If you're thinking of joining a clinical trial for experimental drugs, make sure to get the answers to these questions

By GEETA ANAND
Staff Reporter of THE WALL STREET JOURNAL

Tens of thousands of patients each year enroll in clinical trials of experimental drugs and devices. Some join these studies to advance medicine for future generations, others because they hope to personally benefit from cutting-edge technology before it is available on the market. But the drugs and devices being tested may not work for you and may not be safe. So, before you put your health on the line, ask some tough questions:

* Does the doctor running your study have a financial stake in the outcome of the clinical trial for which you are being recruited?

Your clinical trial's doctor may have stock in the company running the trial, or may be an inventor of the drug or device being tested. If the doctor is an inventor, he or she may stand to earn a royalty from sales if the drug is approved. Or the doctor could be a paid consultant or speaker for the company making the experimental medicine or device. Ethics groups recommend that doctors disclose these kinds of potential conflicts, but it isn't required by any law, so you should ask the doctor.

"You ought to know the depths of the physician's involvement -- if it's just $5,000 in stock, that's one thing and probably doesn't matter. But if it's $50 million or the doctor is a co-inventor of the device, you may want to reconsider your involvement," says Mark Barnes, a lawyer who advises hospital boards and drug companies on clinical trials. A financial interest can cloud a doctor's judgment, but it doesn't necessarily mean you shouldn't participate in the trial. Indeed, "for devices in the early stages of development, the inventor often knows best how to use the device safely," says Greg Koski, former director of the Office for Human Research Protections, a part of the Department of Health and Human Services that oversees the national system for protecting people in research.

* Ask for a copy of the protocol -- the document your trial sponsor gives the doctor that describes in detail the experimental medicine and the study planned.

Your doctor will give you what is known as a "consent form," a legal document to sign agreeing to participate in the trial. The form contains basic information on the experimental medicine and potential side effects. But doctors participating in clinical trials also have a protocol, a much more detailed document prepared by the sponsor of the trial -- the company or the researcher. If you read the protocol, you will be much better informed about the potential risks and benefits of participating in the study. The protocol may detail the criteria for qualifying for the trial, as well as the criteria for being dropped from the study. It should also tell you the results of previous animal tests and human trials, the number of people expected to enroll and the facility where the trial will be conducted. You can also find the names of the doctors who are the principal investigators for the trial and more detail on side effects and potential benefits.

* Is your doctor the principal investigator in the trial? If not, who is the principal investigator?

"It is probably better to be connected to someone at the top of the trial or someone deeply involved rather than someone peripherally involved," Mr. Barnes says. This is because the principal investigator often has the most information on how the trial is going. He or she is usually informed first of reports of adverse events -- health problems that could be linked to the medicine. You want to be directly supervised by the best-informed person so that if you have a bad side effect, it's likely to be picked up as quickly as possible. If you can't get yourself in a position to be supervised by the principal investigator, you should at least get the name and contact information for this person. Then if you or your treating physician have a question, either of you can contact the principal investigator or his or her core research staff for information.

* Ask the doctor which Institutional Review Board is in charge of your trial and who the chairman and members of the board are.

The protocol, or rules, for your clinical trial had to be approved by a panel made up mostly of medical professionals. This panel is usually called an Institutional Review Board and is charged with monitoring your trial. The IRB, as it is known, may be attached to the hospital or clinic where your trial is being held. Or the IRB could be one of several regional private boards that monitor clinical trials. It's a good idea to find out the name and credentials of the IRB and the members, or at the very least, the chairman. Then, if you have questions, you can contact the chairman. And you can weigh the credentials of the board members in evaluating whether to participate.

* Is the board monitoring your study part of an accredited human research protection program?

If the board monitoring your trial is at a well-respected hospital where the investigator regularly practices, that's generally a good sign. But if the board is across the country from the hospital or clinic running the trial or at a hospital or clinic you've never heard about, you may need to investigate further.

A good question to ask is whether the monitoring board is part of a research program accredited by either of two national organizations formed in recent years in response to concerns about patient safety. They are the Partnership for Human Research Protection and the Association for the Accreditation of Human Research Protection Programs. If the board monitoring your trial is approved by either group, it is more likely to have additional safeguards in place to protect patients. But bear in mind that because the groups are relatively new, they have accredited only a fraction of research programs so far.

* If the experimental drug helps you, will you be able to continue on the treatment when the trial ends?

Even if the experimental therapy or device works, it is far from certain that you will be able to continue on the therapy after the trial. In treatments for cancer and AIDS, some companies consider making the treatments available to patients for whom standard therapies have failed. So ask your doctor whether you can continue on the treatment when the trial is finished. The trial's protocol or consent form may tell you about future access to the drug. If it isn't clear, ask your doctor for the phone number for the contact person at the company sponsoring the trial and make the call yourself.

* How quickly will you be informed whether you were given the experimental drug, a placebo or some other treatment during the trial?

When you finish the trial, it could be important to know which therapy you were given, because -- whether it worked well or failed to do so -- it might affect future decisions about your treatment. But don't assume you'll learn what you got as soon as your participation in the clinical trial ends. That's often not the case. Companies often insist on waiting until all patients have finished the trial and the data have been collected and reviewed before disclosing who was on which therapy. That means you might have to wait months or even years before knowing what therapy you were taking. "Sometimes there may not even be a firm plan in the protocol for patients to find out," Mr. Barnes says. Ask your clinical trial's doctor about this before you enroll to avoid an unpleasant surprise later.

* What will happen if something goes wrong? Who will pay?

This information may be laid out in the "consent form" you are asked to sign. But if it isn't clear, you should ask the doctor to show you where in the consent form or protocol there is information about who pays if your health gets harmed and to interpret it for you. Often, the trial sponsor will pay for your immediate care, and you or your insurer will be expected to cover longer-term costs. Also, remember that you do not give up your ability to file claims in court if your care is mishandled in a clinical trial.

* How much will participating in the trial cost?

Sometimes, it costs nothing to participate in a trial. But it could cost you tens of thousands of dollars. That's because even though companies usually supply the experimental medicines at no cost, you could be charged for tests and procedures that are required by the clinical trial. So be sure to ask the doctor running the trial to detail in advance all of the costs you will be expected to pick up.

* How does your doctor answer all of these questions?

Listen carefully to how your doctor answers your questions. Assess the depth of his or her knowledge of the trial details. Consider his or her willingness to talk to you. It is particularly important when you are taking an untested drug to be supervised by an attentive, caring doctor. "If your doctor doesn't have the time to personally explain the research," Dr. Koski says, "he shouldn't be doing the clinical trial."