Millions of people around the globe participate in clinical trials each year. These final stages of the research process are vital for testing the safety of new medications, evaluating efficacy of therapies, and gathering data needed for regulatory approval. At JDRF, clinical trials are an important part of our strategic research plan to bring a continuous flow of life-changing therapies and, ultimately, a cure for type 1 diabetes (T1D). Maybe you’ve heard about JDRF’s trials on the diabetic macular edema drug Lucentis or the testing of artificial pancreas systems and wondered how people with T1D are selected for these studies or even how you, too, can participate in a clinical trial.
You don’t have to be sick to participate in a clinical trial, but nearly all studies focus on a specific medical condition. Some trials seek volunteers who have the illnesses or conditions that will be studied, while other trials look for healthy participants. Each study has detailed requirements that you must meet in order to enroll. If you’re thinking about participating, it’s a good idea to talk to your health care provider.
There are several types of clinical studies that involve volunteers, but most fall under four categories: prevention, treatment, screening, and quality of life/supportive care. Every clinical study is led by a principal investigator (or PI, in clinical lingo) who leads a research team that can be made up of medical doctors, scientists, academic researchers, nurses, social workers, or other health care professionals. The PI takes direct responsibility for the study, guides the research, and reports its progress and findings to the funding agency. It is up to the PI to ensure the study’s protocol is being followed and that all participants have given informed consent—your written permission to participate in the study that acknowledges you understand all aspects of the trial, as well as its risks and benefits.
Once you are enrolled, a medical professional involved in the trial will typically perform a physical exam and review your medical history. Participants will be randomly assigned to a study group, and you’ll be given information about your study visits, schedule, medications and/or testing procedures. You may be assigned to the control group (participants who get standard treatment or, if the study is testing a new medication, receive a placebo) or you may be in the experimental group (volunteers who get the treatment or medication that is being tested). Your normal healthcare providers will still be in charge of your routine care while the research team follows you at separate visits throughout the study.
Whether you take part in a short, months-long study or a multiyear one, you will not know which focus group you are assigned to or which treatment you are getting. Sometimes, at the end of your participation, you may be given the experimental treatment or drug while further stages of the study continue, until the results are known.
It’s important to note that the sponsor of the trial—normally an academic institution, research foundation, or pharmaceutical company—usually pays for your costs associated with tests and procedures related to the study. Of course, it’s always smart to ask the PI or research team about what patient care costs will be provided before enrolling. And if you do take part but do not want to continue in the study for any reason, you may stop at any time—clinical trial participation is always voluntary.
In general, you will not know the results of the study until the PI has published the results in peer-reviewed medical or scientific journals. Sometimes, if the findings are particularly interesting or important, the study sponsor or institute conducting the research will make a public announcement prior to publishing the results. The PI or the trial health care team will keep you up to date on when the findings will be released and can answer any questions you may have regarding the study outcome.
If the new medication or treatment proves to work better at the end of the trial, it may be approved by federal regulators and become available to everyone with the condition. And that’s the whole goal of clinical testing—helping doctors and scientists find new medications or therapies to better treat, prevent, or even cure a condition.