to demonstrate efficacy and gather additional safety data. This phase traditionally includes dose-finding studies to establish the potential doses and forumulations of the product that would have the great efficacy in various patient populations. Phase II stuides are used to deterimine if further effort should be invested to develop the product into a marketable treatment for patients.
Phase III studies are often referred to as Development-For-Launch or Confirmatory studies. They tranditionally involve large numbers of patients who are administered the investigational product, a placebo or a comparator drug as a treatment for their targeted condition to conclusively determine the product’s efficacy. In the context of pharmaceuticals, the outcome of Phase III studies serve as the foundation upon which a company seeks product registration with Regulatory Authorities.
Phase IV clinical trials are sometimes also referred to as Post-Marketing or Life Cycle trials. These studies, conducted after a product is approved for registration by regulators, are used to gain a broader understanding of the long-term effects of a product and to research health economics aspects.