What is a clinical trial?
A clinical trial (also clinical research) is a research study in human volunteers to answer specific health questions. Research studies are designed to help determine if a new or modified treatment, such as a medication or device, is safe and effective at treating a specific illness. Carefully conducted clinical trials are an essential component for improving the treatment of medical conditions because they lead to improvements in health care. In the United States all new treatment
products must go through a systematic evaluation process to determine their safety and effectiveness before the Food and Drug Administration (FDA) will approve them for routine use. Prior to FDA approval, these treatments are available only through clinical trials; after FDA approval they become commercially available to ensure that they have an acceptable level of safety and demonstrate benefit to helping patients with a specific disease before they become commercially available to other patients.
Why should I participate in a clinical trial?
By participating in a clinical trial you can play a more active role in your own health care, gain access to new research treatments before they are widely available, and help others by contributing to medical research.
What are the phases of clinical trials?
After being studied in a laboratory and animal studies, most new treatments are examined in a series of clinical trials that follow a progression of steps or phases. Each phase is designed to determine specific information about the potential new treatment such as its risks, safety and effectiveness. Each of the following phases have a different purpose and help scientists answer different questions:
Phase I trials: Tests an experimental 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. Once information is gathered and analyzed a Phase II trial may begin.
Phase II trials: The experimental 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.
Phase III trials: The experimental 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 experimental drug or treatment to be used safely. Subjects participating in Phase III trials are usually assigned to one of three treatment groups:
- The test medication or treatment, or
- The standard (or currently available) treatment, or
- A placebo (an inactive medication or treatment)
Phase IV trials: Conducted after the drug or treatment becomes part of standard therapy (approved by the FDA), also known as post market studies. These studies seek to obtain additional information including the treatment risks, benefits, efficacy and optimal use.
What is informed consent?
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 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.
What are the risks of clinical trials?
Both standard care and clinical trials have risks, side effects and benefits that vary depending on the individual. However, rigorous guidelines are in place to protect the well-being and safety of participants.
- There may be unpleasant, serious or even life-threatening side effects to experimental treatment.
- Experimental treatments must be evaluated for both immediate and long-term side effects.
- The experimental treatment may not be effective for the participant.
- The study may require more of time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements.
How is the safety of the participant protected?
The ethical and legal codes that govern medical practice also apply to clinical trials. In addition, most clinical research is federally regulated with built in safeguards to protect the participants. The trial follows a carefully controlled protocol, a study plan which details what researchers will do in the study. As a clinical trial progresses, researchers report the results of the trial at scientific meetings, to medical journals, and to various government agencies. Note: Individual participants' names will remain secret and will not be mentioned in these reports.
What are the types of costs associated with a clinical crial?
Standard of Care costs: Standard-of-care (SOC), or usual care costs, costs are those costs that would occur regardless of your participation in a clinical trial and are usually covered by your health insurance. SOC costs include doctor visits, hospital stays, clinical lab tests, x-rays. Research Costs: These costs are associated with participation in a clinical trial and are not considered part of your SOC. This includes data collection, research physician and nurse time, study labs and tests performed specifically for the research trial. These costs are usually covered by the company sponsoring the trial. It is important to discuss the potential costs of study participation with the research staff prior to your involvement.
For up-to-date information about Medicare coverage of clinical trials, visit http://www.cms.hhs.gov/
Who can participate in a clinical research trial?
You may choose to participate in a clinical research trial, but the researchers must determine whether you are eligible. Each clinical research trial includes people who have the same or very similar health profiles based on specific inclusion and exclusion criteria. When selecting participants, researchers may consider these or other factors: medical history, medication history, current medical status, and your willingness and ability to follow all of the trial’s instructions and schedules.