Clinical trials are carefully designed research studies that help doctors and researchers test and develop new ways to prevent, diagnose, treat, or manage diseases and health conditions, such as cancer. In these studies, the treatments, tests, procedures, or strategies being studied are often called interventions.

Each clinical trial is designed to answer a specific research question, often focusing on areas where current approaches could be improved or where new options are still needed.

Click here to learn more about clinical trials: Australian Clinical Trials Alliance

There are different types of clinical trials, including:

• Treatment trials test new treatments, new ways of giving existing treatments, or new combinations of treatments.
• Diagnostic trials investigate new methods or tests to accurately detect or diagnose a disease.
• Screening trials look at new ways to find disease early, often before symptoms appear.
• Medical, surgical and device trials evaluate new or improved medical devices, procedures, or surgical techniques.
• Quality of life trials are often conducted alongside treatment trials and focus on improving comfort, daily functioning, and overall wellbeing.
• Preventive and educational trials explore ways to prevent disease or improve health through education, lifestyle changes, or other preventive strategies.

Clinical trials are essential for developing new treatments and approaches to health care that can improve outcomes for people, such as helping them live longer or enhancing their quality of life. When a new treatment or approach is shown to work better than existing options, it may become the new standard of care, benefiting future patients.

Clinical trials also rely on people volunteering to take part. Without them, many advances in treatments and medical devices would not be possible. However, more awareness is needed, as only a small number of eligible people currently participate in clinical trials.

Clinical trials are important because they help advance medical knowledge, improve future healthcare, and, in some cases, may give patients access to new and promising treatments that are not yet widely available.

While there is no guarantee that you will personally benefit from participating in a clinical trial, potential benefits may include:

• Seeing your treatment team more frequently, with your condition, care, or treatment being monitored more closely
• Access to medicines, programs, or other treatments that may not be widely available outside the study or subsidised by the Pharmaceutical Benefits Scheme (PBS), and that may be more effective or have fewer side effects than current standard treatments
• Taking an active role in your health care and gaining a better understanding of your disease or condition
• Learning new ways to support your health and improve your lifestyle
• Maintaining or improving your quality of life
• Feeling reassured that you have explored all available treatment options
• Knowing that your involvement may help improve care and treatments for future patients

Your doctor or the clinical trials team will carefully discuss all potential benefits and risks with you before you decide whether taking part in a clinical trial is right for you. Taking part is entirely voluntary, and you can withdraw at any time without affecting your care.

Clinical trials must meet strict safety rules and standards and be approved by an independent ethics committee to protect participant safety. Before taking part in a clinical trial, and throughout the study, potential risks are identified, carefully monitored, and clearly explained to participants through a process called informed consent.

Some potential risks of taking part in a clinical trial may include:

• The study treatment may not work for you or may be less effective than existing treatments or the current standard of care
• If you receive standard care in the trial, it may turn out to be less effective than the new treatment being studied
• The study treatment may cause side effects, which could be unpleasant, serious, or, in rare cases, life-threatening
• Because the intervention has been used in a limited number of people, there may be unexpected risks that are not yet known. These risks depend on the type and phase of the trial
• Participation may require additional procedures or commitments, such as extra tests, clinic visits, hospital stays, or more complex treatment requirements (for example, keeping a symptom diary, collecting 24-hour urine samples, or wearing a monitor overnight)

Your doctor or the clinical trials team will carefully discuss all potential benefits and risks with you before you decide whether taking part in a clinical trial is right for you. Taking part is entirely voluntary, and you can withdraw at any time without affecting your care.

If you are interested in taking part in a clinical trial, you should discuss this with your doctor.

Before taking part in a clinical trial, participants must give ‘informed consent’. This means you are given clear information about the trial, including alternative treatment options, benefits and risks, and your rights, so you can decide whether taking part is right for you.

This information is usually provided both verbally and in writing through a Participant Information and Consent Form (PICF) and may also be available in other formats such as videos. You are encouraged to ask questions, take time to consider the information, and discuss it with your family, friends, or health care team before deciding.

Taking part is voluntary. You can withdraw from the trial at any time without affecting your care. You will also be kept informed of any new information that may influence your decision about whether to continue.

Each clinical trial follows a detailed plan, known as a protocol, which clearly sets out who can and cannot take part. These rules are called eligibility criteria. They are based on the research question the trial is designed to answer and help ensure the study is conducted safely and produces reliable results.

Eligibility criteria may be based on factors such as:

• Type of cancer
• The stage and spread of the cancer (for example, localised, advanced, or recurrent, meaning the cancer has returned)
• Age
• Other medical conditions and current medications
• Previous cancer treatments
• Performance status, which describes how well a person feels and manages everyday activities
• Other test results, such as blood tests and scans

Randomisation means assigning participants in a clinical trial to different treatment groups (also called study arms) at random or by chance, similar to a lottery. This means each person has a chance of receiving either the new treatment or the comparison treatment. The comparison (or control) treatment is usually the current standard treatment that a participant would receive outside of a clinical trial. In some studies, it may be a placebo. 

