- Clinical trials test safety and effectiveness of new treatments or new ways to use existing therapies.
- Participation in a clinical trial is voluntary and depends on the possible benefits and risks associated with a particular trial. Before any of these medicines can be prescribed, the federal Food and Drug Administration (FDA) has required strict testing through a clinical trial to be sure they are safe and effective at treating a particular disease.
What Is a Clinical Trial?
A clinical trial is a research study performed by physicians, scientists, and other health professionals in which new treatments are tested on volunteers. These studies take place only after laboratory tests show that the new treatment has some chance of being effective and safe. Prior to conducting the study on volunteers, an institutional review board (IRB) must approve the safety of each proposed study and ensure that all federal regulations that protect human volunteers are being followed. Volunteers must show full understanding of the benefits and risks and sign an informed consent.
While participating in a study, patients with a condition (such as rheumatoid arthritis) may be given a new medicine (or a placebo drug) and then monitored very closely. During follow-up, patients are evaluated for any side effects as well as how well the medicine works to control symptoms and disease outcomes. Even after enrolling, patients can leave the clinical trial at any time and for any reason. Leaving the study will not affect their usual medical care in any way.
What Are the Benefits and Risks of Participation?
Benefits of participating in a clinical trial include:
- Access to new and, potentially, more effective medicines before they become available to the general public.
- Free treatment for many clinical trials, including the medicine being evaluated, doctor visits, physical examinations, laboratory, and other tests.
- The opportunity to help advance medical knowledge and expand treatments for particular conditions.
Possible risks of participation include:
- Some patients may receive placebo (inactive ingredients sometimes referred to as “sugar pills”) during the study and not the study medication. Participants and study staff are “blinded” and will not know what you are getting and will not be able to choose as patients are “randomized” to a group. This means there is no guarantee that you will be given the new medication versus a placebo drug.
- Participants may experience side effects from the study medication. While any medicine can cause side effects, doctors generally know much less about medicines still under investigation.
Talk with your rheumatologist about clinical trials and whether they are right for you. You can learn about clinical trials on the website clinicaltrials.gov.
Updated March 2023 by Kristen Lee, MD, and reviewed by the American College of Rheumatology Committee on Communications and Marketing. This page is updated biannualy.
This information is provided for general education only. Individuals should consult a qualified health care provider for professional medical advice, diagnosis and treatment of a medical or health condition.