Clinical trials

Doctors and researchers use clinical trials to find new ways of preventing, diagnosing or treating conditions and diseases. Without clinical trials, our ability to improve the health of Victorians and Australians would be hindered. Most of the current treatments and devices that we take for granted would not be available if clinical trials had not tested them first.

Victoria’s research and health facilities are some of the best in the world, and this makes Victoria and Australia an attractive place for researchers to conduct clinical trials. This means that Victorians can have access to cutting-edge treatments. There are benefits and risks for participants in clinical trials.

The regulations governing clinical trials in this country make the process as safe as possible for clinical trial participants. All research involving humans must be assessed and approved by a Human Ethics Research Committee (HREC).

Clinical trials can differ depending on what they are designed to achieve. Trials of drugs and treatments will pass through a number of phases as they progress from a laboratory experiment to a new treatment.

Types of clinical trials

The three major types of clinical trials focus on:

• treatment – testing new medications, devices or surgical techniques
• prevention – looking at ways of preventing disease including potential treatment (medications, vaccines or vitamins) or lifestyle changes
• screening or diagnosis – studying new diagnostic tests to see if they are effective at detecting diseases or conditions.

Phases of clinical trials

Drugs and treatments must pass through phases of testing. Knowing about these phases will help you to understand who can participate in a given phase of a trial, and the risks and benefits of different stages.

The phases include:

• Pre-clinical studies – the safety and effectiveness of drugs and treatments is tested in animals.
• Phase I – the safety and side effects of a drug are tested in a small number of healthy volunteers or participants.
• Phase II – the safety and effectiveness of a drug are tested in 100 to 300 volunteers who usually have the condition that the drug is designed to treat.
• Phase III – the effectiveness of the treatment is tested (and compared with standard treatments or an inactive placebo) in a larger number of people who have the condition that the drug is designed to treat.
• Phase IV – the drug or treatment is tested for long-term effects after it is released for doctors to use.

Consent to participate in a clinical trial

People who are deciding whether to enter a clinical trial must understand all the essential facts, benefits and risks. This information should be clearly written in a Patient Information Statement that is approved by an ethics committee. Understanding and agreeing to all the correct information is called informed consent.

You will need to know all the details, including:

• the purpose of the trial
• how long the trial will take
• the procedures involved
• the risks and benefits of the trial
• the effects on you and your family or carers
• the costs involved in the trial
• your key contact people during the trial.

You need to ask questions if anything is unclear. Writing down a list of questions to ask your doctor is a good way to make sure that all your concerns are covered. It can also be helpful to thoroughly research clinical trials online.

There are special considerations for specific groups of people including:

• pregnant women and the unborn fetus
• children and young people
• people in dependent or unequal relationships
• people highly dependent on medical care who may be unable to consent for themselves
• people with an intellectual or mental impairment
• people involved in illegal activities
• Aboriginal and Torres Strait Islander people
• people in other countries.

Participants in clinical trials

Clinical trials are voluntary and you should not feel pressured into participating. If you are seeing a doctor for a condition, your care will not be affected if you do not choose to enter a clinical trial. If you are in a trial, you can choose to leave at any time and your standard treatment will not be affected.

Before the trial begins, researchers must choose particular types of people to participate in the clinical trial so that meaningful results are obtained. The characteristics of the people who can enter the trial are known as inclusion criteria, and the characteristics that prevent entry are known as exclusion criteria.

Benefits for clinical trial participants

There are risks and benefits associated with clinical trials. The benefits include:

• having access to cutting-edge research treatments that are not yet available to the general public
• having access to leading experts and facilities
• playing an active role in your own health and treatment
• having access to new research treatments if you have a rare condition that is difficult to treat
• knowing that you are contributing to potential future treatments for people around the world.

Risks for clinical trial participants

It is important that you understand the risks of a trial before agreeing to participate. Speak with your doctor about all the potential risks.

Potential risks include:

• The experimental treatment might have side effects that are different or more serious and unpleasant than the side effects experienced with the current standard treatment.
• The trial might involve inconvenience (such as keeping a diary or collecting urine samples for 24 hours) or additional tests, hospital visits or complicated medications.
• The experimental treatment might not help your condition (if you have one) or you might be put into the group that receives the standard treatment or a placebo (inactive) treatment.

Conduct of clinical trials

The conduct of clinical trials is tightly regulated in Australia. The guidelines covering clinical trials are:

• National Statement on Ethical Conduct in Human Research
• Australian Code for the Responsible Conduct of Research

The Therapeutic Goods Administration also has a number of guidance notes and documents, including notes for good clinical practice.

A Human Research Ethics Committee must also approve all clinical trials before they can proceed.

Where to get help

• Your GP (doctor)