Randomly assigning participants to treatment groups helps reduce bias. This means the study results are less likely to be influenced by factors unrelated to the treatment itself, such as personal preferences, expectations, or other differences between participants. It also helps ensure that the groups are as similar as possible at the start of the trial. This is important so that any differences in outcomes are more likely due to the treatment itself, rather than differences between the people in each group, such as age or how severe their illness is.

Randomisation is usually done by a computer and is used in all Phase 3 trials and some Phase 2 trials.

Some studies are blinded, which means the participant does not know which treatment group (or study arm) they are in, or which treatment they are receiving.

In a double-blind study, neither the participant nor the research or medical team knows which treatment the participant is receiving, whether it is the study treatment or the control treatment.

Blinding helps reduce bias and ensures that the study results are less likely to be influenced by expectations, opinions, or personal beliefs.

Treatments and medicines are usually studied in laboratories, and sometimes in animal studies, for many years before being tested in people.

After this, clinical trials involving people begin and are conducted in stages, known as phases. These phases are designed to carefully assess the safety, benefits, and risks of a new treatment or approach step by step.

Each phase has a specific purpose, and not all treatments go through every phase:

• Phase 1: focuses on safety and finding a safe dose
• Phase 2: looks at how well the treatment works (efficacy) while continuing to assess safety
• Phase 3: compares the new treatment with standard treatment or placebo
• Phase 4: monitors long-term effectiveness and safety in general use

See the table below for more information.

A clinical trial team is made up of a group of health care and research professionals who work together to run the study and care for participants. The exact team can vary depending on the trial, but it commonly includes:

• Principal Investigator:
  A medical specialist or senior doctor responsible for the overall conduct of the study at the site and for the safety of participants.
• Co-investigators (or sub-investigators):
  Doctors or other qualified health professionals who work with the Principal Investigator to help conduct the study and care for participants.
• Clinical trial coordinator (or study coordinator):
  A member of the research team who manages the day-to-day running of the study at the site. They coordinate study visits, explain study procedures, and act as a point of contact for participants.
• Clinical trial or research nurses:
  Registered nurses who are part of the study team and may be involved in your care during the trial. They assist with study procedures, provide clinical care, and help collect study information.
• Pharmacist (or clinical trials pharmacist):
  A pharmacist responsible for the safe storage, preparation, dispensing, and management of study medicines in line with the study protocol.
• Data managers and research scientists:
  Members of the research team who collect, manage, and analyse study data to help answer the research questions. They do not provide direct care to participants.
• Allied health professionals (if relevant):
  Health professionals such as dietitians, physiotherapists, psychologists, or other specialists who may be involved in study-related assessments or care, depending on the trial.
• Ethics and safety oversight bodies:
  Independent committees, such as Human Research Ethics Committees (HRECs) and safety monitoring committees, that review and monitor the study to help ensure it is conducted ethically and that participant safety is protected. These groups do not provide direct care.

Taking part in a clinical trial is generally free for Australian and New Zealand citizens and residents. Costs related to the trial itself, such as study treatments, tests, and additional check-ups, are usually covered by the organisation funding or conducting the research (often called the sponsor).

Participants are not usually paid to take part, but reasonable out-of-pocket expenses, such as travel, parking, or light refreshments, may be reimbursed. This can be particularly helpful for people who need to travel long distances or live in regional or rural areas.

Any costs, reimbursements, or requirements will be explained clearly before you decide whether to take part, as part of the Participant Information and Consent Form.

Participants are still responsible for any health care costs they would normally pay as part of their standard treatment outside the trial.

Cancer clinical trials take place in a range of settings, including hospitals, treatment centres, universities, and laboratories These are often called participating centres.

Some parts of a trial may also be completed from home, such as surveys or appointments conducted by phone or video.

ANZUP trials are undertaken at many different participating centres across Australia, New Zealand and internationally.

Your doctor or trial team will let you know where the study visits will take place.

Clinical trial results often take time to become available, usually a few years after the study ends, and sometimes longer. Results are typically published in medical journals or presented at scientific meetings.

Participants can ask their doctor or clinical trial team about the results and how they will be shared. Results are usually reported for the whole study group rather than for individual participants, and some studies provide an easy-to-understand summary of the findings.

