About clinical trials

When researchers discover a new drug or treatment, they must test it through clinical trials. This shows whether it is:

• safe and effective for people to use
• better than the current standard treatment.

Most big advances in treatment are the result of clinical trials with people in Australia and overseas.

Your treating team may suggest you take part in a trial. Before you decide, it’s important to understand the potential benefits and risks.

Visit the websites that publish clinical trial opportunities at Find a clinical trial.

Common fears about 'new' drugs

People often think they will be a guinea pig if they take part in a clinical trial. This is not the case. 

Some trials are 'first-in-human' or Phase 1 trials. But many trials (usually Phase 1 to 3) use drugs in new situations or groups of people that are already proven safe and effective. 

For example: abemaciclib was first approved for patients with metastatic breast cancer. Further clinical trials tested its use for people with early breast cancer. We now know that using it in high-risk early breast cancer patients, after surgery, reduces the risk of the cancer coming back and helps prolong life. 

New brands of drugs also often need to go through a trials process. These days, trials often look at the effectiveness of a 'new brand' of a drug that we already know works. These type of trials (often Phase 1 or 2) are quite common. They give people access to classes of drugs already proven effective, but not yet available to the public.

Understand the different phases and types of trials at the Australian Government's Clinical Trials site.

Safety and ethics of clinical trials

All clinical trials go through an ethics review process. This process includes feedback from people affected by the health condition, to ensure the safety of patients.

Barriers to accessing clinical trials 

Only around 5% of Australians diagnosed with any type of cancer take part in clinical trials.

This is the result of several factors, such as:

• low awareness of clinical trials
• doctors don’t always suggest trials to people who may be eligible
• unequal access.

Some people may find it hard to access a clinical trial. For example:

• Trials often happen in major cities, not rural, regional or remote areas. The Australian Teletrial Program aims to help by using telehealth technology. A local doctor can help patients take part in a clinical trial under the supervision of a lead doctor from a major centre.
• People who are culturally and linguistically diverse may not have access to information in their language.

In BCNA’s Member Survey in 2017, 45% of respondents with metastatic disease felt they haven’t had opportunities to talk about clinical trials that may be of benefit. 

Eligibility for clinical trials

To take part in a clinical trial, you first need to be eligible. 

The eligibility criteria depend on what the trial is assessing. These criteria are often quite specific. Researchers must prove that the results are due to their treatment or procedure, not other factors. 

Eligibility criteria may include:

• age
• type and subtype of cancer
• stage of cancer
• previous treatment
• for metastatic breast cancer, where in the body the cancer has spread
• any other health problems (‘comorbidities’)
• use of some complementary or alternative medicines

If you are eligible, your doctor will give you information to explain: 

• what treatment the clinical trial is studying
• how this option compares with other treatments available to you
• all probable benefits and risks including side effects
• what participants need to do, and how often, such as go to appointments for treatments or tests
• if you can take part at your current treatment centre or if you have to change
• your rights – including the most important right to opt out of the trial. You can opt out at any time, for any reason, without this affecting the medical care you receive in future.

How trials are run

Clinical trials are run in public and private hospitals. They may be run by researchers from:

• universities
• hospitals
• private institutions
• pharmaceutical companies
• collaborative groups - a mix of all these researchers. 

Two groups of patients usually take part, to compare the new treatment to the existing treatment: 

Intervention group – people who receive the new treatment 

Control group – people who receive the current standard treatment. 

Participants usually can’t choose which group they are in.

Some people worry they may receive poor treatment or no treatment if they are in the control group. It’s important to know that all clinical trials are carefully regulated. All patients have at least the level of treatment they would have received without taking part in the trial.

It can take months or even years for the trial data to show how effective the drug is, compared to current standard therapy. Some trials may run for a few years so more people can take part in the trial. This leads to more reliable information.

When the results show that a new treatment is more effective than an old treatment, the new one often becomes the standard treatment. People in the control group may then be offered the new treatment.

Consent to take part

You do not have to participate in a clinical trial, even if your doctor suggests it.

If you choose to take part, you will need to sign a consent form. This is a long document that explains what is involved and your rights and responsibilities.

Take time to understand everything you’re agreeing to. If you wish, discuss it with your family and friends or ask for a second opinion from another doctor. 

You are free to leave the trial at any time without giving a reason. Your doctor will talk to you about the treatment options available to you.

Whatever happens, a trial should not affect your relationship with your doctor or the quality of your future treatment. 

Benefits of taking part in clinical trials 

There are several possible advantages of joining a clinical trial.

Access to new treatments 

You may access new treatments or new ways of receiving treatment that you could not normally access.

This might be important if:

• existing treatments are not working well for you
• a new treatment in a trial looks effective at treating your subtype of cancer, is not easily available, or is very expensive. 

Close monitoring and excellent care

During clinical trials, breast cancer specialists, research nurses and other cancer experts monitor patients closely. This can be a positive and a negative, as you may have more medical visits than for standard treatment. But you will have an excellent standard of care.

Better outcomes 

Research shows that patients treated in clinical trials usually fare better than those who are not involved.

Helping future patients

Researchers need people to take part so they can find new treatments. These will hopefully benefit you and other people in the future.

Risks and concerns of taking part

The decision about whether or not to join a clinical trial is a personal one. You need to weigh up the benefits against any potential challenges – short and long term. 

You may receive the standard treatment

You might not be in the group that receives the new treatment. Sometimes you and your doctors won’t know if you’re having the new or current recommended treatment. 

Extra time for appointments

You may need extra appointments or you may have to visit the clinic more often than for standard treatment. You may need to fill in more forms and surveys.

Possible costs 

You don’t pay for treatment you receive as part of the clinical trial. But you may have extra out-of-pocket costs, such as travelling further to the trial site. This may also affect your working hours, which can be a problem for people who are self-employed or casual. 

The treatment may be no better 

The new treatment might not be more effective than the current treatment. Your doctors will talk to you if it appears the new treatment is not working for you. They will discuss other options with you.

Side effects 

The new treatment might have more or different side effects than the standard treatment. You can leave a trial at any time if side effects become too much for you. 

Extra tests 

You may need more tests during and after treatment than with standard treatments. 

Your medical team may change

Not all medical oncologists and oncology centres participate in clinical trials. You may need to change oncologists to take part in a trial. Your regular oncologist will be kept up to date with how you are doing. You can return to them at the end of the trial.

Treatment advances through clinical trials 

Most improvements in breast disease outcomes have come from clinical trials. Discoveries include: 

• Lumpectomy then radiotherapy can be a safe, possibly better, alternative to mastectomy for early breast cancer. 
• The use of trastuzumab (Herceptin) to treat HER2-positive breast cancer. 
• Adding a CDK4/6 inhibitor, such as ribociclib, palbociclb or abemaciclib, to hormone-blocking therapy can slow the progression of HR+, HER2-negative metastatic breast cancer.
• For women with HR+ early breast cancer, using aromatase inhibitors for more than 5 years can give more protection against recurrence.
• A shorter, more intense course of radiotherapy (‘hypofractionated radiotherapy’) may be as effective as standard radiotherapy for some women aged 50 and over with an early stage cancer and clear margins following lumpectomy.