1. What is a clinical study?
2.   Who can be in a clinical study?
3.   What is informed consent?
4.   How is a drug approved for testing?
5.   How are experimental drugs tested in humans?
6.   What is the difference between the phases of a study?
7.   Who pays for clinical research?
8.   Should I volunteer?

 
 
Q.   What is a clinical study?   [back to top]
 
A.      A clinical study or clinical trial is designed to test the effects of medication, medical treatment or device on a  group of volunteers.   These studies are used to measure the drug's ability to treat a condition, safety, and a drug's possible side effects.
 
Q.   Who can be in a clinical study?    [back to top]
 
A.      People with the condition the drug is developed for as well as people who are healthy can volunteer to participate in a study.  The FDA has strict requirements as to whether a study will involve healthy people or just people with the condition being studied.

          Each study has its own requirements and they can include requirements like age, sex, or a medical condition  for a person to qualify.  In addition to the study requirements the physician will review each participants medical history and the study requirements to determine who can participate.  Known risks and discomforts will be explained by the study physician prior to a volunteers participation in a study.  In addition to the known risk, there may also be some unknown medication side effects involved in participating in a clinical study.  The procedures, risks, and benefits are explained to potential participants during the informed consent process.

 
Q.    What is informed consent?   [back to top]
 
A.      Informed consent is a process designed to ensure that volunteers have all the information they need to decide whether or not they want to participate in the study.  This process allows the volunteer to ask questions  and exchange information with the clinical investigator.  The clinical investigator is responsible  for ensuring that informed consent is obtained before a volunteer is allowed to participate in the study.
 
Q.   How is a drug approved for testing?    [back to top]
 
A.      The U.S. Food and Drug Administration or FDA typically must authorize a drug company's proposal to conduct a clinical study.  Before the FDA gives authorization for a study a drug company must do years of  preclinical or laboratory research before they can begin testing in humans.  This research often involves years of  experiments with animal and human cells.  The compounds are also extensively tested in animals.  If this stage is successful the pharmaceutical company provides this data to the FDA requesting approval to begin testing in humans.
 
Q.   How are experimental drugs tested in humans?     [back to top]
 
A.      The clinical testing of experimental drugs is normally done in three phases, each successive phase will involve a larger number of volunteers.
 
Q.   What is the difference between the phases of a study?    [back to top]
 
A.      Phase I Study
             Phase I studies studies are concerned with the drug's safety.  This phase of testing is performed on a small number of  healthy volunteers.  The study is designed to determine what happens to the drug in the human body -- how it is absorbed, metabolized, and excreted.  A Phase I study also investigates side effects as the dosage level is increased.  This initial phase of testing generally takes several months.

          Phase II Study
           Phase II studies are concerned with the drug efficacy or effectiveness.  This phase is only conducted after the drug is shown to be safe.  The second phase of testing can involve several hundred patients.  Most Phase II studies are randomized trials.  One group of patients will receive the experimental drug while the second  "control" group will receive the standard treatment or placebo.  These studies are often blinded which means neither the  patients nor the researchers know who is getting the experimental drug.  When a study is blinded it can provide the pharmaceutical company and the FDA with the relative safety and effectiveness of the new drug. 

          Phase III
           In a Phase III study, a drug is tested in several hundred to several thousand patients.  The large scale of this phase provides the pharmaceutical company as well as the FDA with a more thorough understanding of the new drug's effectiveness, benefits, and the range of possible adverse reactions.  Most Phase III trials are blinded and randomized.  Once the Phase III trial is completed the pharmaceutical company can request FDA approval to market the new drug.

          Phase IV
            Sometimes pharmaceutical companies will initiate another study which can be called a post-marketing or a Phase IV study.  In Phase IV studies pharmaceutical companies have several objectives.

  1. The study will often compare a drug with other drugs already in the market.
  2. The study is often designed to monitor the drug's long-term effectiveness and impact on a patients quality of life.
  3. Many of the studies are designed to determine the cost effectiveness of a drug therapy relative to other traditional and new therapies. 
 
Q.   Who pays for clinical research?    [back to top]
 
A.      Funding for clinical research comes from both the government (through the National Institutes of Health) and private industry (the pharmaceutical and biotech companies).  The sponsor of the research  hires the physicians who may work in a wide variety of health car settings, to conduct the clinical trial.  The physicians are typically paid on a per patient basis.  The medical care is provided for free to the patient.  Patients may also be paid for their participation for in the clinical trial.
 
Q.   Should I volunteer?    [back to top]
 
A.      Volunteering to participate in a clinical drug study is one of the best ways you can contribute to the understanding of diseases that affect people and the development of new therapies.  Volunteers are important to the clinical study phase of the drug approval process.  As a volunteer, you are the most criticallink in a long chain of research and testing in the development of new medications for a broad range of health problems.