What is a clinical study? 

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 and observational studies. In a clinical trial (also called an interventional study), participants receive specific interventions according to the research plan or protocol created by the investigators. These interventions may be medical products, such as drugs or devices; procedures; or changes to participants' behavior, for example, diet. Clinical trials may compare a new medical approach to a standard one that is already available or to a placebo that contains no active ingredients or to no intervention. Some clinical trials compare interventions that are already available to each other. When a new product or approach is being studied, it is not usually known whether it will be helpful, harmful, or no different than available alternatives (including no intervention). The investigators try to determine the safety and efficacy of the intervention by measuring certain outcomes in the participants. For example, investigators may give a drug or treatment to participants who have high blood pressure to see whether their blood pressure decreases.
New drugs are first developed in research labs, and then tested on animals. Only then are clinical studies done in humans. Clinical trials of new drugs are done in different phases:

  • Phase I studies test a new drug for the first time in a small group of people (about 20-80) to see if it is safe, to find the right dose, and to know the side effects.
  • Phase II studies are done in more people (about 100-300) to see how well the new drug treats a disease.
  • Phase III studies are done in large groups of people (about 1000 to 3000) to see if the new drug works well, has side effects, and how it compares to other drugs.
  • Phase IV studies are done after the treatment is approved by the U.S. Food and Drug Administration (FDA).

For more information, visit http://clinicaltrials.gov/ct2/about-studies/learn#ClinicalTrials


Why Should I participate in Clinical Trials?

People who volunteer in clinical trials help to advance medical discoveries that can provide new drugs, new surgical procedures or devices to prevent, detect, or treat diseases.  Clinical trials offer hope for many people and an opportunity to help researchers find better treatments for others in the future.
For more information, visit http://www.nih.gov/health/clinicaltrials/whyparticipate.htm

How are participants in clinical trials protected?

There are several ways study participants are protected during a clinical trial. Informed consent is a process in which researchers provide potential and enrolled participants with information about a clinical study. This information helps people decide whether they want to enroll, or continue to participate, in the study. The informed consent process is intended to protect participants and should provide enough information for a person to understand the risks of, potential benefits of, and alternatives to the study.
In addition to being monitored by an Institutional Review Board (IRB) – a board comprised of physicians, researchers and members of the community who ensure the study is ethical and the rights and welfare of the participants are protected, some clinical studies are also monitored by data monitoring committees, a group of independent scientists who monitor safety and scientific integrity of a clinical trial.  Various Federal agencies, including the Office of Human Subjects Research Protection (OHRP) and FDA, have the authority to determine whether sponsors of certain clinical studies are adequately protecting research participants.
For more information, visit  http://clinicaltrials.gov/ct2/about-studies/learn#HowAreParticipants

What should you know about a participating in a study?

Anyone interested in participating in a clinical study should know as much as possible about the study and feel comfortable asking the research team questions about the study.

  • What is being studied?
  • Why do researchers believe the intervention being tested might be effective? Why might it not be effective? Has it been tested before?
  • What are the possible interventions that I might receive during the trial?
  • How will it be determined which interventions I receive (for example, by chance)?
  • Who will know which intervention I receive during the trial? Will I know? Will members of the research team know?
  • How do the possible risks, side effects, and benefits of this trial compare with those of my current treatment?
  • What will I have to do?
  • What tests and procedures are involved?
  • How often will I have to visit the hospital or clinic?
  • Will hospitalization be required?
  • How long will the study last?
  • Who will pay for my participation?
  • Will I be reimbursed for other expenses?
  • What type of long-term follow-up care is part of this trial?
  • If I benefit from the intervention, will I be allowed to continue receiving it after the trial ends?
  • Will results of the study be provided to me?
  • Who will oversee my medical care while I am in the trial?
  • What are my options if I am injured during the study?