Trials are performed for all medications, medical devices and procedures before getting FDA approval.  These trials are performed in different phases and all data and information is recorded and sent to the FDA, prior to approval.  After laboratory testing and animal testing, the trials enter human phases.

Phase I Studies

Phase I studies is the first step after the investigational product has entered human trials.  This phase includes a small number (20-80) of participants. These are usually healthy volunteers, but in some cases can be patients with advanced disease (e.g. cancer, mental health disorders).  Phase I studies can last anywhere from a few days to several months.  Approximately 70% of Phase 1 studies continue to Phase 2.

Phase I studies seek to:

• Evaluate safety of the drug or device
• Identify side effects
• Determine a safe dose range
• Learn how the investigational product is absorbed and handled by the body (pharmacokentics/dynamics)

Phase II Studies

Phase II studies enroll a larger selection of participants, about 100 to 300.  These participants generally have the condition that the drug or device seeks to improve.

In Phase II studies, researchers further evaluate safety and determine if the investigational product has the intended effect in humans. These studies are sometimes randomized controlled trials where one group of patients receives the investigational product, while another group (the “control group”) receives a standard treatment or placebo. In order to provide unbiased comparative information, these study are often “blinded” which means neither the patients nor the researchers know who is receiving the investigational product.

Phase II studies usually take up to two (2) years to complete. About 33% of Phase 2 conducted successfully move on to Phase 3.

Phase III Studies

In Phase III, larger numbers (thousands) of patients with the disease are included to:

• Confirm or further evaluate effectiveness of the drug, device or treatment
• Further monitor side effects, including those stemming from longterm usage
• Compare it to current common treatments
• Collect other information that will be used to determine whether the agent should be approved and marketed

Phase III studies are usually randomized and blinded trials that last anywhere from about 6 months up to 5 years to complete. About 25% to 30% of Phase III studies are successfully completed.  The data gathered from the trials in this phase is submitted to the FDA by the pharmaceutical company to gain approval to market the drug or device in the United States.

Phase IV Studies

Phase IV studies, often called Post-Marketing Surveillance Trials, are conducted after the investigational product has been granted approval from the FDA for consumer sale. These are not required, but can be requested by the FDA to obtain more information.  These studies involve people in various populations and are generally intended to collect additional information after the investigational product is approved and marketed regarding its risks, benefits and long-term effects. Results from Phase IV studies can influence whether an investigational product should be taken off the market or if restrictions should be applied to the use of the investigational product.

WHY SHOULD I PARTICIPATE IN A CLINICAL TRIAL?

By taking part in a clinical trial, you have an opportunity to try a new treatment that may or may not be better than those that already exist.

As a study participant, you can help medical professionals better understand how the treatment works in people of different races, genders and age ranges.  Regulatory agencies such as the FDA seeks to ensure that people of different ages, races, ethnicities, and genders are included in clinical trials.

Some reasons to participate in a clinical trial:

• Access to new treatments not available on the market.
• Access to free medication.
• Compensation or reimbursement for participating (in some trials).
• Satisfaction in helping advance medical research.
• Your participation is voluntary which means you can leave the study at any time.

Each clinical research study has specific eligibility criteria that a participant must meet in order to be enrolled in the study. Therefore, not everyone who applies for a clinical research study will be accepted.

By taking part in a clinical trial, you have an opportunity to try a new treatment that may or may not be better than those that already exist.

As a study participant you can also help others better understand how the treatment works in people of different races and genders because different people may respond differently to the investigational product. Regulatory agencies such as the FDA seeks to ensure that people of different ages, races, ethnicities, and genders are included in clinical trials.

Some reasons to participate in a clinical trial:

• Access to new treatments not available on the market.
• Access to free medication.
• Compensation for participating (in some trials).
• Satisfaction in helping advance medical research.
• Your participation is voluntary which means you can leave the study at any time.

Each clinical research study has specific eligibility criteria which a participant must meet in order to be enrolled in the study. Therefore, not everyone who applies for a clinical research study will be accepted.

There are many different types of clinical research studies that call for participants of all ages, gender, race, and degrees of health:

• Prevention studies look for better ways to deter disease or inhibit it from returning. These treatments may include medicines, vitamins, vaccines, minerals, or lifestyle changes.
• Studies that test new treatments, new combinations of drugs, or new approaches for therapy.
• Diagnostic studies help find better tests or procedures for diagnosing a particular disease or condition.
• Screening studies test the best way to detect certain diseases or health conditions.
• Quality of Life studies (or Supportive Care studies) explore and measure ways to improve comfort and the quality of life for individuals with a chronic illness.

