About Clinical Trials

A clinical trial is a research study conducted to answer specific questions about new treatments, or to investigate new ways of using known treatments. In most cases, the therapies and drugs investigated in clinical trials are not approved by Regulatory Agencies (such as the Food and Drug Administration (FDA) in the United States), and the benefits and risks of taking the treatment are not yet confirmed. Clinical trials are used to determine whether new treatments are safe and effective. The knowledge gained from clinical trials regarding the safety and efficacy of new treatments or drugs ultimately may allow pharmaceutical companies to offer new and better options to patients.

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Preclinical Studies

Before testing of a potential therapeutic begins in humans, a pharmaceutical company conducts laboratory and animal studies to determine the product's safety and biological activity. These studies provide information on potential side effects, the amount of the product that is absorbed by the body, the amount of time the product stays in the body, and the product's therapeutic effect.

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Clinical Evaluation

After the preclinical studies are complete, the company approaches the FDA with a request to test the product candidate in people. The company files an Investigational New Drug (IND) application, which contains the data from preclinical studies and other information. It also submits a proposal to study the product's safety and dosage in people.

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Clinical Trials Phase I: Safety

A phase I clinical trial is the first step in a clinical trial program for a product candidate. This trial is designed to assess safety and establish dosing for future trials.

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Phase II/III: Safety and Efficacy

Phase II and III studies determine the dose and efficacy in treating the targeted medical condition. Often, a pharmaceutical company conducts multiple phase II or III trials to study a product in a variety of patient populations or disease indications, e.g., prevention of bleeding in adults and children, treatment of an acute bleeding episode in adults and children, or maintenance of hemostasis in surgery. Phase II or III studies may include multiple arms that compare various treatment or dosing approaches.

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Risks and Safety Measures

By participating in a clinical trial, a patient may experience unpleasant, serious, or even life-threatening side effects of the drug or treatment being studied. The drug or treatment may not be effective for patients. Every clinical trial has a protocol which describes exactly what the physician and patient must do during the trial. The patient may be required to visit the clinical trial investigation site often and undergo numerous tests to evaluate the drug or therapy.

To minimize the risks and protect patients' rights, all clinical trials must be approved and monitored by an Institutional Review Board or Ethics Committee. These review boards/committees also examine the study's protocol to validate that the patient's rights are protected, and that the study does not present an undue or unnecessary risk to the patient.

In addition, anyone participating in a clinical trial in the United States is required to provide informed consent, this is typically documented when the patient signs and dates an "informed consent form". This form explains the conduct of the clinical trial in detail, the risks involved, the potential benefit of the experimental treatment or drug, what a patient can expect during the clinical trial, and assures the patient that he/she has the right to leave the study at any time with no consequence to their health.

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Benefits

Patients participating in a clinical trial may benefit by having access to the latest research and treatments not available to the general public. These patients often are able to get advanced medical care at a leading health care center for little or no cost to them. Participation in a clinical trial also allows patients to have an active role in their own health care, as well as help others by contributing to medical research.

Patients considering participation in a clinical trial should discuss it first with their doctors. Patients should also be sure that they understand all risks and potential benefits by asking questions and ensuring the questions are answered to their satisfaction. Below is a list of suggested questions to help guide a patient's conversation with a study doctor or a delegate to the study doctor (nurse coordinator):

• What is the purpose of the trial?
• How long will the trial last?
• How often will I be required to visit the study site?
• What types of tests or procedures will I have to undergo?
• Where is the trial being conducted?
• How is my health/safety monitored?
• Are there any risks?
• What are the projected benefits of my participation in the trial?
• Are there alternatives to the treatment being tested in the trial?
• Who is sponsoring the trial?
• Will I be expected to pay for any part of the trial?
• What happens if I experience a side effect during the course of the trial?