Frequently Asked Questions About Clinical Trials

How do I get involved?

Every clinical trial has criteria that patients must meet before they can be enrolled. Considerations of eligibility for a study can require specific information about the person’s condition, previous treatment (e.g., tumor type, stage, age, gender, medical history and medications regularly taken) and additional criteria that may not apply to you. Have your health care provider make the initial contact with the group performing the trial to get the complete list of eligibility criteria.

Are clinical trials safe?

All clinical trials must be approved by the federal government and adhere to strict regulations protecting the rights of participants. To help ensure the relative safety and effectiveness of the treatment, each clinical trial must undergo preclinical testing (e.g., cell studies and animal testing) before large-scale testing in people may begin. However, as with any treatment, there are inherent risks:

• The new therapy may not be as effective as previous research indicated.

• There may be unexpected side effects.

• The study may require more time and effort than the standard treatment.

What are phases of clinical trials?

There are four phases of clinical trials.

• In phase I clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time.  They evaluate safety, determine a safe dosage range and identify side effects.

• In phase II clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.

• In phase III studies, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.

• In phase IV studies, the post marketing studies delineate additional information, including the drug's risks, benefits, and optimal use.

If someone is in a phase III trial and a clear advantage results for the participants in the other group, what happens?

If early results show a clear advantage for one of the groups, the sponsor of the study could choose to end the trial early or allow a crossover.  A crossover involves  those not receiving the more effective treatment  to receive it.

What happens if someone wants to stop participating in a trial?

Before joining a clinical trial, participants are given full information about the trial and then asked to sign an informed consent document stating that they understand what is involved.  Under the informed consent process, a person has the right to discontinue participation in a trial at any time. A participant's decision to leave a clinical trial does not jeopardize future treatment, and the participant will have a chance to discuss other treatments or care with a doctor from the trial. The person may be referred back to his or her primary doctor for standard care.

ould I get a placebo (“sugar pill”)?

Clinical trials for cancer are designed to compare a new treatment to the one currently being used as standard therapy. If you choose to enroll in a clinical trial, you will be placed in a control group or in the experimental group to receive the trial drug. The very least you will be offered is the standard treatment already in use. When there is no standard treatment, a placebo may be used.

Why are there eligibility criteria?

Meeting criteria helps protect the participant's safety. Some people have other health problems that could be made worse by the treatments in a study.

Trial participants must be as similar as possible so that doctors can be sure of the study results. For example, some clinical trials will not take participants who already have been treated for cancer. Otherwise, doctors could not be sure whether the participant's results were due to the treatment being studied or the earlier treatment.

Remember that the treatment being tested in the clinical trial is believed to be as good as the standard treatment, but no one knows whether it is better than the standard treatment.  That is what is being studied.

Why don't all doctors tell people that clinical trials are an option?

Health care professionals sometimes do not refer people to clinical trials due to one or more of the following.

• They are unaware of available trials.

• They are unwilling to lose control of a person's care.
  

• They believe that standard therapy is best.

A physician's decision on any treatment question is based on what he or she believes will be best for the patient. However, every patient has the right to consider all possible options and to make a personal decision on what is best.

If someone agrees to participate in a cancer treatment trial, will the cost be any more than the standard treatment? Will the person's health plan cover the costs?

Two kinds of costs are associated with clinical trials, patient care costs and research costs.

The trial's sponsor usually covers research costs. Patient care costs may be covered by a participant's health plan. They include doctor visits, hospital stays, laboratory tests and x-rays, all of which are part of participating in a trial or receiving standard treatment. Clinical trial participation sometimes involves the requirement of additional tests that might not be covered by either patient care costs or research costs.

Although some health plans cover clinical trials, others will not reimburse costs for "experimental therapies." However, decisions often are made on a case-by-case basis, and costs for some treatments are reimbursed. Some clinical trial sponsors make a special effort to work with health plans to reimburse costs.

In considering options, interested persons should ask trial personnel what the experience to date has been in the trial they are considering. They should ask specifically about the type of insurance involved. People may even be able to get examples of how their employer or managed care plan has responded.

What happens to clinical trial results?

After a clinical trial is completed, researchers analyze the collected data and make decisions about further testing.
After a phase I trial is completed, researchers decide whether:

• There is enough data to support further study with a phase II trial, or

• Further research will not be conducted because the agent was not safe.

After a phase II trial is completed, researchers decide whether:

• There is enough data to support further study with a phase III trial or

• Further research will not be conducted because the agent was not safe or effective.

After a phase III trial is completed, the researchers must look at the data and decide whether the results have medical importance. When the analysis is complete, the researchers will inform the medical community and the public of the trial results.

In most cases, a trial's results are first reported in peer-reviewed scientific journals. But if the results have significant medical importance, a public announcement may be made while the formal report is being submitted to ensure that patients can quickly benefit from the new advancement. Particularly important results are likely to be featured by the media and discussed at scientific meetings and by advocacy groups.
Once an intervention is proven safe and effective in a clinical trial, it may become the new standard of practice for physicians.

Are people who don't have cancer eligible to participate in a clinical trial?

Three types of cancer trials are open to people without cancer:

• Prevention trials

• Early detection/screening trials

• Diagnostic trials

October 2007