(A scientific study in people that helps to determine if and how a drug or treatment works.)
are the backbone of drug development.
Have you ever thought closely about the contents of your medicine cabinet? The medicines inside may have different purposes, but they have one big thing in common: before reaching you, they underwent years of scientific studies to ensure they were safe for you to take. These research studies are called clinical trials.
Clinical trials provide critical information about an investigational drug’s safety and effectiveness, and generate in-depth data that can even lead to the creation of other innovative treatments. Every potential drug undergoes multiple phases (In Phase 1 trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects. In Phase 2 trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety. In Phase 3 trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely. In Phase 4 trials, studies look at real-world experience over a long time and provide additional information on the drug’s risks, benefits, and optimal use.) of research, each with a different goal.
Ultimately, clinical trials make a difference not just for today’s patients, but also potentially to those who’ll be diagnosed tomorrow, next month, or even a decade from now.
People participate for different reasons.
Choosing to participate in a clinical trial is a personal and individual decision. Everyone is different. In a study by the Center for Information and Study on Clinical Research Participation (CISCRP), the following factors were common motivations for participants:
• Medical care
• Access to medical professionals
• Learn about their disease
• Receive regular updates about the research while they’re enrolled
• Receive information about the study results after the study has ended
• Feel part of a community
Participant health is important to the medical professionals who conduct clinical trials. One of the responsibilities of medical professionals who run clinical trials is to care for participants (Also called a volunteer, is someone who takes part in a clinical research study.) . These professionals may go by unfamiliar titles like, Principal Investigator or Study Coordinator. But they are, also, doctors, nurses, and trained research professionals. As such, these professionals monitor participant safety and well-being. Trial professionals will adjust study-related care when they feel it's in the participant's best interest. This may mean withdrawing a participant from a clinical trial. They will also often coordinate care delivery with the participant’s personal physician and healthcare team.
Clinical trials have a regulatory and ethical framework to protect participants. Everyone conducting a clinical trial has strict regulatory and ethical duties. To ensure these duties are followed and that participants are treated well, a team of people, who are independent from the research conduct, is asked to review and maintain oversight over each clinical trial. These teams of people are called Institutional Review Boards (IRBs). An IRB reviews every clinical trial to ensure appropriate steps are taken to protect the rights and welfare of the people participating. The IRB must review the clinical trial before participants can enroll. IRB representatives are available to participants if they have any questions.
The risks and benefits are different for everyone. Each clinical trial has different risks and benefits. And because all clinical trial participants are different, they will experience these risks and benefits in different ways. For example, one participant may experience a side effect that another participant does not.
Since clinical trials are experiments, the exact risks and benefits can be difficult to predict. But researchers only move forward with clinical trials when they are optimistic about the potential benefits and believe the risks for participants are acceptable. In addition, an Institutional Review Board (IRB) must review the research protocol, or plan, before researchers can enroll participants. Researchers inform participants about potential risks and benefits before they enroll in a clinical trial. This discussion takes place during what's called the Informed Consent process, which helps participants understand what's involved so that they can make the decision that’s right for them.
All clinical trials have rules about who can and cannot participate
Factors that allow someone to participate in a clinical trial are Inclusion Criteria.
Those that disqualify someone from participating are called Exclusion Criteria. These criteria are based on things such as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions.
Inclusion and Exclusion Criteria are developed with much thought and consideration. They are necessary to prove whether medicines work or not in a specific patient population and make sure the right participants enter the trial and stay safe. These criteria are applied to all potential participants equally before they start the study.
Participants can drop out of clinical trials any time. Participants can drop out of a clinical trial even after they have agreed to participate. And they do not need to give a reason. No matter what stage of a clinical trial, participants have the right to change their mind. If a participant decides to stop participating, the Principal Investigator will remove them from the trial in a way that is safe for the participant.
Participation usually involves no cost and may involve payment.
For most clinical trials, study medication is provided and visits are conducted at no cost to the participant. However, that's not always the case.
Some clinical trials also pay participants. These payments, if any, tend to vary based upon the phase of the trial. For example, payment for a Phase 1 trial (Researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.) is often more substantial, while payment for Phase 4 trial (Studies that look at real-world experience over a long time and provide additional information on the drug’s risks, benefits, and optimal use.) is often only reimbursement for time and travel.
Participants learn about any costs or payments before enrolling in a clinical trial, during the informed consent process (Before joining, the study is explained to all participants and they’re given the chance to ask questions. The purpose of the informed consent process is to protect the rights of study participants and to make sure they are adequately informed of important details about the clinical study.)
Clinical trials take place in different settings. Clinical trials can be conducted in a variety of places, including hospitals, universities, doctors' offices, and community clinics all around the world. Some clinical trial facilities are fully dedicated to research, while other facilities house both research and healthcare delivery. Similarly, some medical professionals who conduct clinical trials devote all of their time to research, while other professionals split their time between research and healthcare delivery.
The majority of clinical trial participants would do it again. An estimated 2.2 million people participated in clinical trials in 2013. During that same year, a survey by the Center for Information and Study on Clinical Research Participation (CISCRP) found that 95% of clinical trial participants would consider enrolling in another clinical trial. And 91% thought the experience they had in their clinical trial was “Excellent” or “Good.”