Usually, clinical trials compare a new product or therapy to existing treatments to see if it works as well or better to treat or prevent a disease or condition. In a blinded study, a participant may be randomly assigned to receive the test product, or a different existing, approved therapy.

In some studies, participants may receive a placebo which offers no therapeutic action, yet looks or acts like the test product. Comparison with a placebo can be the best way to test the effectiveness of a new product.  Potential participants are informed before they enter a trial whether placebos are going to be used in the study and the risks and benefits of the study medication(s).

A clinical research study is conducted according to a research plan known as a study protocol that is designed to answer specific research questions and to assure the safety of the participants. The protocol is put out by the pharmaceutical company and includes the following information:

• The reason for conducting the study
• Who may participate in the study (inclusion/exclusion criteria)
• The number participants needed
• The schedule of tests, procedures, investigational product and their dosages
• The length of the study
• What information will be collected about the participants

from the overall participation in the study.

What happens during a study?

The clinical research study follows the general outline below:

Pre-Screening

The clinical research group will review their existing patient databases or medical charts to identify patients who may be eligible to participate in the clinical research study. Participants can also be found by advertisements on the internet, newspaper, radio, or television.  The research staff may also conduct seminars, free health screenings, or forums.

Responders to these pre-screening efforts are contacted and briefly interviewed to confirm if they are possibly eligible to participate in the clinical research study.

Informed Consent

If you are eligible to participate in a clinical research study, an appointment will be scheduled for you to come to the clinical research site and meet with a research staff member. Before any study related procedures are performed you will be required to sign an informed consent form which details the study design, indicates the risks and benefits, your rights as a study participant, and who to contact in case of an emergency. During the informed consent visit, you will have an opportunity to ask any questions you may have about the study and your participation. You should receive a copy of the signed informed consent form for your records.

Screening Visit

Once you agree to participate in the clinical research study and have signed the informed consent form, the study screening procedures will begin. The purpose of the screening visit is to determine if you meet the specific inclusion and exclusion criteria for the specific clinical research study in which you agreed to participate.  During the screening visit, you will be asked to answer questions regarding your medical history, medications and other treatments you are taking, and to complete questionnaires. The research team member may also assess your general health by performing a physical examination, EKG and collect blood/urine samples.

The investigational product is not administered during the screening visit.

Study Visits (Treatment Visits)

If you meet the specific criteria, to include specified medical and laboratory results, for the study you will be asked to return to the clinic for a series of study visits sometimes referred to as Treatment Visits. It is during these study visits that a study participant will receive the investigational product or the comparator product (which sometimes can be a placebo). The number of study visits and the interval in which the visits occur can vary from study to study.

End of Study Visit

Once you have reached the end of the treatment visits, an End of Study Visit takes place. During this visit, the same or similar assessments conducted during the screening visit (prior to you receiving the investigational product) is repeated. The research team will also discuss with you any follow-up treatment options (which may include receiving standard of care treatments for your illness).  You may be asked to return to the research site for a follow-up safety visit, depending on the requirements of the study.

Follow-up Safety Visit

The number of follow-up safety visits and the interval in which the visits occur can vary for each study. The purpose of these visits is to ensure you are not experiencing any lingering side-effects from the investigational product or

Risks are involved in clinical research, as in routine medical care and activities of daily living. Most clinical research studies pose side effects that are temporary and go away when the treatment is stopped. However, some research subjects experience side effects that can be permanent or require medical attention. Some side effects appear during treatment, while others may not show up until after the treatment is over. Some investigational products that are being tested have side effects that can be unpleasant, serious or even life-threatening. Because the investigational products being studied are new, researchers don’t always know what the side effects will be in humans. The specific risks associated with any research protocol are described in detail in the informed consent document, which you are asked to sign before taking part in research. In addition, the major risks of participating in a study will be explained to you by a member of the research team, who will answer your questions about the study. Before deciding to participate, you should carefully weigh these risks. Although you may not receive any direct benefit as a result of participating in a clinical research study, the information collected by your participation may help others.

To help you decide if you want to be in a study, the FDA requires that study participants are given complete information about the study before they agree to take part.

Informed consent forms should be written so the participant can easily understand it and should include:

• purpose of the research
• how long the study will take
• what will happen in the study and which parts of the study are experimental
• possible risks or discomforts
• possible benefits
• other procedures or treatments that the participant might want to consider instead of the treatment being studied
• that FDA may look at study records, but the records will be kept secret
• whether any medical treatments are available if the participant is hurt, what those treatments are, where they can be found, and who will pay for the treatment
• the person to contact with questions about the study, participant rights, or if the participant can get hurt
• the participant can quit at any time

If you don’t understand the information included in the informed consent form, be sure to ask the doctor or other research staff member to explain it. This discussion should take place in private, and you must be given enough time to make an informed decision. In most studies, you should be allowed to take the consent form home for further discussion with others, such as family, caregivers and primary care physicians. Make sure you understand all of it before you agree to be in the study.

