Research Administration

A clinical trial (also called clinical research) is a research study in human volunteers to answer specific health questions. Carefully conducted clinical trials are the fastest and safest way to find treatments that work in people and ways to improve health. Interventional trials determine whether experimental treatments or new ways of using known therapies are safe and effective under controlled environments. Observational trials address health issues in large groups of people or populations in natural settings

Most clinical trials are sponsored by pharmaceutical companies, federal health agencies, medical foundations, medical centers, and universities; however, physicians may sponsor some clinical trials, particularly when the trial evaluates a currently available treatment for a condition other than for what it was previously approved.

Clinical trials are performed around the world in various settings, including doctors’ offices, hospitals, research centers and teaching institutions. Since most trials require periodic visits to the study physician, volunteers should take the location of the facility into consideration when deciding whether to participate in a clinical trial.

Informed consent is the process by which a potential study participant agrees to participate in a trial after being provided with information about the study and treatment. It includes an explanation of what’s involved in the trial, the duration of the study, the frequency and length of the required visits, as well as the known risks and benefits associated with the study treatment.

Participation in a clinical trial is completely voluntary. All questions about the study should be answered to the participant’s satisfaction before an informed consent form (ICF) is signed. Participants have the right to refuse participation or to withdraw their consent at any time during the study without penalty.

The wishes and best interest of the participant are considered at all times. Throughout the trial, participants will be provided with any new information that may affect their decision to continue their involvement in the study. In addition, the study physician may withdraw a participant at any time, without consent, if it becomes evident that it’s not in their best interest to continue.

Before you and your child decide whether to participate in research, you must be fully “informed.” It is important for you to understand:

• Your participation is voluntary
• Why the research is being done
• What the researchers want to accomplish
• What will be done and for how long
• What risks are involved
• What possible benefits can be expected
• What other treatments are available
• You have the right to leave the study at any time without interfering with you regular care.

Research at UTMB includes children as well as adults. The law states that children cannot make their own decision about research. We, however, feel it is important that children receive an explanation of the research and have a chance to ask questions. If your child's doctor tells you that your son or daughter must "assent" to participate, it is because we respect his or her rights as a person. "Assent" refers to the process by which we ask a child's agreement to participate. In most cases, investigators will obtain a child's assent if he or she is over the age of 7. It is important that the information shared with your child be understandable. The researcher will consider your child's age and maturity level when presenting information. Doctors and parents need to work together to help the child understand what a study involves.

Adults who cannot legally make decisions for themselves may also agree to participate through an assent process. Researchers must also obtain the consent of an individual who represents such. Investigators want to make sure that the person whose mental abilities are impaired has some understanding of what will happen to him/her and can say no if he/she so chooses.

Sometimes assent is not required. In situations where the research may be the only way to help a patient with a life-threatening illnesses, assent is not required if the research is expected to be beneficial to the individual. Even so, researchers are encouraged to discuss the research with the individual patient.

A placebo is an inactive substance, which may be given to some of the study participants in a clinical trial as a control, so that researches can verify that any results can be attributed to the drug being tested assist in comparing the effects of the active study treatment.

There’s no cost for joining a clinical trial. Study medication, as well as study-related tests and procedures are usually provided at no charge to study participants. In some cases, you may be compensated for your time and travel expenses related to study participation.

In the interest of safety, the participant must fully inform the study physician about past and current medical conditions, illnesses, and medications, follow the prescribed treatment plan and attend scheduled visits. Study personnel will explain other study-specific responsibilities.

Children are not little adults. Without research, doctors cannot know how best to diagnose and treat babies and children. About two out of three medicines used for sick children have not been tested in children for correct dosage, safety and side effects. Also, research involving healthy kids adds important information to improve children’s health by learning more about their bodies and how they work.

Participating in research, including “healthy child research,” may lead to knowledge that could benefit your child as well as others. Depending upon the research, participants may also learn information about their condition. Research may help determine:

• Correct dose
• Medication safety
• How well the medicine works
• Side effects

Research participation is not right for every child or family. It is a personal choice. You should not feel bad if you or your child choose not to participate in research.

You may consider taking part in a study because:

• You may benefit from the best possible treatment or an experimental treatment that would otherwise not be available to you.
• The physicians and nurses will closely monitor your progress throughout the trial.
• You would be helping researchers to improve the treatments for future patients.

You might consider not taking part in a study because:

• The experimental treatment may not work for you, or it could make your condition worse.
• The experimental treatment may cause side effects that no one anticipated.
• Your health insurance may not cover research procedures.
• If the trial is randomized and includes a placebo (an inactive, dummy pill), you may not be given the experimental treatment.
• The amount of testing for efficacy and safety purposes may involve too many trips to the research office and take too much time.

When deciding whether to participate, you should ask the following questions and you should feel comfortable with the answers:

• If I am ill, will this research help me?
• What are the risks?
• What is involved? What will I have to do?
• Will I be charged anything or compensated for my participation?
• How can I end my participation if I change my mind?
• What will happen when the study is over? Will I be told the results?
• Is the study controversial?
• Whom do I contact to express concerns or obtain information?

The UTMB Institutional Review Board (IRB) protects people in research studies. The IRB includes scientists, non-scientists, and community members. The IRB reviews, approves, and monitors all research at UTMB in which people take part. This oversight keeps risks to research participants as low as possible. The IRB also keeps track of ongoing studies to make sure they are being done in the right way. The IRB requires that all researchers treat research participants with respect. The IRB protects your rights and welfare if you take part in a research study.

