Clinical Trials 101

Picture: http://blog.patientslikeme.com/2016/06/01/patients-as-partners-john-and-david-share-their-clinical-trial-experiences/

If you have a complex condition, you might have heard the phrase “clinical trial” at some point in your medical journey – if not in a doctor’s office, then perhaps in a news segment or a commercial. Clinical trials are a major part of medical care, treatment, and helping science move forward. So what is a clinical trial?

The National Institute of Health defines clinical trials as “A clinical study involves research using human volunteers (also called participants) that is intended to add to medical knowledge. There are two main types of clinical studies: clinical trials (also called interventional studies) and observational studies.”” If that’s a little too general, keep reading…

How clinical trials are approved?

Clinical trials do not pop out of thin air. Before a trial can start enrolling patients there are many steps that researchers and labs must take. Any new drugs/therapies go through a painstaking process of preclinical studies to show that they are likely safe and effective.

Usually, this process starts with studies of basic cancer biology. For example, a researcher might find a certain gene mutation in a kind of cancer, and then might show how this gene mutation could cause cancer. In especially promising cases, this can lead to the development of a drug to target this gene mutation (or other feature that provides an “Achilles heel” to the cancer). Then researchers investigate how the treatment works on either human or animal cancer cells that are grown in the lab. If cell studies seem promising, a drug may sometimes move forward to an animal study.

During an animal study the treatment is tested on cancer in rats or mice. Researchers must earn approval for all animal studies by demonstrating that their drug has a reasonable enough chance of helping humans to justify testing in animal models. This animal model testing can be a good indication of how the treatment will work in humans. That said, an animal model does not always show how a new treatment will be processed, absorbed, or expelled by a human. It is important to note that anywhere along this path of testing, a drug that seemed promising can fail to meet its goals and thus not move forward in the clinical trial process.

However, if the cell and animal studies suggest that the new treatment may work, researchers may complete an Investigational New Drug (IND) application through the FDA. In the application researchers describe results from pre-clinical studies, details about how the drug is made, who makes it, physical qualities of the drug, and outline what they hope will be clinical trial protocols. Applicants must also agree to get consent from all trial participants, have the study reviewed by an Institutional Review Board (IRB) and follow many rules that come with studying a new drug in humans.

If the IND is approved the clinical trial can finally move forward to Phase 1.

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Clinical trial phases

Clinical trials are completed in phases. If a new drug does not pass any phase then it cannot move to the next. Each phase generally increases the number of subjects and may move from a treatment of last resort with a general population of advanced cancer patients to a treatment of choice with a more targeted population in which the drug has been shown to work especially well.

The goal of Phase 1 clinical trials is to test a drug’s safety. In this phase, doctors can also learn more about correct dosage, side effects, when and how often it should be taken, and how the cancer responds to the treatment. Typically, there are anywhere from 10 to 30 people in a Phase 1 clinical trial, which can last from months to a year

Phase II clinical trials refine researchers’ understanding of drug safety and efficacy. Sometimes participants are separated into two or more groups. This is done to see how a new therapy compares to the standard of care or to try different dosing levels. Participants on a Phase II clinical trial may be asked to keep logs about how they are feeling. These trials last about two years and have under 100 patients.

Phase III clinical trials compare the new therapy to the current standard of care. In this phase, the trial evaluates side-effects and effectiveness and tries to define whether one treatment works better than the other. In many cases people on Phase III clinical trials are chosen at random to either be treated with the new therapy or with the standard of care (most cancer clinical trials do not include a placebo). Many trials are designed such that if the first cohort of patients shows benefit, the trial design changes so that the “control” group is also offered the new drug. The FDA will typically not approve a drug to be used in the general public unless it passes a Phase III clinical trial.  Commonly, Phase III clinical trials include more than 100 people.

The last phase of clinical trials is Phase IV. During this phase, drugs that are newly approved by the FDA are tested in hundreds or thousands of participants. This allows doctors to collect more information about the drug over time and in a large population to watch for late-onset side-effects and to refine their use of the drug, perhaps in combination with other drugs, at different disease stages, and in more focused patient populations.

For more information about clinical trial phases click here.

Only if a drug succeeds in clinical trials does it become available for use outside the trial (with a few special exceptions). This means that new treatments are often available in clinical trials years before they become available outside the academic medical centers that commonly host trials.

Generally, a clinical trial isn’t for everyone – many patients have forms of cancer that are successfully controlled by current treatments. But for patients with rare or especially aggressive forms of cancer, or if standard-of-care treatments don’t result in long-term disease control, clinical trials can provide additional options. Likewise, even if a patient’s cancer is controlled by current treatments, many clinical trials exist that study aspects of cancer other than the disease itself, for example studies of new strategies to support wellbeing or survivorship issues.

Clinical trials allow doctors and researchers to test treatments that will extend lives and, increasingly, even lead to cures for some forms of the disease. Some patients will benefit from some clinical trials. Other patients understand that their involvement contributes to easing the burden of cancer for future patients. From basic laboratory researchers to the doctors administering treatments, no one takes clinical trials lightly. In these essential tests, it is the bravery and hope of human patients that allows cancer science to truly reach its goal of providing new treatments.

FAQ about Clinical Trials

  1. Does my insurance pay for clinical trials?
    1. Typically all study related testing and out-of-pocket costs are paid for by the trial’s sponsor. However, patients are often encouraged to ask their insurance provider to pay for routine tests/procedures/etc…that would fall under the standard of care.
  2. Will this drug be administered close to my home by my local oncologists?
    1. This truly depends where you live and where the trial is taking place. Clinical trials are offered at many different hospital, clinics, doctors’ offices, etc…That being said sometimes a specific trial may require travel as not every healthcare institution will provide the same trials.
  3. Will I get reimbursed for my expenses?
    1. Please consult with your doctor for specifics about reimbursement.

Working towards a better future

At the University of Colorado Cancer Center our patients have access to clinical trials that are paving the way in cancer care. The CU Cancer Center is also the only NCI-designated cancer center in Colorado, so our patients can feel confident in our research and doctors.

About the author: Taylor Abarca

Taylor Abarca (Bakemeyer) is the Social Media Web Specialist at the University of Colorado Cancer Center. Contact her at Taylor.Bakemeyer [at] ucdenver.edu.

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