How Are Clinical Trials Designed?

Clinical trials of investigational treatments (sometimes called "experimental treatments") go through three phases:

• In Phase I trials, researchers test a new treatment in a small group of people. The goals of Phase I trials are to:
  - judge if the treatment is safe,
  - find safe dosage levels, and
  - find out about any side effects.
• In Phase II trials, the new treatment is given to a larger group of people. The goals of Phase II trials are to:
  - see if the new treatment helps and
  - further judge its safety.
• In Phase III trials, the new treatment is given to an even larger group of people. The goals of Phase III trials are to:
  - compare the new treatment to the standard treatment,
  - get more information about whether the new treatment helps,
  - keep track of side effects, and
  - collect information for using the new treatment safely.

In most Phase III trials and some Phase II trials, patients are divided into at least two groups or "arms." One group of patients gets the new treatment. This group is called the "investigational group." The treatment they get is called the "investigational treatment." Another group of patients gets the standard treatment. This group is called the "control group." The standard treatment is the treatment you would get if you did not take part in the clinical trial. Groups are compared to see which treatment works better.

In well-designed clinical trials, patients are assigned to different "arms" of the trial using a system similar to flipping a coin. In other words, patients do not choose whether they will get the new treatment investigational group or the standard treatment control group. Grouping patients by chance is called "randomization."

In some trials, patients know which arm they are in. In other trials, patients do not know which arm they are in. This is called a "masked study." In some trials, neither doctors nor patients know which arm the patients are in. These trials are called "double-masked studies."

Should I Join a Clinical Trial?

There are three main reasons why you should think about taking part in a clinical trial:

1. Clinical trials may be the best way to be sure that you get good treatment. Many studies have found that just being in a clinical trial raised patients' chances of surviving. This was true no matter which arm the patients were in.⁶
2. Clinical trials are the only way we will ever find new ways to detect, treat, and finally cure breast cancer. Right now, only 3% of adult cancer patients take part in clinical trials. The more patients that join clinical trials, the more quickly research moves forward.
3. Being in a clinical trial means that you might get a promising new treatment earlier. Of course, you can't be sure that the investigational treatment will help you.

How Can I Find Trials?

There are many ways to find clinical trials. You can ask your doctors or call the National Cancer Institute. You can also search the Internet. No one can give you a list of all the clinical trials you might join. For that reason, you should use all the tools you can.

• First, ask your doctor. Your doctor may not know of any trials for you. But talking about it will let her or him know that you are interested in clinical trials.
• The National Cancer Institute maintains a database of cancer clinical trials.
• The Internet is the fastest-growing source of clinical trials information. No one web site is complete. But you can get a lot of information from a few sources. One of the best web sites is sponsored by the National Library of Medicine. It lists all National Cancer Institute trials as well as other trials.

How Do I Know If It is a Quality Clinical Trial?

NBCCF has standards for judging clinical trials. A good trial needs at least these three things:

• Approval by an Institutional Review Board--This means that a committee has checked that the study meets ethical standards, does not put patients at great risk, and includes safeguards for patients.
• Meaningful Informed Consent--This means that you understand what you are doing and what can happen to you in the trial.
• A good design--This means that the trial is well-designed to answer an important question about breast cancer. Questions may concern how best to prevent, detect, diagnose, or treat breast cancer.

Remember that for many clinical trials, doctors and informed consent forms can't tell you what might happen to you. The purpose of the clinical trial is to see if a new treatment works better than the standard treatment. Sometimes new treatments don't work as well as the standard treatment. Or the new treatment may have serious side effects. This is why it is important that clinical trials are watched closely.

For more information, contact NBCCF and ask for a copy of our "Criteria to Evaluate Quality Clinical Trials." Do you need help judging the quality of a clinical trial? An NBCCF-trained advocate may be able to help you. NBCCF's trained advocates are not medical professionals. They cannot give you medical advice. But they may be able to help you sort out the details and make an informed choice. Contact NBCCF for more information.

What Will It Cost to Take Part in a Clinical Trial?

The trial sponsor usually pays for the cost of the investigational drug or other treatment that is being studied. The trial sponsor may be a drug company or the National Cancer Institute. In addition, routine patient care costs of qualifying clinical trials are now covered by Medicare. Routine costs include all items and services covered by Medicare and typically provided outside clinical trials (such as doctor visits, and routine tests), in addition to items or services associated with the investigational treatment (such as treatment administration, monitoring for treatment effects, and prevention of medical complications.) Patient care costs due to medical complications (side effects) associated with clinical trial participation are also covered by Medicare. To determine whether a particular clinical trial qualifies for Medicare coverage of routine costs, contact the Centers for Medicare and Medicaid Services.

There may be other costs involved in taking part in a clinical trial, and sometimes patients must pay for some or all of these costs. This is often because their health insurance or health plan won't pay for them. And sometimes health plans only agree to pay these costs under certain conditions. For example, your health plan may cover the costs for your blood work and other tests. But it may only cover them if you have the tests done at a certain care center. This may be a problem if the care center where you must have your tests done is far away from the place where you go for the clinical trial. Sometimes patients can work out a deal with the trial sponsor so that they will cover the costs of the blood work and other tests at a more convenient location.

Several states require health insurance companies to cover routine patient care costs associated with clinical trial participation. To see if your state has such legislation, contact the National Cancer Institute for their publication called, "States That Require Health Plans to Cover Patient Care Costs in Clinical Trials."

NBCCF thinks all health insurance companies and health plans should pay for the costs involved for patients to take part in clinical trials. Whether you will have to pay for some of the care you get in a clinical trial depends on a lot of things. It depends on:

• who is sponsoring the trial,
• what your health insurance or health plan covers,
• how your doctor gets paid for your care, and
• many other things.

So it is important that you find out what costs related to the clinical trial your health insurance or plan will cover. Ask your doctor or nurse to explain exactly what the trial sponsor will cover.

6. Gnant M. Impact of participation in randomized clinical trials on survival of women with early-stage breast cancer: an analysis of 7985 patients. Proc ASCO 2000; 19 (Abstract 287).