Clinical Trials — What Are They?

One of the primary purposes of The Triumph Fund is to raise money to support the Clinical Trials Office at the Medical College of Wisconsin.  Logical questions that may arise are: i)  What are clinical trials?;  and  ii)  Why are clinical trials important?  Although the answers are interrelated, let us attempt to first understand – from a lay person’s perspective (and that is what I am) – what exactly is a clinical trial.  We can then in future posts discuss the value of clinical trials and why they are a worthy cause for charitable giving .

1)  What is a Cancer Clinical Trial:

Websites of cancer centers across the country discuss clinical trials.  A clinical trial is frequently expressed as a means of developing today the standard of care for the patients of tomorrow.  It is a catchy statement that has some real meaning.    Clinical trials can give existing patients access more quickly to innovative cancer treatments that may be beneficial to that patient.  Clinical trials, therefore, may promote the treatment of both existing cancer patients, who are involved in the trial, and future cancer patients who may enjoy the benefits of new treatments and interventions that a trial has shown are safe and effective.

Conducting a clinical trial is complex, starting with the admission of patients into a trial.   Any patient participation is voluntary.  Clinical trials are closely scrutinized by independent committees and federal agencies, including the FDA, to ensure maximum safety for the patient and proper study methods.  At the outset, there is a determination as to whether a patient is eligible for a trial, that is, generally, if the patient meets the specified eligibility criteria, including whether he or she has the type and stage of cancer needed for the trial.  The patient then needs to provide informed consent before he or she may participate.   A clinical trial may not involve the testing of a drug or other treatment, such as a radiation procedure.  They may involve trials relating to diagnosis, screening, or prevention.

2)  Phases of Clinical Trials:

There are various “phases” to clinical trials.  Phase O, which is not widely used, generally involves the use of a low dose of a new drug in a patient and evaluates how that new drug may work, such as if it reaches a tumor and how the body responds.  Because of the low dosage, the potential benefits and risks to the patient are minimal.  Phase 1 clinical trials determine if the treatment is safe and follows the use of the treatment in a laboratory setting.  This phase generally involves a small number of patients.  One of the purposes of a Phase 1 trial is to determine the highest dosage that can be given without serious side effects.  A Phase 2 trial may result if a Phase 1 trial shows a treatment is reasonably safe.   This phase, which generally involves more patients than a Phase 1 trial, is designed primarily to determine if the treatment is effective against a particular cancer.  Researchers may look, for example, to see if a tumor shrinks or disappears or if there is a period when it does not grow.  Assuming the treatment is found not to have too serious of side effects and is seen as potentially effective, then it may proceed to a Phase 3 clinical trial, during which even more patients are studied.  Phase 3 involves an assessment as to whether the treatment may have fewer side effects and / or may work better than what is currently available.  In essence, in a Phase 3 trial, there is a comparison between the new treatment and the treatments then viewed as standard. There is also a Phase 4 clinical trial, which seeks more information on a drug that has been approved by the FDA.

These phases of Cancer Clinical Trials are widely discussed on various websites, including on the FDA website (Stages of Drug Development and Review), The National Cancer Institute’s website (Phases of Clinical Trials),  and the American Cancer Society’s website (Clinical Trials: What You Need to Know).

If a drug has been used in a clinical trial and it has proven safe and effective, then the researchers and scientists can submit an FDA application for approval of the drug.  The FDA relies on data established at clinical trials. Depending on the results, the FDA may then approve the drug, allowing other cancer patients to use it.  That is, the results of a clinical trial allow the FDA to make decisions about the approval of a drug and, thus, whether it can be used outside the reach of the clinical trial.  The FDA’s website explains its drug review process.  (FDAs Drug Review Process)

3.  Better Understanding Clinical Trials:

For one who is not a doctor or researcher to understand these trials, a good starting point may be the Fact Sheet on Cancer Clinical Trials published by the National Cancer Institute.   That document discusses the importance of clinical trials, the types of clinical trials, where clinical trials may take place, the phases associated with clinical trials, and the benefits and potential risks of clinical trials.  It explains that the treatments tested at clinical trials may include new drugs, combinations of currently used drugs, radiation treatment, and new surgery techniques.

The website for the Medical College of Wisconsin contains an extensive Director’s Update on the Clinical Trials Office at the College.  A helpful page in that update answers many questions regarding Clinical Trials Office at the College and its mission (CTO FAQs).  The College and Froedtert Hospital also have a primer on clinical trials (Clinical Trials Basics), which also discusses the phases.

The American Cancer Society’s website contains a wealth of information on cancer clinical trials, including a guide that assists patients in discussing clinical trials with their doctors (Clinical Trials:  What You Need To Know) and the use of placebos in clinical trials (Placebo Effect).

Finally, the Coalition of Cancer Cooperative Groups is an organization with the sole mission of improving patient awareness of cancer clinical trials, facilitating access to trials, and promoting participation in them.  The Coalition’s website contains a great deal of information on clinical trials (Learn About Cancer Clinical Trials) and articulates what it describes as myths and facts about such trials (Myths and Facts About Cancer Clinical Trials).  An added benefit is that this website provides a Glossary that gives definitions of many of the terms used in cancer research and clinical trails.

4.  Let’s Review:

As we consider these pronouncements on cancer clinical trials, we see, among other things, that:  i)  they are voluntary; ii)  their main focus is on patient safety;  iii)  they serve to provide a basis for researchers to determine, on an expedited basis, if new treatments, be they drugs or devices, are effective and are safe; iv)  when drugs are evaluated in a trial, they may not be new drugs, but instead may be an existing drug that is evaluated in use with another existing drug or may be an existing drug being tested on a cancer for which it has not been previously used;  v)  there are potential risks for those who volunteer, because the efficacy of a drug or device is unknown; vi)  there are potentially significant benefits, both to the participant in the trial and to others in the future who will be aided by the results of the tests and analysis undertaken during and as a result of a clinical trial;  vii)  the FDA is tuned into clinical trials and its approval of a drug may be influenced and hence hastened by the results of a clinical trial, thus making the drug available to others before it otherwise would have been.


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