A Primer on Clinical Trials

May 16, 2022

A Primer on Clinical Trials

Is a clinical trial right for you?  That’s not an easy question and there are many things to consider before you decide to join or not to join a clinical trial.  Clinical trials are how advances in medicine are made.  It all starts with an idea, one that likely was drawn from previous research.  Let’s say that a chemotherapy drug has been previously tested in a clinical trial for lung cancer patients and was found to have moderate benefits.  The doctor in charge of the research, after studying the mechanism of how the drug works, thought it may be beneficial for patients with stomach cancer as well.  She therefore decides to test the drug in stomach cancer patients.

For her to test the drug on stomach cancer patients, she will need to apply to the government agency in charge (the FDA) for permission to start a new clinical trial.  Her idea will undergo many layers of detailed review at the agency before she will be allowed to proceed.  The agency will ensure that her trial is scientifically sound, addresses any safety issues and that the potential benefits outweigh the possible risks.  Once the agency has approved the trial, it then undergoes an additional review by an independent review board, called the Institutional Review Board, or IRB.  Only after IRB approval will the doctor be able to start enrolling patients into the trial.

You are sitting in your doctor’s office and he explains to you that he has a clinical trial he thinks may benefit you.  What do you do?  First, you should know that all clinical trials have come through a very rigorous process before being offered to patients.  Let’s take the chemotherapy drug that is about to be tested in patients with stomach cancer.  Years previously, the drug started out as an idea in a lab.  It first would be tested in a lab dish, then likely in an animal model.  Then, it would be put through three “phases”: phase 1 being tested in just a few volunteers to make sure of its safety.  It progresses to a Phase 2 trial which tests to determine its benefits and risks.  Finally, it is included in a Phase 3 trial, the “gold standard” of drug testing where it is tested against the standard of care or sometimes a placebo.

Second, the decision whether to participate or not is completely up to you.  Your doctor or other health care provider will carefully explain the entire process to you, including all procedures, any possible side effects and potential benefits to you.  You will never be forced or coerced to participate in a clinical trial, and you are free to leave it whenever you wish.  You will be required to sign an informed consent form before you can be enrolled.

Third, you should consider the time commitment to some clinical trials.  Clinical trials range in complexity from quite simple to very complex.  Generally, clinical trials do require more of your time than standard of care as more testing and appointments will likely be needed.  Clinical trials can also result in increased costs that may not be covered by study funds or insurance, such as travel to the extra appointments.

After thinking about all these things, and talking with family, friends and your health care team, you decide you may be interested.  What are the possible benefits of participating in the trial?  Clinical trials offer the newest state-of-the-art treatments before they are available anywhere else, that may help treat your cancer.  You will be more closely monitored than you would otherwise by the research team. Many patients who participate in clinical trials feel that they are helping other patients and contributing to medical knowledge.  Patients also feel empowered by taking an active role in their health care decision making process by considering all possible treatments, including the experimental ones.

What are the risks?  Almost every clinical trial involves some risks.  The experimental treatment may not work or may not work on all patients.  There may be more side effects than standard treatment, and some of these may be serious and in rare cases, fatal.

Ultimately, the decision to participate or not is between you and your doctor after considering all possible risks and benefits to you.  Ask questions until you have all the information you need to make an informed decision about whether or not to participate in a clinical trial.

For more information on clinical trials, please visit https://www.cancer.gov/publications/patient-education/cancer-treatment-research-studies