The Clinical Research Process

Clinical trials are conducted to allow safety and efficacy data to be collected for new drugs, medical devices, or treatment techniques. Depending on the type of product and the stage of its development, healthy volunteers and/or patients enroll into small pilot studies initially, followed by larger scale studies in patients that often compare the new product with the currently prescribed treatment.

As positive safety and efficacy data is gathered, more patients are typically involved. Clinical trials can vary in size from a single center in one country to multi-center mega-trials across several countries.

Clinical trials are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups, and pharmaceutical companies, in addition to federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD), and the Department of Veteran’s Affairs (VA). Trials can take place in various locations, such as hospitals, universities, doctors’ offices, or community clinics.

Participants in clinical trials can play a more active role in their own health care, gain access to new research treatments before they are widely available, and help others by contributing to medical research.

All clinical trials have guidelines about who can participate and criteria that must be met. The factors that allow someone to participate in a clinical trial are called inclusion criteria and those that disallow someone from participating are called exclusion criteria.

These criteria are based on such factors as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions. Before joining a clinical trial, a participant must qualify for the study. Some research studies seek participants with illnesses or conditions to be studied in the clinical trial, while others need healthy participants.

People should know as much as possible about the clinical trial and feel comfortable asking the members of the health care team questions about it, the care expected while in a trial, and the cost of the trial. The following questions might be helpful for the participant to discuss with the health care team. Some of the answers to these questions are found in the informed consent document.

• What is the purpose of the study?

• Who is going to be in the study?

• Why do researchers believe the new treatment being tested may be effective?

• Has it been tested before?

• What kinds of tests and treatments are involved?

• How do the possible risks, side effects, and the benefits in the study compare with my current treatment?

• How might this trial affect my daily life?

• How long will the trial last?

• Will hospitalization be required?

• Who will pay for the treatment?

• Will I be reimbursed for other expenses?

• What type of long-term follow up care is part of this study?

• How will I know that the treatment is working?

• Will results of the trials be provided to me?

• Who will be in charge of my care?

Informed consent is the process of learning the key facts about a clinical trial before deciding whether or not to participate. It is also a continuing process throughout the study to provide information for participants. To help someone decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. Then the research team provides an informed consent document that includes details about the study, such as its purpose, duration, required procedures, and key contacts. Risks and potential benefits are explained in the informed consent document. The participant then decides whether or not to sign the document. Informed consent is not a contract, and the participant may withdraw from the trial at any time.

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 can 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.

Side effects are any undesired actions or effects of drug or treatment. Negative or adverse effects may include headache, nausea, hair loss, skin irritation, or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects.

The ethical and legal codes that govern medical practice also apply to clinical trials. In addition, most clinical research is federally regulated with built in safeguards to protect the participants. The trial follows a carefully controlled protocol, a study plan which details what researchers will do in the study. As a clinical trial progresses, researchers report the results of the trial at scientific meetings, to medical journals, and to various government agencies. Individual participants’ names will remain secret and will not be mentioned in these reports (See Confidentiality Regarding Trial Participants).

A control is the standard by which experimental observations are evaluated. In many clinical trials, one group of patients will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.

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

Most clinical trials provide short-term treatments related to a designated illness or condition, but do not provide extended or complete primary health care. In addition, by having the health care provider work with the research team, the participant can ensure that other medications or treatments will not conflict with the protocol.

Yes. All medical records and research materials that identify you will be held confidential so far as permitted by law. Only individuals with study affiliation will have access to your personal information

Yes. A participant can leave a clinical trial at any time. When withdrawing from the trial, the participant should let the research team know so that we may retrieve any remaining medication and do an exit visit if possible.