Clinical Study Basics

What is a clinical study?

A clinical study is a scientific study of a medicine to confirm whether the medicine is safe and effective for a particular disease or condition.

Why are clinical studies needed?

A new medicine cannot be approved for use in Australia / New Zealand without clinical study data demonstrating that the medicine is effective and safe for use in humans.

How are clinical studies approved?

Ethics committees, independent from the clinical study sponsor, review the clinical study procedures and decide if the clinical study is appropriate and conform to the Ethical Principles of the Declaration of Helsinki and to international Good Clinical Practice guidelines. These independent ethic committees operate according to the guidelines issued by the National Health and Medical Research Council.

Who is the Principal Investigator?

Each clinical study site is led by a doctor, identified as the Principal Investigator for the study. The doctor, with his/her team of nurses, pharmacists and other health care professions, is responsible for checking the health of the study participants at all stages of the study and completing the clinical study procedures according to the clinical study design.

What are the benefits from participation in a clinical study?

  • Gaining early access to new medicines not otherwise available
  • Obtaining clinical study medicine at no cost during the study
  • Receiving extensive medical care associated with the study
  • Contributing to the development of new medicines and new life-enhancing treatments

What are the risks from participation in a clinical study?

  • The participant may experience potential side effects from the study medicine
  • The study medicine may not work for the clinical study participant
  • The clinical study participant may receive the control medicine and not the test medicine

How can a person participate in a clinical study?

A potential participant can contact a principal investigator to see if they qualify for the study.

After discussing the clinical study design with the principal investigator the study participant will receive a full written plain language explanation of the clinical study design. The written explanation will include information on, but not limited to:

  • Eligibility criteria
  • The possibility of receiving the new medicine or control medicine
  • Possible risks and benefits of the new medicine
  • The risks of any side-effects
  • The type, frequency and risks of any medical tests or procedures defined in the clinical study
  • The participants rights and responsibilities
  • The participant right to withdraw from a clinical study at any time, without consequence to ongoing medical care.

Only after careful consideration should a person provide written consent to participate in a clinical study.

What happens if side effects occur from taking a medicine in a clinical study?

By the time a medicine has been approved for use in a clinical study, it has already been tested extensively in laboratories for likely side effects or the medicine has already been used safely in humans. However, if unexpected side effects do occur, the participant or carer is asked to notify the principal investigator so appropriate care will be provided to the participant.

What happens with the results from clinical studies?

The results are evaluated for scientific validity of the benefits and risks of the new medicine. The results may also be published in medical journals and other relevant publication. All details of the participant's treatment and individual identity are kept confidential even when the results of the study are published.