What is the clinical research of medicines?

Clinical research of new medicines is the testing of them on people. Every new medicine must undergo detailed clinical testing before it can be registered and made available to the community.

All pharmaceutical treatments in use today had to be proven effective and safe in clinical trials before they could be made available for widespread use within the community.

By the time a study treatment gets to a stage where it is given to patients in clinical trials, it has been thoroughly researched in the laboratory.Clinical trial research is conducted by experienced medical staff. All clinical trials must be approved by an independent Ethics Committee that monitors the conduct of the trial.A new medicine takes 5–10 years to develop. After the discovery of a new medicine the initial testing will be done in laboratories using specialised ‘ex-vivo’ tests (tests in animal tissues but not in living animals or humans). The next phase of testing is ‘pre-clinical’ – meaning in living animals. These animal tests are regulated by an Animal Ethics Committee to ensure that the tests are appropriate and ethical. Animal testing is required on all new medicines before they can be tested on people.

Why do people take part in Clinical Trials?

An individual’s decision to enter a clinical trial is a very personal one. Having hope for a future cure, understanding more about a disease that is affecting a loved one, and contributing to society are just a few reasons for taking part in a study.
By participating in a clinical trial at the Centre for Clinical Studies, you are playing an important part in the advancement of medical knowledge and helping to bring new medicines to people who need them.
Participation in clinical trials is completely voluntary. Clinical Trials provide volunteers with a free thorough medical examination (or Medical Screening). Most Clinical Trials provide a financial reimbursement to compensate for volunteer’s time and inconvenience.

What are the different phases?

Clinical research of new medicines has three main phases:

phase I
Initial testing of the safety, tolerability and absorption of the new medicine. Phase I studies often involve healthy volunteers and start with very low doses. Typical Phase I studies may involve giving a single dose of the new medicine followed by repeated blood samples to see how long the new medicine stays in your body. Once a single dose study is completed, multiple dose studies are undertaken to see how long the new medicine stays in the blood after repeated dosing. These types of studies are what ultimately lead to the ‘prescribing information’ for a new medicine. For example if a medicine says “take 2 tablets twice per day, morning and night” this has been determined from the studies that have measured how long it takes for the medicine to leave the body system. Other phase I studies may include testing for interaction with other medicines and food. For example if a medicine’s ‘prescribing information’ says “take on an empty stomach” or “not to be taken with heart medications”, this is because the clinical tests have shown that the medicines are affected by eating food or by taking another medication at the same time. Understanding interactions with other medications is particularly important because many people may be on multiple medications at the one time. Phase I studies usually involve one or more overnight stays in the clinical research centre to ensure that each participant is subject to the same conditions (food, clinical tests, exclusion of other drugs – alcohol, caffeine, recreational drugs) and that they are observed for any adverse effects. Each clinical test is taken at specific time points in order to record the effects of the medicine over time. In most studies some participants receive the new medicine and some others receive a similar looking inactive dummy medicine, called a ‘placebo’. As a participant you will not know whether you have had the new medicine or the placebo. There are usually only 10–40 participants in a study. The majority of the clinical research trials done at the Centre for Clinical Studies are Phase I trials.

phase II
These trials are conducted in patients who have the disease that the new medicine is trying to treat. The aim of these trials is to test the safety and effectiveness of the new medicine in the ‘target population’. Generally these trials will involve multiple doses of the medication and are often carried out on out patients rather than in hospital. Phase II studies may also require overnight stays in the clinical research centre to undertake blood tests to measure how long the medicine lasts in the blood of patients. In certain diseases, such as cancer, a very successful trial in phase II can allow for the drug to be submitted for registration. There are usually between 20–100 patients involved in a phase II study.

phase III
These trials are conducted in patients and may involve hundreds or thousands of participants. These are called registration trials. In general a new medicine needs to show that it is safe and effective in two independently-run trials before it can be submitted for approval by a health authority.

late phase III/Phase IV Studies

In late Phase III/Phase IV studies, studies often compare an investigational drug with other drugs already available on the market. This is called a “bio-equivalence study”.

Some late phase studies are designed to monitor a study drug's long-term effectiveness as well as the impact on a patient's quality of life. Others are designed to look at the cost-effectiveness of an investigational drug therapy relative to other traditional or existing treatments.