CLINICAL TRIALS

About Clinical Trials

The process that brings a new drug from the research laboratory to the marketplace is a long and careful one. Long, because it takes years to complete all the laboratory, animal and human tests; careful, because all those involved in bringing it to market must ensure patient safety and follow strict government regulations in evaluating the drug's safety and efficacy.

One of the most important steps in the development process is the clinical trial, which is a study with people who volunteer to receive the drug and be observed for its effects. It is through these trials that investigational drugs are tested to determine whether they can benefit human beings. But before this can happen, people must volunteer and participate - a choice that may benefit not only the individual but many, many others as well.

Whether or not you qualify for a clinical trial depends on a number of factors, including your medical condition. Your physician will discuss these factors with you and provide comprehensive information on the investigational drug and the trial itself.

Still, you may have questions. Participation in a clinical trial can be a great undertaking, and there are no guarantees that the investigational drug will work. This page, however, will help you understand some of the potential benefits and risks of participation, and give you some idea of what to expect if you decide to participate.

What is a Clinical Trial?

A clinical trial is carefully supervised research that is done in humans prior to the availability of an investigational drug to the general public.

The term clinical refers to research performed in a hospital or clinic setting in which a physician and other healthcare professionals observe a participant. A trial is a study, whereby the drug is analyzed for its effects on a group of participants.

However, before a drug enters the clinical trial phase, it is extensively tested through basic or preclinical research in the laboratory, both in lab experiments and in animals. The type of experiments depends on the disease being studied, and if animals also can have the same disease. This type of research is extremely important in gathering information on the drug's possible benefits and risks before it is tested in humans.

It is important to understand that both basic research and research done through clinical trials are carefully supervised, monitored and documented. Investigational drugs must demonstrate their safety and efficacy to government regulators (such as the Food and Drug Administration in the United States) before they are approved. Clinical trial volunteers play a very big role in this process.

Why Do People Become Involved in Clinical Trials?

The reasons why people become involved in clinical trials are as varied as the individuals themselves.

Some people have an illness that currently has no treatment. A clinical trial may offer the opportunity to try an investigational medication, which may help.

Others may become involved because their current medication has too many side effects and they want to see if they can tolerate the investigational drug being tested.

And others volunteer because they want to help find answers to scientific or medical questions that will help others benefit in the future.

Only you can decide whether or not to participate in a clinical trial. That's why you should take plenty of time to think about the trial and ask all the questions you want. You may even consider asking a close friend or family member to help you in your decision—they may have questions or concerns that you may not have considered.

Above all, remember that there are risks associated with clinical trials. And yes, it's a tough decision. Have all the facts before you make up your mind.

"I decided to enter a clinical trial because I hoped that the research would be of help to future patients."
—Clinical trial participant

What are the Risks Involved in Clinical Trials?

Investigational drugs are tested in clinical trials because not everything is yet known about them. So there may be risks to receiving the drug, which will not be uncovered until after a clinical trial begins. Your physician will discuss these potential risks with you. You should carefully weigh them against the possible benefits, and ask as many questions as you need to. Keep the following in mind when evaluating risks:

Sometimes the risks, or side effects, may seem no worse than those of your current treatment options. Other times, the side effects may seem greater.
You may react differently from another individual when given the same drug. Preclinical testing in the laboratory can often give scientists and physicians an idea of what individuals may experience in the clinical setting. But every person is different. Your physician will ask you a lot of questions ahead of time to learn as much as possible about your medical history in order to reduce the potential risks.
In addition, the risks associated with clinical trials can vary depending upon the disease being treated. These risks are carefully weighed and balanced by your physician, the study physician, the national regulatory agency (such as the Food and Drug Administration [FDA] in the US), and an Institutional Review Board (IRB)/Independent Ethics Committee (IEC). An IRB/IEC is an independent body that includes medical, scientific and non-medical representatives. The IRB/IEC reviews and approves a clinical study for safety issues and protects the rights and well-being of study participants.
If something shows up that is a risk to participants in the clinical trial, the participants will be notified by the investigator and, under some circumstances, the trial may be stopped or placed on hold until more information is known.

