By Angela Spivey

Before medications or treatments for any disease are put on the market, scientists test them in studies known as clinical trials. These studies determine whether a medication or treatment is safe and effective in fighting a specific disease or condition, and how and in what dosage the treatment should be administered.

Patients provide the essential ingredient in those trials. In fact, some of those patients come from FirstHealth Moore Regional, which enrolls patients in national clinical trials such as those sponsored by the National Cancer Institute.

Offering enrollment in national clinical trials is a sign of quality for a health care institution, says John Byron, M.D., a board certified OB/GYN specialist with the Southern Pines Women’s Health Center, since a hospital must go through a rigorous approval process to qualify to participate.

“If you’re going to have cancer treatment, and you’re looking at a place to have it done, if the institution is offering clinical trials, that means they’ve gone through all the regulatory evaluations and have the facilities to take care of you,” says Dr. Byron, the principal investigator for Gynecologic Oncology Group trials at Moore Regional. “The hospital has met the same requirements that Duke or Chapel Hill have to fulfill.”

The National Cancer Institute sponsors the GOG.

According to Dr. Byron, patients who are enrolled in national clinical trials receive treatment as part of a standardized plan that’s been reviewed by many doctors and scientists. All patients enrolled in the trial receive the same treatment, whether they’re being treated at Moore Regional or at the Mayo Clinic.

“All the smartest people looking at this particular cancer and the treatments have come up with what they think is the best way to treat it, and everybody has to follow the same recipe,” he says.

New medications or therapies undergo at least three phases of clinical trials. Phase I assesses the medication’s safety in a small group of 20 to 80 healthy people. This is usually the first time the medication is tested in humans. This phase also determines a safe dosage and identifies side effects.

Phase II studies test the medication in 100 to 300 people who have a specific condition or disease that the medication is designed to treat. This phase helps determine how well the medication works to treat that condition, and it further evaluates the medication’s safety.

Next, large groups of 1,000 to 3,000 people take the medication in Phase III studies. At this stage, researchers want to find out how well the medication works compared to standard treatment or, sometimes, compared to a placebo, which is an inactive substance.

After Phase III studies are successfully completed, the Federal Drug Administration (FDA) reviews the results and considers the medication for FDA approval. Phase IV studies—which can be conducted after a drug is already on the market—provide further information about a medication’s risks, benefits and optimal dosage.

Especially for patients with life-threatening diseases such as cancer, participating in clinical trials can offer hope. “A clinical trial offers you a way to get the newest experimental medications in development,” Dr. Byron says.

Phase II trials offered at Moore Regional, for instance, often test new agents that have been proven to work against cancers in general but may not be offered to the general public to treat a particular cancer.

Before a trial enrolls patients, a committee made up of doctors, scientists and at least one non-scientist community member reviews the study plan. This committee, called an Institutional Review
Board, can either approve a trial, approve

it providing that changes are made to the plan or deny approval. Before approving a trial, the IRB is charged with ensuring that the trial’s potential benefits outweigh its potential risks.

National clinical trials offered at Moore Regional are reviewed by the Sandhills IRB as well as by the IRB of the national institution sponsoring the trial. “Even if it’s been reviewed 20 times, when we enroll patients locally, we review it again,” Dr. Byron says.

Despite all the built-in protections, patients are accepting some risk when they enroll in a clinical trial. Before enrolling, patients are given detailed consent forms outlining the trial’s study plan and its potential risks and benefits. Ultimately, it’s up to the individual to decide whether to enroll and participants can withdraw from a trial at any time.

What are some of the risks? For one, the investigational treatment may not necessarily work better than standard treatments, or even as well. Or the new treatment may cause unexpected side effects.

And participating in the trial might require more time than the patient’s standard care. “Patients need to consider that a trial is going to involve rigorous follow-up, and that they’re going to need to allow the appropriate amount of time for that,” Dr. Byron says.

For example, some trials ask patients to keep a diary to assess quality-of-life during cancer treatment.

Often when a patient is enrolled in a trial that is testing a new investigational drug, the drug itself is provided at no cost. However, some trials may include extra expense, says Pat Young, R.N., clinical trials coordinator at Moore Regional.

One national clinical trial is currently enrolling patients from Moore Regional who are at higher risk for ovarian cancer. Women who choose to have their ovaries removed preventively are compared to those who choose to keep their ovaries but undergo regular screening procedures such as ultrasound and testing for a blood marker of ovarian cancer.

The trial covers the cost of the blood screening, but the patient and her insurance cover the rest of the cost.

In this trial, Young says, women choose whether they will undergo screening only or will have their ovaries removed. And at any time during the study, they can opt to switch to the ovary-removal group. But in other trials, patients are randomized (chosen randomly to receive either the investigational medication or the standard treatment.)

Virtually all of the trials at Moore regional include an additional layer of protection, because national cooperative groups such as the GOG run them. Data monitoring committees review the results of these national trials, and every six months, the committee recommends whether to close the trial or keep it open. A trial may close if a medication shows serious side effects in a significant number of patients, or if it shows dramatic benefits.

“So if a study closes early, because an particular arm that was getting the real drug has shown a benefit, all the patients are told which medication they have been given, which may have been the drug being developed, and are given the option to continue receiving the drug,” Young says. “Patients who were getting standard care are offered the opportunity of receiving the treatment that shows an advantage over the standard treatment.”

Many of the women in the ovarian cancer prevention trial, for instance, are thinking of their daughters and granddaughters.

“Patients who are participating in clinical trials deserve the best and should be protected in any way and every way possible,” Young adds. “They are doing such a service to not only themselves but to humankind. A lot of people participate in a clinical trial not only to help themselves possibly but to help people in the future.”