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Clinical Trials:
Sometimes Good, Sometimes Not
A Very Difficult Decision

by Jules Reichel

 

I am the Secretary of the URF Board and a patient of Dr. Catalona. He performed my radical prostatectomy in September 1997. Iíve written in Quest for over 5 years. I study, write, counsel, and lecture, but I am not a doctor.


An Experimental Approach

Quest readers know about the enormous progress in effective screening for prostate cancer.

Advances have also been made with prostate cancer therapies including: early use of surgery, or radiation, or surgery followed by radiation (especially in the adjuvant setting Ė i.e. before much rise in PSA).

Other therapies, especially hormonal treatment, can also be very helpful for some patients.

If a patient is satisfied with his care and it is working, there is certainly no reason for change. But when therapies are not working or when conventional therapies can be predicted to have outcomes that are poor, the patient should consider the possibility of experimental treatment in an environment where good science is combined with good patient care.

The formal, government approved version of an experimental approach, especially with drug therapies, is a clinical trial. I know patients who had life-saving success from clinical trials. However, itís important that patients not be encouraged to ignore the risks. Great caution is needed when a person may be transitioning from being a patient to be cured, to being a test subject for science.


The Controversy

Clinical trials have varied structures but most patients encounter them as drug-company sponsored tests.

To provide for patient safety, the clinical trial is divided into 3 or 4 phases.

The first phase uses few patients and looks for best dose and least side effects.

Phase II trials are performed on larger groups and focus on effectiveness as well as continuing the safety studies of Phase I. It is common for drugs to fail in Phase II.

Later is Phase III with its randomized multicenter testing.

Phase IV studies gather data after approval by the United States Food and Drug Administration. It takes a drug company about 10 years to develop and test a drug, and it takes at least another 5 or more years to obtain FDA approval. A 17-year development cycle is a typical number. Very few drugs that are given to patients in clinical trials are ultimately approved.


Questions And Answers: Some of the issues

Q#1: If I am going to try a new drug, arenít I better off in the hands of trial experts?

Ans#1: ďNo strong evidence was found for a harmful or beneficial effect of participating in a Randomized Controlled Trial compared with receiving the same or similar treatment outside such trials.Ē (British Medical Journal, May 2005)

All doctors are not able to treat patients using new drugs and many send their patients to the trial for this reason. However, other doctors can indeed deliver such treatment outside of a trial.

Q#2: Wonít I get the benefit of free trial drugs; whereas, my insurance company may not pay for experimental drugs?

Ans: #2: Drug companies usually supply drugs for trials at no cost. They do not, however, pay for many other medical costs of treatment such as: doctors, hospitals, or transportation.

Insurance company payments are hard to understand. However, major cancer centers seem to be able to get drugs and tests covered outside of a trial that would otherwise be left to the patient.

Q#3: If I happen to succeed with the new drug, wonít I have access to a life saving drug before others know about it?

Ans: #3: You might indeed have benefit. But a lot of hurdles are in the path of such success. Being transformed into a test subject and not a patient is a huge loss. The doctor/researcher has the conflict that heís trying to find scientific truth and also treating a patient. Thatís not the same thing. Two examples of resulting troubles are:

  1. Even though you are succeeding medically as a patient, you can be encouraged to leave the trial because your extent of benefit is not good enough to meet the scientific plan (called a protocol) that was submitted prior to the trial. Thatís enormously disheartening.
  2. You may be one of those who fully succeed at the trial. You will get well by using the drug. Then, you learn that drugs for trials are produced in a lab as a one-time effort. It is not a production drug that you buy in the pharmacy. If the overall trial was not successful, the drug company may never produce any more of the drug and you canít use it because it doesnít exist.

Q#4: Despite their shortcomings, arenít clinical trials an effective way to prove that new drugs are useful?

Ans: #4 Iíve been emphasizing the problems of the patient, but clinical trials offer many problems for researchers as well. One of their more obvious problems is that itís hard to recruit participants.

It is then statistically impossible to be confident that their claimed benefit is true. Soon enough the researchers resort to combining data taken elsewhere (this approach is called a meta-analysis). Itís what critics call an underpowered trial, and is of less value.

The FDA approved only 20 drugs in 2006. Prostate cancer has had only one approved drug that is thought to extend life.

It is undeniable that clinical trials follow the methods of science and minimize the risk of false results. However, itís hard to say that they are effective when they are so expensive, slow, and unresponsive to patient needs for drugs that work.


Observational Trials

 

Trials run in two ways. One is the clinical trial, and the other is an observational trial in which a doctor, unconnected to a clinical trial, administers drugs to a patient and observes the correlation between the drugs that he gave and the health status of the patient. An observational trial may introduce errors because the doctor may overlook the real causes of the disease.

However, observational trials are quick and in the era of rapid Internet communication, itís not easy to understand why a patient should have to wait forever for the results of clinical trials when panels of reliable expert doctors can offer the same life-saving choices based on their clinical practice.

Still, the clinical trial paradigm is strong and no change is likely any time soon. The patientís best alternative option is then to use a major cancer treatment center where, at least, the huge staff at the center can share patient experimental information and the results from their own practice.


My Bottom Line

It is not correct to say that patients shouldnít participate in clinical trials. It is an available option and sometimes your doctor says that he has no more options for you, or that he believes that it is your best option.

Iím suggesting caution only because these widely hyped trials have risks that are much higher than patients imagine and successful results have been very limited.

Dr. Mark Moyad (University of Michigan) wrote his opinion of clinical trials. I agree with him and I have cited a small fragment of his views below:
ďOne must admit that thousands of men have volunteered to be part of clinical trials because it (was) the right thing to do and it represented hope to us and themÖ They trusted that their doctors would do the right thing for them and future patients and this is how and why many of them were recruited to be in these trials.
ď(Let us remember) the thousands of patients that we have recruited in clinical trials because we sold them on hope, and for what, only to see one drug approved by the FDA (for hormone refractory prostate cancer) in my 42 years of lifeÖ So many have sacrificed up to this point with the hope that we would deliver on the promise of (better) treatments. Let us bring back the human side of medicine. It is desperately needed.Ē

Dr. Moyad says that doctors prescribe thousands of drugs every day that have not been approved by the FDA to extend life. Doctors attempt to extend the quality and quantity of life despite not having FDA approval. And, he says, ďThis is exactly what makes doctors so wonderfully human and not just 100% science oriented.Ē

I wish to thank Dan Miller who helped me with the research for this article.

 

Feedback
Feedback is encouraged. Contact Dr. Catalona at www.drcatalona.com (see “contact us”), or send me an e-mail at jules105@aol.com. All 16 prior articles are available on the above Website (Either enter my name in the search window or click on Quest Articles and then under my picture).

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