Frequently asked questions

- What are clinical trials?

A clinical trial is a research study in human volunteers to answer specific health questions.
Carefully conducted clinical trials are the safest and fastest way to find treatments that work in
people, and new ways to improve health.

There are different kinds of clinical trials, including those to study:

  • prevention options
  • new treatments or new ways to use existing treatments
  • new screening and diagnostic techniques
  • options for improving the quality of life for people who have serious medical conditions

Clinical trials are conducted according to a plan called a protocol. The protocol describes what
types of patients may enter the study, schedules of tests and procedures, drugs, dosages, and
length of study, as well as the outcomes that mil be measured. Each person participating in the study must agree to the rules set out by the protocol.

- Who should consider clinical trials and why?

Some people participate in clinical trials because they have exhausted standard (approved)
treatment options – which either did not work for them, or they were unable to tolerate side effects. Clinical trials may provide another option when standard therapy has failed.
Other people participate in trials because they want to contribute to the advancement of medical knowledge.

For each clinical trial, researchers develop eligibility criteria, such as the age, sex, type and stage of disease, previous treatment history, and other medical conditions. These criteria help to reduce the amount of variation in the study, without threatening the scientific integrity of the trial, by removing medical variations that might complicate analyzing the results.

Not everyone who applies for a clinical trial will be accepted. Volunteers may be excluded based on the eligibility criteria and/or the number of participants needed by the researchers to collect enough information to determine the safety and effectiveness of a therapeutic agent.

- Are clinical trials safe?

The FDA works to protect participants in clinical trials and to ensure that people have reliable information as they decide whether to join a clinical trial. The federal government has regulations and guidelines for clinical research to protect participants from unreasonable risks.

Although efforts are made to control the risks to clinical trial participants, some risks may be unavoidable because of the uncertainty inherent in medical research studies involving new medical treatments.

The government requires researchers to give prospective participants complete and accurate information about what will happen during the trial. Participants must sign an “informed consent” document before joining the study indicating they understand that the trial is research, and that they can leave the clinical trial at any time. This informed consent is part of a process that ensures a prospective participant in a clinical trial understands what known risks might be associated with the study, and whether there are potential, but as yet unknown risks that may be associated with the product being studied. This information permits someone deciding whether or not to enter a clinical trial to make an informed decision about the level of risk they are willing to accept before they enter the trial.

- What are the potential benefits of participating in a clinical trial?

Participating in well-designed and well-executed clinical trials is one approach for eligible
patients/volunteers to:

  • get actively involved in their health care.
  • gain access to potentially new research treatments
  • have access to expert medical care for the condition being studied, since investigators are often specialists in the disease area being studied.
  • help others by contributing to medical research.