Clinical Trials 101

Many people who struggle with illness wonder what experimental therapies are available to them. Researchers (e.g. doctors and scientists) are constantly trying to discover more effective and/or safer treatment options for patients. In order to benefit from the latest research therapies, enrolling in a clinical trial may be an option for you. This article will provide answers to questions you might have about clinical trials.

What is a clinical trial?

A clinical trial is a research study that uses human volunteers to study the safety and effectiveness of a certain medical intervention. Examples of medical interventions being studied include:

  • A drug
  • A medical device (e.g. pacemaker)
  • A procedure
  • Behavioral change (e.g. daily exercise)

Medical interventions may be novel (never used before) or they may be already on the market but are being studied in a different way.

Researchers want to make sure that the intervention they are studying is equal to or better than what is already available. They do this by comparing the intervention to a control group who receives current medical treatment or a placebo. Then they measure outcomes in the participants to evaluate how safe and effective the intervention is.

Why are clinical trials done?

Clinical trials are the key tool that doctors and scientists have to study new and potentially life saving therapies. Not only do they add to the existing medical knowledge but they can lead to better treatments for patients in the long run.

Researchers may have certain questions that can be answered by a clinical trial. Examples include:

  • Is the intervention safe?
  • Is the intervention effective?
  • Is Drug A better than Drug B?
  • What dose of Drug A is the most effective and safe?
  • Does the intervention still work when used in a different population (ie the elderly or children)?
  • Can a certain intervention already in use be used to treat another disease?
  • What is the preferred method of taking Drug A? Orally or by injection?

Who can participate?

Each clinical trial will have a different set of criteria for who can participate in the research. Some trials are interested in healthy volunteers while others recruit those with a particular illness.

  • Other factors that may be considered include:
  • Age
  • Gender
  • Severity of illness
  • State of health
  • Current and past treatments.

You will be required to discuss your eligibility with the researcher prior to participation in the study.

Who runs the trial?

A team of researchers works together to carry out the trial. The team leader is a principal researcher who is often a medical doctor. The research coordinators (also called research assistants/associates) are in charge of managing the day-to-day work of the project. Additional members of the team may include doctors, nurses, pharmacists, social workers and other health care professionals.

Trials are funded by a variety of different sources and may include: pharmaceutical companies, medical centers, government agencies, not-for-profit organizations, as well as individual citizens.

Where does it take place?

The location of the study is specific to each clinical trial. Common sites include hospitals, medical centers and universities.

What does it cost?

You will not be required to pay for the cost of the drug or intervention once a participant in the trial. The costs to you are indirect such as travel costs, parking, childcare and time away from work. Some trials may provide reimbursement and it is important to discuss this prior to signing up.

Do I have to change doctors?

Clinical trials will generally have their own health care team who will look after your needs specific to the research. They typically do not assume responsibility for your other health care needs.

Are clinical trials safe?

Participants are protected on a number of different levels when they sign up for a clinical trial.

  1. Informed consent – Informed consent means that before agreeing to participate, researchers provide individuals with information about the clinical trial including the risks and benefits to participation. This way, patients can make the decision that is best for them. Informed consent also means that individuals can decline or withdraw at any point in the research process if the study is not right for them.
  2. Institutional review boards – Researchers must submit comprehensive documentation and receive approval from a local ethics board (usually affiliated with a hospital or university) before the clinical trial can begin. The ethics board is composed of physicians, researchers, ethicists and community members who review the project to ensure that participants’ rights, safety and wellbeing are upheld.
  3. Government – the government is responsible for inspecting and regulating clinical trials. In Canada, Health Canada ensures that participant’s safety is protected. In the US, the Food and Drug Administration (FDA) oversees clinical trials.

What are the advantages of participating?

By participating in a clinical trial, you may receive a cutting-edge intervention that has the potential to improve your health. You may also benefit from the expert care and additional medical work-up you receive as part of the study.

By being a part of research, you are part of forming the future of medicine, which can be of great help to other people in a similar situation as you.

What are the disadvantages of participating?

Although clinical trials try to minimize the risk to the participants as much as possible, there is always some level of risk involved. You may experience side effects to the drug or intervention being tested.

Each individual has to weigh the risk and benefits for themselves before making a decision about participating in the trial.

How do I get involved?

If you are aware of a clinical trial that you would like to learn more about, it is a good idea to discuss it with your family physician and/or specialist physician. Your health care professional may not be aware of the particular intervention so it important to bring detailed information to your appointment. Have a discussion with them about whether participating in a clinical trial is right for you.

Be sure to contact the research coordinator or contact person assigned to the clinical trial of interest to you. Don’t be afraid to ask questions about the research, remembering that your health and safety come first.