What is the purpose of investigational studies?

Testing a new medication through clinical research

Clinical pharmacology studies are required by governing and regulatory bodies, such as the Food & Drug Administration (FDA), to ensure the safety and efficacy of a new drug.

Clinical trials are conducted in phases. First, new medications are developed in a laboratory and tested in animal studies. If initial laboratory research is successful, a Phase 1 trial is conducted with healthy volunteers. The goal of Phase I trials is to assess the safety and tolerability of the new drug, and determine a safe dosage range. Phase II and Phase III trials establish the effectiveness of the medication with individuals diagnosed with the disease being treated.

Phase I trials

Phase I studies with healthy volunteers assess the following:

>How the drug is tolerated and what side effects it has on the healthy volunteer.

>Evaluating the pharmacokinetics of the drug (the process by which a drug is absorbed, distributed, metabolized and eliminated by the body)

>Compare the different forms of the medication: tablet, drop, capsule...

The purpose of these preliminary investigational studies is to understand how the medication interacts in healthy volunteers before treating patients. Doses of the medication are escalated in a step-wise fashion to ensure volunteer safety. This means that the safety, tolerability, and side effects of a medication are evaluated at the lowest dosage before a higher dosage is administered to a volunteer.

Phase II and III trials

Following a Phase I trial, Phase II and III trials are carried out with patients affected with the disease/ailment that the medication is attempting to treat. These studies are designed to investigate the efficacy, dosing and tolerability of a medication with this population.