Frequently Asked Questions (FAQs) About Clinical Trials

How do I participate in a Penn cardiovascular clinical trial?
What is a clinical trial?
Are clinical trials safe?
What is an Institutional Review Board (IRB)?
What is "Informed Consent"?
Who participates in a clinical trial?
Why volunteer for a clinical trial?
What questions should I ask when volunteering for a clinical trial?
Where can I find more information about clinical trial rules and regulations?
How do I report a complaint about a clinical trial to the Food and Drug Administration (FDA)?
What is a protocol?
What do the terms "inclusion" and "exclusion" mean?
What does it mean: Phase One, Phase Two, Phase Three clinical trials?

How do I participate in a Penn cardiovascular clinical trial?

• Read these Frequently Asked Questions (FAQs)
• Ask your doctor – he or she may know of new trials relating to your condition
• Search the Penn Cardiovascular Clinical Trials database
• Search other clinical trial databases, such as:
CenterWatch Clinical Trials
ClinicalTrials.gov
National Heart, Blood and Lung Institute (NHLBI)
Pediatric Heart Network Current Studies (Funded by the NHLBI)
SearchClinicalTrials.org

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What is a clinical trial?

A clinical trial is a carefully designed investigation to measure the effectiveness of a medical treatment on a group of patients.
The medical treatment under investigation could be medicine or drug, medical device, new therapies, vaccines, or new ways of using known treatments.

A clinical trial is conducted under the close supervision of a physician investigator.
It is a carefully proscribed, step-by-step process that assures trial subjects receive careful medical attention.

Learn more about clinical trial participation…
• University of Pennsylvania Office of Regulatory Affairs - Human Research Participants
• University of Pennsylvania Office of Human Research - Research Volunteers
• National Institutes of Health (NIH) ClincalTrials.Gov – Understanding Clinical Trials
• NIH Clinical Center – Participate in Clinical Studies
• NIH Clinical Center – Are Clinical Studies for You?
• Center for Information and Study on Clinical Research Participation – Facts and Figures
• CenterWatch Clinical Trials Listing Service – Patient Resources

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Are clinical trials safe?

People considering participating in a clinical trial should discuss it with their physician or medical caregivers.
They should understand the credentials and experience of the individuals and the facility conducting the study.

In March 2007, the University of Pennsylvania received full accreditation for its human research protection program from the Association for the Accreditation of Human Research Protection Programs, Inc. (AAHRPP). To achieve this status, the University demonstrated a program of participant safeguards that surpasses state and federal requirements. AAHRPP requires additional protections, such as conflict-of-interest rules and community education. Accreditation is valid for three years.

The AAHRPP offers accreditation to organizations that conduct or review research with humans. Responding to increased public concern for protecting research participants, AAHRPP seeks not only to ensure compliance with federal regulations, but to raise the bar in human research protection by helping organizations reach performance standards that surpass the threshold of state and federal requirements. » Learn More

Patient rights and safety are protected in two important ways:

First, any physician awarded a research grant by a private company or the National Institutes of Health MUST obtain approval from an Institutional Review Board before conducting the study. The Institutional Review Board, usually composed of physicians and lay people, examines the study to ensure that a patient’s rights are protected, and that the study does not present undue or unnecessary risk to the patient.

Second, anyone participating in a clinical trial in the United States is required to sign an Informed Consent Form.
The Informed Consent Form details the nature of the study, the risks involved, and what may happen to a patient in the study.

The Informed Consent Form is NOT a contract; participants may leave a study at any time.

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What is an Institutional Review Board (IRB)?

The University of Pennsylvania Institutional Review Board (IRB), overseen by the University of Pennsylvania Office of Regulatory Affairs, is an independent committee that protects the rights and welfare of human research subjects. The IRB may approve, disapprove, or require modifications in order to approve studies being conducted by the University of Pennsylvania Health System, or by faculty, staff or students.

The mission of the University of Pennsylvania IRB is:
1. To promote the rights and welfare of human research participants
2. To facilitate excellence in human research by providing timely and high quality review of human research
3. To provide professional guidance and support to the research community

Contact the University of Pennsylvania’s IRB:

Website: University of Pennsylvania Office of Regulatory Affairs - Institutional Review Board »
Phone: (215) 898-2614
Office Hours: 9AM - 5PM, Monday through Friday
Mailing Address:
University of Pennsylvania Office of Regulatory Affairs
3624 Market Street, Suite 301 South
Philadelphia, PA 19104-6006

Other Important Websites:
University of Pennsylvania Office of Regulatory Affairs - Human Research Participants
University of Pennsylvania Office of Regulatory Affairs

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What is "Informed Consent"?

Informed Consent is a process by which people learn the important facts about a clinical trial to help them decide whether to participate. This information includes details about what is involved, such as the purpose of the study, the tests and other procedures used in the study, and the possible risks and benefits. In addition to talking with the doctor or nurse, people receive a written consent form explaining the study.

People who agree to take part in the study are asked to sign the Informed Consent Form. However, signing the form does not mean people must stay in the study. The Informed Consent Form is NOT a contract. Participants can leave a study at any time - either before the study starts or at any time during the study or the follow-up period.

The informed consent process continues throughout the study. If new benefits, risks, or side effects are discovered during the study, the researchers must inform the participants. They may be asked to sign new consent forms if they want to stay in the study.

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Who participates in a clinical trial?

