A clinical trial is a research study designed to evaluate the safety, efficacy and potential side effects (and benefits) of new medical treatments, interventions or procedures on human participants. These trials generate data to determine if an intervention is beneficial, and if so, to what extent. In such trials, volunteers willingly participate in a controlled setting, following specific protocols, to facilitate scientific observation and analysis. These trials play a crucial role in advancing medical knowledge and developing new therapies that can improve patient outcomes.

Participants in clinical trials have the opportunity to assume a more engaged role in their health care, receive early access to emerging research treatments prior to their widespread availability and make valuable contributions to medical research, benefiting others in the process.

Anyone meeting the specific eligibility criteria can participate in a clinical trial. Inclusion criteria are the factors that allow individuals to take part in a clinical trial, while exclusion criteria disqualify individuals from participation. Inclusion criteria typically vary depending on the nature of the study and the specific medical condition being investigated. Potential participants may include a diverse group of different ages, genders and health status. Prospective participants must meet all of the qualifications of the study before enrolling in a clinical trial. Other trials may target specific healthy populations or exclude individuals with certain medical conditions. The final determination of eligibility follows the previously outlined protocol for the trial, and is not determined by your health care provider.

The process of conducting clinical trials varies depending on the specific trial. A clinical trial team includes doctors, nurses, research coordinators and other health care professionals like social workers. At the beginning of the trial, the team assesses the participant’s health and provides them with clear instructions for their role in the study. They closely monitor the participant throughout the trial and stay in touch even after it’s finished. In some clinical trials, participants may have to undergo more tests and see doctors more often than they normally would for their illness or condition. Regardless of the type of trial, participants collaborate with a research team. The key to successful participation in a clinical trial is to carefully follow the trial’s guidelines and maintain regular communication with the research staff.

Before you decide whether or not to take part in a clinical trial, it’s important to learn some important facts about it. This is called informed consent, and it’s an ongoing process that continues throughout the study to provide you with information. To help you make your decision, the doctors and nurses involved in the trial will explain all the details of the study to you. They also will discuss the potential risks and benefits involved, which are described in a document called the informed consent form. It’s crucial to note that informed consent is not a binding contract, and you have the right to withdraw from the trial at any time.

Clinical trials contribute to advancing medical knowledge that guides everyday health care decisions. Some benefits of your participation include:

• Early access to new research treatments before they become widely available
• Access to additional support from experienced research staff working alongside your health care team
• Opportunity to play an engaged role in your health care journey

Treatment could have some unpleasant or potentially serious side effects that may even be life-threatening. It’s important to keep in mind that the treatment may not work as effectively for the participant. Following the treatment protocol might require more time and attention compared with another treatment. You could need to visit the study site more frequently, receive additional treatments, stay at the hospital or have to follow complex dosage requirements.

Side effects are any undesired actions or effects of a drug or treatment. Side effects will vary by treatment plan on each clinical trial. Known and potential risks of an investigational treatment will be included in the informed consent form. Experimental treatments must be evaluated for both immediate and long-term side effects.

Clinical trials must adhere to ethical and legal codes, much like the rules that govern medical practice. Clinical research is regulated by the federal government and includes safety measures to keep participants protected. Participants’ safety is monitored throughout the course of the clinical trial. Researchers may share the trial’s findings at scientific gatherings, in medical publications and with different government organizations.

It’s important for individuals to learn all they can about the clinical trial and feel at ease before choosing to participate. Some of your questions may be answered in the informed consent document. Here are some questions that might be useful for participants to talk about with the research team and/or your health care provider.

• What is the purpose of the study?
• Who is going to be in the study?
• Why do researchers believe the new treatment being tested may be effective? Has it been tested before?
• What kinds of tests and treatments are involved?
• How do the possible risks, side effects and benefits in the study compare with my current treatment?
• How might this trial affect my daily life?
• How long will the trial last?
• Will hospitalization be required?
• Who will pay for the treatment?
• Will I be reimbursed for other expenses?
• What type of long-term follow-up care is part of this study?
• How will I know that the treatment is working?
• Will results of the trials be provided to me?
• Who will be in charge of my care?

Prepare in advance by making a list of potential questions to ask. (Review suggested questions above.) Ask a friend or relative to come along to offer support and hear the responses to your questions. Bring a recording device (or use your voice memo app on your smartphone) to record the conversation for future reference.

Every clinical trial in the U.S. must be approved and monitored by an institutional review board (IRB) to make sure the risks are as low as possible and are worth any potential benefits. An IRB is an independent committee of physicians, statisticians, community advocates and others that ensures a clinical trial is ethical and the rights of study participants are protected. All institutions that conduct or support biomedical research involving people must, by federal regulation, have an IRB that initially approves and periodically reviews the research.

Yes. Most clinical trials offer short-term treatments for a specific illness or condition, but they do not provide comprehensive primary care that covers all aspects of a person’s health. It’s important to note that when a health care provider collaborates with the research team, they can ensure that the trial’s protocol won’t interfere with any other medications or treatments the participant may be receiving.