Before you can be in the study, you must sign the informed consent form, showing that you have been given this information and understand it. The informed consent form is NOT a contract and you can leave the study at any time, for any reason. You may ask questions at any time throughout the study.

In addition, you must also be informed of any new information learned during the study that may affect your willingness to continue to participate in the study.

Here are some questions to ask your doctor to help you decide if you want to take part in a clinical trial:

• What is the study trying to find out?
• What kinds of test and exams will I have to take while I’m in the study? How much time do these take? What is involved in each test?
• How often does the study require me to go to the doctor or clinic?
• Will I be hospitalized? If so, how often and for how long?
• What are the costs to me? Will my health insurance pay for it?
• What follow-up will there be?
• What will happen at the end of the study?
• What are my other treatment choices? How do they compare with the treatment being studied?
• What side effects can I expect from the treatment being tested? How do they compare with side effects of standard treatment?
• How long will the study last?

The study protocol outlines who can participate in a clinical research study referred to as the Inclusion/Exclusion Criteria. The criteria that allows a person to participate in a clinical research study are called inclusion criteria; and the criteria that disqualifies a potential research subject are called exclusion criteria. These criteria are based on factors such as age, gender, the type and stage of a disease, previous/current treatment history, and other medical conditions. A research subject is a person who meets the inclusion/exclusion criteria and agrees to take part in a clinical research study. A research subject may be either a healthy individual or an individual with a specific disease or condition.

Every clinical research study is spearheaded by a Principal Investigator.  Often this is a licensed medical doctor who leads a team of other doctors, nurses, social workers, and other health care professionals. The clinical research team roles and responsibilities include:

• Principal Investigator (PI): A person responsible for the ethical conduct of the research study. This includes protecting human subjects’ rights, safety and welfare, study compliance with the protocol set forth by the sponsor, and adherence to institutional, state and federal regulations and guidelines. The PI is responsible for ensuring informed consent received from each participant and for appropriately maintaining study records. The PI is also responsible for ensuring the study complies with all financial and administrative policies and regulations, directing overall fiscal management of the study, and obtaining any conflict of interest disclosures that are relevant. The PI oversees all aspects of a clinical trial from protocol design, recruitment of participants, data collection, analysis and interpretation of results.  The PI is also responsible for ensuring that all research team members have appropriate education, training and qualifications to assume delegated tasks.
• Sub-Investigator: The Sub-Investigator may perform some or all of the PI functions, but they do not accept primary responsibility for the research study. The sub-investigator is under the supervision of the PI and is responsible for performing study–related procedures. The Sub-Investigator will often make important study-related decisions to be in compliance with the ethical guidelines of the study.
• Research Coordinator: The Research Coordinator oversees and coordinates the daily activities of clinical research studies. They work closely with the clinical teams and investigators to ensure that all procedures and visits required by the protocol occur according to specific guidelines set forth by the same protocol. Research Coordinators manage participant enrollment and ensure compliance with the protocol and other applicable regulations. This includes, but is not limited to, participant recruitment, obtaining informed consent, educating participants on the details of the research study, assessing participant eligibility, facilitating participant care and follow-up according to the protocol, creating source documentation, supporting the assessment of toxicities/adverse events and reporting serious adverse events per IRB and sponsor requirements. The coordinators are considered the point of contact for all participants.

All study team members are responsible for ensuring that the conduct of the study is compliant with institutional, state, federal and industry guidance and regulations.

The clinical research team will make every effort to ensure that your Personal Health Information (PHI) is kept extremely confidential. Your information will not be shared without your permission, except if required by law. If you choose to submit your information to use through this website or by phone, with your permission, this information will be entered into our clinical research database for both current and future study opportunities. Your submission of information is voluntary, and you can request to have your information removed from the database at any time.

In most clinical research studies all visits, tests, medications, and procedures related to the study are free of charge. In addition, if you qualify for one of our research studies, you may be compensated for your time and travel. The amount of compensation (as well as any expenses not covered by the study) will be reviewed during the informed consent process.

The number of study visits, duration, and frequency will vary depending on the study design. Typically, the study visits range from 30 minutes to several hours. The initial visit (screening visit) will usually be longer as it will include the informed consent process, an assessment of your medical history, collection of laboratory samples, and various study related questionnaires.