Participants may be adults, children or families who may be healthy or with disease, depending on the study.
Trial participants volunteer for a clinical study and sign an Informed Consent Form.

Participants have the option to withdraw at any point. In some clinical trials, volunteers may be reimbursed for their participation.

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Why volunteer for a clinical trial?

By taking part in a clinical trial, you can try a new treatment that may or may not be better than those that already exist.
You can also help others better understand how the treatment works in people of different races and genders.
You can become a partner in scientific discovery and help accelerate the pace of medical advances.

Points to Consider:
• A research study may or may not help you personally.
• In the future, the results could help others who have a health problem.

The Center for Information and Study on Clinical Research Participation - CISCRP - provides the latest facts and statistics on clinical research participation in easy-to-understand terms. CISCRP educates, informs and empowers patients, the public, medical and research professionals, the media and policymakers about clinical research participation.

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What questions should I ask when volunteering for a clinical trial?

Get answers to these questions when you meet with a clinical trial physician:

Download Questions PDF

• Why is the research being done?
• How long will the study last?
• Who should I call if I have a question about the study?
• Will the research help me personally?
• Could the research hurt me?
• Who pays if I'm unexpectedly injured in the study?
• What will the researcher do with my information?
• What kinds of test and exams will I have to take?
• How much time do these take?
• What will be done to me as part of the research?
• How often does the study require me to go to the doctor or clinic?
• What are the costs to me? Will my health insurance pay for it?
• What are my other treatment choices? How do they compare with the treatment being studied?
• Are there any side effects to expect as a result of the research?
• How do they compare with side effects of standard treatment?
• Will there be any follow-up after the study?
• What happens at the end of the study?

Points to Remember
• Clinical trials are tests of medical treatments to see if they are safe and if they work.
• Before you agree to take part in a study, you must be given complete information about
  the study, including possible side effects and benefits.
• You should ask lots of questions to be sure you understand the study.
• You must sign a special agreement called an "Informed Consent Form" before taking part in the study.
• The Informed Consent Form is NOT a contract. You may leave the study at any time.

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Where can I find more information about clinical trial rules,
regulations, and federal protections?

• University of Pennsylvania Office of Regulatory Affairs - Human Research Participants
• National Institutes of Health (NIH) ClinicalTrials.gov
• FDA Clinical Trial Consumer Information
• Office for Human Research Protections (U.S. Dept. of Health and Human Services)
• Center for Information and Study on Clinical Research Participation (CISCRP)

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How do I report a complaint about a clinical trial to the
Food and Drug Administration (FDA)?

You may report a complaint related to FDA-regulated clinical trials at this FDA website:
Reporting Complaints Related to FDA-Regulated Clinical Trials

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What is a protocol?

A protocol is a research study plan.
The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions.

All protocols must be approved by an oversight committee called the Institutional Review Board (IRB).

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What do the terms "inclusion" and "exclusion" mean?

Clinical trial descriptions provide "inclusion" and "exclusion" criteria; they are required conditions for study participation.
These conditions help researchers to select particpants who provide the best match for research being conducted.

Inclusion criteria describe conditions researchers are seeking from study participants.
For example, the researcher only wants to recruit study participants who are over the age of 50.

Exclusion criteria describe conditions researchers are NOT seeking from study participants.
For example, the researcher does not want to recruit study participants who smoke.

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What does it mean: Phase One, Phase Two, Phase Three clinical trials?

When a drug or other product has undergone successful preclinical testing, a pharmaceutical company provides this data to the Food and Drug Administration (FDA), requesting approval to initiate testing of the treatment in humans. This is called an Investigational New Drug application (IND). Receiving FDA-approval, the company will initiate a Phase One clinical study.

A Phase One Study is the earliest use of an FDA-approved pharmaceutical in people. Phase I studies require specialized monitoring and lab facilities as well as a skilled investigative group to ensure safety of the product and to perform the sophisticated data analyses needed. A Phase I study usually involves a small number of healthy patients, perhaps 20 or more, and also investigates side effects that may occur as dosage levels are increased.

A Phase Two Study enrolls a carefully screened group of patients into detailed protocols to study a product’s clinical efficacy. This testing phase may last up to two years, and involve several hundred patients. Many Phase II studies are randomized trials , in which one group of patients receives the experimental drug, while a second "control" group receives a standard treatment or placebo. Often these studies are blinded , so neither the patient nor the physician knows who is receiving the experimental drug. In this manner, the study can yield comparative information about the relative safety of the new drug and its effectiveness.

A Phase Three Study usually involves multiple sites and greater numbers of patients (perhaps several thousand). Patients are carefully screened to meet stringent protocol requirements, and studies are usually randomized, blinded, and last several years. This large-scale testing provides the pharmaceutical company and the FDA with a more thorough understanding of the drug’s effectiveness, benefits, and the range of possible adverse reactions. This expanded safety and efficacy database is the last stage before a company can request FDA approval for marketing the drug.

Once the FDA has granted a New Drug Approval (NDA) to market the drug, a post approval Late Phase Three and/or Phase Four Study might continue assessment of the drug or device’s performance in actual clinical use, without the strict criteria employed in earlier phase trials. The company may also want to compare a drug with other drugs already available, and monitor a drug’s long-term effects and impact on quality of life. Statistical information about the costs of treating illness, compared to alternate therapies, and the measurements of outcomes are derived from large-scale databases.

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