Clinical Trial FAQ

A clinical trial involves research using human volunteers (also called participants) that is intended to add to medical knowledge.
In a clinical trial, 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, such as diet. Clinical trials may compare a new medical approach to a standard one that is already available, 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.

There are many reasons people volunteer for clinical trials. Healthy volunteers may hope to contribute to advancing science. Patients with an illness or disease participate with the hope the investigational treatment may benefit their condition or treatment outcome. While there are no guarantees that the investigational treatment will benefit you, each person who participates in a clinical trial contributes to scientific progress. Your participation helps researchers advance therapies that have the potential to improve and save lives.

Clinical trials that are well-designed and well-executed are the best approach for participants to:

• Play an active role in their own health care.
• Help others by contributing to medical research.

There are also risks to clinical trials:

• There may be unpleasant, serious or even life-threatening side effects to treatment.
• The treatment may not be effective for the participant.
• The protocol may require more of their time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements.

Clinical trials need to be approved before they start, and there are several layers of safeguards to protect the people who participate in them:

• Principal Investigators. Every clinical study is led by a principal investigator, who is often a medical doctor. Clinical studies also have a research team that may include doctors, nurses, social workers, and other health care professionals.
• Institutional Review Boards. Each federally supported or conducted clinical study and each study of a drug, biological product, or medical device regulated by FDA must be reviewed, approved, and monitored by an institutional review board (IRB). An IRB is made up of doctors, researchers, and members of the community. Its role is to make sure that the study is ethical and that the rights and welfare of participants are protected. This includes making sure that research risks are minimized and are reasonable in relation to any potential benefits, among other responsibilities. The IRB also reviews the informed consent document.

In addition to being monitored by an IRB, some clinical studies are also monitored by Data Monitoring Committees (also called Data Safety and Monitoring Boards).

• A Data Monitoring Committee (DMC)/Data Safety and Monitoring Board (DMSB) is a group of independent clinicians and scientists who monitor the safety and scientific integrity of a clinical trial. The DMC/DSMB can recommend to the sponsor that the trial be stopped if it is not effective, is harming participants, or is unlikely to  serve its scientific purpose. Members are chosen based on the clinical experience, scientific skills, and knowledge needed to monitor the particular trial.

Informed consent is the process of learning the key facts about a clinical trial before deciding whether or not to participate. It is also a continuing process throughout the study to provide information for participants. To help someone decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. If the participant’s native language is not English, translation assistance can be provided. Then the research team provides an informed consent document that includes details about the study, such as its purpose, duration, required procedures, and key contacts. Risks and potential benefits are explained in the informed consent document. The participant then decides whether or not to sign the document. Informed consent is not a contract, and the participant may withdraw from the trial at any time.