Frequently asked questions

On this page:

• Participating in clinical research studies and clinical trials
• From bench to bedside...
• What are clinical trials?
• What steps are involved and why do they take so long?
  • Step1: pre-clinical
  • Step 2: Phase I
  • Step 3: Phase II
  • Step 4: Phase III
  • Step 5: Drug/treatment registration
  • Step 6: Phase IV
• How long does it take?
• Why participate in a clinical trial?
• Who can participate in a clinical study?
• Will there be pay for our participation?
• Conclusion

Participating in clinical research studies and clinical trials

Here we hope to answer some of your questions about participating in clinical research and clnical trials. If you have any further questions please call:

The Australian Childrens' Clincial Trials Centre
(02) 9845 2358

From bench to bedside...

We often hear about the latest scientific discovery on TV or in the newspaper. It always sounds as though they are just days away from a cure. But what really happens after these “scientific breakthroughs”? How long before sick people can benefit from this discovery and why does it have to take so long? This article will help explain the steps involved in turning a new idea or discovery into a new medical treatment and how you (or your children) can be part of the journey.

What are clinical trials?

Clinical trials are the process by which new therapies or new ways of using existing treatments are tested to see if they are safe and effective. It is an important part of our health system and the only way that new drugs, medical devices or treatment protocols can be approved for human use in our country. This is a good way to protect consumers and make sure that patients are offered the safest and most effective treatments.

What steps are involved and why do they take so long?

Step1: pre-clinical

A clinical trial usually starts after researchers have spent many years developing and testing new therapies or drugs in the laboratory. The experiments usually move from the test tubes, to cells growing in culture and eventually to small animals such as mice. Sometimes there might be tests in larger animals that are closer to humans, such as monkeys. These experiments are called "pre-clinical" trials. Only once "pre-clinical" trials have conclusively shown that the treatment could be effective and safe will permission be granted to start tests in humans.

Step 2: Phase I

The next step in the journey is called "PHASE I". Phase I clinical trials are when the drug or treatment is first tried in humans. It is tested on a small number of people to determine how safe the treatment is and whether there are any side-effects. The investigators are looking to see if their new idea is feasible in humans.

The participants have to be carefully monitored (this is called the "follow up"). The participants may be healthy volunteers and all participants must have signed a consent form.

A consent form is a document that every participant must sign before they can be included in a trial. They are signing to say that they understand all aspects of the trial especially all of the possible risks and benefits. It is important that each participant signs the consent form of their own free will.

Clinical trials are voluntary and no-one should feel pressured to participate.

Step 3: Phase II

PHASE II trials are the next rung on the ladder. They are similar to Phase I trials but involve many more participants (100-300). The aim is to see if the treatment/drug is effective and to further test its safety. It is usually tested on patients with the target disease or illness.

Step 4: Phase III

The next stage is obviously called PHASE III. In Phase III trials, many more people are given the drug/treatment to confirm its effectiveness. The investigators will compare the new treatment/drug with the current commonly used therapies or with a "placebo".

A placebo is like giving someone a pretend drug or treatment. For example, the participant may receive a tablet that looks and tastes like the real drug being tested but it is really just a harmless sugar tablet. A placebo is used to test whether the effects being seen in the trial are really due to the drug/treatment itself or just because the patient "thinks" that they are being treated. The human mind is very powerful so investigators must be sure that their patients are reacting to the drug/treatment and not to the power of positive thinking! Placebos are not usually used in cancer trials.

If placebos are used in a trial then each participant must be aware of this before signing the consent form. The investigators will continue monitoring for any possible side effects and will collect information about the optimum way to use the drug or device safely.

Step 5: Drug/treatment registration

The sponsors are very happy when Phase III trials have been successfully completed. This usually means that the Therapeutics Goods Administration (TGA) will register their drug/treatment.

This gives them permission to market their new therapy to the Australian public and make it available to everyone. For large drug companies, this is a chance to get a return on the money that they invested during all those years of research. For the investigators the job is not over just yet...

Step 6: Phase IV

The final step in new drugs/treatments being fully accepted as the new standard is to convince the public and the doctors that the drug is safe over the long term. Hence PHASE IV studies are conducted after a drug has been released onto the market.

The TGA is particularly interested in being informed about any long-term side-effects or side-effects in specific groups of people. For example, women may have different responses to men or people already taking another drug may respond differently.

How long does it take?

In Australia, this process generally takes three to six years from the start of the Phase I trials to the registration of a new drug or treatment.

The time from drug discovery to the end of Phase IV, however, may be 12 to 15 years. Obviously this process is very expensive, costing the sponsor sometimes hundreds of millions of dollars from beginning to end. Just getting to Phase I following successful pre-clinical trials may take months to years to organise depending on the complexity of the treatment. It may take several years just to get the treatment or drug manufactured in such a way that it is fit for humans. It also takes a lot of planning and paper-work to get all of the regulatory documentation in place to run a clinical trial.

Investigators cannot begin human trials without going through a very strict review process. Each trial testing a new drug or treatment in humans must be thoroughly reviewed and approved by the Therapeutic Goods Administration (TGA), an approved Human Research Ethics Committee (HREC) and sometimes other speciality groups such as a Bio-safety Committee or a Gene Therapy Review Committee. These review processes are essential to make sure that each trial has been properly thought through by as many experts as possible. This reduces the risk that people may be asked to participate in trials that are unsafe or unethical.

Why participate in a clinical trial?

Being part of a clinical trial has many advantages, including:

• Access to world-best therapies at the earliest opportunity
• The knowledge that your doctor or health team is interested in always improving the treatments that they have to offer
• The knowledge that you will be very closely monitored and the therapies will be administered using a strict clinical trial protocol
• Knowing that you are helping to add to the pool of knowledge that could help sick people

Who can participate in a clinical study?

In order to predicate in a clinical trial, you must qualify for the study. Potential participants must meet certain pre-determined criteria such as age, gender, disease indication etc. Children of all ages from babies to 16 years old may participate in our studies. Some clinical trials need healthy volunteers whilst others need volunteers with a certain disease or illness.

The factors that allow you to participate in a clinical trial are called "inclusion criteria" and the factors that keep you from participating are called "exclusion criteria". These criteria are not meant to discriminate against people but to keep the volunteers safe and answer the research question most effectively.

Will there be pay for our participation?

Generally there is no pay for participation in clinical trials. This is because participation should be truly free choice and people should not be lured into something they feel uncomfortable about just so they can get paid.

It is usual, however, that volunteers are reimbursed for any expenses related to the trial such as transportation, accommodation, meals, parking etc. Trial participants do not pay for any trial related tests such as blood tests, scans, X-rays or consultations with specialists.

Conclusion

You and your child should only participate in a clinical trial when you completely understand the protocol and feel totally comfortable with the process.

There are no negative repercussions to the standard of clinical care if you choose not to participate.

Getting involved in clinical research is totally voluntary and should only be considered when you feel the benefits are greater than the risks for the trial and for your situation.