MiddletownJournal.com

How can I find out about clinical trials?

Cancer patients can contact 1-800-4-CANCER, an information service sponsored by the National Cancer Institute (NCI) that provides information on the more than 1,500 clinical trials that are NCI funded. The information is available online at http://www.cancer.gov/clinicaltrials. For broader listings of trials that are currently recruiting patients, go to http://www.clinicaltrials.gov. Patient advocacy organizations are another good resource.

What protections are there for clinical trial participants?

There is almost always some risk when you participate in a clinical trial, since many are conducted for the express purpose of determining whether or not a new drug, device or procedure is safe. However, before clinical testing or a new drug is initiated, the FDA must examine lab and animal data to estimate the potential risk to humans. If risk is too high vs. the potential benefit, the agency will stop the study. Moreover, each trial site has an institutional review board (IRB) that evaluates trial proposals for safety. Many trials also have a data safety and monitoring board or committee (DSMB/DSMC) that reviews the data from time to time and will stop the trial if it finds evidence of safety problems. Such oversight does not by any means eliminate risk, but it does ensure trials are conducted as safely as possible.

I have been asked to sign an informed consent document. What does this mean?

When you sign an informed consent document, you are attesting that you fully understand the potential risks and benefits of participating in a clinical trial. If the information in the document is unclear, ask questions to clarify. And remember, an informed consent statement is not a contract to participate in a trial; you can leave the study at any time.

Isn't the care I receive as part of a clinical trial free?

Each trial is unique. Coverage for care provided may vary so make sure the health care team explains to your satisfaction the charges for which you will be responsible before joining a trial. You should also talk to a representative of your health plan before charges are incurred. Some insurers refuse to pay for any treatment or prevention strategy deemed experimental, and many have stringent requirements that you and your health care team must satisfy before coverage is allowed.

Will Medicare pay for any expenses associated with a clinical trial?

Yes, under new guidelines issued in 2000, Medicare now pays for standard of care treatments or procedures the plan would normally cover, even if they were provided as part of a clinical trial. Clinical device trials, however, require pre-approval from Medicare Part A and Part B before a trial begins. If a trial begins before it is approved, you may be required to pay for the treatment.

How can I tell the extent to which an experimental treatment has been tested?

Clinical trials are conducted in several stages. The first stage, Phase I, looks at safety in small trials sometimes enrolling as few as 10 participants. Phase II trials enroll more participants (often hundreds) and study both safety and effectiveness. In order for a treatment to advance to Phase III, it must have demonstrated acceptable safety and produced evidence of effectiveness. Sometimes the FDA requires a Phase IV trial after a drug has been approved, in order to monitor long-term safety and effectiveness. The health care team should also be willing to disclose the extent of testing that has been done to date. Given recent concerns about drug safety, you can expect to see more Phase IV clinical trials conducted.

I want to have children at some point. How can I decide if it's safe to take an experimental medication?

Unfortunately, there is no definitive answer. In the past women of childbearing potential were routinely excluded from clinical trials, but that left large gaps in knowledge of how the medications worked, and after approval, drugs were often prescribed to such women anyway. The prodding of advocacy groups has led researchers to study drugs in broader populations. If you are considering joining a drug trial, ask if there are animal data on reproductive effects (bear in mind, however, that effects in humans often differ dramatically). Most trials of new drugs will require that you abstain from sex or use an effective form of contraception because of unknown effects on a fetus.

I've been invited to join a trial that will measure the effectiveness of stepped-up preventive measures. Will these procedures be covered by my health plan?

Probably not. Even if your health plan covers preventive measures-like an annual Pap smear-it probably will not cover additional screenings.

Why can't I know whether I'm receiving the experimental drug, the standard treatment or a placebo?

Participant expectations can influence your perceptions and make results of a clinical trial difficult to interpret. If you know you are getting a potential breakthrough medication, you may think your symptoms are abating even though they aren't. This effect-the placebo effect-can be very powerful. The only way to overcome this effect, and therefore get a truer reading of a drug's effectiveness, is to keep participants "blind" as to which treatment they are getting. Physicians and other health care professionals can also corrupt trials through bias. A doctor who expects a drug to work may interpret an MRI a bit more positively, for example. Thus, many studies are "double-blinded," with neither participants nor health care professionals knowing who is receiving the experimental treatment and who is receiving the standard treatment or placebo.

Why should I be a human guinea pig and join a clinical trial?

Participation does help advance medical science. Participation in a clinical trial, however, brings a host of potential risks and benefits. The benefits include access to new treatments otherwise unavailable and access to top medical care at elite facilities. If a treatment proves effective, you may be offered the treatment, even if you have been in the control group. There is also some preliminary evidence that suggests participants in Phase III trials have better outcomes overall than those treated with standard care. But the risks of participation are real and should not be slighted. It is important to remember that participation in a clinical trial is voluntary; you may choose not to participate at any time. You have this choice; a guinea pig does not.