Before you participate in a clinical trial, you’ll want to know more about how it works and what to expect. We’ve assembled some frequently asked questions about clinical trials to help you get started. Your physician will be able to tell you more details as they relate to you and your personal situation.
What Is a Clinical Trial?
Clinical trials, also called clinical studies, are research studies with people. Each trial is designed to answer specific scientific questions and to find better ways to prevent, detect or treat diseases or to improve care.
Clinical trials are divided into four phases. Each phase is designed to discover new information about the drug or treatment.
- Phase I trials are the first step in testing a new approach. In phase I trials, a new drug or treatment is tested first in a small group of people to evaluate safety and dosage and identify side effects. This phase of testing is associated with the highest amount of risk.
- Phase II trials further evaluate the effectiveness and safety in a larger group of people.
- Phase III trials evaluate a drug or treatment in a larger group of people to confirm effectiveness and monitor side effects, and compare it to commonly used treatments.
- Phase IV trials further study a drug or treatment, collecting information about the effects in various populations and any side effects associated with long-term use.
Are Research Studies Confidential?
Yes. Confidentiality is guarded for all patients. Investigators’ files and records are secured so that all identifying information is protected.
Does My Health Insurance Cover the Costs of a Clinical Trial?
Possibly. Many insurance plans and Medicare cover costs of routine care in many clinical trials, especially phase III trials. Make sure to check with your health insurer before enrolling in a clinical trial.
Can I Leave the Clinical Trial at Any Time?
Yes. Participating in a clinical trial is voluntary. You can change your mind at any time after entering a trial. You may also refuse to take part in any aspect of the research.
What Protection Do I Have As a Patient in a Clinical Trial?
Clinical trials have the same ethical and legal codes that govern medical practices. In addition, most clinical research is federally regulated or federally funded (at least in part), with built-in safeguards to protect patients. These safeguards include regular review of protocol and the progress of each study. University Health has designated the review of their research protocols to the Institutional Review Board at UT Health San Antonio.
A control group is the term that describes the standard by which researchers evaluate experimental observations. 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.
Randomization is a method based on chance that determines how participants are assigned to a treatment group. Randomization equally distributes people with particular characteristics, minimizing the differences from one group to another.
A double-blind study is a clinical trial designed in which neither the participants nor the study staff knows which participants are receiving the experimental drug and which are receiving a placebo (or another therapy).
A protocol is a study plan for the clinical trial. The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions. 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.
Informed consent means you, as the patient, are given information so you can understand what is involved in a trial, including any potential benefits and risks. Then you can decide freely whether or not to participate. Most studies require you to have informed consent.
Exclusion and inclusion criteria are the medical or social standards that determine if you may or may not be allowed to enter a research study. These criteria are generally based on such factors as age, gender, the type or stage of a disease, previous treatment history or other medical conditions.
An investigational drug is one that is under study but does not have permission from the U.S. Food and Drug Administration (FDA) to be legally marketed and sold in the United States, or used for an unapproved purpose. The FDA is the federal agency that approves and oversees drugs and biologics in the United States.
At University Health, the research pharmacist’s role includes preparing and dispensing investigational drugs, ensuring that you receive proper doses at proper times as defined in the research protocol. The research pharmacist is the only individual who knows if you’re receiving the study drug or placebo. They keep records and ensure research staff are educated on the investigational drugs.
Compassionate use of experimental therapeutics before FDA approval is referred to as compassionate use. This procedure is used with very sick individuals who have no other treatment options, and requires individual approval.
Efficacy refers to the maximum ability of a drug or treatment to produce a result regardless of dosage. A drug passes efficacy trials if it is effective at the dose tested and against the illness for which it is prescribed.
A medical device is "an instrument, apparatus, implement, machine, contrivance, implant, in vitro reagent or other similar article that is intended for use in the diagnosis of disease or other conditions, or in the cure, mitigation, treatment or prevention of disease." Medical devices can be anything from thermometers to artificial hearts to at-home pregnancy test kits. The FDA reviews and approves devices through investigational studies.