What is a Placebo?

A placebo is an inactive product used to compare results of the active drug being studied and to learn more about its actions. It is used in clinical trials as one of many possible treatment assignments. Because the participant and healthcare professionals usually do not know which individual has received the placebo and which has not, observations in the clinical trial can be conducted more fairly, since undue concentration on the people who received the investigational drug is reduced.

What are the Potential Benefits of Participating in a Clinical Trial?

There are a number of potential benefits from participating in a clinical trial. In general, they include:

Becoming involved in the testing of an investigational drug that would not otherwise be available.
The possibility of free study medication and study-related medical care during the clinical trial.
Contributing to medical science, which may help other patients now and in the future.

"I was always willing to try anything that was available to potentially help in finding new treatment options for the disease."
—Matt, Clinical Trial Volunteer

Are Volunteers Required to Pay for Participation in Clinical Trials?

You should not be asked to pay to be in a clinical trial. In most cases, study medication is provided free of charge.

In addition, the costs of many tests and procedures associated with the trial will not be charged to you. And in most instances, the cost of routine tests and procedures that are part of your normal healthcare might be paid under your current insurance plan or covered by your healthcare provider.

If you are considering participation in a clinical trial, discuss the potential financial implications with your physician or other healthcare professional. They can tell you exactly what will be covered as part of the clinical trial and what will be considered part of your normal healthcare routine, including that which may be your responsibility. Remember to ask everything that's on your mind. It's your right.

How Do People Become Involved in a Clinical Trial?

People interested in clinical trial participation should discuss this with their physician. It's the best way to learn whether or not an appropriate trial is available, and where it is being conducted. Potential participants are screened to determine if they are eligible for a given trial. Sometimes screening involves nothing more than a series of questions and answers. Other times it may require laboratory tests. In any case, these tests are performed only with the participant’s consent.

Not every disease will have a clinical trial currently underway. But this situation is constantly changing. Consult your physician or patient support group to learn if research is being planned which may be appropriate for you.

"I found an article on the bulletin board at the hospital...and they were eager to talk to people who were interested in potentially getting involved with a study."
—Alfred, Clinical Trial Volunteer

What are the Participant's Responsibilities?

Your responsibilities will be explained to you by your healthcare team. In general, however, you must see your study physician as scheduled and take the study medication as directed.

You may also be asked to keep a diary of your experience with the study medication; this may help give your study physician information on its safety and effectiveness. You should also contact your study physician as soon as possible if you are hospitalized for any reason.

In addition, it is important to let all of your non-study physicians know that you are participating in a clinical trial. They will need the name and phone number of the study physician in order to let him or her know about any pertinent medical information that may affect your participation in the trial. It is advisable to keep the study physician's name and number with you at all times in case of emergency.

Can People be Enrolled in a Clinical Trial Without Their Knowledge?

No. Clinical research is regulated by federal/national government and by the medical profession. You are protected from unauthorized research without your consent through a process known as informed consent.

Informed consent means that your physician and/or study nurse will thoroughly explain the clinical trial to you and may provide a package of information on the trial along with an informed consent form. The informed consent form provides information on the length of the study, potential side effects (if known) from the drug being tested, your rights to withdraw from the study, and how the study is considered completed.

The consent form will also explain how the study protocol is designed, whether or not a placebo is to be used, and what kind of tests will be required. If you don't understand any of the terms of an informed consent form, it is important that you ask your physician or nurse.

Once you are satisfied that you have a full understanding of the nature and scope of the trial and decide to proceed, and if you qualify to participate, you may decide to sign the informed consent form. However, remember that you always have the right to say no. If you are uncomfortable with what you are being asked to do in the study, or if you don't fully understand it, or if you simply no longer want to participate, do not feel like you have to participate.

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