What are clinical trials?
Clinical research translates basic science and discoveries into safe and effective tests, drugs, devices and procedures, eligible for approval by the U.S. Food and Drug Administration (FDA). Clinical trials are carefully designed and monitored studies intended to test and evaluate investigational drugs and treatment plans.
Why participate in clinical research?
Many participants are patients whose own illnesses are not responding to standard treatment, and who hope that the study treatment – possibly an investigational drug, a device, or a combination of drugs or devices – will work better than standard therapy.
Other participants are healthy people who want to contribute to medical science and to help doctors and researchers find better ways to treat others’ illnesses.
How safe are clinical trials?
Researchers follow strict ethical and scientific principles to ensure that patients are protected and that the results produced are valid. The study’s sponsor – a drug or device company – will draft a plan of how the study will proceed. This plan, or protocol, specifies how many individuals will be enrolled, how much drug will be used and when, what medical tests will be provided, and so on.
Evaluating new treatments involves some risks. Using a small group of volunteers, Phase I trials determine the best dosing and identify side effects. After a drug is shown to be safe and well-tolerated, Phase II and Phase III trials provide treatment to a larger numbers of patients with a specific disease. Side effects can vary, with dangerous complications possible.
To ensure that participants are not be exposed to unreasonable or unethical risks, every protocol is reviewed and approved by the sponsoring organization as well as an Institutional Review Board (IRB), which consists of health professionals, clergy and other community members. At Rochester General, the Office of Human Research Protection fulfills the IRB role.
Do I need a referral from my physician to participate in a clinical trial?
Your doctor’s referral is not necessary, but anyone seriously considering a trial is urged to talk with his or her doctor and family about the potential benefits and risks. In order to participate, you will be evaluated by the medical staff conducting the study to determine whether you meet the medical criteria.
If you’re approved to join a trial, you should continue seeing your regular physician. Trial coordinators will consult with your doctor about how the study’s therapies are impacting you.
Will I have to pay to participate?
In most cases, the drugs and devices used, and any procedures for the study such as blood work, urine samples, x-rays and travel expenses are provided free to participants. Other costs unrelated to the trial may be covered by the patient’s insurance company or Medicare. Be sure to get a full accounting of costs before committing to a trial.
What are the phases of clinical research?
Each phase of clinical research is designed to gather specific information about the study drug, device or treatment.
Phase I
A Phase I trial is designed to determine the best dose of the investigational drug and to identify any potential side effects. Because a Phase I study represents the first time an investigational drug or therapy is being used on humans, the risks can be significant. Such trials typically involve a small number of volunteers.
Phase II
Begun after an investigational treatment is initially shown to be safe and well-tolerated, aPhase II trial studies how well the treatment works, usually in a larger group of patients. Early- and late-stage Phase II trials may be called Phase IIA and Phase IIB.
Phase III
Phase III trials examine how well the drug or therapy works in hundreds or many thousands of patients. In this phase, the study drug may be compared to an existing standard treatment. Early- and late-stage Phase II trials may be called Phase IIIA and Phase IIIB.
Phase IV
After a drug has been approved by the U.S. Food and Drug Administration and marketed, Phase IV trials – typically involving a large number of participants – may evaluate new uses of existing therapies or be used to detect side effects that did not appear during Phase III.
What else do I need to know before participating in a trial?
The National Institutes of Health guides potential participants in deciding whether to participate, and encourages that individuals ask the following questions:
- What is the purpose of the trial?
- What kind of tests and treatments are involved?
- What is likely to happen in my case with, and without, this new research treatment?
- What are alternative treatment options and their advantages and disadvantages?
- How might this trial affect my daily life?
- What side effects might I expect from the study?
- How long will the trial last?
- Will hospitalization be required?
- If I were harmed as a result of the trail, what treatment would I be entitled to?
- What type of long-term follow-up care is part of this study?
Where can I learn more about clinical research?
The following organizations provide additional information:
- Food and Drug Administration Consumer Health Information
- The Center for Information and Study on Clinical Research Participation (1-888-CISCRP3)
- ClinicalTrials.gov – a registry of federally and privately supported clinical trials currently being conducted in the United States and around the world
- VeritasMedicine.com – the source for some of the above questions and answers