Hoag’s Clinical Research

Clinical trials are research studies that evaluate the safety and effectiveness of new ways to diagnose, treat and prevent diseases or conditions.

Treatments studied in clinical trials may be new drugs or new combinations of drugs, new surgical procedures or devices, or new ways to use existing treatments.

These studies may also show which medical approaches work best for certain illnesses or groups of people.

Clinical trials follow strict scientific standards to protect patients and help produce reliable study results that can lead to new advances in medical care.

Clinical trials are conducted in four different phases with each phase serving a specific purpose to researchers.

Phase I: Assess the safety of a new drug or treatment. The phase is usually done with a small group of participants and is designed to determine the effects of the drug or treatment on humans including safe dosage ranges and side effects.

Phase II: The drug or treatment is given to a larger group to determine effectiveness and to continue to evaluate safety. Most phase II studies are randomized trials in which one group of participants receives the experimental drug, while a second “control” group receives a standard treatment or placebo. Often these studies also use blind testing, which means that neither the participants nor the researchers know who has received the experimental drug.

Phase III: Several hundred or even thousands of participants may receive a new drug or treatment and be followed for several years. This phase usually involves randomized and blind testing to help confirm the effectiveness of the drug, gauge side effects and gather information so the drug or treatment can be used safely.

Phase IV: The drug or treatment has received FDA approval, which enables researchers to gather additional safety and effectiveness data on an even larger group of people to determine its impact on quality of life. The drug or treatment may also be evaluated for cost and against other similar drugs already on the market. Phase IV studies can result in a drug or device being taken off the market, or restrictions of use could be placed on the product, depending on the findings in the study.

Each clinical trial has specific guidelines for who is eligible to take part in the study. Often, these criteria are related to:

• Age

• Gender

• Type and stage of a disease

• Previous or current medications

• Existing medical conditions

• Recent participation in a clinical trial

Some studies enroll people who have a specific disease or condition. Others enroll healthy people to test new approaches to prevention, diagnosis, or screening.

Only people who fit the clinical trial’s eligibility criteria are able to participate. It’s important to keep in mind that these criteria are used to protect your safety during a clinical trial.

Before deciding to participate in a study, you will receive an informational document called an informed consent form. This form provides important facts about the study to help you decide if participating is right for you.

The research team will review this form with you and answer your questions. Although you must sign the informed consent form in order to participate in the study, it is not a contract. You may leave the study at any time for any reason.

Your study records are private and confidential. Only the research team and your physician or nurse will have access to your records.

There are many potential benefits for clinical trial participants. Patients with existing diseases or conditions are able to take a more active role in their own health care, and gain access to new research treatments before they are widely available.

If you do not receive the new strategy being tested, you may get the current standard care for your condition. This treatment might be as good as, or better than, the new approach. You also will have the support of a multidisciplinary team of experts, who will monitor your health closely.

Regardless of any direct benefit from the clinical trial you take part in, the information gathered can help others and add to scientific knowledge. Many people who take part in clinical trials want to help others, and be part of the process of improving medical care.

New treatments being studied are not always better than, or even as good as, standard care. As with standard treatments, the new treatment may not work for you, even if it works for other patients.

In addition, a new treatment may have side effects that are unexpected, or that are worse than those of standard care. This is especially true during phase I and phase II clinical trials. The risk of side effects might be even greater for trials with innovative approaches, such as gene therapy or new biological treatments.

Also, insurance companies do not always cover all patient care costs in a study. What is covered varies by plan and by study.

This is why it’s important to understand the risks and benefits of any clinical trial before you agree to volunteer. If you decide to participate, you will be given contact information in case of severe side effects or an emergency. If the treatment you receive in the study turns out to be harmful, the study will be stopped.

It is vital for patients to be well informed about the clinical trial before agreeing to participate.

The following questions may be helpful for the participant to discuss with the health care team, and may be found in the informed consent document.

• What is the purpose of the study?

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

• Has it been tested before?

• What kinds of tests and experimental treatments are involved?

• What are the possible risks, side effects, and benefits of the new treatment, and how do these compare with my current treatment?

• Why do I want to participate in a clinical trial?

• Have I considered other possible options?

• What are my goals and expectations if I decide to take part? How realistic are these?

• How might this trial affect my daily life?

• How long will the trial last?

• Will hospitalization be required?

• Who will pay for the experimental 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 experimental treatment is working?

• Will results of the trials be provided to me?

• Who will be in charge of my medical care?

Although some of these questions may not have clear-cut answers, they should help you in thinking about some important issues. Each person’s reasons for wanting or not wanting to take part in a study may be different. Asking questions and taking time to consider what’s best for you is an important part of the decision-making process.

Yes. A participant can leave a clinical trial at any time, for any reason.

Randomized Controlled Trials (RCTs) are the gold standard for testing the efficacy and safety of new medical interventions, and represent three important elements of the scientific design:

• Randomized: Randomization is important in a clinical trial in order to prevent the results from being skewed. When participants are assigned at random (by chance alone) to the treatment or control arm of a clinical trial, the results are free from a specific type of selection bias.

• Controlled: In the control group, the participants do not receive the new treatment being tested, but receive a reference treatment or placebo instead. The control group is used for comparison or reference.

• Trial: During an RCT, the drug or treatment being studied is on trial. Based on the results, it will be approved or disapproved. In order to be approved for wider use, the results must indicate that there is a worthwhile level of efficacy, which must be balanced against an acceptable level of adverse effects.

As with any clinical trial, it is the patient’s choice to be in a randomized trial.

A placebo is an inactive substance or treatment that has no beneficial value. In clinical trials, experimental treatments are often compared with placebos to assess the experimental treatment’s effectiveness.

The term “placebo effect” is used to describe a beneficial effect that is produced by a placebo drug or treatment that cannot be attributed to the actual properties of the placebo itself. Instead, these positive effects are the result of a patient’s belief in the treatment as beneficial.