Intro to Clinical Trials: FAQs

The idea of joining a clinical research study may be exciting, yet unfamiliar. Choosing to participate in a clinical research study is an important personal decision. It is often helpful to talk with your physician, family members or friends before deciding to participate in a research study.

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Learn about the risks and benefits of medical research participation:

FAQ

What is a clinical trial?
Why do a clinical trial?
Are clinical trials safe?
What is Informed Consent?
Who makes sure my rights are protected?
What are the risks?
Who sponsors clinical trials?
Who conducts a clinical research study?
Should I participate in a clinical research study?
Can I participate in a clinical trial?
What will happen if I volunteer for your trial?
Will I be compensated?
Will participation in a clinical study cost me anything?
What if I enter a study and change my mind?
What are the different types of clinical trials?
How are experimental drugs tested in humans?
What is a “Phase”?
What is a protocol?
What is a control or a control group?
What is a placebo?
Who should I contact for more information?

What is a clinical trial?
A clinical trial is a research study designed to answer specific health questions related to the development, effectiveness, production and evaluation of new disease treatments and therapies.

Before a pharmaceutical company can bring a new drug to market, the investigational drug must be tested in several clinical research studies. A clinical research study is carefully designed to test the safety, effectiveness, and side effects of an investigational medication on a group of volunteers. Clinical research studies are regulated by the Food and Drug Administration (FDA) and are closely monitored by an independent review board and sponsoring pharmaceutical company.

Doctors and other health professionals run the clinical trials according to strict rules set by the FDA. The FDA sets the rules to make sure that people who agree to be in the studies are treated as safely as possible. New therapies are tested on people only after laboratory and animal studies show promising results.

Additionally, many studies are to obtain approval to use an older drug for a new use or for a new patient population. During a clinical trial, the volunteer is carefully monitored and cared for by a research team, including a physician (called a principal investigator) and a research coordinator.

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Why do a clinical trial?

Reasons to participate in a clinical trial include access to new and/or alternative treatments, free medication, access to expert medical attention and even just the desire to leave a legacy by helping the advancement of medicine.  For many trial volunteers, clinical trials are an opportunity to get a peek at upcoming, and in many cases, very promising medications and treatments.

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Are clinical trials safe?
The government has put in place strict guidelines and safeguards to protect those who choose to participate in clinical trials. Before a prospective treatment is tested on humans, it is thoroughly tested through laboratory and model studies to determine if it’s safe. The tests are run by health professionals and doctors according to strict rules set by the Food and Drug Administration (FDA).

While we conduct trials and collect data for the pharmaceutical industry, our foremost concern is the health and safety of you, our research participants. We count on you to provide us with the feedback needed to pass along to the pharmaceutical companies that insures the new medications are safe, effective and side effects well known. Research participants are an essential part of the clinical trial process and we value your feedback and participation.

Participation in a clinical trial is voluntary, and you may withdraw from the study at any time. Your medical condition will be closely monitored throughout the study by experienced research staff and physicians. There are established safety parameters for each study.

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What is Informed Consent?
Informed Consent is a Food and Drug Administration (FDA) requirement that says you must be given complete information about the study before you agree to take part. Each study candidate is given an Informed Consent form to read, which describes in detail what is involved if you participate in the study. It describes the most common risks for the medication being studied and the potential benefits of participating in the study. You will be able to discuss the Informed Consent and ask questions prior to signing up for the study. The study staff and physician will help you fully understand the risks and benefits and your rights as a research participant. If you decide to participate in the research study, you will be asked to sign the consent form.

The FDA requires that you receive and understand the following information:

  • The study involves research of a drug or device
  • The purpose of the research
  • How long the study will take
  • What will happen in the study and which parts of the study are experimental
  • Possible risks or discomforts
  • Possible benefits
  • Other procedures or treatments that you might want to consider instead of the treatment being studied
  • The FDA may look at study records, but the records will be kept secret
  • Whether any medical treatments are available if you are hurt, what those treatments are, where they can be found, and who will pay for the treatment
  • The person to contact with questions about the study, your rights, or if you get hurt
  • You can quit at any time
  • Make sure you understand all of the information before you agree to be in the study

It is a good idea to take the consent documents home and discuss them with family members and your regular physician if you have one. Talking about your options can help you to feel comfortable with your decision. If you decide to join the clinical trial, be sure to ask for a copy of the Informed Consent documents so you can review them at any time. If English is not your native language, you can ask for the consent documents in languages other than English.

