Clinical Trials

• Disease prevention — using drugs, vitamins, foods to reduce risk
• Treatments — new drugs or combinations of drugs; new ways of giving treatment, new types of treatment
• Diagnosing disease — new tests or scans
• Controlling symptoms — new drugs or complementary therapies

Trials aim to find out if a new experimental drug or procedure

• Is safe
• Has side effects
• Works better than the currently used treatment
• Helps you feel better

New treatments must be thoroughly tested. A new drug, for example is investigated first in the laboratory. If it looks promising, it is carefully studied in people. There are three different types of clinical trials. These are called Phase 1, 2 and 3.

Phase 1 is the earliest trials in the life of a new drug or treatment. They are usually small trials, recruiting up to 30 patients.

About 70% of new treatments tested at Phase 1 make it to Phase 2 trials. Phase 2 trials are often larger than Phase 1. There may be up to 50 people taking part. If the results of Phase 2 trials show that a new treatment may be as good or better than existing treatment, it then moves to Phase 3.

Phase 3 trials compare new treatments with the best currently available treatment (the standard treatment). Phase 3 trials are usually much larger than Phase 1 or 2. This is because differences in success rates may be small, so many results are required to show the difference. Sometimes Phase 3 trials involve thousands of patients in many different hospitals and even different countries. Phase 3 trials are usually randomized. This means the researchers put the people taking part into two groups at random. One group gets the new treatment and the other the standard treatment.

Clinical trials are essential to the development of new interventions. 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. This is because computer simulation and animal testing can only tell us so much about how a new treatment might work and are no substitute for testing in a living human body.

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.

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 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.

You can access further information from the public stem cell resource “A Closer Look at Stem Cells” which has a section on clinical trials.

The Australian Government also has resources for learning about Clinical Trials: https://www.australianclinicaltrials.gov.au This website

• enables you to search for clinical trials in Australia,
• to understand how to enroll in a clinical trial
• the questions you should consider