Clinical trials

Clinical trials are research investigations that evaluate the effects of one or more health-related interventions on health outcomes. If you would like to learn more about Clinical Trials, the Australian Clinical Trials website provides more detailed information and will help you to better understand clinical trials and make an informed decision about your potential participation in research. Australian Clinical Trials – For Consumers

We are extremely grateful to our patients for their participation in our clinical trials, they make this important research possible and an invaluable contribution to improved healthcare for so many others, now and in the years to come.

What is a clinical trial?

Clinical trials are research investigations in which people volunteer to test new treatments, interventions or tests as a means to prevent, detect, treat or manage various diseases or medical conditions.

Some investigations look at how people respond to a new intervention and what side effects might occur. This helps to determine if a new intervention works, if it is safe, and if it is better than the interventions that are already available.

Clinical trials might also compare existing interventions, test new ways to use or combine existing interventions or observe how people respond to other factors that might affect their health (such as dietary changes).

Researchers may also conduct clinical trials to evaluate diagnostic or screening tests and new ways to detect and treat disease.

Phases of clinical trials

Many clinical trials to develop new interventions are conducted in phases. In the early phases, the new intervention is tested in a small number of participants to assess safety and effectiveness. If the intervention is promising, it may move to later phases of testing where the number of participants is increased to collect more information on effectiveness and possible side effects.

Clinical trials of biomedical interventions typically proceed through four phases:

Phase I Clinical Trial

Phase I clinical trials are done to test a new biomedical intervention for the first time in a small group of people (e.g. 20-80) to evaluate safety (e.g. to determine a safe dosage range and identify side effects).

Phase II Clinical Trial

Phase II clinical trials are done to study an intervention in a larger group of people (e.g. several hundred) to determine efficacy (that is, whether it works as intended) and to further evaluate its safety.

Phase III Clinical Trial

Phase III studies are done to study the efficacy of an intervention in large groups of trial participants (from several hundred to several thousand) and often in many countries by comparing the intervention to other standard or experimental interventions (or to non-interventional standard care).  Phase III studies are also used to monitor adverse effects and to collect information that will allow the intervention to be used safely.

Phase IV Clinical Trial

Phase IV studies are done after an intervention has been marketed. These studies are designed to monitor the effectiveness of the approved intervention in the general population and to collect information about any adverse effects associated with widespread use over longer periods of time.  They may also be used to investigate the potential use of the intervention in a different condition, or in combination with other therapies.

Other clinical trials

Researchers may also conduct exploratory studies, sometimes referred to as ‘Phase 0 trials’ or ‘pilot studies’.  These come before Phase I trials and are used to test how the body responds to an experimental drug. In these studies, small doses of the new drug are given once or for a short time to a very limited number of people.

Clinical trials of diagnostic tests are sometimes divided into exploratory phases, challenge phases and advanced phases to see how effective and how accurate the tests are.

Why do we need clinical trials?

Clinical trials are essential to the development of new interventions. Most modern medical interventions are a direct result of clinical research. New interventions for most diseases and conditions — including cancer, heart disease, high blood pressure and asthma — have been developed through clinical research. Clinical trials often lead to new interventions becoming available that help people to live longer and to have less pain or disability. Clinical trials also lead to improvements in quality of life due to their focus on monitoring and improving the quality of patients’ lives, not simply the efficacy of treatments.

For example, without clinical trials, we cannot properly determine whether new medicines developed in the laboratory or by using animal models are effective or safe, or whether a diagnostic test works properly in a clinical setting. Clinical trials also permit testing and monitoring of the effect of an intervention on a large number of people to ensure that any improvement as a result of the intervention occurs for many people and is not just a random effect for a one person.

Clinical trials can also help to improve health care services by raising standards of treatment. Doctors and hospital staff involved in clinical trials are continually trained to provide best practice patient care. Australian clinical trials are recognised internationally for including very high quality patient care.

Why participate in a clinical trial?

The discovery of new interventions that can help people to live longer, have less pain or be free of ill-health or disability is possible through research and the willingness of people to voluntarily participate in clinical trials. Both healthy participants and those diagnosed with a health condition or disease are needed to help in finding new ways to prevent, diagnose, treat and potentially cure diseases or health conditions. By taking part in a clinical trial, you can contribute to the advancement of scientific understanding and, in some cases, to improved health for yourself and others with the same disease or health condition.

If you are healthy or otherwise free of the health condition or disease related to the clinical trial, you may choose to participate to help others, to contribute to improved health care or to contribute to improving scientific knowledge. You may even have a personal interest in the research or the specific trial, or you may even have a family member or friend with the disease or health condition.

Whilst your voluntary participation may help improve the lives of family, friends or others and is an extraordinary gesture of kindness and goodwill, potential participants should think carefully about participation. There are considerations around the demands on your time and the risks and benefits (if any), which should be carefully thought through before providing your informed consent to participate in research.

You may decide to participate if you have the disease or health condition specifically related to a clinical trial because your participation may contribute to a better understanding of, or better treatment or potential cure for this condition.

In some cases, your participation can provide access to new interventions before they are widely available.  Clinical trials may also offer the hope of developing better interventions or tests for a particular disease or condition, so that even if a trial does not directly provide benefit for the individual participant, it may provide benefits for others with the disease in the future. It is important to carefully consider these potential benefits (if any) before you decide whether or not to consent to participation.

As a patient enrolled on a clinical trial, even when you are receiving the highest quality care, you may also benefit from the specialised attention and support provided by the additional clinical trial staff who understand your disease or condition.