Clinical Research

Clinical research is research that involves human subjects or material (e.g. blood, tissue) of human origin. Implicit in the concept of clinical research is a direct interaction between the investigator and the human subject.

Clinical Trials

A clinical trial is a particular type of research study that aims to evaluate the effectiveness and safety of medications or medical devices. Government agencies, such as the FDA, typically approve or disapprove new treatments based on the results of clinical trials.

Clinical trials offer patients an opportunity to receive promising new therapies that would otherwise not be available.

Clinical Trial Phases

Clinical trials often proceed in three steps or stages, referred to as Phases I, II and III. Each phase aims to provide answers to different questions about a potential treatment.

A Phase I trial is also sometimes described as a “first in human” study; the trial represents the first occasion in which a particular drug or device is tested in humans. Often, very little is known about the safety of a drug or device in humans prior to a Phase I trial. Consequently, drugs and devices in Phase I are typically given to only a small number of patients. The primary aim of a Phase I trial is to find out if the treatment is safe. A Phase I trial will often examine different dosages of the experimental drug to find a safe dosage range. If a drug is found to be safe during Phase I, then investigations will proceed to Phase II.

Phase II trials involve a larger number of people and are designed to learn more about the safety of a drug, to begin to understand the effects of the drug on the body (sometimes called pharmacodynamics) and to learn more about how the body metabolizes and eliminates the drug (also called pharmacokinetics). The hope is that Phase II trials may also provide early evidence for the efficacy of the drug (i.e. is there any sign that the drug has the intended effect in terms of improving symptoms or longevity).

Phase III trials are typically randomized and placebo-controlled. They often take place simultaneously at multiple sites and involve large numbers of patients. The primary goal of a Phase III trial is to demonstrate that a treatment is beneficial, or more beneficial than any existing therapy. Phase III trials also provide important information about drug safety and tolerability in a larger number of people. 

Participating in a Clinical Trial

The decision whether to participate in a clinical trial is often a complicated and difficult one. Every person has unique, concerns and priorities, the decision to participate in a clinical trial should be made after careful discussion with the investigator, your physician and your friends and family.

Perhaps it is most important for you to understand the goals of a trial and the potential risks and benefits that may result from your participation. For example, what is already known about the safety and tolerability of the study drug? What are the most common side effects; is there any evidence that the drug may be effective? Will the drug be made available to you once your participation in the trial is over?

You should also clearly understand what will happen during a trial and what you will need to do. Is there a placebo? What proportion of people will receive placebo versus active drug? How will the drug be administered? How often will you need to take the drug? For how long will you need to take the drug? 

You should certainly explore all of your options. If you choose not to participate in a trial, what are the other therapeutic options? If there are multiple trials for which you may be eligible, what are the relative pros and cons of each trial? It will rarely be possible for you to participate in more than one trial at the same time, and it may not even be possible to participate in two trials consecutively. These are questions you should ask the study investigator.

Risks and Benefits

Most clinical trials offer both benefits and risks that you should weigh in your mind as part of your decision whether to participate in a trial.

Here are some of the general benefits to participating in a trial:

• You may have access to a treatment that is not otherwise available (remembering that if the trial is placebo-controlled, then participation in the trial does not guarantee that you will receive this treatment).
• You contribute to scientific knowledge and research, which could help other people who have the same condition.
• Participation in a trial typically involves regular and rigorous follow-up, which means that you may get more attention from your health care team and closer monitoring of your wellbeing and potential side effects than you might receive if you choose not to participate in a trial.
• You may receive the study drug and the care provided during the study either at a reduced cost or at no cost at all.

There are also some downsides to participating in a trial:

• The new treatment may cause unknown side effects or have other risks; this is particularly true for early phase trials.You may not have a choice about which treatment you receive. Many trials are placebo-controlled, so you may not receive the new treatment.
• The new treatment may or may not work, even if you receive it (rather than the placebo).
• Regular follow-up is typically required and the time and travel may be inconvenient. 
• Your insurance may not cover all of the costs associated with participating in a clinical trial.