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Who makes sure my rights are protected?
The IRB is the formal body in the community charged with overseeing medical research involving people. An IRB functions as both a clinical trials clearinghouse and monitor. In other words, it must give approval before any clinical trial can begin and then keep close tabs on the progress of the research while keeping the safety and wellbeing of all research participants their top priority.

During the initial review process, the Institutional Review Board (IRB) will establish how often a study should be monitored—at least yearly and sometimes more frequently. During these review sessions, the IRB examines a progress report provided by the clinical investigator in charge of the project. The report features information about how many people are enrolled in the study and how many have withdrawn, a description of participants’ experiences, including benefits and/or adverse effects, and the overall progress to date.

Based on this material, the IRB decides whether or not the project should continue as described in the original research plan, and, if not, what changes need to be made. An IRB can decide to suspend or terminate approval of the clinical trial if the investigator is not following set requirements, or, if the study appears to be causing unexpected serious harm to participants.

Some clinical trials—especially large Phase III clinical trials, which often involve many institutions—are monitored more closely and more often by Data Safety Monitoring Boards (DSMB). The DSMB is a group made up of physicians, statisticians, and patient advocates, a majority of whom are not connected with the trial. Their priority is patient safety, and they review the data periodically with the following two questions in mind:

  • Are there any unexpected or severely toxic effects?
  • What is the treatment outcome of the trial so far?

The DSMB is required to produce summary reports that provide regular feedback to the IRB at the institutions involved in the study concerning the cumulative toxicities observed in trial participants.

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What are the risks?
As with most healthcare treatments, there are both benefits and risks to every clinical trial. At Clinical Trials of South Carolina, we strive to ensure that all risks are minimized to keep our volunteers healthy and safe. The risks associated with clinical trials may include:

  • There may be side effects or adverse reactions to medications or treatments.
  • The treatment may not be effective for you.
  • The protocol may require a lot of your time for trips to the study site, treatments, hospital stays, or complex dosage requirements

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Who sponsors clinical trials?
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 Veteran’s Affairs (VA). Trials can take place in a variety of locations, such as hospitals, universities, doctors’ offices, or community clinics.

Funding for clinical research comes from both the federal government through the National Institute of Health (NIH) and from private industry, usually pharmaceutical and biotech companies. The sponsor of the research hires physicians to conduct the clinical trial.

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Who conducts a clinical research study?
A research team led by a doctor, known as the Principal Investigator, conducts clinical research studies. The team may also include nurses and research staff. Meet our clinical research staff. 

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Should I participate in a clinical research study?
Each year, thousands of people volunteer to participate in clinical research studies. By volunteering, you might learn more information about your current medical condition and gain access to new research treatments before they are made available to the public. With the careful medical supervision promised to trial volunteers, you may play a more active role in your health care, and receive free study-related medicines and medical exams during the study. Your feedback could help others with similar medical conditions by contributing to medical research.

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Can I participate in a clinical trial?
Before participating in a trial, volunteers must meet specific criteria. The criteria are based on various factors, not limited to but including age, medical history, current health status, current medications, and allergies. Some research studies seek participants with illnesses or conditions to be studied in the clinical trial, while others need healthy participants.

The factors that allow you to participate in a clinical trial are called inclusion criteria, and the factors that keep you from participating are called exclusion criteria. It is important to note that inclusion and exclusion criteria are not used to reject people personally. Instead, the criteria are used to identify appropriate participants and keep them safe. The criteria help ensure that researchers will be able to answer the questions they plan to study.

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What will happen if I volunteer for your trial?
The first step toward qualifying is to meet with our Research Coordinator. If you qualify, you will be asked to come to the office for a visit. There, you will receive a medical examination, and the Research Coordinator will review the Informed Consent document with you and answer any questions you may have. The time frame and requirements of the trial are explained.