Yes, you can leave a clinical trial whenever you want. If you decide to withdraw from the trial, it’s important to inform the research team and explain why you’re leaving the study. There might be some procedures or tests that need to be done when you withdraw, but these are done to ensure your safety.

Clinical trial ideas typically originate from researchers. Once researchers have experimented with new therapies or procedures in the laboratory and in animal studies, the treatments showing the most encouraging results are moved in to clinical trials. Throughout the trial process a greater understanding is gained about the new treatment, including its potential risks and how effective it might be.

Clinical trials are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups and pharmaceutical companies, in addition to federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD) and the Department of Veterans Affairs (VA).

A protocol is like a study plan that explains how a research study will be conducted. It’s designed to keep participants safe and answer important research questions. The protocol tells us who can join the study, when and how often they will have tests and take medicine, and how long the study will last. In a clinical trial, people who follow the protocol visit the research team regularly to make sure they’re healthy and to see if the treatment is working and safe.

A placebo is a kind of medicine that doesn’t actually have any real therapeutic effect. It can come in the form of a pill, liquid or powder. In clinical trials, researchers sometimes use placebos to see if a new treatment really works. They compare the new treatment with the placebo to find out how well it works. In some studies, a group of participants may receive a placebo instead of an active drug or treatment.

A control serves as the benchmark for assessing experimental findings. In many clinical trials, one set of patients receives an experimental drug or treatment while another group, known as the control group, is given either the usual treatment for the illness or a placebo.

Clinical trials come in various forms. Here are some types of clinical trials you might encounter:

1. Treatment trials: These trials focus on evaluating new treatments, such as medications, therapies or surgical procedures, to determine their effectiveness in treating specific conditions. Participants in treatment trials may receive the new treatment being studied or a standard treatment for comparison.
2. Prevention trials: These trials concentrate on finding ways to prevent certain diseases or conditions from occurring. They often involve comparing different strategies, such as medications, vaccines or lifestyle modifications, to assess their ability to reduce the risk of developing a particular ailment.
3. Diagnostic trials: Diagnostic trials aim to improve the accuracy and effectiveness of diagnostic tools, tests or procedures used to identify diseases or conditions. Researchers may compare different diagnostic methods or evaluate new technologies to enhance early detection and improve patient outcomes.
4. Screening trials: These trials assess the effectiveness of screening methods in detecting specific diseases or conditions. By identifying potential health concerns early, screening trials strive to increase the chances of successful treatment or prevention.
5. Quality of life trials: Quality of life trials focus on improving the overall well-being and comfort of individuals living with chronic illnesses or conditions. These trials may investigate the impact of interventions, such as support programs or alternative therapies, on a patient’s physical, emotional and social aspects of life.
6. Observational trials: Observational trials observe individuals in their natural settings to gather data on health-related behaviors, risk factors or disease progression. These trials do not involve any interventions but can provide valuable insights into the relationship between various factors and health outcomes.

Drug trials are conducted in phases. The trials at each phase have a different purpose and help scientists answer different questions:

• Phase I trials: Researchers test a new drug or treatment in a small group of people (20 to 80) for the first time to evaluate its safety, determine a safe dosage range and identify side effects.
• Phase II trials: The study drug or treatment is given to a larger group of people (100 to 300) to see if it is effective and to further evaluate its safety.
• Phase III trials: The study drug or treatment is given to large groups of people (1,000 to 3,000) to confirm its effectiveness, monitor side effects, compare it with commonly used treatments and collect information that will allow the drug or treatment to be used safely.
• Phase IV trials: Post-marketing studies collect and report additional information, including the drug’s risks, benefits and optimal use.

Most human use of investigational new drugs takes place in controlled clinical trials conducted to assess safety and efficacy of new drugs. Data from the trials can serve as the basis for the drug marketing application. Sometimes, patients do not qualify for these carefully controlled trials because of other health issues, age or other factors. For patients who may benefit from the drug use, but don’t qualify for the trials, FDA regulations enable manufacturers of investigational new drugs to provide for “expanded access” use of the drug. For example, a treatment investigational new drug (IND) application or treatment protocol is a relatively unrestricted study. The primary intent of a treatment IND protocol is to provide for access to the new drug for people with a life-threatening or serious disease for which there is no good alternative treatment. A secondary purpose for a treatment IND protocol is to generate additional information about the drug, especially its safety. Expanded access protocols can be undertaken only if clinical investigators are actively studying the new treatment in well-controlled studies, or all studies have been completed. There must be evidence that the drug may be an effective treatment in patients like those to be treated under the protocol. The drug cannot expose patients to unreasonable risks given the severity of the disease to be treated.

Some investigational drugs are available from pharmaceutical manufacturers through expanded access programs listed in ClinicalTrials.gov. The manufacturer generally manages expanded access protocols, with the investigational treatment administered by researchers or doctors in office-based practice. If you or a loved one would be interested in treatment with an investigational drug under an expanded access protocol listed in ClinicalTrials.gov, review the protocol eligibility criteria and location information and inquire at the contact information listed for the protocol.