Clinical Trials FAQ
What is a clinical trial and what happens during one?
A clinical trial is a research study conducted in human beings with the goal of answering specific questions about new therapies, vaccines, diagnostic procedures, medical devices and new ways of using known treatments.
Clinical trials are most often used to determine whether new drugs, diagnostics or treatments are both safe and effective.
Who can participate in a clinical trial?
Anyone can potentially participate in a clinical trial – even young children. Each trial has specific criteria that determine who the trial is for (eligibility criteria), as well as any criteria that would prevent participation (exclusion criteria). These criteria may include factors such as age, type of disease, disease severity, use of certain medications, and other medical conditions.
Can children participate in a clinical trial?
With the consent of their legal guardians, yes.
Are there different types of clinical trials?
There are two main types of clinical trials, interventional trials and observational trials.
Interventional clinical trials test the safety and efficacy of drugs and devices (the intervention) in humans to determine if ‘intervening’ with the drug/device can alter disease. These trials are done in phases of increasing numbers of individuals and number of clinical readouts being examined.
Observational clinical trials are done to follow clinical and other health-related outcomes without assigning them to receive a particular intervention in advance.
What are the phases of interventional clinical trials?
The phases of clinical trials are intended to build on the findings from the previous phase. Not all candidate drugs/devices move into higher phases of clinical trials if a clinical benefit was not observed, or the side effect profile was too concerning.
Phase 1 trials are usually conducted with 20-100 healthy volunteers or volunteers with a certain disease/condition. The purpose is to study the general safety and dosage range of a candidate drug. These trials usually run for several months.
Phase 2 trials are usually conducted with several hundred volunteers with the disease/condition. The purpose is to study efficacy and side effects of the candidate drug with a more targeted dosing approach. These trials usually run for several months to up to 2 years.
Phase 3 trials are usually conducted with 300 to 3,000 volunteers with the disease/condition. The purpose is to study efficacy of the candidate drug and to monitor for adverse effects in a larger group of people. These trials usually run from 1-4 years. Drugs/devices can be submitted for US Food and Drug Administration (FDA) approval after two phase 3 clinical trials have been completed with comparable results.
Phase 4 trials are conducted on several thousand volunteers with the disease/condition. These trials are often called post-marketing surveillance trials. The purpose is to study long-term safety, effectiveness, and optimal use of the approved drug in the real-world. These trials run for a varying amount of time.
What are the potential benefits of participating in a clinical trial?
Participating in a clinical trial can have many benefits including helping yourself and others by contributing to medical research. You may play an active role in your healthcare by having access to cutting-edge treatments that others outside of the trial don’t yet have, and through the trial you may have access to a different level of medical care and follow-up.
Potential drawbacks to participating in a clinical trial including side effects from the test treatment that may be different or worse than those from treatments you are currently on. As with any medication, these side effects can range from minor to serious.
As a clinical trial is an experiment, you may receive a treatment that doesn’t work for you, one that is less effective than your current one, or if you are in a phase 3 randomized trial you could receive the placebo control. Some participants may need to be seen more frequently and appointments may be time consuming.
What is informed consent for a clinical trial?
Informed consent is a process of communication between you and the clinical trial site that often leads to agreement or permission for care, treatment or services.
Informed consent is a mandatory part of the clinical trial process to ensure all participants are aware of what will happen during and after the clinical trial before they agree to participate.
How is the safety of a clinical trial participant protected?
The safety of clinical trial participants is protected in several ways.
First, all clinical trials undergo the process of Institutional Review Board (IRB) approval. The IRB is a group of individuals that are responsible for the review of the processes (protocol) and documentation that will be used in the clinical trial. The IRB determines what is appropriate and acceptable for studies involving human beings to ensure their safety and consent to participate in the research process, BEFORE the study begins. Any alterations to the approved IRB protocol require review and amendment by this group.
Second, informed consent is the process by which you agree to participate in the trial. Informed consent means you are given all the information you need in order to make a decision of whether or not to participate. You are allowed to review the full clinical trial protocol, ask questions, and fully understand what will happen during, and after the clinical trial. Should you agree to participate, you will need to confirm your agreement to the informed consent document.
Third, as you progress through the trial, you will be asked about and/or monitored for side effects, and any other health issues that could be of concern.
How do I find a trial that is suitable for me?
A good place to start is to ask your doctor. You can also search for clinical trials based on location and trial type using NEA’s Clinical Trial finder.
Is there any cost to participate in a clinical trial?
The cost of the study medication and study-related exams and care are covered by the sponsor of the clinical trial. You should ask the trial coordinator before you agree to participate if there will be any out of pocket expenses such as co-pays, and if time and travel expenses will be reimbursed.
Can I withdraw from a clinical trial for any reason should I need to?
Yes. Your consent to participate in a clinical trial can be stopped at any time for any reason. Clinical trial coordinators will work with you should you want to cease participation to ensure you are aware of next steps and any recommended follow-up.
What happens to the treatment I received during the clinical trial after the trial is over?
In many cases, the manufacturer of the treatment will make it available to all volunteers in the study after the study is completed. You should discuss this with the clinical trial coordinators before you agree to participate.