During a trial, you can expect to be given the investigational drug, a placebo or a currently available treatment. You can also expect to be seen by both the doctor and research coordinator at each visit. Study coordinators and physicians closely monitor the participants throughout the study via physical exams, laboratory tests and other medical procedures. Often, these procedures are described as an extension of what is traditionally done to manage the patient’s health and medical condition. Patients in research studies receive study medication, tests and exams at no charge throughout the course of the study. Studies can last for weeks, months or even years, during which time patients can save the enormous cost associated with paying for medications and office visits.

At the end of each trial you should expect to return any unused study drugs, and you will be asked to complete a short questionnaire about your experience. If results of the trials are made public, your input will be published online.

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Will I be compensated?
Some clinical trials will pay you for joining the trial. In some programs, researchers will reimburse you for expenses associated with participating in the research. Such expenses may include transportation costs, childcare, meals, and accommodations.

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Will participation in a clinical study cost me anything?
In exchange for the voluntary participation in our research, we provide professional care, investigational medication and close monitoring for each of our patients at no cost, and there is no need for health insurance.

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What if I enter a study and change my mind?
You can leave the study at any time, for any reason. The Informed Consent form is NOT a contract. If you plan to stop participating, please let the research team know why you are leaving the study, so our office can fill out the proper paperwork for our files.

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What are the different types of clinical trials?
Treatment trials test new treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.

Prevention trials look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes.

Screening trials test the best way to detect certain diseases or health conditions.

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How are experimental drugs tested in humans?
The clinical testing of experimental drugs is normally done in three phases, each successive phase involving a larger number of people. Once the FDA has granted a New Drug Approval (NDA), pharmaceutical companies also conduct post marketing or late phase III/IV studies:

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What is a “Phase”?
Clinical studies are done in a series of four different phases:

Phase I: These studies are primarily concerned with testing the drug’s safety in human volunteers. This initial phase of testing in humans is done in a small number of healthy volunteers (20 to 100), who are usually paid for participating in the study. About 70 percent of experimental drugs pass this initial phase of testing. During this phase, scientists find the best way to give a new treatment, how much they can safely give, and to identify side effects.

Phase II: In Phase II Studies, researchers try to see how well the product works against the disease and to further evaluate the treatment’s safety. This phase lasts from several months to two years. It involves up to several hundred patients (100-300) that have the disease that the product is going to treat. Most of these trials are randomized, i.e. the patients are randomly assigned (similar to flipping a coin) to a “control” group who will receive a standard treatment or placebo (sugar pill). Trials are also typically “blind,” meaning neither the patient nor the physician knows which group the patient has been assigned to. This is done to remove any bias the patient or the physician might subconsciously develop in assisting the patients.

Phase III: In a phase III study, a drug is tested in several hundred to several thousand patients. This large scale testing tries to provide the drug company with a more complete understanding of the drug’s effectiveness, benefits and range of possible side effects, compares it to commonly used treatments, and collects information that will allow the drug or treatment to be used safely. Once a phase III study is successfully completed, the drug company can request FDA approval for marketing the medicine.

Post-Marketing – Late Phase III/IV: In late phase III/phase IV studies, pharmaceutical companies have several objectives: (1) studies often compare a drug with other drugs already in the market; (2) studies are often designed to monitor a drug’s long-term effectiveness and impact on a patient’s quality of life; and (3) many studies are designed to determine the cost-effectiveness of a drug therapy relative to other traditional and new therapies.

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What is a protocol?
All clinical trials are based on a set of rules called a protocol. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical trial, participants are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.

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What is a control or a control group?
A control is the standard by which experimental observations are evaluated. In many clinical trials, one group of patients will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.

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What is a placebo?
A placebo is an inactive pill, liquid, or powder that has no treatment value. In clinical trials, experimental treatments are often compared with placebos to assess the treatment’s effectiveness.

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Who should I contact for more information?
If you would like more information about participating in a clinical trial please contact our office at (843) 637-4010, and a member of our research team will be happy to assist you.

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