A Short Review of Clinical Studies
March 27th, 2017
Robin Jon Zimmerman
A clinical trial is a study designed to answer specific questions about biomedical or behavioral treatments, including new drugs or surgical procedures. They are a vital part of medical research and are a key component in advancement in treatments and patient survival. Many preconceptions are still a part of popular culture, like clinical trials are only for when you have exhausted all their options, or you will have to stop a current treatment to enlist in the trial. Some fear that a clinical trial is risky and may harm them. Many think they are just being used as a guinea pig for a virtually untested therapy. I hope to clear up these misconceptions and I will be making the argument as to why clinical trials are something any patient would want to consider participating in.
Clinical trials are performed because new tools are being created to treat illnesses while old tools can be improved upon. This is especially true with melanoma because the late stages of this cancer is very difficult to survive. A clinical trial does not start without first going through an intense review by ethic committees and agencies. The most recognized for these are probably the Food and Drug Administration (FDA) and the Institutional Review Board (IRB).
When considering a clinical trial the first thing to realize is that the patient is empowered to advocate for themselves and are free to ask questions before, and during the trial. Before starting a trial an informed consent form will need to be signed and should have all the basic information about the trial being offered. There are pros and cons to any trial; pros include access to new treatments before approval, the potential to help future patients, and helping move all research forward; cons can be that trial treatments are not guaranteed to be better than standard, though placebos are rare, there is no guarantee that you will be assigned study treatment. Additionally the treatments are usually only available at a sponsoring institution and treatments may include additional time, visits, and biopsies. These last drawbacks can make the study “Financially Toxic”. That is, the distance and additional treatment time will pull the bread winner away from their job, or it could cost too much to have someone care for the family at home.
Clinical trials are performed in a series of phases. Before even reaching a first phase and any interaction with a human patient a lot has been done already; such as, lab studies and animal models. A phase 0 study on a limited number of patients is conducted to determine just how the body metabolizes a drug. After this, Phase I, a small number of patients will enter the trial to evaluate safety. Phase II will study a larger sampling of patients to assess the drug’s safety and its effectiveness. Phase III will have the largest number and is the last step before approval. In rare cases a treatment will have such high rate of effectiveness and prove itself in Phase I and will jump to Phase III immediately.
`The Hippocratic Oath stated “Primum non nocere“– “First, do no harm”. With that as a basis the first primary goal of a clinical trial is to ensure its safety. Meticulous records are kept during the research to facilitate regulatory approval and to approve the use of the therapy for patients outside of the research. So ultimately clinical are not something to fear but to embrace.