Participation in a clinical trial can provide access to cutting-edge, potentially lifesaving and life-enhancing treatments.
A clinical trial is a research study that helps to determine whether a new drug or device is safe and effective. Each study is designed to answer questions and find better ways to screen, diagnose, prevent or treat a disease or condition. They may be sponsored by drug manufacturers, government, and organisations including the Leukaemia Foundation.
What happens during a clinical trial?
Each study has a plan that maps out the study–what will be done, by whom, when and why. This protocol also explains who is eligible to participate in a trial and what is expected of them. If you are eligible, a team of doctors and nurses will manage your care. Trials are held at hospitals and research centres around the country.
What are the benefits of participating in a clinical trial? Should I consider taking part?
Participation in a clinical trial gives you access to cutting-edge, potentially life-saving and life-enhancing treatments. Your participation contributes to the advancement of medicine and helps others who share or may develop your condition.
Before signing up, learn as much as possible about the trial, then discuss your options with your doctor. Clinical trials are not right for everyone, nor is every patient able to participate. Before starting any trial, you will be given a Patient Informed Consent document to read, which includes a schedule of events and explains what will happen during the study, what is expected of you, the care you will receive and the costs that you may have to cover. You will then be given a consent form to sign. Ask your doctor questions and be sure you have read and understand all aspects before you sign the Informed Consent document.
What will the cost be?
You will not need to pay any money to participate in a clinical trial, over and above what you may pay for standard treatment. Any medical tests and medical care related to the trial are also at no cost. Some studies compensate you for other expenses such as parking and travel. Private insurers often do not cover the costs related to a clinical trial, so you will need to check with your health care provider.
What are the risks – isn’t it dangerous to take an experimental drug?
The risks depend on the type of treatment being studied and the health of the individual patient. For some, there could be unpleasant, even serious, side effects. Often these side effects are temporary and end when the treatment stops. There are both known and unknown risks with any trial. Be sure you understand the known risks before you join any study.
Whilst most clinical trials involve some risk, researchers must follow strict scientific guidelines and ethical and legal codes to ensure that you are protected. Studies need to be approved by an Independent Ethics Committee (IEC) or Institutional Review Board (IRB), or an equivalent, depending upon the regulations of the country where the trial is being carried out.
This committee – made up of scientists, doctors, and other people from the local community (e.g., consumer representative, ethics expert) – reviews each study to see that it is designed to protect the patient and to ensure that the benefits of the study outweigh the risks. Each trial must meet the Good Clinical Practice (GCP) standard. GCP is an ethical and scientific quality standard that ensures that the rights, safety and well being of study participants are protected.
How long will the study last?
The length of each study is different. The trial’s protocol document will provide you with information on how long the trial lasts and what is expected of you.
You can leave the trial at any time. You can withdraw your consent and choose to leave the trial if you no longer wish to participate for any reason.
Will I still get regular medical care?
As a participant of a clinical trial, you would receive excellent medical care from a team of doctors, nurses, researchers, social workers and other health professionals who are on hand to manage your condition. The trial’s protocol may require you to visit the study site more often to check in with your study doctor. Plus you may receive more tests and treatments than usual.
What if I get a placebo?
It is very unusual for a person to receive a placebo in a clinical trial in Australia and you would be warned of this possibility before commencing a trial. As a rule, trials of drugs for cancer do not use a placebo but rather participants receive a standard treatment already in use or the standard treatment plus the drug being studied. In “randomised” trials, researchers use a computer to randomly decide who will get the experimental treatment and who will receive the standard treatment. In a “blinded” trial, neither the researchers nor you will know if you’re receiving the experimental or standard drug. The randomised system ensures the process meets the scientific requirements of the trial.
Does my doctor have to be one of the doctors involved in the trial for me to take part?
No, your doctor does not have to participate in the trial in order for you to join. Trial researchers may provide you with care or they will want your regular doctor to care for you. Whether or not your doctor participates in the trial, you will need to see him or her for general medical care. Sometimes you may have to attend a different treatment centre and have a new doctor to participate in a trial.
How do I find out if I’m eligible?
Each study’s protocol has guidelines stating who can and cannot join the clinical trial. The criteria vary by study and could include your age, gender, medical history, current health status and the particular type or stage of disease you may have. Before you join the trial, you will be asked to sign an informed consent form. Then a doctor or nurse will assess your medical history, perform a physical exam and perform laboratory tests to determine whether you meet the eligibility criteria.
Is everyone with my disease eligible?
No, only people meeting the study’s guidelines, or eligibility criteria, may join the study. If you are found to be ineligible, you should talk to your doctor to see if there is another clinical trial that may be right for you.
What would be required of me if I participate?
The doctor will first talk to you about informed consent. This is a process by which you will learn the details of the trial – what is involved, the purpose of the study, the tests and procedures that will be used, and the risks and benefits. You will then be given a written consent form, which explains the study. If you agree to take part, you will be asked to sign the form. If there is something on the form you do not understand, ask questions. Study doctors and nurses are available to help you understand the risks and benefits of the trial.
What happens at the end of the trial – will I still be able to receive the drug?
After you complete the study, you may or may not be able to continue receiving the drug. In some cases the treatment will not be made available to you again until it is government-approved. Once the trial ends, researchers analyse the data and if the study is considered pivotal and results are positive they will be submitted to the national health authority for approval. During the approval process some pharmaceutical companies choose to continue to make the drug available through a pre-approval access program.
How do I find a clinical trial?
First, talk to your doctor. He or she will be able to access an up-to-date listing of clinical trials. You may also want to call patient advocacy groups and local university medical centres. The Australasian Leukaemia & Lymphoma Group lists on its website (www.allg.org. au) currently open clinical trials in blood and bone marrow cancers including leukaemia, lymphoma and myeloma. Other Australian clinical trials sites include:
- Australian Cancer Trials
- The Australian New Zealand Clinical Trials Registry
- NHMRC Clinical Trials Centre
- Australasian Leukaemia & Lymphoma Group (ALLG)
- Queensland Clinical Trials Portal
Where can I get more information about clinical trials?
- Therapeutic Goods Administration (TGA) website includes ‘at a glance’ information and a list of frequently asked questions.
- The Cancer Council Victoria website – Clinical Trials
- The Cancer Council Western Australia website – Clinical Trials
- ABC Health Matters website – Clinical Trials a good vernacular description, and common questions
- ClinTrial Refer.
Clinical trials overseas
- Cancer Trial Treatment Options
- ClinicalTrials.gov (US)
- National Cancer Institute (US)
- ClinicalTrials.com (US)
Warren’s clinical trial story
Warren’s story isn’t unusual – prior to his 2003 diagnosis, he had no awareness of blood cancer, let alone chronic lymphocytic leukaemia (CLL). After seven rounds of chemotherapy, a bone marrow biopsy indicated he was running out of options. Until he learned of a targeted oral therapy overseas. The therapy was brought to Australia as part of a clinical trial and Warren was fortunate enough to be able to access the treatment that has kept him alive.