JDRF - Juvenile Diabetes Research Foundation

Put simply, a clinical trial is a scientific study of a new medical therapy in humans.

Clinical trials are usually the last stage of the research process, capping off years of laboratory and animal research. Their ultimate aim is to obtain a realistic picture of what would happen if a medical treatment was available to the Australian public.

Three different phases

Whilst clinical research can be conducted into any aspect of the disease process – from preventing the disease to identifying people at risk or stopping complications – all new therapies or medical protocols must go through a strict three-phase process before your health care professional can recommend them to you.

A Phase 1 trial evaluates the best way to use a new therapy and collects vital information on how it impacts humans including how safe it is. As the first step out of the laboratory, these trials usually only enroll a small number of patients that fit a strict selection criteria. They are often conducted in closely monitoring surroundings such as a hospital or clinic.

If a phase 1 trial shows the therapy is safe it moves to Phase 2. These trials usually involve a bigger range of people to identify long term safety and effectiveness under different circumstances (eg is the treatment suitable for people of a certain age or disease stage). They are also used to determine different dosing requirements.

The final stage is a Phase 3 trial which often involves the new treatment being compared to an existing treatment to check that it is useful and worthy of being made widely available. These are trials are often national or international and may involve thousands of people.

It all takes time

According to JDRF Head of Research Development, Dr Dorota Pawlak, the different phases of the clinical trial process can take up to ten years to complete.

“Researchers can and do check the effectiveness of a treatment in the laboratory, but the truth is no one really knows what effect it will have on humans until it’s been taken out of the lab and tested on them.”

“Even if everything goes to plan, the clinical trial phase will usually continue for years after lab research has finished.”

Should I participate?

Without volunteers, trials would not go ahead and the potential new treatments, preventions and cures would never make it to market.

Most clinical trials will recruit people within a specific set of criteria to ensure the best and safest outcome.

“Safety is an issue with any experimental procedure, but approved clinical trials will always be designed to minimise risk and maximise health benefits. In most cases, trial participants receive excellent health care whilst on the trial.”

“There are a number of benefits to joining a clinical trial. For some, the biggest benefit is gaining access to new or expensive treatments or specialized care. For others, knowing they are contributing to life saving research is reward enough.”

Important questions to ask before joining a clinical trial

There are a number of important questions you should ask before considering clinical trial participation.

• Has this trial been approved and registered by an appropriate governing body?
• Why is this trial important and what will the results mean to me?
• What are the possible risks and side effects? Will any side effects happen now or years later?
• What will taking part involve (time off work, medical tests, special diets etc)?
• Is there any travel required and will I be reimbursed for this?
• How will my privacy be protected?
• Will the results of the trial be made available to me and will I be able to find out what treatment regime I was on?

JDRF and the Clinical Trial Network

The recent Australian government commitment of $5 million to a Clinical Trials Network is a strong first step that has been applauded by the type 1 diabetes community. However, delivering increased access to clinical trials will require significantly more investment. JDRF forecasts that $35 million is needed to establish this innovative clinical trials model, which will have comprehensive linkages to Australia’s research landscape as well as international projects.

The case for support is strong, with positive return on investment for the health system and increased international investment in local research some of the economic benefits. The human rationale for access to the latest treatments and technologies is more powerful again. For people with type 1 diabetes, the CTN will enable easier identification of suitable clinical trials, allow participation in a wider range of trials and accelerate the research process to allow therapies to be available sooner.

To help JDRF’s efforts to secure the funding needed for the Clinical Trial Network, join now to become an official JDRF Advocate.