At the end of a study, the researchers analyze the data that were collected from all participants throughout the study. Findings and data collected about you will be compared to other participants. In clinical trials, doctors and specialists in biostatistics would conduct the analysis and report the findings to scientific meetings and medical journals. Data will also be shared with experts and various government agencies responsible for the approval of new drugs, biologics and devices. Not all experimental treatments receive final FDA approval in the end.

What happens when the study is over also varies from study to study. Sometimes patients can remain on the study drug if they are responding to the new treatment; however, this is the exception rather than the rule. Generally, participation ends when the study ends because it might not be safe or effective to continue treatment based on what is known at the time. Clinical trial participants can withdraw from the study at any time without jeopardizing the rest of their standard medical care.

In an observational study, investigators assess health outcomes in groups of participants according to a research plan or protocol. Participants may receive interventions (which can include medical products such as drugs or devices) or procedures as part of their routine medical care, but participants are not assigned to specific interventions by the investigator (as in a clinical trial). For example, investigators may observe a group of older adults to learn more about the effects of different lifestyles on cardiac health.

Randomized is used to describe a research study that hopes to compare two or more different treatments or procedures. Randomized means that you will be assigned to a study group by chance, like flipping a coin.

Most randomized trials will only disclose this kind of information when the study has been completely finalized; this is done to protect the integrity of the research data and results. If the trial is a "double-blind" trial, the doctor will not even know which substance you received. Most protocols will have information with respect to when a study will be "unblinded." If this is something that you want to know more about, ask the investigator what the study's policy is before you agree to participate.

A registry is a database composed of information and/or tissues obtained from individuals. This information is collected in a standardized way so that it can be used in research; in this way, it is different from information collected typically by the patient's doctor. These registries are also called "banks." Registries are established for the purpose of collecting as much information as possible about a particular illness or a group of people. Some registries are very large and may include family members of people with specific illnesses. Agreeing to participate in a registry could be as simple as allowing a research study to collect tissues that would normally be thrown away after surgery; or a registry could be designed to collect blood, tissue specimens, and information from your medical records for many years. A registry could have information on 100 people or it could have information on 10,000 people. It is from registries and banks that many researchers develop new ideas for new research studies.

As part of the Health Insurance Portability and Accountability Act of 1996 (HIPAA), which was originally passed as a means of maintaining healthcare coverage, Congress included Administrative Simplification provisions in HIPAA directed at standardizing how healthcare information and claims are processed and used. The privacy provisions (as well as the security provisions) were then added to HIPAA's Administrative Simplification provisions to protect how patient health information or protected health information (PHI) is stored, transmitted and used.

The Health Insurance Portability and Accountability Act Privacy Rule establishes the conditions under which protected health information (PHI) may be used or disclosed by covered entities for research purposes. The Privacy Rule also defines the means by which individuals will be informed of uses and disclosures of their medical information for research purposes, and their rights to access information about them held by covered entities.

It may be necessary for a researcher to create new health information about you or to review information in your medical records as part of your participation. Before being able to do this, the researcher must obtain your authorization to access your health information. As part of the informed consent process, you will receive a description of the type of health information to be viewed or created as part of the research, understand who will view the information, for what purposes, and for how long. After reviewing this information, you will be asked to grant permission for the use of your information by providing "authorization."

You have the right to revoke or withdraw your authorization for the researcher to use your identifiable health information. If you wish to revoke or withdraw your authorization, you must do so in writing, and provide that written revocation to the researcher to his/her mailing address. The researcher will no longer be able to allow you to participate in the research study and will stop collecting any additional identifiable health information about you. However, the researcher may, by law, continue to use the health information about you previously collected, as necessary to maintain the integrity of the research study and make reports to oversight agencies.

A protocol is 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. While in a clinical trial, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.

Clinical trials are conducted in phases. The trials at each phase have a different purpose and help scientists answer different questions:

• In Phase I trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
• In Phase II trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
• In Phase III trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
• In Phase IV trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.

Most human use of investigational new drugs takes place in controlled clinical trials conducted to assess safety and efficacy of new drugs. Data from the trials can serve as the basis for the drug marketing application. Sometimes, patients do not qualify for these carefully-controlled trials because of other health problems, age, or other factors. For patients who may benefit from the drug use but don't qualify for the trials, FDA regulations enable manufacturers of investigational new drugs to provide for "expanded access" use of the drug. For example, a treatment IND (Investigational New Drug application) or treatment protocol is a relatively unrestricted study. The primary intent of a treatment IND/protocol is to provide for access to the new drug for people with a life-threatening or serious disease for which there is no good alternative treatment. A secondary purpose for a treatment IND/protocol is to generate additional information about the drug, especially its safety. Expanded access protocols can be undertaken only if clinical investigators are actively studying the new treatment in well-controlled studies, or all studies have been completed. There must be evidence that the drug may be an effective treatment in patients like those to be treated under the protocol. The drug cannot expose patients to unreasonable risks given the severity of the disease to be treated. Some investigational drugs are available from pharmaceutical manufacturers through expanded access programs listed in ClinicalTrials.gov. Expanded access protocols are generally managed by the manufacturer, with the investigational treatment administered by researchers or doctors in office-based practice. If you or a loved one are interested in treatment with an investigational drug under an expanded access protocol listed in ClinicalTrials.gov, review the protocol eligibility criteria and location information and inquire at the Contact Information number.

• ClinicalTrials.gov provides patients, family members, and members of the public easy and free access to information on clinical studies for a wide range of diseases and conditions.
• The National Cancer Institute provides accurate, up-to-date information about many types of cancer, information about clinical trials, resources for people dealing with cancer, and information for researchers and health professionals.
• The Center for Information and Study on Clinical Research Participation is an independent non-profit organization dedicated to educating and informing the public and patients about clinical research.