The Rogosin Institute

What Is A Clinical Study?

A clinical study is a research study to answer specific questions about new therapies or new ways of using known treatments or vaccines. Clinical studies (also called medical research and research studies) are used to determine whether new drugs or treatments are both safe and effective.  New therapies are tested on people only after laboratory and animal studies show promising results.  Carefully conducted clinical studies are the fastest and safest way to find treatments that work.

back to top

What Is A Protocol?

All clinical studies are based on a set of rules called a protocol.  A protocol describes what types of people may participate in the study; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical study, participants are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.

back to top

Are There Protections For People Who Participate?

The government has strict guidelines and safeguards to protect people who choose to participate in clinical studies.  Every clinical study in the U.S. must be approved and monitored by an Institutional Review Board (IRB) to make sure the risks are as low as possible and are worth any potential benefits.  An IRB is an independent committee of physicians, statisticians, community advocates, and others that ensures that a clinical study is ethical and the rights of study participants are protected.  All institutions that conduct or support biomedical research involving people must, by federal regulation, have an IRB that initially approves and periodically reviews the research.

back to top

What Is Informed Consent?

Informed consent is the process of learning the key facts about a clinical study before you decide whether or not to participate.  If you are considering joining a clinical study, the research staff will give you informed consent documents that include the details about the study.  Since joining a clinical study is an important decision, you should ask the research team any questions you may have about the study and the consent forms before you make a decision.  It is also a good idea to take the consent documents home and discuss them with family members or friends.  Talking about your options can help you feel comfortable with your decision.  Informed consent is more than signing a form. It is a process that continues throughout the study.  You should feel free to ask the research team questions before, during, and after the study.

back to top

Who Can Participate In A Clinical Study?

All clinical studies have guidelines about who can get into the program.  Before you join a clinical study, you must qualify for the study.  Some studies seek participants with illnesses or conditions to be studied in the clinical study, while others need healthy volunteers.  The factors that allow you to participate in a clinical study are called inclusion criteria and the factors that keep you from participating are called exclusion criteria.  It is important to note that inclusion and exclusion criteria are not used to reject people personally.  Instead, the criteria are used to identify appropriate participants and keep them safe.  The criteria help ensure that researchers will be able to answer the questions they plan to study.
back to top

Who Sponsors Clinical Studies?

Clinical studies are sponsored by government agencies such as the National Institutes of Health (NIH), pharmaceutical companies, individual physician- investigators, health care institutions such as health maintenance organizations (HMOs) and organizations that develop medical devices or equipment.  Studies can take place in a variety of locations, such as hospitals, universities, doctors' offices, or community clinics.

back to top

What Happens During A Clinical Study?
 

The clinical study process depends on the kind of study you participate in.  The team will include doctors and possibly nurses, dietitians and other health care professionals.  They will check your health at the beginning of the study, give you specific instructions for participating in the study, monitor you carefully during the study, and stay in touch with you after the study.   Some clinical studies involve more tests and doctor visits than you would normally have for your illness or condition.  For all types of studies, you will work with a research team.  Your participation will be most successful if you follow the protocol carefully and stay in contact with the research staff.  These are some terms that will help you understand what happens in a study.

back to top

What is a placebo?
 

A placebo is an inactive pill, liquid, or powder that has no treatment value.  In clinical studies, investigational treatments are often compared with placebos to assess the treatment's effectiveness.  In some studies, the participants in in the control group will receive a placebo instead of an active drug or treatment.

back to top

What is a control or control group?

A control is the standard by which investigational observations are evaluated.  In many clinical studies, one group of participants will be given an investigational drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.
back to top

What are single and double-blind studies?

In a single-blind study, participants do not know whether they are in the investigational or control group in a research study.  Those in the investigational group get the medications/treatments being tested, while those in the control group get a standard treatment or no treatment.  

In a double-blind study, neither the participants nor the study staff knows which participants are receiving the investigational treatment and which ones are getting either a standard treatment or a placebo.  These studies are performed so neither the patients' nor the doctors' expectations about the investigational drug can influence the outcome.
back to top

What are side effects and adverse reactions?
 

Side effects are any undesired actions or effects of drug or treatment.  Investigational treatments must be evaluated for both immediate and long-term side effects.

back to top

What are the benefits and risks associated with clinical studies?

There are both benefits and risks associated with clinical studies:
By participating in a clinical study, you can:  Take an active role in your own health care, gain access to new treatments that are not available to the public, obtain expert medical care at leading health care facilities during the study, help others by contributing to medical research.

Clinical studies have risks:  There may be side effects or adverse reactions to medications or treatments, the treatment may not be effective for you, the protocol may require a lot of your time for trips to the study site, treatments, hospital stays, or complex dosage requirements.

back to top

What Should I Know Before I Join A Clinical Study?
 

You should know as much as possible about the research study.  It is important for you to feel very comfortable asking questions and the staff should answer them in a way you can understand.  Plan ahead and write down the questions you want to ask.  Ask a friend or relative to come with you for support and to hear the responses to your questions.


Questions about the research include:  

• Why is this research being done?  
• What is the purpose of the study?  
• Who is sponsoring the study?  
• Who has reviewed and approved this study?
• Why does this research team think the treatment, drug, or medical device will work?
• Questions about participation include:
• Where is the study site? How long is  the study?  How often will I have to go to the
  study site? Will I have to be in the hospital during the study?  Can anyone find out
  that I am in a study?
• What kinds of therapies/procedures/tests will I have during the study? Will they hurt?
  If so, for how long?
• How will the tests in the study compare to tests I would have outside the study?  
• Who will provide my medical care after the study ends?  Will I be able to take my regular medications during the study?  
• What medications, procedures, or treatments must I avoid while in the study? 
• What are my responsibilities during the study?  
• Will the study researchers work with my doctor while I am in the study?  
• Can I talk to other people in the study?  
• Will I be able to find out the results of the study?

Questions about risks and benefits include:  

• How do the possible risks and benefits of the study compare with approved
  treatments for me?  
• What are possible short and long-term side effects?
• Other questions include:  
• What other treatment options do I have?  
• Will I have to pay anything to be in the study?  What may the charges be and
  will my insurance cover those expenses.

back to top

Should I Continue Working With My Primary Health Care Provider If I Participate In A Study?
 

Yes.  Most clinical studies provide short-term treatments related to a designated illness or condition, but not extended or complete primary health care.  Also, by having your health care provider work with the research team, you can ensure that your other medications or treatments will not conflict with the protocol.

back to top

Can I Leave A Clinical Study After It Has Begun?
 

Yes.  You can leave a clinical study at any time. If you plan to stop participating, let the research team know why you are leaving the study.

back to top

Will I Be Paid For Participating In A Clinical Study?
 

Some clinical studies will pay you for joining the study, while others will not.

back to top

Source: National Institute of Allergy and Infectious Diseases,
National Institutes of Health