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December 2000 Volume 4, Issue 2:
ASK THE EXPERTS
By Drs. Morie A. Gertz, Martha Q. Lacy and Angela Dispenzieri
Why Should I Participate In A Clinical Trial?
12.01.00

Why Should I Participate In A Clinical Trial?

It is currently estimated that no more than 2% of patients diagnosed with malignancy participate in cancer clinical trials. The purpose of this article is to discuss what a cancer clinical trial is and the advantages and disadvantages to enrollment. Regularly patients ask, “What is the best treatment for my disease?” Oftentimes they fail to realize that the answer to this question came from previous patients who had the same form of cancer and elected to participate in cancer clinical trials. There is clearly room for improvement in cancer therapies, there always are new questions to ask to address what constitutes “the best therapy for my phase of the disease.” Ideally, if an animal model existed, new agents and combinations of treatments could be tested in animal models, answers obtained quickly and applied to patients with the disorder. Unfortunately, reliable animal models do not exist, for most plasma cell disorders. In addition, studies with cancer cells growing on a plate or in the test tube do not always reflect what happens in patients with the disease. Therefore, the only way to advance knowledge and improve the treatment for patients with malignancy, is for a patient to participate in cancer clinical trials. If you receive a treatment for your disease by your doctor outside a clinical trial and you have a superb response, who will know it besides you or your doctor? The answer is no one. However, when patients are treated in the context of a clinical trial, both good and bad outcomes are reported to other doctors who are interested in your disorder. Your doctor then uses this valuable information to judge the merits of treatment directly applicable to you.

Many patients raise the issue “I will feel like a guinea pig.” In fact nothing can be further from the truth. Clinical trials are developed by panels of experts in this disease area. These individuals have a thorough understanding of the biology, natural history, and current clinical outcomes with the disease. Moreover, these doctors are dedicated to seeing improved outcomes. Think of it this way. Not only do they have the personal gratification of knowing they are helping their own patients, but also the development of a new treatment that dramatically improves patient outcome will lead to professional recognition. This too can be a strong motivator. This ensures that everything is done so a clinical trial will provide meaningful results. Oftentimes patients are concerned about treatment studies where the treatment is determined by a coin toss and they are assigned to one of two treatment arms. This often makes patients uncomfortable but it should not. When studies that involve this type of random assignment to treatment are proposed, the patient is protected by ethics committees, the federal government, and institutional review boards to ensure that the two arms of the study proposed do not expose patients to excessive, unjustified side effects. Moreover you have the assurance that no one truly knows which arm of the treatment is better. If the doctors really knew which treatment was better, they would not need to answer the question in the first place and it would be unethical to assign patients to a treatment arm believed to be inferior.

What advantages are there? New cancer treatments are coming along at an increasing rate. Someone will have to be the first to receive a new treatment. If the new treatment is not effective this is generally recognized relatively quickly and the treatment can be discontinued and other new agents considered. However, consider the first initial studies of thalidomide. Those patients who volunteered to receive thalidomide allowed themselves access to the benefits of this new agent two years before it was finally approved for use in this country by FDA. Therefore, with clinical trial participation when a new treatment is successful, you may be the first to benefit. In addition, most trials mandate that your clinical status must be carefully monitored. You and your doctor may be alerted to changes in your disease status sooner than if you had not participated in the trial. Cancer clinical trials give you a chance to receive the most advanced treatment in development. There is also a societal benefit when you take part in a cancer clinical trial. You are not only potentially helping yourself, but you certainly are providing important answers to future generations of patients who will come along and ask the same question, “Doctor, what is  the best treatment for my disease?”

The purpose of a clinical trial is to answer questions about effectiveness and safety. All clinical trials are monitored to ensure that the trial is adhering to national standards and good clinical practice. The document that describes those patients eligible for the clinical trial, the specifics of the clinical trial, and the testing involved is called a protocol. All patients participating in a clinical trial have to give written consent in a form that describes the advantages and disadvantages of participation.

We would suggest that when discussing treatment options with your doctor you ask whether you are eligible for participation in a clinical trial and where in your area clinical trials are available for your stage of the disease. You may be able to access treatments that are not generally available and in many cases the medications are supplied at no charge. You should not lose sight of the fact that you are doing a service to mankind in increasing knowledge about your disease. The National Cancer Institute has a web site that often discusses clinical trials at www.nci.nih.gov. 

Note: Drs. Gertz, Lacy and Dispenzieri part of the Mayo Clinic's Myeloma program.

Mayo Clinic's Hematology Group's web site has been changed to www.mayo.edu/mmgrg/rst/homepage.htm

You can get more infomration about clinical trials from our website.


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