If you are thinking about taking part in a clinical trial, it can help to ask questions so you understand your options and what to expect. Below are some of the questions you may want to ask:

• About your options
  • What is the usual (standard) treatment for my condition?
  • Are there clinical trials that may be suitable?
  • Will my care be affected if I choose not to take part in a trial?
• About the trial
  • What is the aim of the trial, and what research has been done so far?
  • How does the trial treatment compare with standard treatment (or a placebo)?
  • How are treatments assigned (for example, randomisation)?
  • Who is running and funding the trial?
  • How will the results be measured?
  • If I join the trial but later change my mind, how can I stop? Will this affect my care in any way?
  • How will my privacy and confidentiality be protected?
• What taking part involves
  • What will I need to do, and how much time will it take?
  • Will I need extra tests, hospital visits, or travel?
  • How long will I be involved in the trial?
  • Can I continue my usual medications?
  • Who will look after me during the trial, and who can I contact with questions?
• Risks and benefits
  • What are the possible benefits and risks?
  • How do these compare with my current treatment options?
  • What happens if new safety information becomes available during the trial?
• Costs
  • What costs are covered, and will I be reimbursed for expenses such as travel?
  • How do the costs of participating in a trial differ from standard treatment?
• After the trial
  • What follow-up care will I receive?
  • How and when will results be shared?
  • Will I be able to continue the treatment if it is helpful?

You do not need to ask all these questions at once. Your doctor or the clinical trials team can guide you, and you are encouraged to take time to consider your options.

Speak with your GP or cancer specialist if you would like more information about clinical trials or think you may be eligible to take part.

ANZUP have clinical trials in various stages of progress: open to recruitment, in follow up and completed trials. Click here to learn more about ANZUP’s clinical trials.

 You can also find information about clinical trials in Australia on the Australian Cancer Trials website (www.australiancancertrials.gov.au), and about clinical trials in Australia and New Zealand on the Australian New Zealand Clinical Trials Registry (ANZCTR) (www.anzctr.org.au).

Often described as ‘bench to bedside’ (from the laboratory to patient care), translational research involves turning laboratory and research discoveries into practical applications, such as new treatments, tests, medical devices, or approaches to care, that can be used in clinical practice or, in some cases, made available to patients more broadly.

Translational research may also continue after a clinical trial. In this case, trial results can be used to develop clear treatment pathways, diagnostic processes, or clinical guidelines that support future patient care.

Translational research brings together specialists from different fields to work collaboratively. By involving researchers, doctors, nurses, psychologists, and other health professionals, research can be strengthened through a wide range of expert perspectives.

At ANZUP, translational research is a strong focus. Our goal is to bring together the many professional disciplines and groups involved in researching and treating urogenital cancers. This work is supported through the ANZUP Translational Research Subcommittee, which includes multidisciplinary ANZUP members from Australia and overseas.

Patients who participate in ANZUP trials may be asked to provide consent for the collection of blood and tissues samples. These samples may be used in translational research studies conducted in Australia and/or internationally.

Quality of life (QoL) refers to a person’s overall wellbeing. It includes:

• how well they can carry out everyday activities (physical, psychological, and social wellbeing)
• how satisfied they feel with their ability to carry out daily activities and how their cancer is being managed

Quality of life is an important concern for people with cancer. Symptoms such as pain, fatigue, and anxiety can affect daily life. Managing these symptoms can reduce distress and improve wellbeing.

Quality of Life research looks at how cancer and its treatments affect a person’s everyday life and overall wellbeing.

Many clinical trials include QoL assessments, either as part of the main study or as a separate sub-study. These help researchers understand how treatments affect patients’ symptoms, daily activities, and wellbeing, and ensure that new treatments are not only effective but also manageable for patients.

How is Quality of life measured?

Quality of life is usually measured using standardised questionnaires, often called patient-reported outcome (PRO) measures.

These questionnaires ask people about different aspects of their health and wellbeing, such as:

• symptoms (for example pain, fatigue, or nausea)
• emotional wellbeing (such as anxiety or mood)
• side effects of treatment
• ability to carry out everyday activities

Each response is used to generate a score, allowing researchers and healthcare teams to assess and compare quality of life over time or between treatments.

In clinical trials, you may be asked to complete a questionnaire before treatment (a baseline) and at different points during and after treatment. This helps track how quality of life changes.

Taking part in these questionnaires is voluntary and choosing not to take part will not affect your care.

The information collected, known as patient-reported outcomes (PROs), helps improve understanding of treatments and supports better care for future patients.

Why is Quality of life research important?

Quality of life is now a key focus in cancer care. As treatments improve, more people are living longer with cancer, and for some it becomes a long-term (chronic) condition requiring ongoing treatment. This means it is just as important to understand how treatments affect daily life as it is to understand how well they treat the cancer itself.

Understanding quality of life helps researchers and doctors:

• balance the benefits of treatment with its side effects and overall impact on patients
• identify and better manage symptoms
• develop and select treatments that are more tolerable and acceptable, supporting people to continue them

ANZUP is committed to improving outcomes for people with urogenital cancers and supporting research that focuses on what matters most to patients and their families, including quality of life.

Please talk with your doctor to find out if there is a clinical trial that may be suitable for you.