About clinical trials 

What is a clinical trial?

Clinical trials are a type of research investigation 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. Clinical trials are research investigations that evaluate the effects of one or more health-related
interventions on health outcomes.

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.

If you would like to learn more information 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.

What is a clinical trial phase?

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 to evaluate safety. For example, this might be 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 to determine whether the intervention works as intended, called efficacy, and to further evaluate its safety.

Phase III clinical trial

Phase III clinical trials are done to study the efficacy of an intervention in a larger group of trial participants and often in many countries by comparing the intervention to other standard or experimental interventions, or to non-interventional standard care. Phase III clinical trials 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 clinical trials are done after an intervention has been marketed. These clinical trials 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 long 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 clinical 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 advance phases to see how effective and accurate the tests are.

Why are clinical trials important in health care?

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 clinical trials?

Participating in a clinical trial can be an opportunity to take a more active role in your own health care. You might gain new or increased insights into your own health conditions or concerns, and you may have an opportunity to better understand your health care treatment and management.

In some cases, participation in a clinical trial involves access to trial new treatments under development, new services and models of care, or the opportunity to participate in research better designed to understand your health care experiences.

It is also possible that you might not gain any direct or immediate benefit for yourself by participating in research. Your participation in research may help others, either now or into the future, by enabling the discovery of more effective models of health care, better treatments, and ultimately better
management of health outcomes.

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.

What does research participation involve?

Participation in research is voluntary. Because every research study is different, your level of involvement and the type of participation will depend on the individual research project. In general, the Participant Information and Consent Form (PICF) will explain what is involved and what you can expect.
This will always be explained when you are approached to consider participating, and you can always ask questions to find out more information.

There are many different ways that you can participate in research, including:

• observation of your routine medical care
• permission to collect samples such as blood or urine and undertake diagnostic tests such as an x-ray
• permission to access information from your medical record, for example a blood test or treatment history
• answering a survey online or completing a paper based questionnaire or form
• being involved in an interview either in person or over the phone
• attendance at follow-up sessions or clinical appointments
• attendance at a facility or service not operated by Metro North, for example a clinical research facility

It is important to consider what participation means to you, and how it fits in with your personal circumstances. This involves understanding all of the requirements for participation, such as study visit locations or the time involved.

Are there any risks associated with participation in research?

There may be some risks associated with participation in clinical research, and it is important to read all of the information you are provided carefully, and ask questions so that you feel comfortable with your understanding of the risks involved.

New drugs, devices or procedures are always tested first in a non-clinical setting, and may have even been tested on animals to determine the risk involved. Some side effects may be known, while others may not be, and each person may react differently to a medication or procedure.

You may be required to come off your usual medication, for example, to test the effectiveness of a new medication being tested. There could be risks associated with this choice, and again, it is important to discuss this openly with the researcher and consider for yourself the benefit and risks associated
with participation.

All known risks will be described to you in writing, provided in a document we commonly call a Participant Information and Consent Form (PICF). The researchers will provide this to you before you decide to take part in any research, and answer any questions that you may have about the risks.  It
is possible that side effects not previously reported are experienced during clinical trials. The research team closely monitor the safety of participants and will provide emergency contact details.

Are there any costs involved with participation in research?

Sometimes research will be conducted as a part of your routine care, and may not involve extra visits or services.

Payment for participation in clinical trials does not occur in Australia, however it is common for out of pocket expenses to be covered for a clinical trial. For example: parking, meals for lengthy visits, and travel if you do not live locally.

Clinical trials are generally sponsored by government agencies, universities, medical centres, pharmaceutical companies and device manufacturers. Where they are, there should be no cost to you.

It is important to discuss these considerations with the researcher when you are first invited to participate, and should be outlined clearly when discussing trial participation.

What about time off work to attend clinical trial appointments?

If being involved in a research study requires you to take off work, you are able to request a medical certificate from the research team.

What if I no longer want to participate?

Participation in research is voluntary. It is not compulsory. You are free to withdraw from a research project at any time. If you no longer wish to participate in a research project, you can decide to not continue without it affecting your access to ongoing treatment or ongoing care. You can be sure
that you will always continue to receive the best care we are able to offer you.

How do I participate in a clinical trial?

For anyone who is interested in being a part of a clinical trial, the first step is finding out more about trials in general, and a good starting point is the Australian Clinical Trials website. It is a good idea to seek out information from multiple sources. There is a wealth of information available
on this and other websites about clinical trials in general, why a person may want to be part of a clinical trial, the ethics and regulation of clinical trials in Australia and how to find a trial that is relevant.

Once you have found, or been approached about a clinical trial that is appropriate for you, it is important to seek information about what participation in that particular trial will involve.  At Metro North, we encourage you to feel comfortable to ask questions, and seek the information that
you require to make an informed decision about participation.

You may talk to any of the health professionals involved in your care.  They will be able to provide you with general information about clinical trials, and they may be able to provide you with information on clinical trials that are relevant to you.

Consent

Everyone who participates in a clinical trial must give ‘informed consent’ or have a parent or guardian or other legally authorised person provide consent.

Informed consent means that potential participants are given information about the key facts of a clinical trial before deciding whether or not to take part. Informed consent also means that participants are provided with information on new developments throughout the trial.

You cannot be entered into a trial if you don’t want to be. If you are asked to take part, you are free to say yes or no at any time. There should be no pressure on you to enter into a trial.

To help you decide whether or not to be a part of the trial, a member of the research team will explain the details of the trial to you. The researchers will also provide a document, usually called a Participant Information and Consent Form (PICF)[1], which includes details about the study such as its
purpose, duration, required procedures risks and potential benefits. You can ask anything that is not clear to you or that you do not understand. You can take your time and talk it over with family and friends or your own doctor before deciding whether to take part.

You will then be able to decide whether you would like to participate. If you do sign the consent document and decide to participate, this means that you are agreeing to take part in the trial and have understood what it will involve. The consent document is not a contract, and you may still withdraw
from the trial at any time. If you do withdraw from a clinical trial, the relationship between you and your doctor will not be affected. You can be assured that you will still receive the best treatment that Metro North is able to provide.

If there are any changes to the trial or the protocol, you will be kept informed and you may be asked to give your consent again before proceeding with the trial.