The Cure In a Clinical Trial?

One of the reasons some people consider participating in a clinical trial when they normally wouldn't is because they have a condition that has not responded well to existing treatments. Frustrated with the therapies they have tried or desperate as their condition worsens, the prospect of a cure becomes particularly encouraging. This prospect leads some to seriously consider enrolling in a clinical trial of a new therapy, even when they would not normally take the risks involved because the only way to get the experimental therapy is to participate in the clinical trial.

What risks? Every new therapy carries with it two types of risks: those that are anticipated and those that are not. If the therapy (let's say it's a drug) involves a compound that has been used in people or animals before, for other purposes, then many of the risks (side effects) are known or can at least be anticipated.

If the drug is new, then its effects on people are not yet known, so most of the risks are unknown. Just because a risk is known, doesn't mean it's insignificant. All it means is that it is somewhat more feasible to weigh the risks of taking the drug against the possibility that the drug may ease, or even cure, the condition.

The most uncertain situation involves a truly new compound. In such cases, there is some reason to believe that the compound may help, otherwise it would not make sense to conduct or enroll in the research as a therapy. But the certainty of the compound's effectiveness is very low. At the same time, the safety of the compound is also uncertain. The participant doesn't know whether the therapy will work and doesn't know how much damage the therapy may do.

Is it ethical for the medical community to make a new treatment available only to clinical trial participants?

By definition, a new treatment has not yet been tested, so its safety and effectiveness are unknown. It would not be ethical to market an untested treatment. In addition, it is particularly important to measure the effects of the experimental treatment on individual patients as well as the patient population at large in order to make the case for or against the use of the treatment for a particular condition. And the only way to do that is within the carefully-controlled conditions of a clinical trial.

If someone's condition is bad enough, if their prospects are dim and their symptoms are intolerable, then taking the virtually unknown risks of trying a treatment that may or may not show any effectiveness may be a reasonable course of action. But assuming such risks for unknown benefits is probably not wise for someone who has other - any other - medical options.

-Joe

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Is the Consent Form a Legal Document?

If you have ever considered volunteering to participate in a clinical trial, you know how much paperwork is associated with the Informed Consent process. Hopefully, the research was explained to you by a physician or nurse who also answered any questions you may have had.

Then you were probably handed a daunting document - the Consent Form - to sign. Chances are the Consent Form was quite long, detailed, and did not exactly read like WebMD magazine or USA Today. You may or may not have taken it home with you, read it carefully, and asked questions of the study team or other experts you may know. At some point, if you were enrolled in the clinical trial, you signed the Consent Form.

What exactly does that signature convey and how much legal weight does the Consent Form carry?

While the careful and formal way in which Consent Forms are presented to potential participants may suggest that they are binding legal documents, they're not. All your signature on a Consent Form documents is that you did, in fact, acknowledge that you understand the study and voluntarily agree to participate in the study. It does not compel you to participate. It does not mean you can't change your mind. It does not prevent you from withdrawing later on.

In short, your signature on the Consent Form documents your voluntary participation. It does not legally require you to participate. It just seems a lot more official than it really is.

-Joe

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Should Pregnant Women Be In Clinical Trials?

It's a question that, for years, seemed to have a perfectly obvious answer: "No! Of course pregnant women shouldn't be in clinical trials. They're, well... pregnant, for god's sake! They're probably not in any condition to undergo the risks and rigors of research. Not to mention that subjecting a fetus to the unknown effects of a new drug seems completely unethical!" And let's not underestimate the advice given by company attorneys to avoid the liability for damage done to these women and their unborn babies, even in the most responsibly designed and conducted studies being monitored by the Food and Drug Administration.

That hands-off approach has, for the most part, been abandoned.

Why? What led to the change of philosophy in a relatively short time? It's not due to any less concern over the safety of research subjects. In fact, it's due primarily to a greater concern for the safety of patients.

Back when pregnant women were not included in clinical trials, it was noted that recently-approved medicines were being prescribed for adults - all adults, including those who were pregnant! As a result, pregnant women (and their fetuses) were being exposed to drugs which were never tested for safety or effectiveness in pregnant women in the first place. And that seemed even more unethical than the status quo.

Federal regulations now call for testing of drugs and devices in the populations in which they are anticipated to be used. This may pose somewhat greater risk to clinical trial participants, but it is intended to reduce the risk to many more patients once the drug or device is approved and marketed. Better to be looking for side effects in a population under careful control than in the population of patients at large.

-Joe

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When It's Time To Call It Quits

Sorry for the gap - I've had that sinus-and-chest-and-stomach thing that's been going around lately. But I'm much better now!

One of the questions asked by many clinical trial participants involves terminating their participation. "Can I quit if I want to?" The answer itself is simple: "Yes, if it's safe."

Just as participants voluntarily enroll in a clinical trial, their continued participation is likewise voluntary. No one can be forced to remain a research subject against their will. But no one should withdraw from a study unless it is safe to withdraw.

Individuals thinking about volunteering for a clinical trial should carefully consider why they would want to enroll. Similarly, once in a study, participants who wish to withdraw should be sure they are doing so for good reason.

The best way to examine the pros and cons is to ask the study team. Ask them what would be involved in quitting now, how your health might be affected and what alternatives there might be to quitting. Sometimes a simple lack of communication is responsible for a participant's dissatisfaction with the way a study is going. Talking to the research team may clear up a misunderstanding - which could lead to a change of heart or reassure the participant that quitting is in fact what they want to do.

But the next step is by far the most important: Assess the safety of quitting at this time. What are the risks to your health if you stop? Would it be safer to gradually withdraw than to withdraw all at once? These questions should be asked of the study team, but they should also be asked of, and considered by, your physician. Your physician is in the best position to advise you on these questions because your physician's primary interest is in your health.

So, if you think it's time to call it quits, find out what would be involved, check out the alternatives and, most importantly, figure out whether it is safe! The decision is yours.

-Joe

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How Would YOU Feel?

Let's say you were diagnosed with breast cancer or lung cancer. And let's say you decide to participate in a clinical trial testing a new therapeutic compound - an antibody - that would work by disrupting the blood supply of the tumor cells in your body. You feel it's quite a risk because it's not known for sure whether or not the therapy will work and, even if it does, what the side effects will be. But you convince yourself that, in addition to the possibility that the new therapy might help you, deep down you also want to help future cancer patients by volunteering for this study.

You're nervous and uncomfortable, but you are enrolled in the clinical trial. Your cancer progresses, very slowly, causing you to wonder whether you're doing the right thing. Maybe you should try a very aggressive approach that is already accepted as the standard. Yes, the side effects will probably be unpleasant, but at least you know what to expect and you have some idea of how effective the treatment will be.

The clinical trial is finished. Your participation has ended. You're relieved because your anxiety has diminished, but you still wonder whether or not you made the right decision. Then you decide that you DID make the right decision because you helped contribute to medical science. Others will benefit because you volunteered to take a risk.

Then you find out that the company that sponsored the clinical trial is planning to charge a lot for this new treatment. A lot. Like, over $100,000 per patient! How would you feel about your "contribution" then?

Last week the New York Times reported that Genentech, Inc plans to charge as much for its cancer compound Avastin. Let me know what you think.

-Joe

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