1. What is a clinical trial?
Clinical trials, also called clinical studies, are research studies with people. Each trial seeks 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 phases, each phase designed to discover new information about the drug or treatment.
- Phase I trials are the first step in testing a new approach in humans. 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. The phase the 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, 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.
2. What is a control group?
The standard by which experimental observations are evaluated. In may 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.
3. What is a placebo?
A placebo is an inactive pill, liquid, or powder that has no treatment value.
4. What is randomization?
A method based on chance by which study participants are assigned to a treatment group. Randomization minimizes the difference among groups by equally distributing people with particular characteristics among all trial arms or treatment groups.
5. What is a double-blind study?
A clinical trial designed in which neither the participating individuals nor the study staff knows which participants are receiving the experimental drug and which are receiving a placebo (or another therapy). Double-blind trials are thought to produce objective results, since the expectations of the doctor and the participant about the experimental drug do not affect the outcome.
6. What is protocol?
A study plan on which all clinical trials are based. 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.
7. What is informed consent?
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, and then decide freely whether or not to participate. Informed consent is required in studies that are federally regulated or funded, as well as by many state laws.
8. What is are exclusion/inclusion criteria?
These are commonly referred to as "inclusion" and "exclusion" criteria. These are the medical or social standards determining if a person 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. These criteria are set to identify appropriate participants and keep them safe.
9. What is a contraindication?
These are specific circumstances when the use of certain treatments could be harmful.
1. Are research studies confidential?
Confidentiality is guarded for all patients. Investigators files and records are secured so that all identifying information is protected.
2. Are trial costs covered by health insurance?
Costs of routine care in many clinical trials, especially phase III trials, are now covered by many health insurance plans and Medicare. Make sure and check with your health insurer before enrolling in a clinical trial.
3. Can I leave the trial at any time?
Yes, 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.
4. What protection do I have as a patient in a clinical trial?
The ethical and legal codes that govern medical practices apply to clinical trials. 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 by researchers at other places. University Health System has designated the review of their research protocols to the Institutional Review Board at the UT Health Science Center at San Antonio (UTHSCSA). To learn more about Research at the UTHSCSA, please see http://research.uthscsa.edu.
5. What are Investigational Drugs?
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. This can include any drug that has not yet be approved for a new or additional indication by the FDA. The FDA is the federal agency with oversight responsibility for drugs and biologics in the U.S. They must approve a drug before it can be used. For more information on the FDA please see http://www.fda.gov/cder/index.html.
6. What is the role of the Research Pharmacist?
The research pharmacist at UHS is Javier Palacios, RPh. In this academic health center his role includes preparation and dispensing of the investigational drugs, ensuring that research volunteers receive proper doses at proper times as defined in the research protocol. He is the only individual that knows if the patient is receiving the study drug or placebo. He maintains the records for dispensing these medications and ensuring staff are educated on the investigational drugs.
7. Are investigational drugs ever used prior to FDA approval?
Compassionate use of experimental therapeutics prior to FDA approval is referred to as compassionate use. This procedure is used with very sick individuals who have no other treatment options. Individual approval must be obtained for the "compassionate" use of an investigational drug or therapy.
8. What is efficacy of a drug or treatment?
This is 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. In the procedure mandated by the FDA, phase II clinical trials gauge efficacy and Phase III trials confirm efficacy.
9. What is a Medical Device?
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 to see if:
- the product does what it claims to do effectively, and
- does not present any unreasonable risks to the patient.
For more information from the FDA on medical devices please see http://www.fda.gov/medicaldevices/deviceregulationandguidance/guidancedocuments/default.htm.