All About Clinical Research

On this page, we will answer most of your questions about clinical research. If you have further questions, we encourage you to contact the Clinical Research Centre.

We also invite you to consider participating in a clinical trial. Click here to learn more about ongoing trials and how to participate.

What are clinical trials?

Before an investigational drug or treatment can be considered for approval, it must be shown to be safe and effective. Typically, this is done through clinical trials—carefully designed and monitored studies to test and evaluate investigational drugs and treatment plans.

Why do people participate in clinical trials?

Volunteers may be interested in clinical trials for a variety of reasons:

• Some are motivated by the opportunity to receive the highest level of medical care free of charge.

• Others want to gain access to promising drugs prior to their general availability.

• Many get involved strictly for philanthropic reasons. They want to help their children and grandchildren obtain better medical care in the future.

How are clinical trials conducted?

Researchers develop a study plan, or "protocol," explaining exactly how the trial will be conducted, what information will be gathered and what the researchers hope to learn. It describes the blood tests, X-rays and other tests that will assess how well each patient responds.

Who pays for the study treatment in a clinical trial?

The study drugs, blood work, X-rays and travel expenses are provided at no cost to participants.

How are people in clinical trials protected?

Federal regulations and ethical guidelines require researchers to follow strict ethical and scientific principles to protect participants and ensure valid results. The study plan, or "protocol," must be reviewed and approved by the organization that sponsors the study as well as by an Institutional Review Board.

Are there risks in clinical research trials?

Evaluating investigational treatments involves some risk to subjects. Side effects can vary and complications can occur. Patients are closely monitored throughout the trial, and periodic reviews of test results are done while the trial is underway. Participants are encouraged to ask questions about any concerns they may have.

What is informed consent?

Before enrolling in a clinical trial, potential participants must understand the possible benefits and risks, as well as the procedures to be used. These will be explained by research staff, who will make every effort to answer participants' questions. These issues are summarized in an informed consent document. All participants are asked to read, understand and sign this document before entering the study.

What are eligibility criteria (inclusion and exclusion criteria)?

Participants in a study must meet the eligibility criteria in the protocol, such as gender, age, health problems and previous or current treatments.

Eligibility criteria, which differ from trial to trial, exclude people who might be harmed by the study drugs (such as pregnant women) or procedures used in the research. They also help ensure valid results by limiting the study to only certain types of patients (for example, patients with certain disease characteristics).

Can clinical trial participants leave a research trial?

Yes, trial participants can leave the trial at any time for any reason.

What are trial phases?

New drugs and treatments are tested in phases:

• Phase I trials use a small number of volunteers to determine the best dose of the study drug.

• Phase II trials define proper dose ranges, effectiveness and possible side effects.

• Phase III trials involve a larger number of volunteers who are further observed for effectiveness and possible side effects.

• Phase IV trials are done after the drug or treatment has been approved. These trials determine new uses for a drug or evaluate effects on a different patient population or disease process.

Do all clinical research studies involve a placebo (a substance that looks like the study drug but contains no active drug)?

No, not all studies involve a placebo. Some Phase III trials may compare investigational therapies to the standard therapy used to treat a disease. Where there is no standard treatment, the investigational treatment may be compared to a placebo. When a placebo is used, participants are informed before they agree to take part that they may not receive the study medication but may receive a placebo instead.

What is a blinded or masked research trial?

Most clinical trials have a "treatment group" (which receives the study drug) and a "control group" (which may receive standard treatment or placebo). In a blinded or masked study, neither the participants nor the researchers are told which group a patient is in. A blinded or masked study prevents bias.

Why are participants randomized in some clinical research trials?

In trials where there is both a treatment and a control group, participants are assigned to a group by chance, called "randomization." Patients are randomized to prevent study bias.

Is a referral from my physician required for participation in a clinical research trial?

No, you don't need a referral. It is up to you to decide to participate or not. You will be evaluated by research staff, who will determine if you meet the eligibility criteria. Then the trial will be explained to you. Patients are usually encouraged to discuss the study with their physician and family.

Can clinical research trial participants still see their regular doctor?

Yes. Clinical trials do not replace normal health care. In fact, trial coordinators, with the subject's permission, may update the participant's doctor regularly.

What happens to the trial information?

Throughout the study, researchers collect information to find out how well the investigational treatment is working. The information from a clinical trial is evaluated to determine whether the investigational drug is safe and effective enough to move into the next phase of study. Sometimes results are extremely encouraging, and sometimes the treatment is determined to be ineffective or unsafe.

If a drug or treatment is proven to be safe and effective after a Phase III trial, the results will be presented for review. If it is approved, the investigational drug could become the new standard of care or another treatment option for physicians and patients. In this way, clinical trials help to bring new therapies to patients.